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Item 1. Business

2026-03-05 compared with 2025-03-10 · 403 added, 509 removed, 535 unchanged (63% of the section changed)

We are a clinical-stage C lustered R egularly I nterspaced S hort P alindromic R epeats (“CRISPR”) genome-editing biopharmaceutical company dedicated to developing transformative therapies for patients with devastating diseases.

Our genome-editing platform, including our novel chRDNA ( C RISPR h ybrid R NA- DNA , or “chRDNA,” pronounced “chardonnay”) technology, enables more precise genome editing of allogeneic cell therapies.

Our allogeneic chimeric antigen receptor (“CAR”) -T (“CAR-T”) cell therapy product candidates are manufactured in advance with cells from healthy donors, with the goal of enabling broad patient access, rapid patient treatment, and increased manufacturing scale.

Our allogeneic CAR-T cell therapy product candidates in clinical development are directed at established cell surface targets against which autologous CAR-T cell therapeutics have already demonstrated clinical proof of concept, including CD19 and B cell maturation antigen (“BCMA”), as well as targets such as C-type lectin-like molecule-1 (“CLL-1”).

We use our chRDNA technologies to armor our cell therapies through multiple genome-editing strategies, such as checkpoint disruption, immune cloaking, or a combination of these two strategies, to enhance activity against devastating diseases.

We are advancing our pipeline of allogeneic CAR-T cell therapies with the following four clinical development programs targeting the treatment of hematologic malignancies and autoimmune diseases:

an allogeneic anti-CD19 CAR-T cell therapy, being evaluated in patients with relapsed or refractory B cell non-Hodgkin lymphoma (“r/r B-NHL”) in our ANTLER phase 1 clinical trial

also being evaluated in patients with lupus nephritis (“LN”) and in patients with extrarenal lupus (“ERL”) in our GALLOP phase 1 clinical trial

an allogeneic anti-BCMA CAR-T cell therapy, being evaluated in patients with relapsed or refractory multiple myeloma (“r/r MM”) in our CaMMouflage phase 1 clinical trial

an allogeneic anti-CLL-1 CAR-T cell therapy, being evaluated in patients with relapsed or refractory acute myeloid leukemia (“r/r AML”) in our AMpLify phase 1 clinical trial

CB-010 has received regenerative medicine advanced therapy (“RMAT”) designation for relapsed or refractory large B cell lymphoma (“r/r LBCL”) as well as fast track designations for r/r B-NHL and refractory systemic lupus erythematosus (“SLE”) from the U.S.

Our genome-editing platform is based on our novel chRDNA ( C RISPR h ybrid R NA- DNA , or “chRDNA,” pronounced “chardonnay”) genome-editing technology, which enables more precise genome editing of allogeneic cell therapies.

Our allogeneic, or off-the-shelf, chimeric antigen receptor (“CAR”) -T (“CAR-T”) cell therapy product candidates are manufactured in advance with cells from healthy donors, with the goal of enabling broad patient access, rapid patient treatment, and increased manufacturing scale.

Our allogeneic CAR-T cell therapy product candidates in clinical development are directed at CD19 and B cell maturation antigen (“BCMA”), established cell surface targets against which autologous CAR-T cell therapeutics have already demonstrated clinical proof of concept.

We use our chRDNA technology to armor our cell therapy product candidates through genome-editing strategies, such as checkpoint disruption and immune cloaking, to enhance activity against hematologic malignancies.

We are advancing two clinical-stage allogeneic CAR-T cell therapy product candidates for the treatment of patients with hematologic malignancies:

• Vispacabtagene regedleucel (“vispa-cel,” formerly CB-010):

an allogeneic anti-CD19 CAR-T cell therapy that has been evaluated in patients with relapsed or refractory B cell non-Hodgkin lymphoma (“r/r B-NHL”) in our ANTLER phase 1 clinical trial.

an allogeneic anti-BCMA CAR-T cell therapy that is being evaluated in patients with relapsed or refractory multiple myeloma (“r/r MM”) in our CaMMouflage phase 1 clinical trial.

We believe vispa-cel, if approved, will have several advantages over commercially available autologous CAR-T cell therapies, including broad patient access, shorter time to treatment with an off-the-shelf product, significantly lower manufacturing costs, and a substantially smaller footprint for manufacturing.

Vispa-cel has received regenerative medicine advanced therapy (“RMAT”) designation for relapsed or refractory large B cell lymphoma (“r/r LBCL”), fast track designation for r/r B-NHL, and orphan drug designation for follicular lymphoma (“FL”) from the U.S.

Food and Drug Administration (“FDA”).

CB-011 has received fast track designation for r/r MM from the FDA;

and CB-012 has received fast track designation for r/r AML from the FDA.

We believe CB-011, if approved, will have several advantages over both commercially available bispecific antibody therapies and commercially available autologous CAR-T cell therapies, including a single-dose treatment regimen, potentially low rates of grade 3 or greater infections, and rapid immune recovery compared to bispecific antibody therapies, as well as broader patient access, shorter time to treatment with an off-the-shelf product, significantly lower manufacturing costs, and a substantially smaller footprint for manufacturing compared to autologous CAR-T cell therapies.

CB-011 has received fast track and orphan drug designations for r/r MM from the FDA.

On April 24, 2025, we announced a strategic pipeline prioritization with workforce and cost reduction initiatives to focus resources on our vispa-cel and CB-011 product candidates.

At that time, we disclosed that we had discontinued our GALLOP phase 1 trial of vispa-cel for the treatment of lupus prior to dosing the first patient;

our AMpLify phase 1 clinical trial of CB-012, an allogeneic anti-C-type lectin-like molecule-1 (“CLL-1”) CAR-T cell therapy for the treatment of relapsed or refractory acute myeloid leukemia (“r/r AML”), as additional data would be needed to advance this program;

and our preclinical research.

Patients treated in the AMpLify phase 1 clinical trial continue to be followed as part of our long-term follow-up study.

Additionally, we announced that our workforce was reduced by 47 employees, or approximately 32% of our workforce.

In connection with the discontinuation of the AMpLify phase 1 clinical trial for our CB-012 product candidate, we terminated our Exclusive License Agreement, dated November 13, 2020, with Memorial Sloan Kettering Cancer Center (“MSKCC”) (as amended, “MSKCC Agreement”), effective August 11, 2025.

Allogeneic CAR-T Cell Therapies

Allogeneic, or off-the-shelf, CAR-T cell therapies offer several advantages over commercially available autologous CAR-T cell therapies, including broad patient access, rapid patient treatment, and increased manufacturing scale:

Allogeneic, or off-the-shelf, CAR-T cell therapies offer several advantages over commercially available autologous CAR-T cell therapies, including broad patient access, shorter time to treatment and increased manufacturing scale with a substantially smaller footprint:

• Broad patient access:

Of every 10 patients with second-line relapsed or refractory diffuse large B cell lymphoma (“DLBCL”), only approximately two patients currently receive commercially available autologous CAR-T cell therapies.

Only 25% of second-line (“2L”) relapsed or refractory large B cell lymphoma (“LBCL”) patients currently receive commercially available autologous CAR-T cell therapies.

Many patients cannot wait weeks to months for autologous CAR-T cell therapy or their T cells are too dysfunctional to manufacture their individual therapy.

With allogeneic CAR-T cell therapy, many more patients could be served as these cell therapies are manufactured in advance from healthy donor T cells, making them readily available off-the-shelf for rapid patient treatment.

Additionally, commercially available autologous CAR-T cell therapies are primarily available at academic centers of excellence in metropolitan areas where a patient’s cells can be removed for modification through apheresis versus in the community hospital setting where the vast majority of patients are treated.

Allogeneic CAR-T cell therapies can be administered in academic centers of excellence and, in the future, in appropriate community hospital settings, regardless of apheresis capabilities, suggesting broader patient access to treatment.

Additionally, commercially available autologous CAR-T cell therapies are primarily available at academic centers of excellence in metropolitan areas, whereas most patients are cared for in the community hospital setting and not at academic centers.

Allogeneic CAR-T cell therapies can be administered in academic centers of excellence and in appropriate community hospital settings, suggesting broader patient access to treatment.

• Rapid patient treatment:

Commercially available autologous CAR-T cell therapies are manufactured from the patient’s own T cells and require weeks to months for manufacturing, product release, and delivery back to the patient for treatment.

Given the long wait time, more than 50% of patients who receive autologous CAR-T cells

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require bridging therapy, adding additional therapies to their treatment journey.

In addition, approximately 40% of patients with second line (“2L”) DLBCL are not able to wait for autologous CAR-T cell therapy.

With allogeneic CAR-T cell therapy, patients can begin treatment within days.

• Increased manufacturing scale:

For autologous CAR-T cell therapy, one manufacturing run yields one treatment for one patient, which cannot be further scaled.

There are a limited number of CAR-T cell treatment centers where apheresis capabilities are available, In addition, manufacturing failures can prevent a patient from receiving their treatment.

In contrast, our allogeneic CAR-T cell therapy product candidates are manufactured from cells from healthy donors, prepared in advance of treatment, and the number of doses per manufacturing run can be scaled for broad patient access.

Our CRISPR chRDNA Genome-Editing Technologies

Commercially available autologous CAR-T cell therapies are manufactured from the patient’s own T cells and require weeks to months for apheresis, manufacturing, product release, and delivery back to the patient for treatment.

Given the long wait time, more than 50% of patients who receive autologous CAR-T cells require bridging therapy, adding additional therapies to their treatment.

With allogeneic CAR-T cell therapy, patients can begin the treatment regimen the same day they become eligible.

• Increased manufacturing scale with smaller footprint:

For autologous CAR-T cell therapy, manufacturing requires multiple large plants to meet the needs of the patient population, and one manufacturing batch yields one treatment for one patient, which cannot be further scaled.

In addition, manufacturing failures can prevent a patient from receiving their treatment.

In contrast, our allogeneic CAR-T cell therapy product candidates are manufactured from cells from healthy donors, prepared in advance of treatment in a single suite at a contract manufacturing organization (“CMO”) at significantly lower cost of manufacturing than autologous CAR-T cell therapies.

These manufacturing attributes allow the number of doses per manufacturing batch to be scaled for broad patient access.

Our CRISPR chRDNA Genome-Editing Technology

Genome-editing technologies used to date have limited efficiency, specificity, and versatility for performing the multiple, precise genomic edits necessary for allogeneic CAR-T cell therapy manufacturing.

Our CRISPR chRDNA genome-editing technologies are designed to address these limitations and enable us to apply armoring strategies to enhance allogeneic CAR-T cell therapy activity against diseases.

Using our chRDNA genome-editing technologies, we have successfully demonstrated multiplex genome editing while maintaining genomic integrity.

Our CRISPR chRDNA genome-editing technology is designed to address these limitations and enables us to apply armoring strategies to enhance allogeneic CAR-T cell therapy activity against diseases.

Using our chRDNA genome-editing technology, we have successfully demonstrated multiplex genome editing while maintaining genomic integrity.

Our allogeneic CAR-T cell therapy product candidates incorporate increasing numbers of genome edits, as shown in the figure below.

We believe this level of editing sophistication has the potential to unlock the broad potential of allogeneic cell therapies by:

We believe this level of editing sophistication and editing fidelity can unlock the broad potential of allogeneic cell therapies by:

• Mitigating the risk of graft-versus-host disease (“GvHD”) :

Development of an allogeneic CAR-T cell therapy requires genome editing to remove proteins from donor T cells that may recognize and attack a patient’s tissue, which, without removal, would pose a risk of GvHD.

To mitigate the risk of GvHD, we engineer our allogeneic CAR-T cell therapies with a knockout of the T cell receptor alpha constant (“ TRAC ”) gene in order to eliminate expression of the T cell receptor (“TCR”) from the surface of the CAR-T cells.

• Enhancing the activity of allogeneic cell therapies for potentially durable activity :

Our chRDNA technologies enable us to apply tailored armoring strategies to our allogeneic CAR-T cells, including (i) checkpoint disruption, through the knockout of programmed cell death protein 1 (“PD-1”) to sustain the activity of CAR-T cells by disrupting a pathway that leads to CAR-T cell exhaustion;

(ii) immune cloaking of CAR-T cells to reduce rejection by the patient’s immune system;

and (iii) a combination of these two strategies.

Our chRDNA technology enables us to apply tailored armoring strategies to our allogeneic CAR-T cells, including (i) checkpoint disruption, through the knockout of programmed cell death protein 1 (“PD-1”) to enhance the activity of CAR-T cells by disrupting a pathway that leads to CAR-T cell exhaustion;

and (ii) immune cloaking, through removal of the endogenous beta-2 microglobulin (“B2M”) protein and insertion of a beta-2-microglobulin–human-leukocyte-antigen-E–(“B2M–HLA-E”) peptide fusion, to reduce rejection by the patient’s immune system.

• Improving the genomic integrity of our products:

We have observed that our cell therapy product candidates have significantly lower levels of off-target edits compared to those made using first-generation CRISPR-Cas9 genome editing, and we believe we make multiple edits while maintaining genomic integrity.

We have observed that our cell therapy product candidates have significantly lower levels of off-target edits compared to those made using first-generation CRISPR-Cas9 genome editing, and we believe we maintain genomic integrity, including during multiplex editing.

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We believe that our chRDNA genome-editing technologies have broad potential to generate cell and gene therapies in oncology, autoimmune diseases, and in additional therapeutic areas.

We believe that our chRDNA genome-editing technology has broad potential to generate ex vivo and in vivo cell and gene therapies.

We own a robust worldwide patent portfolio protecting our Cas9 chRDNA and our Cas12a chRDNA compositions and genome-editing methods.

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We are focused on the development of our pipeline of allogeneic CAR-T cell therapies.

We are advancing four clinical-stage programs from our allogeneic CAR-T cell therapy platform targeting the treatment of hematologic malignancies and autoimmune diseases.

We are advancing two clinical-stage allogeneic CAR-T cell therapies for the treatment of patients with hematologic malignancies.

Our pipeline is shown below:

RMAT designation for r/r LBCL

fast track designation for r/r B-NHL

orphan drug designation for follicular lymphoma (“FL”)

4 CB-010:fast track designation for refractory SLE

fast track and orphan drug designations for r/r MM

fast track and orphan drug designations for r/r AML

CB-010 is an anti-CD19 allogeneic CAR-T cell therapy product candidate that is being evaluated in patients with r/r B-NHL in our ANTLER phase 1 clinical trial and in patients with LN and ERL in our GALLOP phase 1 clinical trial.

To our knowledge, CB-010 is the first clinical-stage allogeneic anti-CD19 CAR-T cell therapy with a genome-edited knockout of the PDCD1 gene to prevent PD-1 expression on the CAR-T cell surface.

In the dose escalation portion of our ANTLER phase 1 clinical trial, 16 patients with multiple subtypes of aggressive r/r B-NHL were enrolled and three dose levels of CB-010 were evaluated:

dose level 1 (40x10 6 viable CAR-T cells), dose level 2 (80x10 6 viable CAR-T cells), and dose level 3 (120x10 6 viable CAR-T cells).

Patients received a lymphodepletion regimen that included two chemotherapy agents, cyclophosphamide and fludarabine, which are generally used for lymphodepletion prior to autologous CAR-T cell therapy.

To ensure optimal engraftment of the allogeneic CB-010 cells, we used a deeper regimen of these chemotherapeutic agents, i.e., cyclophosphamide at 60 mg/kg/day for two days and then fludarabine at 25 mg/m 2 /day for five days, than is used with the commercially available autologous CAR-T cell therapies.

Lymphodepletion reduces the number of the patient’s immune cells and creates an inflammatory state that is required for the infused CAR-T cells to effectively expand and persist for disease activity.

Following dose escalation, we began the dose expansion portion of our ANTLER trial and enrolled 30 patients with 2L LBCL to treat this larger patient population earlier in the course of their disease.

During these portions of our ANTLER trial, patients who had received prior CD19-targeted therapies were excluded.

During dose expansion, dose level 2 (80x10 6 viable CAR-T cells) was selected as the recommended phase 2 dose (“RP2D”) for CB-010.

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At a poster presentation during the 2024 American Society of Clinical Oncology (“ASCO”) Annual Meeting in June 2024, we presented safety, efficacy, and translational data for the first 46 patients evaluated in our ANTLER phase 1 clinical trial after a single dose of CB-010.

CB-010 was generally well-tolerated with adverse events as expected for anti-CD19 CAR-T cell therapies.

A retrospective analysis of all patient data demonstrated that patients who received a dose of CB-010 manufactured from a donor with at least four matching human leukocyte antigen (“HLA”) alleles (of 12 total alleles) with the patient (referred to as “partial HLA matching”) resulted in improved progression-free survival (“PFS’) compared to patients who received a single dose of CB-010 from a donor with fewer than four matching HLA alleles.

To confirm the partial HLA matching strategy, we are enrolling approximately 20 additional 2L LBCL patients in the ongoing ANTLER phase 1 clinical trial.

In addition, we are enrolling a proof-of-concept cohort of up to 10 patients in our ANTLER phase 1 clinical trial who have relapsed following any prior CD19-targeted therapy in this population of unmet need.

We have expanded our clinical development of CB-010 to include autoimmune diseases.

CB-010 holds the potential for deep depletion of disease-causing B cells, which could reset the immune system, leading to sustained drug-free remission.

In our ANTLER phase 1 clinical trial, following a single dose of CB-010, the depletion and recovery of patients’ B cells is on par with the duration of B cell aplasia reported in a recent case series in the literature.

We have initiated our multicenter, open-label GALLOP phase 1 clinical trial to evaluate a single infusion of CB-010 at the RP2D for oncology (80x10 6 viable CAR-T cells) but with a different lymphodepletion regimen, also incorporating partial HLA matching, in adult patients with LN and ERL.

CB-011 is an anti-BCMA allogeneic CAR-T cell therapy product candidate that is being evaluated in patients with r/r MM in our CaMMouflage phase 1 clinical trial.

To our knowledge, CB-011 is the first anti-BCMA CAR-T cell therapy incorporating an immune cloaking approach that includes both the removal of the endogenous beta-2 microglobulin (“B2M”) protein and insertion of a beta-2-microglobulin–human-leukocyte-antigen-E–peptide transgene (“B2M–HLA-E”).

This immune cloaking armoring strategy results in no endogenous class I HLA alleles expressed on the CAR-T cell surface.

This reduces the number of potential mismatched HLA alleles to six from 12, resulting in a reduced risk of rapid immunologic clearing of the CAR-T cells by the patient.

In the dose escalation portion of our CaMMouflage phase 1 clinical trial, dose level 1 (50x10 6 viable CAR-T cells), dose level 2 (150x10 6 viable CAR-T cells), dose level 3 (450x10 6 viable CAR-T cells), and dose level 4 (800x10 6 viable CAR-T cells) of CB-011 have cleared without any observed dose-limiting toxicities (“DLTs”).

We have implemented a deeper lymphodepletion regimen that includes an increased dose of cyclophosphamide (up from the original 300 mg/m 2 /day to 500 mg/m 2 /day together with the same fludarabine dose of 30 mg/m 2 /day for three days).

Dose level 3 (450x10 6 viable CAR-T cells) and dose level 4 (800x10 6 viable CAR-T cells) with the deeper lymphodepletion have cleared with no DLTs observed.

We are enrolling additional patients at multiple dose levels with the deeper lymphodepletion regimen in order to further define safety and efficacy and to determine a RP2D.

CB-012 is an allogeneic anti-CLL-1 CAR-T cell therapy product candidate that is being evaluated in patients with r/r AML in our AMpLify phase 1 clinical trial.

To our knowledge, CB-012 is the first allogeneic CAR-T cell therapy armored with both checkpoint disruption and immune cloaking strategies.

Patients in our AMpLify phase 1 clinical trial receive a lymphodepletion regimen prior to CAR-T cell infusion.

The lymphodepletion regimen includes two chemotherapy agents, cyclophosphamide (750 mg/m 2 /day) and fludarabine (30 mg/m 2 /day) for three days.

Patients then have two days of rest, followed by a single CB-012 dose on day zero.

In the dose escalation portion of our AMpLify phase 1 clinical trial, dose level 1 (25x10 6 viable CAR-T cells), dose level 2 (75x10 6 viable CAR-T cells), and dose level 3 (150x10 6 viable CAR-T cells) of CB-012 have cleared without any observed DLTs, and we are enrolling patients at dose level 4 (300x10 6 viable CAR-T cells).

In July 2024, we announced that we had discontinued preclinical research activities associated with our allogeneic CAR-natural killer (“NK”) cell therapy platform to focus resources on our allogeneic CAR-T cell therapy platform and to advance our four clinical programs for oncology and autoimmune diseases.

In addition to our CAR-T cell therapy programs, we are developing in vivo genome-editing capabilities.

Cas12a chRDNA guides may offer improved specificity compared to all-RNA guides for in vivo genome-editing applications.

We are using Cas12a chRDNA guides to optimize the Cas12a mRNA sequence and refine our lipid nanoparticle formulations with a goal of demonstrating functional gene disruptions in animal models.

Our initial research is in non-humanized mouse models to demonstrate genome-editing and gene correction capabilities.

In the future, we aim to take a human sequence target and perform disease-specific edits and demonstrate highly efficient editing across multiple clinically relevant targets to highlight the broad therapeutic potential of this platform.

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Our mission is to develop innovative, transformative therapies for patients with devastating diseases through our novel chRDNA genome-editing technologies.

Our overarching goal is to build an integrated company that discovers, develops, manufactures, and commercializes genome-edited cell therapies that have the potential to treat patients with significant unmet needs.

Our initial focus is on allogeneic CAR-T cell therapies for hematologic malignancies and autoimmune diseases, and our chRDNA technologies offer additional potential applications in the future.

Our mission is to develop innovative, transformative therapies for patients with devastating diseases through the use of our novel chRDNA genome-editing technology.

Our overarching goal is to build an integrated company that develops, manufactures, and commercializes genome-edited cell therapies that have the potential to treat patients with significant unmet needs.

Our current focus is on two clinical-stage allogeneic CAR-T cell therapies for treating patients with hematologic malignancies;

our chRDNA technology also has the potential for additional applications.

Key components of our strategy include:

• Applying our chRDNA genome-editing technology to engineer allogeneic cell therapies from our CAR-T cell platform that have the potential for durable activity against multiple diseases.

Our chRDNA technologies enable us to design allogeneic cell therapies with the potential to achieve enhanced activity against diseased cells through the use of armoring strategies, including (i) checkpoint disruption, such as through a knockout of PD-1 to sustain the activity of CAR-T cells by disrupting a pathway that leads to CAR-T cell exhaustion;

(ii) immune cloaking of CAR-T cells to reduce rejection by the lymphoid compartment of a patient’s immune system;

and (iii) a combination of these strategies.

• Applying our chRDNA genome-editing technology to engineer allogeneic CAR-T cell therapies that have the potential for durable activity against multiple diseases.

Our chRDNA technology enables us to develop allogeneic cell therapies with the potential to achieve enhanced activity against diseased cells through the use of armoring strategies, including (i) mitigation of the risk of GvHD through prevention of TCR expression;

(ii) checkpoint disruption through a knockout of PD-1 to enhance the activity of CAR-T cells by disrupting a pathway that leads to CAR-T cell exhaustion;

and (iii) immune cloaking through removal of the B2M protein and insertion of a B2M–HLA-E-peptide fusion to reduce rejection by the lymphoid compartment of a patient’s immune system.

• Developing allogeneic CAR-T cell therapies against clinically validated targets to derisk our clinical programs.

CB-010, directed to the CD19 antigen, is being evaluated in our ANTLER phase 1 clinical trial in patients with r/r B-NHL and our GALLOP phase 1 clinical trial in patients with LN and ERL.

Vispa-cel, directed to the CD19 antigen, has been evaluated in our ANTLER phase 1 clinical trial in patients with r/r B-NHL.

CB-011, directed to the BCMA antigen, is being evaluated in our CaMMouflage phase 1 clinical trial in patients with r/r MM.

These are the targets of several commercially available autologous CAR-T cell therapies, which reduces target risk as we evaluate the safety and efficacy of our allogeneic CAR-T cell therapies in our ongoing clinical trials.

• Developing allogeneic CAR-T cell therapies against additional targets for diseases with limited treatment options.

We are applying our Cas12a chRDNA platform and insights from our more advanced programs to design allogeneic CAR-T cell therapies against targets for which there are no commercially available autologous CAR-T cell therapies.

For example, CB-012, directed to the CLL-1 target, is being evaluated in our AMpLify phase 1 clinical trial in patients with r/r AML.

• Pursuing select applications of our technology and indications on our own and through strategic collaborations.

We believe that our technology has broad potential to generate cell therapies in oncology, autoimmune diseases, and additional therapeutic areas.

Potential applications include immune cell therapies and in vivo genome-editing therapies.

We may selectively pursue these indications and applications using our internal expertise or through strategic collaborations.

Our team and our culture are critical to our mission to develop innovative, transformative therapies for patients with devastating diseases through our novel CRISPR chRDNA genome-editing technologies.

Our mission-driven team includes leaders who have significant experience, including driving global clinical and regulatory strategies for commercially available autologous CAR-T cell therapies through all phases of development and pivotal trials, and some of the scientists who invented the chRDNA genome-editing technologies we use today in our programs and who continue to drive our future innovations.

CD19 and BCMA antigens are the targets of several commercially available autologous CAR-T cell therapies, which reduces target risk as we evaluate the safety and efficacy of our allogeneic CAR-T cell therapies in clinical trials.

• Pursuing select applications of our technology and indications on our own and through new strategic collaborations.

We believe that our technology has broad potential to generate cell and gene therapies in oncology, autoimmune diseases, and additional therapeutic areas.

Potential applications include in vivo and ex vivo therapies.

We may selectively pursue these indications and applications through new strategic collaborations.

Our team and our culture are critical to our mission to develop innovative, transformative therapies for patients with devastating diseases through the use of our novel CRISPR chRDNA genome-editing technology.

Our mission-driven team includes leaders who have significant experience in the development of cell and gene therapies, including driving global clinical and regulatory strategies for commercially available autologous CAR-T cell therapies through all phases of development.

We were founded in 2011 by globally-recognized pioneers in CRISPR genome editing and nucleic acid biology:

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Haurwitz, Ph.D., who has served as our president and chief executive officer since our formation.

Doudna and Jinek serve on our scientific advisory board (“SAB”), which also includes world experts in CAR-T cell development, immunotherapies, oncology, autoimmune diseases, patient care, and clinical trial development to support commercialization.

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Doudna and Jinek serve on our scientific advisory board (“SAB”), which also includes world experts in CAR-T cell development, immunotherapies, oncology, patient care, and clinical trial development to support commercialization.

Genome-Editing Background

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These insertions and deletions (“indels”) generally disrupt the coding sequence for the targeted gene, resulting in the knockout of the targeted sequence.

HDR, by contrast, is a more controlled repair system where the cell incorporates donor DNA delivered during the experiment into the DSB, resulting in the site-specific insertion of the provided DNA sequence.

HDR, by contrast, is a more controlled repair system where the cell incorporates donor DNA delivered during the editing process into the DSB, resulting in the site-specific insertion of the provided DNA sequence.

The canonical CRISPR system utilizes a CRISPR-associated (“Cas”) protein Cas9, which can cut genomic DNA.

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Additionally, the simultaneous occurrence of both on-target and off-target edits may lead to genomic rearrangements, including chromosomal translocations, that may be problematic for immune cell therapeutics, especially for ones requiring multiple edits.

Our CRISPR chRDNA Technologies

We deploy a new, next-generation CRISPR genome-editing platform, our novel chRDNA technologies, which use hybrid guides containing both RNA and DNA for editing genomic DNA to engineer our allogeneic CAR-T cell therapies.

Our CRISPR chRDNA Technology

We deploy a new, next-generation CRISPR genome-editing platform, our novel chRDNA technology, which uses hybrid guides containing both RNA and DNA for editing genomic DNA to engineer our allogeneic CAR-T cell therapies.

The presence of DNA in a chRDNA guide significantly improves editing specificity relative to an all-RNA guide.

The addition of DNA into the guide increases the fidelity by reducing the binding of the guide to the target sequence, thereby resulting in even less binding of the chRDNA guide to non-target sequences that may be similar to, but different from, the intended target sequence.

Our chRDNA technologies use the canonical Streptococcus pyogenes Cas9 protein or the Acidaminococcus sp .

Our chRDNA technology uses the canonical Streptococcus pyogenes Cas9 protein or the Acidaminococcus sp .

Cas12a protein and a guide that is composed of a mixture of RNA and DNA nucleotides in both the region that interacts with the chromosomal target DNA and in the region that does not interact with the target DNA.

Like Cas9, Cas12a is a Cas protein used to edit genomic DNA site-specifically.

The advantages of our chRDNA technologies include:

The advantages of our chRDNA technology include:

• Specificity:

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however, these guides have lower affinity for potential off-target sites to reduce the likelihood of a genome edit at an unintended location.

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evaluated the integrity and performance of chRDNA guides by employing two unique assays, the SITE-Seq® assay and the VINE assay, on two genes known from the scientific literature to exhibit high rates of off-target editing with either the Cas9 or Cas12a protein and all-RNA guides.

We evaluated the integrity and performance of chRDNA guides by employing two unique assays, the SITE-Seq® assay and the VINE assay, on two genes known from the scientific literature to exhibit high rates of off-target editing with either the Cas9 or Cas12a protein and all-RNA guides.

All-RNA guides generated both robust on-target and off-target editing.

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We developed chRDNA guides that target the exact same genomic locations that achieve equivalent on-target editing compared to the all-RNA guides.

We published research in Molecular Cell , a peer-reviewed journal, on the mitigation of off-target editing using Cas9 chRDNAs (Donohoue, P.D.

et al., Molecular Cell 81, 3637–3649, September 2, 2021).

• High efficiency:

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To insert genes into T cells with our chRDNA technology, we transduce the cells with adeno-associated virus serotype 6 (“AAV6”), which contains the DNA template of interest to facilitate the integration of the DNA into the double-stranded break generated by the Cas9 chRDNA complex or the Cas12a chRDNA complex via the HDR pathway.

Our chRDNA genome-editing technologies do not rely on lentiviral or retroviral genome-editing methods, which are used in commercially available autologous CAR-T cell therapies to insert the CAR gene randomly into the genome and which may increase the risk of genomic mutagenesis.

Our chRDNA genome-editing technology does not rely on lentiviral or retroviral genome-editing methods, which are used in commercially available autologous CAR-T cell therapies to insert the CAR gene randomly into the genome and which may increase the risk of genomic mutagenesis.

• Versatility :

Our chRDNA guides are compatible with, and offer utility across, multiple cell types, including but not limited to T cells, NK cells, and induced pluripotent stem cells (“iPSCs”).

Our chRDNA guides are compatible with, and offer utility across, multiple cell types, including T cells.

• Simplicity :

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CAR-T cells will generally proliferate in response to antigen engagement via the specificity of their respective CAR.

However, allogeneic CAR-T cells are rapidly rejected by a patient’s immune system due to their divergent donor-derived genetic profile and cell surface HLA presentation.

However, allogeneic CAR-T cells are rapidly rejected by a patient’s immune system due to their divergent donor-derived genetic profile and cell surface human leukocyte antigen (“HLA”) presentation.

We believe engineering CAR-T cells to achieve enhanced target cell killing activity is necessary for the realization of the full potential of allogeneic cell therapies.

Furthermore, development of an allogeneic CAR-T cell therapy requires genome editing to remove proteins from donor T cells that may recognize and attack a patient’s tissue that, without removal, would pose a risk of graft versus host disease (“GvHD”).

Checkpoint Disruption with PD-1 Knockout Strategy

Furthermore, development of an allogeneic CAR-T cell therapy requires genome editing to prevent TCR expression on donor T cells that would otherwise pose a risk of GvHD.

Checkpoint Disruption Strategy

One of the approaches we deploy to increase the activity of CAR-T cells is to remove PD-1 from the CAR-T cell surface.

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Diseased cells and the patient’s own cells can express PD-L1, leading to interaction with PD-1 and subsequent exhaustion of the CAR-T cells.

We use our chRDNA technology to knock out the PDCD1 gene to prevent PD-1 expression on the CAR-T cell surface, thereby disrupting PD-1/PD-L1-mediated exhaustion.

We believe that knocking out PD-1 will maintain the CAR-T cells in a higher activity state for an extended period of time, and we believe this will result in greater diseased cell debulking in the patient.

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We use our chRDNA technology to knock out the programmed cell death protein 1 (“ PDCD1”) gene to prevent PD-1 expression on the CAR-T cell surface, thereby disrupting PD-1/PD-L1-mediated exhaustion.

We believe that knocking out PD-1 results in greater activity against disease in the patient, thereby enabling a potentially better therapeutic index relative to PD-1-expressing CAR-T cells.

Immune-Cloaking Strategy

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Allogeneic CAR-T cells are foreign to the patient’s immune system and, unless modified, will be rapidly rejected.

We use our Cas12a chRDNA technology to make multiple edits to T cells to immune cloak them and limit the rapid rejection by both the patient’s cytotoxic T cells and NK cells.

We use our Cas12a chRDNA technology to make multiple edits to T cells to immune cloak them and limit the rapid rejection by both the patient’s cytotoxic T cells and natural killer (“NK”) cells.

Our edits remove all endogenous HLA class I antigens from the CAR-T cell surface and lead to the overexpression of HLA-E, a minor HLA antigen, on the CAR-T cell surface.

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Our Clinical Programs

We are advancing four clinical programs from our pipeline of allogeneic CAR-T cell therapies focused on the treatment of hematologic malignancies and autoimmune diseases.

Our current focus is on advancing two clinical-stage allogeneic CAR-T cell therapies for treating patients with hematologic malignancies.

Strategy and Rationale

CB-010 is an allogeneic CAR-T cell therapy targeting CD19-positive hematologic malignancies and autoimmune diseases.

CB-010 is being evaluated in our ongoing first-in-human, open-label, multicenter ANTLER phase 1 clinical trial (NCT04637763) in the United States, Australia, and Israel in adults with r/r B-NHL as well as in our open-label, multicenter GALLOP phase 1 clinical trial (NCT06752876) in the United States in adults with LN and ERL.

To our knowledge, our CB-010 product candidate is the first allogeneic CAR-T cell therapy in a clinical trial with a PD-1 knockout, and we believe the PD-1 knockout enhances the potential for durable activity of an allogeneic CAR-T cell therapy.

Our vispa-cel CAR-T cell therapy product candidate is an allogeneic CAR-T cell therapy targeting CD19-positive hematologic malignancies.

Vispa-cel has been evaluated in our first-in-human, open-label, multicenter ANTLER phase 1 clinical trial (NCT04637763) in the United States, Australia, and Israel in adults with r/r B-NHL.

To our knowledge, our vispa-cel product candidate is the first allogeneic CAR-T cell therapy in a clinical trial with a PD-1 knockout, and we believe the PD-1 knockout enhances the potential for durable activity of an allogeneic CAR-T cell therapy.

Other CAR-T cell therapies that express endogenous PD-1 could become rapidly exhausted and lose activity due to the interaction between PD-1 and its ligand PD-L1.

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B cell tumors and the patient’s own cells express PD-L1, leading to interaction with PD-1 and subsequent exhaustion of the CAR-T cells.

We prevent PD-1 expression on the CB-010 CAR-T cells, thereby disrupting PD-1/PD-L1-mediated exhaustion.

We prevent PD-1 expression on the vispa-cel CAR-T cells, thereby disrupting PD-1/PD-L1-mediated exhaustion.

More than half of B-NHL tumors express PD-L1, correlating with poorer outcomes.

We believe that knocking out PD-1 will maintain the CAR-T cells in an enhanced activity state for an extended period of time, which we believe will result in greater activity against disease in the patient, thereby enabling a potentially better therapeutic index relative to PD-1-expressing CAR-T cells.

CB-010 has received RMAT designation for r/r LBCL, fast track designations for r/r B-NHL and for refractory SLE, and orphan drug designation for FL from the FDA.

Target Indications

We are developing CB-010 for the treatment of r/r B-NHL, with a focus on 2L LBCL, as well as for the treatment of LN and ERL.

NHL is the most common hematologic malignancy with an estimated 80,620 individuals, or 4% of all cancers, diagnosed in the United States in 2024 according to the National Cancer Institute SEER database.

We believe that knocking out PD-1 results in greater activity against disease in the patient, thereby enabling a potentially better therapeutic index relative to PD-1-expressing CAR-T cells.

Vispa-cel has received RMAT designation for r/r large B cell lymphoma (“LBCL”), fast track designation for r/r B-NHL, and orphan drug designation for FL from the FDA.

Target Indication

We are developing vispa-cel for the treatment of r/r B-NHL, with a focus on 2L LBCL patients who are primary refractory patients or who have relapsed after receiving one prior line of therapy.

NHL is the most common hematologic malignancy with an estimated 80,350 individuals, or approximately 4% of all cancers, diagnosed in the United States in 2025 according to the National Cancer Institute SEER database.

LBCL is a subtype of B-NHL and typically presents as a rapidly growing mass or enlarging lymph nodes in a nodal or extranodal site.

LBCL subtypes include diffuse large B cell lymphoma not otherwise specified (“DLBCL NOS”), high-grade B cell lymphoma (“HGBL”), primary mediastinal large B cell lymphoma (“PMBCL”), transformed follicular lymphoma (“tFL”), and transformed marginal zone lymphoma (“tMZL”).

The most common form of lymphoma is DLBCL, a type of LBCL, with approximately 25,000 patients diagnosed each year in the United States.

In the United States in 2025, approximately 34,000 people with LBCL were treated with first-line therapies and approximately 12,000 patients received second-line treatments.

Overall, for aggressive r/r B-NHL, newer immunologically-mediated therapies under investigation include checkpoint inhibitors, bispecific antibodies, and CAR-T cells.

Commercially available autologous anti-CD19 CAR-T cell therapies have shown encouraging complete response (“CR”) rates, PFS, and overall survival;

however, there are many barriers, including limited patient access, length of time to treatment, and manufacturing capacity and scale limitations.

Commercially available autologous anti-CD19 CAR-T cell therapies have shown encouraging complete response (“CR”) rates, progression-free survival (“PFS”), and overall survival;

however, there are many barriers preventing most 2L LBCL patients from receiving autologous CAR-T cell therapy, including limited patient access, length of time to treatment, and manufacturing capacity and scale limitations.

Thus, there remains a significant unmet medical need for patients with r/r B-NHL.

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LN and ERL are sub-categories of SLE, the most common form of lupus.

Lupus is a chronic autoimmune disease characterized by B cell dysfunction in which the immune system attacks the patient’s own tissues, causing widespread inflammation and organ damage.

There are over 200,000 individuals with SLE in the United States according to the Centers for Disease Control and Prevention.

It has been estimated that about 50% of patients with SLE will develop lupus nephritis and, of those, roughly 10-30% of patients will progress to end-stage renal disease, which requires dialysis or kidney transplant.

Treatments for lupus include antimalarials, steroids, non-steroidal anti-inflammatories, immunosuppressives, blood thinners, and monoclonal antibodies (“mAbs”), among others.

Investigational approaches such as autologous CAR-T cell therapies are being evaluated for lupus;

however, issues related to scale and access, as well as the need for a washout of lupus drugs prior to apheresis and cell therapy infusion, may present challenges due to an extended time in which the SLE disease may flare while the patients are taken off medications.

ANTLER Phase 1 Clinical Trial for CB-010 in r/r B-NHL

We are evaluating CB-010 in our ANTLER phase 1 clinical trial for the treatment of adult patients with aggressive forms of r/r B-NHL.

In dose escalation, CB-010 was evaluated in patients with several aggressive subtypes of r/r B-NHL, the majority of which were third-line or later treatments.

In the ongoing dose expansion portion of ANTLER, CB-010 is being evaluated in 2L LBCL patients.

Patients in our ANTLER phase 1 clinical trial receive a lymphodepletion regimen prior to CAR-T cell infusion.

The lymphodepletion regimen includes two chemotherapy agents, cyclophosphamide and fludarabine, which are generally used for lymphodepletion prior to autologous CAR-T cell therapy.

To ensure optimal engraftment of the allogeneic CB-010 cells, we use a deeper regimen of these chemotherapeutic agents, i.e., cyclophosphamide at 60 mg/kg/day for two days and then fludarabine at 25 mg/m 2 /day for five days, than is used with the commercially available autologous CAR-T cell therapies.

The objective of our ongoing ANTLER phase 1 clinical trial is to further assess the safety and overall objective response rate (“ORR”) of CB-010 in r/r B-NHL patients at the RP2D and in patients who are receiving a partially HLA matched dose of CB-010.

Our ANTLER phase 1 clinical trial consists of two parts:

Part A was the dose escalation portion that followed a standard 3 + 3 design, with sequential, increasing single doses of CB-010, and was completed with 16 patients dosed at dose level 1 (40x10 6 viable CAR-T cells), dose level 2 (80x10 6 viable CAR-T cells), or dose level 3 (120x10 6 viable CAR-T cells).

Part B was the dose expansion portion where CB-010 was evaluated in larger numbers of patients to determine the RP2D in 2L LBCL patients.

ANTLER Phase 1 Clinical Trial for vispa-cel in r/r B-NHL

We evaluated vispa-cel in our ANTLER phase 1 clinical trial for the treatment of adult patients with aggressive forms of r/r B-NHL.

Our ANTLER phase 1 clinical trial consisted of two parts:

Part A was the dose escalation portion that followed a standard 3 + 3 design, with sequential, increasing single doses of vispa-cel, and was completed with 16 patients dosed at dose level 1 (40x10 6 viable CAR-T cells), dose level 2 (80x10 6 viable CAR-T cells), or dose level 3 (120x10 6 viable CAR-T cells).

Part B was the dose expansion portion where vispa-cel was evaluated in additional patients to determine the recommended phase 2 dose (“RP2D”) in 2L LBCL patients.

Dose level 2 (80x10 6 viable CAR-T cells) was selected as the RP2D.

In these portions of our ANTLER trial, patients who had received prior CD19-targeted therapy were excluded.

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1 Subtypes include:

DLBCL, HGBL, tFL PMBCL, MCL (mantle cell lymphoma), FL (follicular lymphoma, aggressively behaving with POD24 (high risk)), MZL (marginal zone lymphoma).

The ANTLER phase 1 clinical trial study design was as follows:

1 B-NHL subtypes include:

DLBCL, HGBL, tFL, PMBCL, FL (follicular lymphoma, with POD24 (high risk)), MCL, MZL

2 LBCL subtypes include:

DLBCL NOS, HGBL, transformed DLBLC from FL or MZL, and PMBCL.

3 Clin Cancer Res.

doi:10.1158/1078-0432.CCR-11-0116.

ANTLER Phase 1 Trial Clinical Data for CB-010 in r/r B-NHL

At a poster presentation during the ASCO annual meeting in June 2024, we presented safety, efficacy, and translational data for the first 46 patients evaluated in our ANTLER phase 1 clinical trial demonstrating safety and efficacy after a single dose of CB-010 with partial HLA matching.

A retrospective analysis of all patient data demonstrated that patients who received a dose of CB-010 manufactured from a donor with at least four HLA alleles matched resulted in improved PFS compared to patients who received a single dose of CB-010 from a donor with fewer than four HLA allele matched, as shown below.

DLBCL NOS (not otherwise specified), HGBL, transformed DLBCL from FL or MZL, and PMBC

diffuse large B cell lymphoma;

follicular lymphoma;

progression of disease within 24 months;

mantle cell lymphoma;

marginal zone lymphoma

On November 3, 2025, we announced results from our ANTLER phase 1 clinical trial evaluating vispa-cel in 2L LBCL patients, including the first data disclosure on 22 patients enrolled in the phase 1 confirmatory cohort to prospectively evaluate patients who received a dose of vispa-cel manufactured from a donor with at least four matching HLA alleles (of 12 total alleles) with the patient (“partial HLA matching”) as well as on additional LBCL patients from dose escalation and dose expansion.

As of September 2, 2025, the safety data cutoff date, our ANTLER clinical trial had enrolled 84 patients, including the confirmatory cohort of 22 2L LBCL patients designed to prospectively confirm the positive outcomes of partial HLA matching observed in earlier retrospective analyses.

Following a lymphodepletion (“LD”) regimen of 60 mg/kg/day of cyclophosphamide for two days and 25 mg/m 2 /day of fludarabine for five days, each patient in the confirmatory cohort received a dose of vispa-cel at the RP2D (80x10 6 viable CAR-T cells) such that the patient and the T cell donor shared at least four of 12 HLA alleles.

As of September 29, 2025, the efficacy data cutoff date, the results for the 22-patient confirmatory cohort were as follows:

• 82% (18/22) overall response rate (“ORR”)

• 64% (14/22) CR rate

• 51% 12-month PFS

Median follow-up time for the confirmatory cohort was 6.0 months.

The announced results included an analysis of the 35 CD19-naïve, LBCL patients enrolled our ANTLER phase 1 clinical trial who received a dose of vispa-cel that met the criteria for our optimized product profile, which criteria are a young T cell donor under the age of 30 and a minimum of two of 12 HLA alleles matched between the patient and the T cell donor.

Twenty of these 35 patients were enrolled in the confirmatory cohort, and the remaining 15 patients were enrolled in dose escalation or dose expansion.

Thirty-two of the 35 patients were 2L patients and three of the 35 patients were third-line or later (“3L+”) patients.

As of September 29, 2025, the efficacy data cutoff date, the results for the 35-patient optimized product cohort were as follows:

• 86% (30/35) ORR

• 63% (22/35) CR rate

• 53% 12-month PFS

Median follow-up time for the optimized cohort was 11.8 months.

The following is a summary of results for the confirmatory cohort and the optimized profile cohort for vispa-cel as of the September 29, 2025, efficacy data cutoff date:

1 2L LBCL 4+ HLA matched, dosed with 80x10 6 viable CAR-T cells

2 2L (N=32) and 3L+ (N=3) LBCL patients treated 40x10 6 viable CAR-T cells, 80x10 6 viable CAR-T cells, or 120x10 6 viable CAR-T cells, optimized for multiple factors, including 2+ HLA matched and young donor

3 Median follow up 6.0 months for confirmatory;

11.8 months for optimized

4 Median follow up 5.1 months for confirmatory;

7.9 months for optimized

duration of response;

not evaluable;

confidence interval

not estimable;

partial HLA matching:

patient has at least four HLA alleles that match

donor T cells used for CB-010 manufacturing.

* Retrospective analysis of HLA allele matching for class I and class II antigens.

ANTLER phase 1 clinical trial as of April 1, 2024, cutoff date;

data collection ongoing.

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At the 2024 ASCO annual meeting, translational research data on CB-010 were also presented, including pharmacokinetic (“PK”) and pharmacodynamic (“PD”) data.

PK data showed that higher numbers of matched HLA alleles between the CB-010 donor and recipient patient correlated with increased CAR-T cell expansion and persistence compared to lower numbers of matched HLA alleles.

PD data showed that a single dose of CB-010 resulted in extended B cell aplasia (~114 days) and a rapid recovery of the patient’s endogenous T and NK cells (approximately three weeks).

CB-010 was generally well tolerated with adverse events (“AEs”) as expected for anti-CD19 CAR-T cell therapies.

No grade 3+ cytokine release syndrome (“CRS”) and no GvHD cases were observed, and AEs of special interest are shown below.

immune effector cell–associated neurotoxicity syndrome;

not reported.

1 Prolonged cytopenias are defined as grade 3 or higher events lasting beyond 30 days following CB-010 infusion;

37/46 (80%) recovered from cytopenias to grade ≤2 by day 35 post CB-010 treatment.

2 Median time of onset was three days (range 0-22), and median duration was three days (range 1-19).

3 Infection events reported were on or after CB-010 infusion, with highest grade reported per patient;

median time of onset was eight days (range 0-279) and media duration was 14 days (range 1-239).

4 Median time of onset was 7.5 days (range 6-34), and median duration was two days (range 1-27).

5 Two grade 3 and one grade 4 ICANS;

all resolved with supportive care.

Median time of onset was eight days and median duration was two days.

ANTLER phase 1 clinical trial as of April 1, 2024, cutoff date;

data collection ongoing.

To confirm that the partial HLA matching strategy can improve outcomes in patients who receive a single dose of CB-010 from a donor with at least four HLA alleles matched, we are enrolling approximately 20 additional 2L LBCL patients in the ongoing ANTLER phase 1 clinical trial.

In addition, we are enrolling a proof-of-concept cohort of up to 10 patients who have relapsed following any prior CD19-targeted therapy in this population of unmet need.

GALLOP Phase 1 Clinical Trial for CB-010 in LN and ERL

We are evaluating CB-010 in our GALLOP Phase 1 trial for the treatment of adult patients with LN or ERL.

The objective of our GALLOP phase 1 clinical trial is to evaluate the safety, PK profile, and initial clinical activity of a single dose of CB-010 (80x10 6 viable CAR-T cells) following a lymphodepletion regimen of cyclophosphamide at 20mg/kg/day for two days and fludarabine at 25mg/m 2 /day for three days.

Patients are screened for donor-specific anti-HLA antibodies and administered CB-010 manufactured from a donor with partial HLA matching.

The primary endpoint is safety.

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lymphodepletion;

Systemic Lupus Erythematosus Disease Activity Index.

The results are further summarized in the following swimmer plot for the optimized profile cohort 1 :

1 2L (N=32) and 3L+ (N=3) LBCL patients treated with 40x10 6 viable CAR-T cells, 80x10 6 viable CAR-T cells, or 120x10 6 viable CAR-T cells, optimized for multiple factors, including 2+ HLA matching and young donor-derived

* Patient diagnosed with lung adenocarcinoma after day 28 scan revealed a non-responsive lung nodule and was enrolled in our long-term follow-up study.

As of the data cutoff date, patient was in continued response without additional anti-lymphoma therapy at one year post-vispa-cel treatment

Additional information:

• Long-term follow-up data reflect the last known response;

marked time points indicate confirmation of no disease progression

• One vispa-cel-related grade 5 IEC-HS occurred on day 25 post-infusion

• Median duration of complete response (“DoCR”) was not reached in this cohort

• Certain patients converted from CR or partial response (“PR”) to progressive disease (“PD”) at various assessment time points as indicated in the chart above

• Efficacy data cutoff date:

September 29, 2025

immune effector cell-associated hemophagocytic lymphohistiocytosis-like syndrome

Vispa-cel was generally well tolerated.

As of September 2, 2025, the safety data cutoff date, treatment emergent adverse events (“TEAEs”) at any grade in 25% or greater of all 84 patients who received vispa-cel were thrombocytopenia (62%), cytokine release syndrome (“CRS”) (55%), anemia (52%), neutropenia (39%), hypokalemia (26%), and leukopenia (26%).

In the confirmatory and optimized profile cohorts, there were no cases of GvHD or grade 3 or greater immune effector cell-associated neurotoxicity syndrome (“ICANS”), 1% of patients experienced grade 3 or greater CRS, and 28% (out of 80 patients) experienced prolonged cytopenias.

The following is a summary of notable adverse events for all three cohorts in the ANTLER phase 1 clinical trial as of the September 2, 2025, safety data cutoff date:

1 2L LBCL 4+ HLA matched, dosed with 80x10 6 viable CAR-T cells

2 2L (N=32) and 3L+ (N=3) LBCL patients treated with 40x10 6 viable CAR-T cells, 80x10 6 viable CAR-T cells, or 120x10 6 viable CAR-T cells, optimized for multiple factors, including 2+ HLA matching and young donor-derived

3 Prolonged cytopenias are defined as grade 3 or grade 4 neutropenia, thrombocytopenia, or anemia ongoing at day 28 (+/- 5 days) post-CAR-T cell infusion, based on laboratory data, distinct from investigator-reported clinical adverse events

4 Includes one vispa-cel-related grade 5 IEC-HS that occurred day 25 post-infusion

A cohort of 3L+ LBCL patients with prior exposure to CD19-targeted therapy enrolled five patients as of the September 2, 2025, safety data cutoff date.

Enrollment in this cohort has been paused to focus on CD19-naïve patients.

Planned Pivotal Phase 3 Clinical Trial in 2L r/r LBCL

Based on these data, we are planning to conduct a randomized, controlled pivotal phase 3 clinical trial of vispa-cel in approximately 250 2L LBCL CD19-naïve patients who are ineligible for autologous stem cell transplant and autologous CAR-T cell therapy.

We believe there is a meaningful commercial opportunity within this population, as approximately 60% of the 12,000 2L LBCL patients treated annually in the United States are considered ineligible for autologous stem cell transplant and autologous CAR-T cell therapy.

Patients randomized to the study arm would receive a single dose of vispa-cel at the RP2D of 80x10 6 viable CAR-T cells following lymphodepletion with 60 mg/kg/day of cyclophosphamide for two days and 25 mg/m 2 /day of fludarabine for five days.

Patients randomized to the comparator arm would be treated with the investigator’s choice of standard of care immunochemotherapy agents.

The primary endpoint would be PFS, and an interim analysis is planned.

Secondary endpoints would include ORR, CR rate, DoR, DoCR, overall survival, quality of life, and safety.

We are in ongoing engagement with the FDA regarding the design of our pivotal trial for vispa-cel;

however, we cannot provide any assurance that our proposed pivotal trial design will ultimately meet FDA requirements and/or recommendations or lead to regulatory approval.

Strategy and Rationale

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We acquired a novel humanized single-chain variable fragment (“scFv”) directed to BCMA that we use for the generation of the BCMA-specific CAR in CB-011.

To our knowledge, CB-011 is the first anti-BCMA CAR-T cell therapy incorporating an immune cloaking approach that includes both the removal of the endogenous B2M protein and insertion of a B2M–HLA-E peptide transgene.

To our knowledge, CB-011 is the first anti-BCMA CAR-T cell therapy incorporating an immune cloaking approach that includes both the removal of the B2M protein and insertion of a B2M–HLA-E-peptide fusion.

This immune cloaking armoring strategy results in no endogenous class I HLA alleles expressed on the CAR-T cell surface.

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We are developing CB-011 for the treatment of r/r MM.

In 2024, 1.8% of all cancers were MM in the United States according to the National Cancer Institute SEER database.

In 2025, 1.8% of all cancers in the United States were MM according to the National Cancer Institute SEER database.

The median age of diagnosis is 69 years, and there were an estimated 36,110 new cases in 2025 in the United States with an estimated 12,030 deaths in 2025.

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Commercially available anti-BCMA autologous CAR-T cell therapies have shown encouraging CR rates, PFS, and overall survival;

however, there are many barriers, including limited patient access, length of time to treatment, and manufacturing capacity and scale limitations.

Of every 10 patients with r/r MM, only approximately one patient currently receives an autologous CAR-T cell therapy.

however, there are many barriers limiting the number of patients who receive autologous CAR-T cell therapy, including restricted patient access, length of time to treatment, and manufacturing capacity and scale limitations.

Approximately 10% of patients with r/r MM receive an autologous CAR-T cell therapy.

Commercially available bispecific antibodies require patients to receive frequent treatments and are associated with high infection rates.

Additionally, many treatments for r/r MM are multidrug regimens comprising varying routes of administration and/or complicated dosing schedules;

Additionally, many treatments for r/r MM are multi-drug regimens comprising varying routes of administration and/or complicated dosing schedules;

these regimens can be complex and burdensome for both patients and physicians.

Due to limited patient access and treatment burden, there is a need for an off-the-shelf, readily available, single-dose treatment for patients with r/r MM.

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CaMMouflage Phase 1 Clinical Trial for CB-011 in r/r MM

We are evaluating CB-011 in our CaMMouflage phase 1 clinical trial in adult patients with r/r MM.

These patients have a documented diagnosis of active MM according to International Myeloma Working Group diagnostic criteria.

The patient population includes individuals for whom three or more lines of therapy, including a proteasome inhibitor (“PI”), an immunomodulatory drug (“IMiD”), and an anti-CD38 antibody, have failed.

Patients who have received a BCMA-targeted therapy within the last three months are excluded from the trial.

Patients in our CaMMouflage phase 1 clinical trial receive a lymphodepletion regimen prior to CAR-T cell infusion.

The lymphodepletion regimen includes two chemotherapy agents, cyclophosphamide and fludarabine, which are generally used for lymphodepletion prior to autologous CAR-T cell therapy.

We began the clinical trial utilizing 300 mg/m 2 /day of cyclophosphamide and 30 mg/m 2 /day of fludarabine, each for a total of 3 days.

We have recently implemented a lymphodepletion regimen that includes a deeper dose of cyclophosphamide (increased from 300 to 500 mg/m 2 /day) together with the same fludarabine dose.

The objective of the ongoing CaMMouflage phase 1 clinical trial is to assess safety, including the incidence of AEs defined as DLTs after CB-011 infusion, identify a maximum tolerated dose (“MTD”), if appropriate, assess the ORR at active dose levels, and identify the RP2D.

Our CaMMouflage phase 1 clinical trial is being conducted in two parts:

Part A is dose escalation following a standard 3 + 3 design, with sequential, increasing single doses of CB-011 with the ability to add additional patients at safe dose levels to further evaluate activity and safety.

Part B is the expansion portion where patients receive CB-011 at the dose level(s) determined in Part A to determine the RP2D.

In the dose escalation portion of our CaMMouflage phase 1 trial, dose level 1 (50x10 6 viable CAR-T cells), dose level 2 (150x10 6 viable CAR-T cells), and dose level 3 (450x10 6 viable CAR-T cells) with the initial lymphodepletion have cleared with no DLTs observed.

Dose level 3 (450x10 6 viable CAR-T cells) and dose level 4 (800x10 6 viable CAR-T cells) with the deeper lymphodepletion have cleared with no DLTs observed.

We are enrolling additional patients at multiple dose levels with the deeper lymphodepletion regimen in order to further define safety and efficacy and to determine a RP2D.

1 LD regimen:

recommended dose for expansion.

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Strategy and Rationale

CB-012 is an allogeneic CAR-T cell therapy targeting CLL-1 that is being evaluated in the ongoing first-in-human, open-label, multicenter AMpLify phase 1 clinical trial (NCT06128044) in the United States in adults with r/r AML.

We have exclusively licensed, in the field of allogeneic cell therapy, the fully human scFv targeting CLL-1 used in CB-012 from Memorial Sloan Kettering Cancer Center (“MSKCC”).

We believe CLL-1 is a compelling target for the treatment of AML due to its expression on myeloid cancer cells, its enrichment in leukemic stem cells, and its absence on hematopoietic stem cells (“HSCs”).

The absence of expression on HSCs indicates that these bone marrow cells will not be targeted by the CLL-1-directed CB-012 CAR-T cells, thereby preventing a patient from loss of a critical compartment of their immune system vital to generating immune cells required for fighting infections and cancer.

To our knowledge, CB-012 is the first allogeneic CAR-T cell therapy with both checkpoint disruption, through a PD-1 knockout, and immune cloaking, through a B2M knockout and B2M–HLA-E fusion protein insertion.

CB-012 has received fast track and orphan drug designations for r/r AML from the FDA.

Target Indication

AML is a cancer of the bone marrow currently treated with chemotherapy, radiation, targeted therapies, and/or HSC transplant.

There were an estimated 20,800 new cases of AML in the United States in 2024 according to the National Cancer Institute SEER database.

Five-year survival in these patients is 32%.

Intensive induction chemotherapy, known as 7 + 3, consisting of cytarabine and an anthracycline, is the most effective therapy for adults newly diagnosed with AML, although the treatment has significant associated toxicities.

There remains significant unmet need in the treatment of patients with AML.

AMpLify Phase 1 Clinical Trial for CB-012 in r/r AML

We are evaluating CB-012 in our AMpLify phase 1 clinical trial in adult patients with r/r AML.

Our AMpLify clinical trial includes patients who have not responded to or relapsed after standard treatment and excludes patients who have been treated with more than three prior lines of therapy and patients with proliferative disease.

Patients who received prior allogeneic stem cell transplant are allowed to participate in our AMpLify clinical trial.

Patients in our AMpLify phase 1 clinical trial receive a lymphodepletion regimen prior to CAR-T cell infusion.

The lymphodepletion regimen includes two chemotherapy agents, cyclophosphamide (750 mg/m 2 /day) and fludarabine (30 mg/m 2 /day) for three days.

Patients then have two days of rest, followed by a single CB-012 dose on day zero.

The objective of our ongoing AMpLify trial is to assess safety, including the incidence of AEs defined as DLTs after CB-012 infusion, identify an MTD, if appropriate, assess the overall ORR at active dose levels, and identify the RP2D.

Our AMpLify phase 1 clinical trial is an open-label study being conducted in two parts:

Part A is the dose escalation portion following a standard 3 + 3 design, with sequential, increasing single doses of CB-012.

Part B is the expansion portion where patients will receive CB-012 at the dose level(s) determined in Part A to determine the RP2D.

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In the dose escalation portion of our AMpLify trial, dose level 1 (25x10 6 viable CAR-T cells), dose level 2 (75x10 6 viable CAR-T cells), and dose level 3 (150x10 6 viable CAR-T cells) have cleared with no DLTs observed, and we are enrolling patients at dose level 4 (300x10 6 viable CAR-T cells).

stem cell transplant.

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CB-012 Preclinical Data

We evaluated CB-012 in preclinical mouse models, which demonstrated that CB-012 significantly reduced tumor burden and increased overall survival.

As shown in the left graph below, in a CLL-1+ AML xenograft model, a single dose of CB-012 significantly reduced an orthotopically established tumor burden over a long duration compared to vehicle, or negative control, treatment.

In a second model, we evaluated CLL-1-specific CB-012 CAR-T cells compared to equivalent CAR-T cells that lacked the PD-1 knockout in a xenograft model of CLL-1+ PD-L1+ tumor cells to evaluate the impact of the PD-1 knockout in CB-012.

As shown in the right graph below, the CLL-1-specific CB-012 CAR-T cells statistically significantly increased overall survival in the tumor-bearing mice compared to mice that received either control CAR-T cells expressing PD-1 (cells engineered with the same edits of CB-012 except the PDCD1 KO) or the vehicle control.

Part A was the dose escalation part following a standard 3 + 3 design, with sequential, increasing single doses of CB-011 with the ability to add additional patients at safe dose levels to further evaluate activity and safety.

Part B is the dose expansion portion where anti-BCMA treatment-naïve and anti-BCMA treatment-exposed r/r MM patients receive CB-011 at the recommended dose for expansion (“RDE”) determined in Part A.

The CaMMouflage phase 1 clinical trial study design is as follows:

1 300 mg/m 2 cyclophosphamide and 30 mg/m 2 fludarabine daily for three days

2 500 mg/m 2 cyclophosphamide and 30 mg/m 2 fludarabine daily for three days

immunomodulatory drug;

maximum tolerated dose;

proteasome inhibitor

On November 3, 2025, we announced results from the dose escalation portion of our ongoing CaMMouflage phase 1 trial evaluating CB-011 in r/r MM patients.

As of the data cutoff date of September 24, 2025, 48 patients were enrolled in the dose escalation portion of the trial, which is now completed.

The dose escalation portion enrolled patients who were fourth- or later-line (“4L+”) and all patients had received prior treatment with a proteasome inhibitor, an immunomodulatory drug, and an anti-CD38 antibody.

In dose escalation, prior treatment with an autologous CAR-T cell therapy was not permitted and no BCMA-targeted therapy was permitted within the prior three months.

We evaluated two different LD regimens and multiple CAR-T cell dose levels.

Thirty-five patients were treated with an LD regimen of 500 mg/m 2 cyclophosphamide and 30 mg/m 2 fludarabine daily for three days (the “selected LD regimen”).

A single dose of CB-011 preceded by the selected LD regimen resulted in responses at all dose levels evaluated (150x10 6 viable CAR-T cells, N=6;

300x10 6 viable CAR-T cells, N=13;

450x10 6 viable CAR-T cells, N=13;

and 800x10 6 viable CAR-T cells, N=3).

Also on November 3, 2025, we announced that the 450x10 6 viable CAR-T cell dose with the selected LD regimen is our RDE.

1 150x10 6 , 300x10 6 , 450x10 6 , or 800x10 6 viable CAR-T cells

2 MRD negative at ≤10 -5

3 MRD not evaluable in one patient

4 MRD available for three patients

minimal residual disease;

stringent complete response;

very good partial response

The results for the 12-patient, BCMA-naïve cohort treated with the selected LD regimen and the 450x10 6 viable CAR-T cell dose were as follows:

• 92% (11/12) ORR

• 75% (9/12) CR/sCR rate

• 91% (10/11) evaluable patients achieved MRD negativity (≤10 -5 )

• Seven of the 12 patients remain on study as of the data cutoff date in VGPR or better six months or longer following receipt of a single dose of CB-011

The median follow-up for patients dosed with the RDE was 8.3 months, and the longest responding patient in this cohort was, as of the data cut off date, in an sCR at 15 months post-infusion.

The results are further summarized in the following swimmer plot for the 12-patient, BCMA-naïve cohort treated with the selected LD regimen and the 450x10 6 viable CAR-T cell dose:

1 MRD negative at ≤10 -5

Additional information:

• One patient who had previously withdrawn from the trial died on day 90 of treatment-related ICAHT;

one patient died of pneumonia on day 50 (not treatment-related)

• Data shown are from BCMA-naïve patients dosed at the 450x10 6 viable CAR-T cell dose with the selected LD regimen

extramedullary disease;

immune effector cell-associated hematotoxicity;

CB-011 had a manageable safety profile across all dose levels and lymphodepletion regimens (N=48), with no cases of GvHD, immune effector cell-associated enterocolitis (“IEC-EC”), parkinsonism, or cranial nerve palsies.

TEAEs in 25% or greater of all patients treated with CB-011 following the selected LD regimen (N=35) were as follows:

neutropenia (80%), anemia (60%), thrombocytopenia (49%), infections (49%), dizziness (31%), cytokine release syndrome (31%), fatigue (31%), leukopenia (29%), decreased appetite (29%), constipation (26%), and pyrexia (26%).

Notable adverse events in the RDE cohort included one CB-011-related grade 5 ICAHT on day 90, one grade 5 pneumonia not related to CB-011 on day 50, and one grade 4 CB-011-related Guillain-Barré Syndrome on day 129, which is resolving.

In the cohort evaluating the 300x10 6 viable CAR-T cell dose level following the selected LD regimen, there was one grade 5 respiratory syncytial virus not related to CB-011 on day 73.

Prophylactic measures for cytopenias and infections and early intervention for IEC-HS have been successfully implemented in our clinical protocol.

The following is a summary of notable adverse events in the CaMMouflage phase 1 clinical trial as of the September 24, 2025 data cutoff date:

1 LD regimen of 500 mg/m 2 cyclophosphamide and 30 mg/m 2 fludarabine daily for three days

2 Any continued ≥ grade 3 cytopenia based on laboratory data at ≥ day 35;

denominator is those evaluable at day 35 (+/-5 days)

We initiated the dose expansion portion of the CaMMouflage phase 1 clinical trial in late 2025.

Strategic Agreements

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We granted certain registration rights to Pfizer under the Securities Purchase Agreement covering the resale of the shares.

Unless otherwise agreed by Pfizer, we have agreed to use the proceeds from the Pfizer Investment solely in connection with (i) the development program for our allogeneic anti-BCMA CAR-T cell therapy known as CB-011 that is being evaluated in our CaMMouflage clinical trial and/or (ii) any other single-targeted anti-BCMA CAR-T cell therapy using an anti-BCMA single-chain variable fragment owned or controlled by us (collectively, cell therapies described in clauses (i) and (ii) are referred to as a “BCMA Product Candidate”), for 36 months beginning on June 29, 2023.

On June 29, 2023, in connection with the Pfizer Investment, we and Pfizer also entered into an Information Rights Agreement, having a thirty-six (36)-month term.

Unless otherwise agreed by Pfizer, we have agreed to use the proceeds from the Pfizer Investment solely in connection with (i) the development program for our allogeneic anti-BCMA CAR-T cell therapy known as CB-011 that is being evaluated in our CaMMouflage clinical trial and/or (ii) any other single-targeted anti-BCMA CAR-T cell therapy using an anti-BCMA scFv owned or controlled by us (collectively, cell therapies described in clauses (i) and (ii) are referred to as a “BCMA Product Candidate”), for 36 months expiring on June 29, 2026.

On June 29, 2023, in connection with the Pfizer Investment, we and Pfizer also entered into an Information Rights Agreement, having a 36-month term and expiring on June 29, 2026.

Under the Information Rights Agreement, we granted Pfizer a 30-calendar day right of first negotiation (“ROFN”) if we commence or engage with any third party with respect to a potential grant of rights to develop and/or commercialize a BCMA Product Candidate, including, without limitation, a license agreement, a co-promotion/co-commercialization agreement, a profit share agreement, a joint venture agreement, or an asset sale agreement (a “Grant of Program Rights”).

If we and Pfizer do not reach an agreement with respect to a Grant of Program Rights within the 30-day period, then we may pursue negotiations and enter into an agreement with any third party.

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and such third party do not reach agreement on the Grant of Program Rights within a specified time period, Pfizer’s right of first negotiation will be reinstated.

If we and such third party do not reach agreement on the Grant of Program Rights within a specified time period, Pfizer’s right of first negotiation will be reinstated.

Under the Information Rights Agreement, we also agreed to grant Pfizer the right to designate one representative to serve on our SAB.

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Additionally, we agreed to provide Pfizer access to any preclinical or interim or final clinical data (including raw data) and results generated as part of the development program for a BCMA Product Candidate at the same time that we provide such data to a third party (other than to our service providers or the FDA or other regulatory authorities), subject to certain confidentiality exceptions.

Memorial Sloan Kettering Cancer Center

On November 13, 2020, we entered into an Exclusive License Agreement with MSKCC (as amended, “MSKCC Agreement”), under which we exclusively licensed from MSKCC know-how, biological materials, and related patent families to fully human scFvs targeting CLL-1 (also known as CD371) for use in T cells, NK cells, and genome-edited iPSCs for allogeneic CLL-1-targeted cell therapy.

We use one of the licensed scFvs in our CB-012 product candidate.

We paid an upfront payment of cash and shares of our common stock and will owe annual license maintenance fees until we have commercial sales.

For each licensed CLL-1 product, we will owe potential clinical, regulatory, and commercial milestone payments totaling up to $111.0 million and, if regulatory approval for a licensed CLL-1 product is received, we will owe low- to mid-single-digit percent royalties on net sales of licensed products.

Our license includes the right to sublicense through multiple tiers and we will owe MSKCC a percentage of upfront cash or equity received from our sublicensees.

The sublicensing percentage owed decreases as our licensed CLL-1 product candidates move through development, starting at a low-double-digit percentage if clinical trials have not yet begun and decreasing to a mid-single-digit percentage if the licensed CLL-1 product candidate is in later clinical trial stages.

We are also responsible for a percentage of the licensed patent costs.

The MSKCC Agreement includes certain diligence milestones that we must meet;

provided, however, that these may be extended by us upon payment of additional fees.

MSKCC is entitled to certain success payments if our stock value increases by certain multiples.

The potential payments are based on multiples of the fair value of our common stock compared with a split-adjusted initial stock price of $5.1914 per share, as subject to future adjustments for stock splits, during a specified time period described below.

Our common stock price will be determined by reference to the 45-trading day volume weighted-average trading price of our common stock immediately preceding the date of determination.

At our option, payments may be made in cash or common stock.

The relevant time period commences when the first patient is dosed with our first CLL-1 product candidate (CB-012) in the first phase 1 clinical trial and ends upon the earlier of the third anniversary of approval of our biologics license application (“BLA”) by the FDA or 10 years from the date the first patient was dosed with our first CLL-1 product candidate in the first phase 1 clinical trial.

Under the terms of the MSKCC Agreement, the aggregate success payments will not exceed $35.0 million.

Additionally, if we undergo a change of control during the relevant time period, a change of control payment may be owed, depending upon the increase in our stock price due to the change of control and also to what extent success payments have already been paid.

In no event will the combination of success payments and any change of control payment exceed $35.0 million.

We may terminate the MSKCC Agreement upon 90 calendar days’ prior written notice to MSKCC.

MSKCC may terminate the agreement in the event of our uncured material breach, bankruptcy, or criminal activity.

If MSKCC materially breaches the MSKCC Agreement in certain circumstances (for example, granting a third party a license in our field), then during the time of such uncured material breach, MSKCC will not be entitled to receive any success payments or any change of control payment.

ProMab Biotechnologies, Inc.

On January 31, 2020, we entered into a Sale and Assignment Agreement with ProMab (as amended, “ProMab Agreement”) under which we purchased a humanized scFv targeting BCMA and a patent family related thereto for an upfront cash payment of $0.4 million and the potential payments of future royalties to ProMab.

To date, five U.S.

To date, six U.S.

patents have granted (U.S.

and 12,435,152) in this patent family.

Our anti-BCMA CB-011 product candidate contains this BCMA scFv.

Under the terms of the ProMab Agreement, in the event that CB-011 is approved by the FDA, we will owe ProMab low-single-digit percent royalties on net sales until the expiration, abandonment, or invalidation of the last patent within the assigned patent family (i.e., 2040 for U.S.

patents, without patent term adjustment (“PTA”) or patent term extension (“PTE”)).

Our anti-BCMA CB-011 CAR-T cell therapy product candidate contains this BCMA scFv.

Such royalties may be reduced by no more than 50% if we must pay royalties to a third party for other intellectual property covering our product.

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If ProMab terminates the ProMab Agreement due to our uncured material breach or bankruptcy, we must cease the manufacture, use, and sale of any products or product candidates incorporating the purchased anti-BCMA scFv.

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Pioneer Hi-Bred International, Inc.

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As consideration for the assignment, we made an upfront payment of $0.5 million and are obligated to pay all patent prosecution and maintenance costs going forward;

up to $2.8 million in regulatory milestones for therapeutic products, up to $20.0 million in sales milestones over a total of four therapeutics products, and a percentage of sublicensing revenues received by us for licensing the chRDNA patent family.

up to $2.8 million in regulatory milestone payments and up to $20.0 million in sales milestones for any therapeutic products that are covered by the chRDNA patent family;

and a low-single-digit percentage of licensing revenue we receive for licensing the chRDNA patent family in the therapeutic field after December 2020.

The sublicensing agreements that we entered into prior to December 18, 2020 (for example, the Intellia Agreement discussed below) are not subject to these economics.

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An interim award from the arbitration panel in 2019 determined that both patent families are included in the Intellia Agreement, but the panel granted us an exclusive leaseback to Cas9 chRDNA guides under economic terms to be negotiated by the parties.

On June 16, 2021, we entered into a leaseback agreement with Intellia (“Leaseback Agreement”), which resolved the arbitration

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Pursuant to the Leaseback Agreement, in exchange for Intellia’s grant to us of an exclusive license to certain intellectual property relating to CRISPR-Cas9, including Cas9 chRDNAs, for use solely in the manufacture of our CB-010 product candidate, we paid Intellia an upfront cash payment of $1.0 million and we will owe up to $23.0 million in potential future regulatory and sales milestones.

Additionally, we will owe Intellia low- to mid- single-digit percent royalties on net sales of our CB-010 product candidate until the expiration, abandonment, or invalidation of the last patent within the intellectual property relating to CRISPR-Cas9, including that relating to Cas9 chRDNAs (i.e., 2036, without PTA or PTE).

On June 16, 2021, we entered into a leaseback agreement with Intellia (“Leaseback Agreement”), which resolved the arbitration proceeding.

Pursuant to the Leaseback Agreement, in exchange for Intellia’s grant to us of an exclusive license to certain intellectual property relating to CRISPR-Cas9, including Cas9 chRDNAs, for use solely in the manufacture of our vispa-cel product candidate, we paid Intellia an upfront cash payment of $1.0 million and we will owe up to $23.0 million in potential future regulatory and sales milestones.

Additionally, we will owe Intellia low- to mid- single-digit percent royalties on net sales of our vispa-cel product candidate until the expiration, abandonment, or invalidation of the last patent within the intellectual property relating to CRISPR-Cas9, including that relating to Cas9 chRDNAs (i.e., 2036, without PTA or PTE).

The Regents of the University of California (“UC”) and the University of Vienna (“Vienna”)

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Without PTA or PTE, the CVC IP will expire in 2033.

The UC/Vienna Agreement includes certain diligence milestones that we must meet.

For products and services sold by us that are covered by the CVC IP, we will owe low- to mid-single-digit percent royalties on net sales, subject to a minimum annual royalty.

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If we do not include intellectual property owned or controlled by us in such sublicense, we pay UC/Vienna 50% of sublicensing revenues received under the sublicense.

To date, we have entered into over 30 sublicensing agreements in a variety of fields such as human therapeutics, forestry, agriculture, research reagents, transgenic animals, certain livestock targets, internal research, bioproduction, cell lines, and microbial applications that include the CVC IP as well as other Cas9 intellectual property owned or controlled by us.

To date, we have entered into over 30 sublicensing agreements in a variety of fields such as human therapeutics, animal therapeutics, agriculture, forestry, research reagents, transgenic animals, certain livestock targets, internal research, bioproduction, cell lines, and microbial applications that include the CVC IP as well as other Cas9 intellectual property owned or controlled by us.

We are obligated to reimburse UC for its prosecution and maintenance costs of the CVC IP.

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Intellectual Property

We strive to protect and enhance the genome editing technologies that we believe are important to our business by seeking patents to cover our platform technologies.

We also rely on trade secrets to protect aspects of our business that are

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not amenable to, or that we do not consider appropriate for, patent protection.

Our success will depend significantly on our ability to obtain and maintain patent and trade secret protection for our technologies, our ability to defend and enforce our intellectual property rights, and our ability to operate without infringing any valid and enforceable intellectual property rights of third parties.

As of March 1, 2025, we owned 63 granted U.S.

We seek and obtain patent protection for our chRDNA genome-editing technology and product candidates.

We also rely on trade secrets to protect aspects of our business that are not amenable to, or that we do not consider appropriate for, patent protection.

Our success will depend significantly on our ability to obtain and maintain patent and trade secret protection for our chRDNA genome-editing technology and product candidates, our ability to defend and enforce our intellectual property rights, and our ability to operate without infringing any valid and enforceable intellectual property rights of third parties.

As of February 27, 2026, we owned 66 granted U.S.

patents, 254 granted foreign patents, and 147 pending patent applications throughout the world.

The patent portfolio owned by us includes U.S.

and foreign patents and patent applications covering methods and compositions relating generally to our Cas9 chRDNA and Cas12a chRDNA technologies (which, for granted U.S.

and foreign patents and patent applications covering methods and compositions relating generally to our Cas9 chRDNA and Cas12a chRDNA technology (which, for granted U.S.

patents, without PTA or PTE, will expire in 2036).

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In general, we file our patent applications in the United States and Europe as well as in numerous other foreign patent jurisdictions.

We have exclusively in-licensed intellectual property covering the anti-CLL-1 scFv of our CB-012 product candidate from MSKCC (which, upon grant, without PTA or PTE or other extensions, will expire in 2040).

Additionally, we have substantial patent protection on CRISPR Type I systems, CRISPR-Cas9 methods and compositions, and other genome-editing technologies.

Additionally, we have substantial patent protection on CRISPR-Cas9 methods and compositions, CRISPR Type I systems, and other genome-editing technologies.

The patent term in the United States and other countries is 20 years from the date of filing of the first non-provisional application to which priority is claimed.

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Additionally, under the Drug Price Competition and Patent Term Restoration Act of 1984 (“Hatch-Waxman Amendments”), the term of a patent that covers an FDA-approved biologic may also be eligible for a PTE of up to five years, which is designed to compensate for the patent term lost during clinical trials and the FDA regulatory review process.

A PTE cannot extend the remaining term of a patent beyond a total of 14 years from the date of product approval and only one patent claiming the drug product, methods of use, or methods of manufacturing may be restored.

A PTE cannot extend the remaining term of a patent beyond a total of 14 years from the date of product approval and only one patent claiming a drug or biologic, methods of use, or methods of manufacturing may be restored.

Moreover, a patent can only be restored once and, if a single patent is applicable to multiple products, it can only be extended based on one product.

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patents is in 2043.

As of March 1, 2025, we owned 25 trademark registrations worldwide, including 7 U.S.

trademark registrations, and 2 pending trademark applications worldwide.

We have registered “CARIBOU,” “CARIBOU BIOSCIENCES,” “SITE-SEQ,” and the Caribou logo as trademarks in relevant classes and jurisdictions in the United States, European Union, and certain other jurisdictions.

Furthermore, we rely upon trade secrets, know-how, continuing technological innovation and potential in-licensing opportunities to develop and maintain our competitive position.

As of February 27, 2026, we owned 27 trademark registrations worldwide, including 7 U.S.

trademark registrations.

We have registered “CARIBOU,” “CARIBOU BIOSCIENCES,” “SITE-SEQ,” and the Caribou logo as trademarks in relevant classes in the United States, European Union (“EU”), and certain other jurisdictions.

We also rely upon trade secrets, know-how, continuing technological innovation and potential in-licensing opportunities to develop and maintain our competitive position.

We seek to protect these trade secrets and other confidential information, in part, by entering into confidentiality agreements with parties who have access to them.

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We currently compete across the fields of cell therapy and genome editing.

We believe that our novel CRISPR-Cas12a chRDNA genome-editing platform has broad potential applicability across human therapeutic indications, and our strategy is to demonstrate our platform’s capability by first developing improved allogeneic cell therapies in hematologic oncology and autoimmune diseases.

We believe that our novel CRISPR-Cas12a chRDNA genome-editing technology has broad potential applicability across human therapeutic indications, including in vivo and ex vivo applications.

The biopharmaceutical industry, in particular the cell therapy and genome editing fields, is characterized by intense investment and competition aimed at rapidly advancing new technologies.

Our platform and therapeutic product candidates are expected to face substantial competition from multiple technologies, marketed products, and numerous other therapies being developed by other biopharmaceutical companies, larger and better funded pharmaceutical companies, academic research institutions, governmental agencies, and private research institutions.

Many of our competitors, either alone or with their collaborators, have substantially greater financial, technical, and other resources, such as larger research and development staff, and/or greater expertise in research and development, preclinical testing, conducting clinical trials, established manufacturing capabilities and facilities, and experienced marketing organizations with well-established sales forces.

Our vispa-cel and CB-011 product candidates will face substantial competition from multiple technologies, marketed products, and numerous other therapies being developed by other biopharmaceutical companies, larger and better funded pharmaceutical companies, academic research institutions, governmental agencies, and private research institutions.

Many of our competitors, either alone or with their collaborators, have substantially greater financial, technical, and other resources, such as larger research and development staff, and/or greater expertise in research and development, nonclinical testing, conducting clinical trials, established manufacturing capabilities and facilities, and experienced marketing organizations with well-established sales forces.

In addition, there is extensive patent infringement litigation in the biopharmaceutical industry and, in the future, we may bring or defend such litigation against our competitors.

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We believe that our CAR-T cell therapy product candidates have the potential to offer beneficial products to patients due to genome edits we make with our chRDNA technology with the goal of extending robust CAR-T cell activity in patients.

Because of the promising therapeutic effect of cell therapies, and the potential benefit of allogeneic treatment alternatives, we expect increasing competition from new and existing companies, which include, among others:

We believe that, if approved, our CAR-T cell therapy product candidates have the potential to offer beneficial products to patients due to genome edits we make with our chRDNA technology with the goal of extending robust CAR-T cell activity in patients.

Because of the promising therapeutic effect of cell therapies, including the potential benefit of allogeneic treatment alternatives, we expect increasing competition from companies such as:

• Autologous T cell therapy :

Autologous T cell therapies directed at CD19 have been commercialized by Novartis AG (Kymriah®), Kite Pharma, Inc., a Gilead Sciences, Inc.

company (Yescarta®, Tecartus®), and Bristol-Myers Squibb Company (Breyanzi®) and are witnessing increased adoption in the marketplace.

Autologous cell therapies directed at BCMA have been commercialized by 2seventy bio, Inc.

with their partner, Bristol-Myers Squibb Company, (Abecma®) and Legend Biotech Corporation with their partner, Janssen Biotech, Inc., a Johnson & Johnson company, (Carvykti®).

Both Abecma and Carvykti cell therapies have succeeded in pivotal trials in earlier lines of r/r MM and are expected to gain label extensions into this market.

Autologous T cell therapies are being developed by a number of additional companies, including but not limited to, 2seventy bio, Inc., Adaptimmune Therapeutics PLC, Alaunos Therapeutics, Inc., Arcellx, Inc., Arsenal Biosciences, Inc., Astellas Pharma Inc., Autolus Therapeutics plc, AvenCell Therapeutics, Inc., Bristol-Myers Squibb Company, Cabaletta Bio, Inc., CARGO Therapeutics, Inc., Eureka Therapeutics, Inc., Gracell Biotechnologies Inc., an AstraZeneca PLC company, Iovance Biotherapeutics, Inc., Janssen Biotech, Inc., Kite Pharma, Inc.

company (Yescarta®, Tecartus®), and Bristol-Myers Squibb Company (Breyanzi®).

Autologous cell therapies directed at BCMA have been commercialized by Bristol-Myers Squibb Company (Abecma®) and Legend Biotech Corporation with their partner Johnson & Johnson (Carvykti®).

Autologous T cell therapies are being developed by a number of additional companies, including but not limited to AbelZeta Pharma Inc., Arcellx, Inc., Arsenal Biosciences, Inc., Astellas Pharma Inc., Autolus Therapeutics plc, AvenCell Therapeutics, Inc., Bristol-Myers Squibb Company, Cabaletta Bio, Inc., Eureka Therapeutics, Inc., Gracell Biotechnologies Inc., an AstraZeneca PLC company, Iovance Biotherapeutics, Inc., Johnson & Johnson, Kite Pharma, Inc.

(a Gilead Sciences Inc.

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research pipeline), F.

Hoffman-La Roche Ltd (through its acquisition of Poseida Therapeutics, Inc.), TCR² Therapeutics Inc., Triumvira Immunologics Inc., TScan Therapeutics, Inc., and Vor Biopharma Inc.;

• In vivo T cell therapy :

Companies such as Abintus Bio, Inc., Capstan Therapeutics, Inc., GigaMune, Inc., Interius BioTherapeutics, Inc., Kelonia Therapeutics, Inc., Myeloid Therapeutics, Inc., and Umoja Biopharma, Inc.

are developing in vivo T cell therapies;

Hoffman-La Roche Ltd (through its acquisition of Poseida Therapeutics, Inc.), TCR² Therapeutics Inc., Triumvira Immunologics Inc., and TScan Therapeutics, Inc.;

• Allogeneic T cell therapy:

Other companies are developing allogeneic T-cell therapies, including Allogene Therapeutics, Inc., Atara Biotherapeutics, Inc., AvenCell Therapuetics, Inc., Cellectis S.A., Celyad Oncology SA, CRISPR Therapeutics AG, Fate Therapeutics, Inc., Gracell Biotechnologies (an AstraZeneca PLC company), Imugene Limited, Kite Pharma, Inc.

Other companies are developing allogeneic T cell therapies, including Allogene Therapeutics, Inc., Atara Biotherapeutics, Inc., AvenCell Therapuetics, Inc., Cellectis S.A., CRISPR Therapeutics AG, Fate Therapeutics, Inc., Gracell Biotechnologies (an AstraZeneca PLC company), Imugene Limited, Kite Pharma, Inc.

(a Gilead Sciences, Inc.

company), Legend Biotech Corporation, March Biosciences, Inc., F.

Hoffman La-Roche Ltd (through its acquisition of Poseida Therapeutics, Inc.), Sana Biotechnology, Inc., and Vor Biopharma Inc.;

Hoffman La-Roche Ltd (through its acquisition of Poseida Therapeutics, Inc.), and Sana Biotechnology, Inc.;

• In vivo T cell therapy :

Companies such as AbbVie Inc.

(through its acquisition of Capstan Therapeutics, Inc.), Bristol-Myers Squibb Company (through its acquisition of Orbital Therapeutics), CREATE Medicines, Inc., GigaMune, Inc., Kite Pharma, Inc.

(a Gilead Sciences Inc.

company) (through its acquisition of Interius BioTherapeutics, Inc.), Kelonia Therapeutics, Inc., Legend Biotech Corporation, and Umoja Biopharma, Inc.

are developing in vivo T cell therapies;

• Allogeneic NK cell therapy:

Companies are developing allogeneic NK cell therapies, including Artiva Biotherapeutics, Inc., Celularity Inc., Century Therapeutics, Inc., Fate Therapeutics, Inc., ImmunityBio, Inc., Nkarta, Inc., NKGen Biotech, Inc., Oncternal Therapeutics, Inc., Senti Biosciences, Inc., and Takeda Pharmaceutical Company Limited;

• Other cell therapies:

Other companies are developing CAR-expressing immune cell therapies derived from macrophages, including Carisma Therapeutics Inc.;

from regulatory T cells, including Kyverna Therapeutics, Inc.;

and from gamma-delta T cells, including Adicet Bio, Inc., CytoMed Therapeutics Limited, IN8bio, Inc., TC BioPharm (Holdings) PLC, and Takeda Pharmaceutical Company Limited;

• Other oncology therapeutics:

Multiple biotechnology and pharmaceutical companies are developing other directly competitive technologies, such as small molecule, antibody, bi-specific antibody, and antibody-drug conjugates;

• Non-oncology therapeutics :

Several companies are also exploring the use of CAR-T cell therapies for the treatment of autoimmune diseases, often including against the same targets as in the oncology field (e.g., CD19, BCMA).

Such autoimmune diseases include LN, SLE, pemphigus vulgaris, myasthenia gravis, and multiple sclerosis.

These companies include BRL Medicine Inc., Fate Therapeutics, Inc., Kite Pharma, Inc.

(a Gilead Sciences, Inc.

company) Kyverna Therapeutics, Inc., Luminary Therapeutics, Inc., Nkarta, Inc., and Sana Biotechnology, Inc.

in allogeneic cell therapies;

and Atara Biotherapeutics, Inc., Autolus Therapeutics plc, Bristol-Myers Squibb Company, Cabaletta Bio, Inc., Cartesian Therapeutics, Inc., Century Therapeutics, Inc., iCell Gene Therapeutics Inc., JW (Cayman) Therapeutics Co.

Ltd, Kyverna Therapeutics, Inc., Lyell Immunopharma, Inc., and Novartis AG in autologous cell therapies.

We also face competition from non-cell-based treatments offered by companies such as Amgen Inc., AstraZeneca PLC, Bristol-Myers Squibb Company, F.

Hoffman-La Roche Ltd, GlaxoSmithKline Capital plc, Merck & Co., Inc., and Pfizer Inc.

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Compared to first-generation genome-editing approaches, our chRDNA platform has shown improved specificity, a reduction in off-target edits and translocations and, when paired with Cas12a, an advanced capability to perform multiplexed edits, in particular multiplexed insertions.

Although we believe that our scientific expertise, novel technologies, and intellectual property position offer competitive advantages, we face competition from multiple other genome-editing technologies and companies.

Other companies developing CRISPR-based technologies include, among others, Arbor Biotechnologies, Inc., Beam Therapeutics Inc., CRISPR Therapeutics AG, Editas Medicine, Inc., Intellia Therapeutics, Inc., Mammoth Biosciences, Inc., Metagenomi, Inc., and Scribe Therapeutics, Inc.

Other companies are developing CAR-expressing immune cell therapies derived from regulatory T cells, including Cabaletta Bio and Kyverna Therapeutics, Inc., and from gamma-delta T cells, including Adicet Bio, Inc., CytoMed Therapeutics Limited, and IN8bio, Inc.

Additionally, multiple biotechnology and pharmaceutical companies are developing other directly competitive technologies, such as small molecules, antibodies, bispecific antibodies, trispecific antibodies, and antibody-drug conjugates.

Bispecific antibodies have received regulatory approval and are also in development for different lines of therapy for patients with relapsed or refractory DLBCL, including those from AbbVie Inc.

(Epkinly®), F.

Hoffman-La Roche Ltd (Columnvi® and Lunsumio®) and for different lines of therapy for patients with r/r MM, including those from Johnson & Johnson (Tecvayli® and Talvey®), Pfizer (Elrexfio®), and Regeneron Pharmaceuticals, Inc.

(Lynozyfic®).

Compared to first-generation CRISPR genome-editing approaches, our chRDNA genome-editing technology has shown improved specificity, a reduction in off-target edits and translocations and, when paired with Cas12a, an advanced capability to perform multiplexed edits, in particular multiplexed insertions.

Although we believe that our novel technologies and intellectual property position offer competitive advantages, we face competition from multiple other genome-editing technologies and companies.

Companies developing other genome-editing technologies include, among others, Allogene Therapeutics, Inc., Cellectis S.A., Precision BioSciences, Inc., Prime Medicine, Inc., Sangamo Therapeutics, Inc., and Wave Life Sciences Ltd.

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Manufacturing CAR-T cell therapies requires multiple components.

Allogeneic CAR-T cell therapies are manufactured with cells from healthy donors and clinical product candidates are prepared, qualified, and released in advance.

After manufacture, allogeneic CAR-T therapies are cryogenically stored in freezers and are readily available for patient treatment.

To date, we have successfully scaled our manufacturing processes so that one manufacturing run from a healthy donor can produce sufficient cell yield for approximately 150 doses of CB-010.This is in contrast to commercially available autologous CAR-T cell therapy where one manufacturing run is required for each patient to be treated using the patient’s cells that may have already been depleted by prior treatments and this process can take several weeks to months to deliver product for patient treatment.

For our CAR-T product candidates, we have optimized the manufacturing process that we developed in-house and have transferred the manufacturing to CMOs that manufacture current good manufacturing practices (“cGMP”)-grade material for our clinical trials.

Additionally, we have developed different analytical methods to understand the integrity and potency of our cells based upon our manufacturing process.

We have made a significant investment in process development to control our product candidate characteristics and to also improve our supply chain capabilities.

For the manufacturing of our allogeneic CAR-T cell therapy product candidates, we have developed a platform process that is scaled to eventually support commercially manufacturing.

Our allogeneic CAR-T cell therapies are manufactured starting with cells from healthy donors, and clinical product candidates are prepared, qualified, released, and available on an off-the-shelf basis prior to patient dosing.

Our allogeneic CAR-T therapies are cryogenically stored in freezers, making them readily available for patient treatment on demand.

To date, we have successfully scaled our manufacturing processes so that one manufacturing run from a healthy donor can produce sufficient cell yield for approximately 200-300 doses of vispa-cel and 50-100 doses for CB-011.This is in contrast to commercially available autologous CAR-T cell therapy where one manufacturing run is required for each patient to be treated using the patient’s cells that may have already been depleted by prior treatments, a process that can take several weeks to months to deliver product for patient treatment.

For our CAR-T product candidates, we have optimized the manufacturing process that we developed in-house and have transferred these processes to CMOs that manufacture current good manufacturing practices (“cGMP”)-grade material for our clinical trials.

Additionally, we have developed analytical methods to characterize and test the safety, purity, and potency of our cells based upon our manufacturing processes and regulatory requirements.

We have made a significant investment in process development to optimize and control our product candidate characteristics and to also improve our supply capabilities.

For the manufacturing of our allogeneic CAR-T cell therapy product candidates, we have developed a platform process that is scaled to support commercial manufacturing.

Our process development and manufacturing core competencies and advantages include:

• optimization and learnings across all of our product candidates and preclinical research programs, allowing for consistency and increased process robustness;

• optimization and learnings across all of our product candidates, allowing for consistency and increased process robustness;

• internal process development to facilitate optimization of manufacturing processes and technical transfers to manufacturing sites;

• readily available and established equipment that further enables the transfer from our process development lab to cGMP operations;

• readily available and commercially-established equipment that further enables the transfer from our process development lab to cGMP operations;

• custom engineering and development to create the necessary specific requirements for our product candidates, while leveraging the broader platform to ensure robust processes;

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• process understanding and cell manufacturing control for continuous optimization of productivity and product candidate quality;

• product candidate optimization from translational learnings across one of the largest allogeneic clinical efficacy and safety data sets;

• closed manufacturing systems;

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• process control and optimization, allowing for increased retention of early memory T cell phenotypes;

• platform scale and efficiency to accommodate high dose yield per batch, with optimization for further commercial supply processes.

The contract manufacturing organizations (“CMOs”) that are manufacturing the phase 1 clinical supplies of our product candidates are located in the United States and are subject to cGMP requirements.

We have dedicated cGMP suites

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at our CMOs for the manufacture of our cell therapy product candidates.

• platform scale and efficiency to accommodate commercial high yield per batch, with further yield optimization processes available.

The CMOs that are currently manufacturing the clinical supplies of our product candidates are located in the United States and are subject to cGMP requirements.

We have dedicated cGMP suites at our CMOs for the manufacture of our cell therapy product candidates.

We use multiple CMOs to individually manufacture the critical and starting materials for our product candidates, including cGMP chRDNA guides, Cas9 and Cas12a proteins, and AAV6 vectors used in the manufacture of our CAR-T cells.

Our chRDNA genome-editing is a next-generation CRISPR technology that does not rely on lentiviral or retroviral genome-editing methods, which are used in commercially available autologous CAR-T cell therapies to insert the CAR gene randomly into the genome, which may increase the risk of genomic mutagenesis.

We expect to rely on our CMOs for manufacturing our product candidates to expedite readiness for future clinical trials, and most of these CMOs have demonstrated capabilities for commercial manufacturing.

We expect to rely on CMOs for manufacturing our product candidates to expedite readiness for future clinical trials, and most of the CMOs we currently contract with have demonstrated capabilities for commercial manufacturing.

Additionally, we may decide to build our own manufacturing facility in the future, or we may deploy a hybrid approach to manufacturing, to provide us with greater flexibility and control over our clinical or commercial manufacturing needs.

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Relevant regulatory authorities include, but are not limited to, the FDA;

the European Medicines Agency (“EMA”), an agency of the European Union (“EU”) in charge of the evaluation and supervision of medicinal products;

the European Medicines Agency (“EMA”), an agency of the EU in charge of the evaluation and supervision of medicinal products;

the European Commission, which is the executive arm of the EU;

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In the United States, our product candidates are regulated as biological products, or biologics, under the Public Health Service Act (“PHSA”), and the Federal Food, Drug, and Cosmetic Act (“FDCA”), and their implementing regulations promulgated by the FDA.

Failure to comply with the applicable requirements at any time during the product development process, including nonclinical testing, clinical testing, the approval process, or post-approval process, may subject us to delays in the conduct of a clinical trial, regulatory review and approval, and/or subject us to administrative or judicial sanctions.

Failure to comply with the applicable requirements at any time during the product development process, including nonclinical testing, clinical testing, the approval process, or post-approval process, may subject us to delays in the conduct of a clinical trial, delays in regulatory review and approval, and/or administrative or judicial sanctions.

Such sanctions may include, but are not limited to, the FDA’s refusal to allow us to proceed with clinical testing of our product candidates, refusal to approve pending applications, license suspension or revocation, withdrawal of an approval, receipt of untitled or warning letters, adverse publicity, product recalls, product seizures, total or partial suspension of production or distribution, injunctions, fines, and civil or criminal investigations and penalties brought by the FDA, U.S.

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• approval by an independent institutional review board (“IRB”), representing each clinical trial site before each clinical trial may be initiated, or by a central IRB if appropriate;

• performance of adequate and well-controlled human clinical trials required to establish the safety and efficacy of the product candidate for each proposed indication, in accordance with the FDA’s current Good Clinical Practice (“cGCP”) regulations including, but not limited to, informed consent and investigator disclosure requirements;

• preparation and submission to the FDA of a BLA seeking approval of our product candidates for one or more proposed indications, including submission of detailed information on the manufacture and composition of our product candidates and proposed labeling;

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• preparation and submission to the FDA of a biologics license application (“BLA”) seeking approval of our product candidates for one or more proposed indications, including submission of detailed information on the manufacture and composition of our product candidates and proposed labeling;

• review of the BLA by an FDA advisory committee, where applicable;

• satisfactory completion of one or more FDA inspections of the manufacturing facility or facilities, including those of any third-party manufacturers, at which the product, or components thereof, are produced in order to assess compliance with cGMP requirements and to ensure that the facilities, methods, and controls are adequate to preserve and ensure the product’s safety, purity, and potency, and, if applicable, the FDA’s current Good Tissue Practice (“cGTP”), for the use of human cell and tissue products;

• satisfactory completion of any FDA audits of the nonclinical study and clinical trial sites to ensure compliance with cGLPs and cGCPs, respectively, and the integrity of nonclinical and clinical data in support of the BLA;

• payment of user fees and securing FDA approval of the BLA;

• payment of any user fees and securing FDA approval of a BLA;

• compliance with any post-approval requirements, including the potential requirement to implement Risk Evaluation and Mitigation Strategy (“REMS”), adverse event reporting, and compliance with any post-approval studies required or requested by the FDA.

Nonclinical Studies and Investigational New Drug Applications

Before testing any investigational biologic product candidate in humans, we must submit an IND application and receive clearance from the FDA to initiate a clinical trial.

Nonclinical Studies and IND Applications

Before testing any investigational biologic candidate in humans, we must submit an IND application and receive clearance from the FDA to initiate a clinical trial.

The results of our nonclinical testing, including laboratory evaluation of product chemistry, formulation and toxicity, as well as animal trials to assess the characteristics and potential safety and efficacy of the product, together with manufacturing information, are submitted to the FDA as part of the IND application.

An IND is an exemption from the restrictions of the FDCA, which would otherwise preclude an unapproved biologic product candidate from being shipped in interstate commerce.

Under a cleared IND, the unapproved biologic product candidate may be shipped in interstate commerce for use in an investigational clinical trial, provided that the product candidate meets certain quality and labeling requirements.

An IND is an exemption from the restrictions of the FDCA, which would otherwise preclude an unapproved biologic candidate from being shipped in interstate commerce.

Under a cleared IND, the unapproved biologic candidate may be shipped in interstate commerce for use in an investigational clinical trial, provided that the product candidate meets certain quality and labeling requirements.

An IND automatically becomes effective 30 calendar days after receipt by the FDA, unless before that time the FDA places the trial on clinical hold.

In such a case, the IND sponsor must correct the deficiencies cited in the hold letter or otherwise satisfy the FDA that the investigation may proceed before the clinical trial can begin.

When the sponsor submits a response to the issues identified in the hold letter, the FDA must respond in writing to the sponsor within 30 days of the complete response by either removing or maintaining the clinical hold.

When the sponsor submits a response to the issues identified in the hold letter, the FDA should respond in writing to the sponsor within 30 days of the complete response by either removing or maintaining the clinical hold.

The FDA may impose a partial or full clinical hold with respect to our product candidate.

In certain cases, we may not be able to proceed at all with our proposed clinical trial.

A partial clinical hold is a delay or suspension of fewer than all of the investigational clinical trial, or certain parts of the investigational clinical trial, subject to the IND.

Human Clinical Trials in Support of a BLA

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A protocol for each clinical trial and subsequent protocol amendments must be submitted to the FDA as part of the IND.

If we wish to conduct a clinical trial outside of the United States, we may, but need not, obtain FDA authorization to conduct the clinical trial under an IND application.

When a foreign clinical trial is conducted under a foreign equivalent to an IND application, FDA IND applications requirements must be met unless waived.

If we wish to conduct a clinical trial outside of the United States, we may, but need not, obtain FDA authorization to conduct the clinical trial under an IND.

When a foreign clinical trial is conducted under an IND, FDA IND requirements must be met unless waived.

If a non-U.S.

clinical trial is not conducted under an FDA IND application, we may submit data from a well-designed and well-conducted clinical trial to the FDA in support of our BLA as long as the clinical trial is conducted in compliance with cGCP and the FDA is able to accept the data from the clinical trial and/or through an onsite inspection if the FDA deems it necessary.

clinical trial is not conducted under an FDA IND, we may submit data from a well-designed and well-conducted clinical trial to the FDA in support of our BLA as long as the clinical trial is conducted in compliance with cGCP and the FDA is able to validate the data from the clinical trial through an onsite inspection if the FDA deems it necessary.

For clinical trials conducted in the United States, each clinical trial must be reviewed and approved by an IRB, either centrally or individually at each institution at which our clinical trials will be conducted.

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The IRB must operate in compliance with FDA regulations governing IRBs.

The FDA, the applicable IRB, or we may suspend or terminate a clinical trial at any time for various reasons, including a finding that the clinical trial is not being conducted in accordance with FDA requirements or that the subjects or patients are being exposed to an unacceptable health risk.

The FDA, the applicable IRB, or we may suspend or terminate a clinical trial, in whole or in part, at any time for various reasons, including a finding that the clinical trial is not being conducted in accordance with FDA requirements or that the subjects or patients are being exposed to an unacceptable health risk.

Some clinical trials receive additional oversight by an independent group of qualified experts organized by us, known as a data safety monitoring board or committee.

This group receives and reviews data from the clinical trial on an ongoing basis and may recommend continuation of the clinical trial as planned, changes in clinical trial conduct, or cessation of the clinical trial at designated check points based on such data.

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In addition to the submission of an IND to the FDA before initiation of a clinical trial in the United States, certain human clinical trials involving recombinant or synthetic nucleic acid molecules may be subject to oversight of institutional biosafety committees (“IBCs”), as set forth in the National Institutes of Health (“NIH”) Guidelines for Research Involving Recombinant or Synthetic Nucleic Acid Molecules (“NIH Guidelines”).

In addition to the submission of an IND application to the FDA before initiation of a clinical trial in the United States, certain human clinical trials involving recombinant or synthetic nucleic acid molecules may be subject to oversight of institutional biosafety committees (“IBCs”), as set forth in the National Institutes of Health (“NIH”) Guidelines for Research Involving Recombinant or Synthetic Nucleic Acid Molecules (“NIH Guidelines”).

Under the NIH Guidelines, recombinant and synthetic nucleic acids are defined as:

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or (iii) molecules that result from the replication of those described in (i) or (ii).

Specifically, under the NIH Guidelines, supervision of human gene transfer trials includes evaluation and assessment by an IBC, a local institutional committee that reviews and oversees research utilizing recombinant or synthetic nucleic acid molecules at that institution.

Specifically, under the NIH Guidelines, supervision of qualifying trials includes evaluation and assessment by an IBC, a local institutional committee that reviews and oversees research utilizing recombinant or synthetic nucleic acid molecules at that institution.

The IBC assesses the safety of the research and identifies any potential risk to public health or the environment, and such review may result in some delay before initiation of a clinical trial.

Although the NIH Guidelines are not mandatory unless the research in question is being conducted at or sponsored by institutions receiving NIH funding of recombinant or synthetic nucleic acid molecule research, many companies and other institutions not otherwise subject to the NIH Guidelines voluntarily follow them.

Furthermore, the Food and Drug Omnibus Reform Act of 2022 (“FDORA”) requires a clinical trial sponsor to submit a diversity action plan for clinical trials, unless a waiver is granted by the FDA for reasons such as prevalence of the disease or condition, impracticality of implementing such a diversity action plan, or if such implementation would be against the interest of public health during a public health emergency.

Unless the FDA has granted a waiver, a sponsor must submit such action plan by the time the sponsor submits a protocol for a phase 3 clinical trial or other pivotal clinical trial.

Furthermore, the Food and Drug Omnibus Reform Act of 2022 (“FDORA”) requires a clinical trial sponsor to submit a diversity action plan for pivotal clinical trials, unless a waiver is granted by the FDA.

The action plan must include information such as the sponsor’s goal for enrollment (by sex, ethnic characteristics, age), the rationale behind the enrollment goals, the subject patient population, potential barriers for enrollment, among others.

This requirement is not yet in effect as it will become applicable to all clinical trials that begin enrollment 180 days after FDA publishes its final guidance on this topic.

In January 2025, the FDA removed its June 2024 guidance on clinical trial diversity action plans from its website, and it is not clear how the FDA will enforce the statutory requirement under FDORA.

The final guidance has not yet been published.

In January 2025, the FDA removed its June 2024 draft guidance on clinical trial diversity action plans from its website, and it is not clear how the FDA will enforce the statutory requirement under FDORA.

Clinical trials typically are conducted in three sequential phases.

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We may conduct multiple phase 2 clinical trials to obtain information prior to beginning larger and costlier phase 3 clinical trials.

In appropriate circumstances, a phase 2 clinical trial may serve as the basis for an application, in which case a separate phase 3 clinical trial will not be necessary.

• Phase 3 clinical trials are undertaken within an expanded patient population to gather the additional information about effectiveness and safety that is needed to evaluate the overall benefit-risk relationship of the drug and to provide an adequate basis for physician labeling.

In appropriate circumstances, a phase 2 clinical trial may serve as the basis for an application, in which case a separate phase 3 clinical trial may not be necessary.

• Phase 3 clinical trials are undertaken within an expanded patient population to gather the additional information about effectiveness and safety that is needed to evaluate the overall benefit-risk relationship of the product candidate and to provide an adequate basis for physician labeling.

These phases may overlap or be combined.

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Approval on the basis of a single trial may be subject to a requirement for additional post-approval studies.

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In addition, the manufacturer of an investigational biologic in a phase 2 or phase 3 clinical trial for a serious or life-threatening disease is required to make available, such as by posting on its website, its policy on evaluating and responding to requests for expanded access to such investigational drug or biologic.

During all phases of clinical development, regulatory agencies require extensive monitoring and auditing of all clinical activities, clinical data, and clinical trial investigators.

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Additionally, we must notify FDA within seven calendar days after receiving information concerning any unexpected fatal or life-threatening suspected adverse reaction.

Other external events may occur that can affect the conduct of our clinical trials, such as pandemics or government shutdowns.

Guidance Governing Gene Therapy Products

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potential implications of off-site gene editing;

The FDA also has issued other guidance documents that relate to gene therapies, such as “Human Gene Therapy for Rare Diseases,” and it is likely that the FDA will continue to issue additional guidelines in the future that may affect our product candidates.

Although guidance documents are not legally binding, we believe that following FDA’s recommendations set forth in them is likely necessary to gain approval for any product candidate we may develop.

The guidance documents provide additional factors that the FDA will consider at each of the above stages of development and relate to, among other things, the proper preclinical assessment of gene therapies;

the CMC information that should be included in an IND application;

the proper design of tests to measure product potency in support of a BLA application;

and measures to observe delayed adverse effects in subjects who have been exposed to investigational gene therapies when the risk of such effects is high.

Further, the FDA usually recommends that sponsors observe subjects for potential gene therapy-related delayed adverse events.

The FDA also has issued other guidance documents that relate to gene therapies, such as “Human Gene Therapy for Rare Diseases,” and it is likely that the FDA will continue to issue additional guidelines with respect to the development and regulatory submission of cell and gene therapy products in the future that may affect our product candidates.

The guidance documents provide additional factors that the FDA will consider at each of the above stages of development and relate to, among other things, the proper nonclinical assessment of gene therapies;

the CMC information that should be included in an IND;

the proper design of tests to measure product potency in support of a BLA;

and measures to observe delayed adverse effects in subjects who have been exposed to investigational gene therapies when the risk of such effects is elevated.

The FDA usually recommends that sponsors observe subjects for potential gene therapy-related delayed adverse events.

Depending on the product type, long term follow up can be up to 15 years or as short as five years.

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Clinical Trial Registry

There also are requirements governing the reporting of ongoing clinical trials and completed clinical trial results to public registries, such as such as www.ClinicalTrials.gov .

There also are requirements governing the reporting of ongoing clinical trials and completed clinical trial results to the U.S.

government-sponsored database, www.ClinicalTrials.gov, within certain timeframes.

We are required to register and disclose certain clinical trial information, including the product information, patient population, phase of investigation, clinical trial sites and investigators, and other aspects of the clinical trial on www.ClinicalTrials.gov.

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The FDA will not approve a BLA unless it determines that the manufacturing processes and facilities are in compliance with cGMP requirements and adequate to ensure consistent production of the product within required specifications.

The PHSA emphasizes the importance of manufacturing control for products such as biologics whose attributes cannot be precisely defined.

Material changes in manufacturing equipment, location, or process post-approval may result in additional regulatory review and approval.

The FDA also will not approve the product if we are not in compliance with cGTPs, which are requirements found in FDA regulations that govern the methods used in, and the facilities and controls used for, the manufacture of human cells, tissues, and cellular and tissue-based products (“HCT/Ps”), which are human cells or tissue intended for implantation, transplant, infusion, or transfer into a human recipient.

The PHSA emphasizes the importance of manufacturing controls for products such as biologics whose attributes cannot be precisely defined.

Material changes in manufacturing equipment, location, or process post-approval may result in additional regulatory review and require approval.

The FDA also will not approve a product if our manufacturing is not in compliance with cGTPs, which are requirements found in FDA regulations that govern the methods used in, and the facilities and controls used for, the manufacture of human cells, tissues, and cellular and tissue-based products (“HCT/Ps”), which are human cells or tissue intended for implantation, transplant, infusion, or transfer into a human recipient.

The primary intent of the cGTP requirements is to ensure that cell- and tissue-based products are manufactured in a manner designed to prevent the introduction, transmission, and spread of communicable disease.

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The results of product candidate development, nonclinical testing, and clinical trials, including negative or ambiguous results as well as positive findings, are submitted to the FDA as part of a BLA requesting a license to market the product.

The BLA must contain sufficient manufacturing information and detailed information on the composition of the product candidate and proposed labeling as well as payment of a user fee, unless the criteria for a waiver or exemption are met.

A BLA must contain sufficient manufacturing information and detailed information on the composition of the product candidate and proposed labeling as well as payment of a user fee, unless the criteria for a fee waiver or exemption are met.

Under the PHSA, the FDA will approve a BLA if it determines that our product candidate is safe, pure, and potent and the manufacturing facility meets standards designed to ensure that our product continues to be safe, pure, and potent.

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Once the submission has been filed, the FDA begins an in-depth review of the application.

Under the goals agreed to by the FDA under the Prescription Drug User Fee Act (“PDUFA”), for a new molecular entity, the FDA has 10 months from date that the FDA filed the BLA in which to complete its initial review of a standard application and respond to us, and six months from such filing date for a priority review of the application.

Under the goals agreed to by the FDA under the Prescription Drug User Fee Act (“PDUFA”), for a new molecular entity, the FDA has 10 months from the date that BLA is filed in which to complete its initial review of a standard application and respond to us, and six months from such filing date for a priority review of the application.

The FDA does not always meet its PDUFA goal dates for standard and priority BLAs.

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The FDA may refer our BLA to an advisory committee for review, evaluation, and recommendation as to whether our BLA should be approved.

In particular, the FDA may refer to an advisory committee application for biologic products that present difficult questions of safety or efficacy.

In particular, the FDA may refer to an advisory committee application for a biologic that present difficult questions of safety or efficacy.

Typically, an advisory committee is a panel of independent experts, including clinicians and other scientific experts, that reviews, evaluates, and provides a recommendation as to whether the application should be approved and under what conditions.

The FDA is not bound by the recommendations of an advisory committee, but it considers such recommendations carefully when making decisions.

On the basis of the FDA’s evaluation of the application and accompanying information, including the results of the inspection of the manufacturing facilities and any FDA audits of nonclinical study and clinical trial sites to ensure compliance with cGMPs and cGCPs, respectively, the FDA will issue an approval letter or a complete response letter.

On the basis of the FDA’s evaluation of the application and accompanying information, including the results of the inspection of the manufacturing facilities and any FDA audits of nonclinical study and clinical trial sites to ensure compliance with cGMPs, cGLPs, and cGCPs, respectively, the FDA will issue an approval letter or a complete response letter.

An approval letter authorizes commercial marketing of our product candidate with specific prescribing information for specific indications.

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The classification of a resubmission is based on the information submitted by us in response to the complete response letter.

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Under the goals and policies agreed to by the FDA under the PDUFA, the FDA has two months to review a class 1 resubmission and six months to review a class 2 resubmission.

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Alternatively, if we receive a complete response letter, we may either withdraw our BLA or request a hearing.

If the FDA approves any one of our products, it may limit the approved indications for use of our products.

As a condition of a BLA approval, the FDA may limit the approved indications for use of our products.

The FDA may also require that contraindications, warnings, or precautions be included in the product labeling.

In addition, the FDA may call for post-approval studies, including phase 4 clinical trials.

In addition, the FDA may require or request post-approval studies, including phase 4 clinical trials.

The FDA may also require testing and surveillance programs to monitor the product after commercialization, or impose other conditions, including distribution restrictions or other risk management mechanisms, including REMS, to help ensure that the benefits of the product outweigh the potential risks.

REMS can include medication guides, communication plans for healthcare professionals, and elements to assure safe use (“ETASU”).

REMS can include medication guides, communication plans for healthcare professionals, and elements to ensure safe use (“ETASU”).

ETASU can include, but are not limited to, specific or special training or certification for prescribing or dispensing, dispensing only under certain circumstances, special monitoring, and the use of patient registries.

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After approval, many types of changes to the approved product, such as adding new indications, certain manufacturing changes, and additional labeling claims, are subject to further testing requirements and FDA review and approval.

Fast Track, Breakthrough Therapy, Priority Review, and Regenerative Medicine Advanced Therapy Designations

The FDA is authorized to facilitate and expedite development and review of new drugs intended to address an unmet medical need in the treatment of a serious or life-threatening disease or condition.

These programs include fast track designation, breakthrough therapy designation, priority review, and regenerative medicine advanced therapy designation.

Fast Track, Breakthrough Therapy, Priority Review, and RMAT Designations

The FDA is authorized to facilitate and expedite development and review of new biologics intended to address an unmet medical need in the treatment of a serious or life-threatening disease or condition.

These programs include fast track designation, breakthrough therapy designation, priority review, and RMAT designation.

These designations are not mutually exclusive, and our product candidates may qualify for one or more of these programs.

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We must also provide, and the FDA must approve, a schedule for the submission of the remaining information, and we must pay applicable application user fees.

However, the FDA’s goal for reviewing a rolling submission does not begin until the last section of the application is submitted.

However, the FDA’s goal timeline for reviewing a rolling submission does not begin until the last section of the application is submitted.

In addition, the fast track designation may be withdrawn by the FDA if the FDA believes that the designation is no longer supported by data emerging in the clinical trial process, or if our designated product candidate development program is no longer being pursued.

Our product candidates may obtain breakthrough therapy designations if they are intended, either alone or in combination with one or more other products, to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that our product candidates may demonstrate substantial improvement over available therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.

The FDA may take certain actions with respect to product candidates with such designations, including holding meetings with us throughout the development process, providing timely advice to us regarding development and approval, involving more senior staff in the review process, assigning a cross-disciplinary project lead for the review team, and taking other steps to provide guidance on the design of the clinical trials in an efficient manner.

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The FDA makes such determination on a case-by-case basis, compared with other available therapies.

Significant improvement may be illustrated by evidence of increased effectiveness in the treatment of a condition, elimination or substantial reduction of a treatment-limiting adverse reaction, documented enhancement of patient compliance that may lead to improvement in serious outcomes, and evidence of safety and effectiveness in a new subpopulation.

A priority review designation is intended to direct overall attention and resources to the evaluation of such applications, and to shorten the FDA’s goal for acting on a marketing application from 10 months to six months.

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The FDA may grant product candidate RMAT designations if such product candidates are regenerative medicine therapies intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition and preliminary clinical evidence indicates that they have the potential to address unmet medical needs for such disease or condition.

Significant improvement may be illustrated by evidence of increased effectiveness in the treatment of a condition, elimination or substantial reduction of a treatment-limiting adverse reaction, documented enhancement of patient compliance that may lead to improvement in serious outcomes, or evidence of safety and effectiveness in a new subpopulation.

The FDA may grant product candidates RMAT designations if such product candidates are regenerative medicine therapies intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition and preliminary clinical evidence indicates that they have the potential to address unmet medical needs for such disease or condition.

RMAT designation provides potential benefits that include early interactions and more frequent meetings with the FDA to discuss the development plan for the product candidate and eligibility for rolling review and priority review.

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RMAT-designated products that receive accelerated approval may, as appropriate, fulfill their post-approval requirements through the submission of clinical evidence, clinical trials, patient registries, or other sources of real-world evidence such as electronic health records, through the collection of larger confirmatory data sets as agreed with the FDA, or via post-approval monitoring of all patients treated with such therapy prior to approval of the therapy.

Regenerative medicine advanced therapy designation may be rescinded if our product candidate no longer meets the qualifying criteria.

RMAT designation may be rescinded if our product candidate no longer meets the qualifying criteria.

Accelerated Approval Pathway

The FDA may grant accelerated approval to our product candidates for a serious or life-threatening condition that provides meaningful therapeutic advantage to patients over existing treatments based upon a determination that our product has an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit.

The FDA may also grant accelerated approval for such a condition when our product candidate has an effect on an intermediate clinical endpoint that can be measured earlier than an effect on irreversible morbidity or mortality (“IMM”), and that our product candidate is reasonably likely to predict an effect on IMM or other clinical benefit, taking into account the severity, rarity, or prevalence of the condition, and the availability or lack of alternative treatments.

The FDA may also grant accelerated approval when our product candidate has an effect on an intermediate clinical endpoint that can be measured earlier than an effect on irreversible morbidity or mortality (“IMM”) and that is reasonably likely to predict an effect on IMM or other clinical benefit, taking into account the severity, rarity, or prevalence of the condition, and the availability or lack of alternative treatments.

Product candidates granted accelerated approval must meet the same statutory standards for safety and efficacy as those granted traditional approval.

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Failure to conduct required post-approval studies, or confirm a clinical benefit during post-marketing studies, would allow the FDA to withdraw the product from the market on an expedited basis.

All promotional materials for product candidates approved under accelerated regulations are subject to prior review by the FDA unless the FDA informs us otherwise.

FDORA included provisions related to the accelerated approval pathway.

Pursuant to FDORA, the FDA is authorized to require a post-approval study to be underway prior to approval or within a specified time period following approval.

FDORA also requires the FDA to specify conditions of any required post-approval study, which may include milestones such as a target date of study completion and requires sponsors to submit progress reports for required post-approval studies and any conditions required by the FDA not later than 180 calendar days following approval and not less frequently than every 180 days thereafter until completion or termination of the study.

FDORA enables the FDA to initiate enforcement action for the failure to conduct with due diligence a required post-approval study, including a failure to meet any required conditions specified by the FDA or to submit timely reports.

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All promotional materials for product candidates approved under accelerated approval are subject to prior review by the FDA unless the FDA informs us otherwise.

The FDA is authorized to require a post-approval study to be underway prior to approval or within a specified time period following approval and is required to specify conditions of any required post-approval study, which may include milestones such as a target date of study completion.

Sponsors are required to submit progress reports for required post-approval studies and any conditions required by the FDA not later than 180 calendar days following approval and not less frequently than every 180 days thereafter until completion or termination of the study.

The FDA may initiate enforcement action for the failure to conduct with due diligence a required post-approval study, including a failure to meet any required conditions specified by the FDA or to submit timely reports.

Post-Approval Regulation

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Manufacturers of our products are required to register their establishments with the FDA and certain state agencies and are subject to periodic announced or ad hoc inspections by the FDA and certain state agencies for compliance with ongoing regulatory requirements, including cGMP regulations, which impose certain procedural and documentation requirements upon these manufacturers.

Accordingly, we and our third-party manufacturers must continue to expend time, money, and effort in the areas of production and quality control to maintain compliance with cGMP regulations and other regulatory requirements.

Our products may also be subject to official lot release, meaning that the manufacturer of our products is required to perform certain tests on each lot of the product before the product is released for distribution.

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Pharmaceutical products may be promoted only for the approved indications and in accordance with the provisions of the approved labeling.

Orphan Drug Designation

Orphan drug designation is available for drugs that are intended for rare diseases or conditions, defined as (i) a disease or condition that affects fewer than 200,000 individuals in the United States or (ii) a disease or condition that affects more than 200,000 individuals in the United States and for which there is no reasonable expectation that the cost of developing and making available a biologic for the disease or condition will be recovered from sales of the product in the United States.

If a drug becomes the first drug that is approved for the same indication for which the FDA has granted the designation, the drug will be entitled to exclusivity, which means the FDA may not approve any other application to market the same drug for the same orphan indication for a period of seven years following the date of our product’s marketing approval, except in certain circumstances, such as a showing of clinical superiority to the product with orphan drug exclusivity by means of greater effectiveness, greater safety, or providing a major contribution to patient care, or in instances of drug supply issues.

In the case of a biological product, sameness is based on the principal molecular features of the product.

Orphan drug exclusivity does not prevent FDA from approving a different drug for the same disease or condition, or the same drug for a different disease or condition.

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Orphan Drug Designation and Exclusivity

Orphan drug designation is available for drugs and biologics that are intended for rare diseases or conditions, defined as (i) a disease or condition that affects fewer than 200,000 individuals in the United States or (ii) a disease or condition that affects more than 200,000 individuals in the United States and for which there is no reasonable expectation that the cost of developing and making available a biologic for the disease or condition will be recovered from sales of the product in the United States.

If a drug or biologic becomes the first product that is approved for the same disease or condition for which the FDA has granted the designation, that product will be entitled to orphan drug exclusivity, which means the FDA may not approve any other application to market the same drug or biologic for the same orphan disease or condition for a period of seven years following the date of marketing approval, except in certain circumstances, such as a showing of clinical superiority to the product with orphan drug exclusivity by means of greater effectiveness, greater safety, or providing a major contribution to patient care, or in instances of product supply issues.

In the case of a biologic, sameness is based on the principal molecular features of the product.

Orphan drug exclusivity does not prevent FDA from approving a different drug or biologic for the same disease or condition, or the same drug or biologic for a different disease or condition.

To obtain an orphan drug designation, we must make a request before submitting our BLA for a particular product candidate.

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The FDA may, on its own initiative or at the request of the sponsor, grant deferrals for submission of some or all pediatric data until after approval of the product for use in adults, or full or partial waivers from the pediatric data requirements, under specified circumstances.

Unless otherwise required by regulation, the pediatric data requirements do not apply to products with orphan designation except a product with a new active ingredient that is a molecularly targeted cancer product intended for the treatment of an adult cancer and directed at a molecular target determined by the FDA to be substantially relevant to the growth or progression of a pediatric cancer.

Pediatric exclusivity is another type of non-patent marketing exclusivity in the United States and, if granted, provides for the attachment of an additional six months of marketing protection to the term of any existing non-patent regulatory exclusivity, including orphan drug exclusivity.

Unless otherwise required by regulation, the pediatric data requirements do not apply to products with orphan drug designation except a product with a new active ingredient that is a molecularly targeted cancer product intended for the treatment of an adult cancer and directed at a molecular target determined by the FDA to be substantially relevant to the growth or progression of a pediatric cancer.

Pediatric exclusivity is another type of nonpatent regulatory exclusivity for drugs and biologics in the United States and, if granted, provides for the attachment of an additional six months of marketing protection to the term of any existing nonpatent regulatory exclusivity, including orphan drug exclusivity.

This six-month exclusivity may be granted if pediatric data is submitted that sufficiently responds to a written request from the FDA for such data.

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rather, if the clinical trial is deemed to be responsive to the FDA’s request, the additional protection is granted.

If reports of requested pediatric studies are submitted to and accepted by the FDA within the statutory time limits, whatever statutory or regulatory periods of exclusivity or patent protection cover the product are extended by six months.

This is not PTE;

instead, this grant of exclusivity extends the regulatory period during which the FDA cannot approve another application.

If reports of requested pediatric studies are submitted to and accepted by the FDA within the statutory time limits, whatever statutory periods of exclusivity that cover the product are extended by six months.

Biosimilars and Exclusivity

The Patient Protection and Affordable Care Act, as amended by the Health Care and Education Reconciliation Act of 2010 (“Affordable Care Act”) includes a subtitle called the Biologics Price Competition and Innovation Act of 2009 (“BPCIA”), which created an abbreviated approval pathway for biological products that are biosimilar to or interchangeable with an FDA-licensed reference biological product in the United States.

The Patient Protection and Affordable Care Act, as amended by the Health Care and Education Reconciliation Act of 2010 (“Affordable Care Act”) includes a subtitle called the Biologics Price Competition and Innovation Act of 2009 (“BPCIA”), which created an abbreviated approval pathway for biologics that are biosimilar to or interchangeable with an FDA-licensed reference biologic in the United States.

Starting in 2015, the FDA commenced licensing biosimilars under the BPCIA, and there are currently numerous biosimilars approved in the United States as well as a number of interchangeable biosimilar products.

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Even after the FDA approves a biosimilar product, the product, its manufacturing processes, post-approval clinical data, labeling, advertising, and promotional activities for the product will be subject to continuous requirements of and review by the FDA or other regulatory authorities.

These requirements include submissions of safety and other post-marketing information and reports, including mandatory post-

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marketing safety reporting;

These requirements include submissions of safety and other post-marketing information and reports, including mandatory post-marketing safety reporting;

registration and listing requirements;

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and requirements regarding recordkeeping.

Under the BPCIA, an application for a biosimilar product may not be submitted to the FDA until four years following the date of approval of the reference product.

The FDA may not approve a biosimilar product until 12 years from the date on which the reference product was approved.

Under the BPCIA, an application for a biosimilar product may not be submitted to the FDA until four years following the date of first licensure of the reference product.

“First licensure” typically means the initial date the particular product at issue was licensed in the United States.

The FDA may not approve a biosimilar product until 12 years from first licensure of the reference product.

The FDA does not assign a date of first licensure or award a new period of exclusivity for a biologic if the licensure is for a supplement for the biologic or for a subsequent application by the same sponsor or manufacturer (or licensor, predecessor in interest, or other related entity) of the biologic for a change that results in a new indication, route of administration, dosing schedule, dosing form, delivery system, delivery device or strength, or for a modification to the structure of the biologic that does not result in a change in safety, purity, or potency.

Even if a product is considered to be a reference product eligible for exclusivity, another company could market a competing version of that product if the FDA approves a full BLA for such product containing our own nonclinical data and data from adequate and well-controlled clinical trials to demonstrate the safety, purity, and potency of the product.

Patent Term Extension

A patent claiming a new biologic product may be eligible for a limited PTE under the Hatch-Waxman Amendments, which permits a patent restoration of up to five years for patent term lost during product development and FDA regulatory review.

A patent claiming a new biologic may be eligible for a limited PTE under the Hatch-Waxman Amendments, which permits a patent restoration of up to five years for patent term lost during product development and FDA regulatory review.

The restoration period granted on a patent covering a product is typically one-half the time between the effective date of an IND and the submission date of a BLA, plus the time between the submission date of a BLA and the ultimate approval date, less any time during which due diligence was not conducted.

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§ 156, only one patent covering an approved product, or the use or manufacture thereof, is eligible for PTE, and the application for the extension must be submitted prior to the expiration of the patent in question and within 60 calendar days after regulatory approval.

A patent that covers multiple products for which approval is sought can only be extended in connection with one of the approvals.

The patent, even if it covers multiple products for which approval is sought, can be extended only once.

The USPTO reviews and approves the application for any PTE in consultation with the FDA.

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Specifically, the process governing approval of medicinal products in the EU generally follows the same lines as in the United States, although the approval of a medicinal product in the United States is no guarantee of approval of the same product in the EU, either at all or within the same timeframe as approval may be granted in the United States.

The process entails satisfactory completion of preclinical studies and adequate and well-controlled clinical trials to establish the safety and efficacy of a product candidate for each proposed indication.

The process entails satisfactory completion of nonclinical studies and adequate and well-controlled clinical trials to establish the safety and efficacy of a product candidate for each proposed indication.

It also requires the submission to the EMA or the relevant member state competent authorities, of a marketing authorization application and granting of a marketing authorization by the EMA or these authorities before the product can be marketed and sold in the EU.

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Treasury Department’s Office of Foreign Assets Controls could apply to any international activities we may undertake.

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Coverage, Pricing, and Reimbursement

Significant uncertainty exists as to the coverage and reimbursement status of any product candidates for which we may seek regulatory approval by the FDA or other government authorities.

Significant uncertainty exists as to the coverage and reimbursement status of any product candidates for which we may obtain regulatory approval by the FDA or other government authorities.

In the United States and other countries, patients who are prescribed treatments for their conditions and providers performing the prescribed services often rely on third-party payors to reimburse all or part of the associated healthcare costs.

Patients are unlikely to use any product candidates we may develop unless coverage is provided and reimbursement is adequate to cover a significant portion of the cost of such product candidates.

In addition, direct or indirect governmental price regulation may affect the prices that we may charge for product candidates.

Patients are unlikely to use any products we may develop unless coverage is provided and reimbursement is adequate to cover a significant portion of the cost of our products.

In addition, direct or indirect governmental price regulation may affect the prices that we may charge for our products.

United States

Even if any product candidates we may develop obtain approval, sales of such product candidates will depend, in part, on the extent to which third-party payors, including government healthcare programs in the United States, such as Medicare and Medicaid, commercial health insurers, and managed care organizations provide coverage and establish adequate reimbursement levels for such product candidates.

Even if any product candidates we may develop obtain regulatory approval, sales of such products will depend, in part, on the extent to which third-party payors, including government healthcare programs in the United States, such as Medicare and Medicaid, commercial health insurers, and managed care organizations provide coverage and establish adequate reimbursement levels for such products.

In general, factors payors consider in determining coverage and reimbursement are based on whether the product is:

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• neither experimental nor investigational.

In the United States, no uniform policy of coverage and reimbursement for biological products, including gene and cell therapy products, exists among third-party payors.

As a result, obtaining coverage and reimbursement approval for such a product from a government or other third-party payor is a time-consuming and costly process that could require us to provide to each payor supporting scientific, clinical, and cost-effectiveness data regarding the product’s clinical benefits, medical necessity, and risks on a payor-by-payor basis, with no assurance that coverage and adequate reimbursement will be obtained.

In the United States, no uniform policy of coverage and reimbursement for biologics, including cell and gene therapy products, exists among third-party payors.

As a result, obtaining coverage and reimbursement approval for such a product from a government or other third-party payor is a time-consuming and costly process that could require us to provide to each payor supporting scientific, clinical, and cost-effectiveness data regarding the product’s clinical benefits, medical necessity, and risks on a payor-by-payor basis, and we cannot provide any assurance that coverage and adequate reimbursement will be obtained.

A decision by a third-party payor not to cover any product candidates we may develop could reduce physician utilization of such product candidates once approved and have a material adverse effect on our sales, results of operations and financial condition.

Additionally, a payor’s decision to provide coverage for a product does not imply that an adequate reimbursement rate will be approved, and inadequate reimbursement rates, including significant patient cost sharing obligations, may deter patients from selecting our product candidates.

One payor’s determination to provide coverage for a product does not ensure that other payors will also provide coverage and reimbursement for the product, and the level of coverage and reimbursement can differ significantly from payor to payor.

Third-party reimbursement and coverage may not be available to enable us to maintain price levels sufficient to realize an appropriate return on our investment in product development.

Coverage policies and third-party reimbursement rates may change at any time.

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In the EU, the approval process and requirements governing pricing and reimbursement for any product candidate vary greatly between countries and jurisdictions.

Some countries allow biological products to be marketed only after a reimbursement price has been agreed.

Some countries may require the completion of additional testing or studies that compare the cost effectiveness of a particular biological product to currently available treatments, or so-called health technology assessments, in order to obtain reimbursement or pricing approval.

Some countries allow biologics to be marketed only after a reimbursement price has been agreed.

Some countries may require the completion of additional testing or studies that compare the cost effectiveness of a particular biologic to currently available treatments, or so-called health technology assessments, in order to obtain reimbursement or pricing approval.

The EU HTA Regulation 2021/2282 became effective in January 2022, is applicable from January 2025 with a phased implementation until 2030, and aims to harmonize clinical and scientific aspects of HTA across the EU.

Some countries, including several EU member states, set prices and reimbursement for biological products, with limited participation from the marketing authorization holders.

For example, the EU provides options for its member states to restrict the range of biological products for which their national health insurance systems provide reimbursement and to

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control the prices of biological products for human use.

EU member states may approve a specific price for a biological product or may instead adopt a system of direct or indirect controls on the profitability of the company providing the biological product.

Recently, many European countries have increased the level of discounting required in relation to the pricing of biological products and these efforts could continue as countries attempt to manage healthcare expenditures.

Some countries, including several EU member states, set prices and reimbursement for biologics, with limited participation from the marketing authorization holders.

For example, the EU provides options for its member states to restrict the range of biologics for which their national health insurance systems provide reimbursement and to control the prices of biologics for human use.

EU member states may approve a specific price for a biologic or may instead adopt a system of direct or indirect controls on the profitability of the company providing the biologic.

Many European countries have increased the level of discounting required in relation to the pricing of biologics and these efforts could continue as countries attempt to manage healthcare expenditures.

Healthcare Law and Regulation

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federal health care programs, and/or criminal penalties.

The current administration has signaled that in 2026 healthcare providers and suppliers can expect to see an increase in enforcement efforts based on these laws.

Restrictions under applicable U.S.

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Physician Payments Sunshine Act, or Open Payments program, created under the Affordable Care Act, which requires certain manufacturers of drugs, devices, biologics, and medical supplies to report annually to the Centers for Medicare & Medicaid Services (“CMS”) information related to payments, including certain product development activities such as clinical trials, and other transfers of value made by that entity to covered recipients, currently defined to include doctors, dentists, optometrists, podiatrists, chiropractors, teaching hospitals, physician assistants, nurse practitioners, and certain other healthcare providers and requires certain manufacturers and applicable group purchasing organizations to report ownership and investment interests held by physicians or their immediate family members;

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price reporting laws, which require companies to calculate and report complex pricing metrics in an accurate and timely manner to government programs.

Such laws may not only affect coverage, reimbursement, and pricing for our product candidates, but can also result in civil penalties for late or incorrect reports;

Such laws may not only affect coverage, reimbursement, and pricing for our products, but can also result in civil penalties for late or incorrect reports;

consumer protection and unfair competition laws, which broadly regulate marketplace activities and activities that potentially harm consumers;

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Numerous federal and state laws and regulations, including federal health information privacy laws, state data breach notification laws, state health information privacy laws and federal and state consumer protection laws (e.g., Section 5 of the Federal Trade Commission Act), that govern the collection, use, disclosure, and protection of health-related and other personal information could apply to our operations or the operations of our collaborators and third-party providers.

In December 2023, HHS finalized its Health Data, Technology and Interoperability rule, establishing new standards for transparency, information exchange, and interoperability for health information technology.

In December 2023, the U.S.

Department of Health and Human Services (“HHS”) finalized its Health Data, Technology and Interoperability rule, establishing new standards for transparency, information exchange, and interoperability for health information technology.

On February 25, 2025, the current administration issued an executive order requiring HHS to issue guidance and otherwise enforce regulations requiring meaningful price transparency and standardized, required disclosures aimed to allow patients to make well-informed healthcare decisions.

California has enacted the California Consumer Privacy Act (“CCPA”).

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government rebate programs and additional downward pressure on pharmaceutical product prices.

Several healthcare reform proposals culminated in the enactment of the Inflation Reduction Act (the “IRA”) in August 2022, which, among other things, eliminated, beginning in 2025, the coverage gap under Medicare Part D by significantly lowering the enrollee maximum out-of-pocket costs and requiring manufacturers to subsidize, through a newly established manufacturer discount program, 10% of Part D enrollees’ prescription costs for brand drugs below the out-of-pocket limit, and 20% once the out-of-pocket limit has been reached.

The IRA also allows HHS to directly negotiate the selling price of a statutorily specified number of drugs and biologics each year that CMS reimburses under Medicare Part B and Part D.

There has been increasing legislative and enforcement interest in the United States with respect to drug and biologic pricing practices since the ACA was enacted.

Several healthcare reform initiatives culminated in the enactment of the Inflation Reduction Act (the “IRA”) in 2022, which, among other things, eliminated, beginning in 2025, the coverage gap under Medicare Part D by significantly lowering the enrollee maximum out-of-pocket costs and requiring manufacturers to subsidize, through a newly established manufacturer discount program, 10% of Part D enrollees’ prescription costs for brand drugs and biologics below the out-of-pocket limit, and 20% once the out-of-pocket limit has been reached.

The IRA also requires HHS to directly negotiate the selling price of a statutorily specified number of drugs and biologics each year that CMS reimburses under Medicare Part B and Part D.

The negotiated price may not exceed a statutory ceiling price.

Only high-expenditure single-source

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biologics that have been approved for at least 11 years (seven years for single-source drugs) are eligible to be selected by CMS for negotiation, with the negotiated price taking effect two years after the selection year.

Only high-expenditure single-source biologics that have been approved for at least 11 years (seven years for single-source drugs) are eligible to be selected by CMS for negotiation, with the negotiated price taking effect two years after the selection year.

For 2026, the first year in which negotiated prices become effective, CMS selected 10 high-cost Medicare Part D products in 2023, negotiations began in 2024, and the negotiated maximum fair price for each product has been announced.

CMS has selected 15 additional Medicare Part D drugs for negotiated maximum fair pricing in 2027.

For 2028, an additional 15 drugs, which may be covered under either Medicare Part B or Part D, will be selected, and for 2029 and subsequent years, 20 Part B or Part D drugs will be selected.

A drug or biological product that has an orphan drug designation for only one rare disease or condition will be excluded from the IRA’s price negotiation requirements, but will lose that exclusion if it receives designations for more than one rare disease or condition, or if is approved for an indication that is not within that single designated rare disease or condition, unless such additional designation or such disqualifying approvals are withdrawn by the time CMS evaluates the drug for selection for negotiation.

The IRA also imposes rebates on Medicare Part B and Part D drugs whose prices have increased at a rate greater than the rate of inflation.

The IRA also extends enhanced subsidies for individuals purchasing health insurance coverage in ACA marketplaces through plan year 2025.

These negotiations resulted in significant price reductions for the products from their 2023 list prices, ranging from 38% to 79%, with an average price reduction of 59.4%.

In addition, CMS selected and announced the negotiated maximum fair price for 15 additional Medicare Part D drugs, which will become effective in 2027.

For 2028, CMS has selected an additional 15 drugs, comprised of drugs covered under Medicare Part D and, for the first time, drugs payable under Medicare Part B.

For 2029 and subsequent years, 20 Part B or D drugs will be selected.

The negotiated prices have represented, and will continue to represent, a significant discount from average prices to wholesalers and direct purchasers.

Currently, a drug or biologic that has an orphan drug designation for only one rare disease or condition will be excluded from the IRA’s price negotiation requirements, but will lose that exclusion if it receives designations for more than one rare disease or condition, or if is approved for an indication that is not within that single designated rare disease or condition, unless such additional designation or such disqualifying approvals are withdrawn by the time CMS evaluates the drug for selection for negotiation.

However, as a result of a statutory amendment enacted in July 2025, beginning with the 2028 negotiated price applicability year, a drug or biologic may be designated for more than one rare disease or condition and still be excluded from price negotiation, as long as the only approved indications are for such rare diseases or conditions.

The IRA also imposes rebates on Medicare Part B and Part D drugs whose prices have increased at a rate greater than the rate of inflation, and, in 2024, CMS finalized regulations for the Medicare Part B and Part D inflation rebates.

The IRA permits the Secretary of HHS to implement many of these provisions through guidance, as opposed to regulation, for the initial years.

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The outcome of these lawsuits is uncertain, and some IRA drug discount provisions have not been challenged in litigation.

Thus, while it is unclear how the IRA will be implemented, it will likely have a significant impact on the pharmaceutical industry and the pricing of our products and product candidates.

It is unclear what policies the new Administration will advance with respect to IRA implementation and other drug pricing proposals.

Thus, while it is unclear how the IRA will be implemented, it will likely have a significant impact on the pharmaceutical industry and the pricing of products.

It is unclear what policies will be advanced with respect to IRA implementation and other drug pricing proposals.

In May 2025, the administration published an executive order regarding most favored nation (“MFN”) drug pricing, which is sometimes referred to as “international reference pricing.” This executive order directs the Secretary of HHS to communicate MFN price targets to pharmaceutical manufacturers and, if significant progress toward MFN pricing is not delivered, to propose a rulemaking plan to implement MFN pricing.

Recently, on December 23, 2025, CMS issued proposed regulations to establish, under the Center for Medicare and Medicaid Innovation, two mandatory MFN pricing demonstration models under Medicare Part B and Part D.

If these rules or other MFN pricing rules are finalized, they are likely to mandate reduced pricing of at least some of the drugs and biologics in the United States if they are also sold in comparator countries.

At the state level, legislatures have increasingly passed legislation and implemented regulations designed to control pharmaceutical product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product access, and marketing cost disclosure and transparency measures, and in some cases, designed to encourage importation from other countries and bulk purchasing.

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Workforce Demographics

As of March 1, 2025, we had 147 total employees, all of which were full-time.

Of our employees, 39 hold Ph.D., M.D., and/or Pharm.D.

degrees, and 107 are engaged in research, development, and technical operations.

Most of our employees are located in Berkeley, California.

As of February 27, 2026, we had 97 total employees, all of whom were full-time.

The majority of our employees are located in Berkeley, California.

Our employees are not represented by labor unions or covered by collective bargaining agreements.

We believe that our employee morale is healthy and consider our relationship with our employees to be good;

however, in July 2024 and in April 2025, we announced workforce reductions, which may have negatively impacted our relationship with employees.

Talent Acquisition, Development, and Total Rewards

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We require our managers and employees to play an active role in the performance management process.

Our performance management

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process is designed to increase clarity and accountability for roles and responsibilities, strengthen communication, and build trust, all while championing personal and professional growth, learning, and success.

Our performance management process is designed to increase clarity and accountability for roles and responsibilities, strengthen communication, and build trust, all while championing personal and professional growth, learning, and success.

Great teamwork is a critical foundation for Caribou.

Our peer-to-peer rewards and recognition program helps us maintain a culture of recognition and teamwork by offering various options for our employees and managers to recognize and reward colleagues across the organization.

We plan to continue to refine our efforts related to optimizing our use of human capital as we grow, including improvements in the way we hire, develop, reward, and retain employees.

We plan to continue to refine our efforts related to optimizing our use of human capital, including improvements in the way we hire, develop, reward, and retain employees.

The Herd at Caribou

Our employees are our greatest asset, and we aim to create an inclusive and collaborative environment with the overall goal of engaging our workforce to support our current pipeline, develop new technologies, and support future business goals, while protecting the long-term interests of our stockholders.

Our employees are our greatest asset, and we aim to create an inclusive and collaborative environment with the overall goal of engaging our workforce to support our current clinical product candidates and future business goals, while protecting the long-term interests of our stockholders.

Our culture is built around our core values:

we are driven by patient needs, innovation is in our chRDNA, together we are stronger, and integrity and ethics guide our decision making.

we are driven by patient need, innovation is in our chRDNA, together we are stronger, and integrity and ethics guide our decision making.

We have built a culture where employees are empowered, their ideas are taken seriously, and their contributions are recognized.

We at Caribou refer to ourselves as “the herd.” We encourage and value social interactions among the herd.

We hold bi-monthly herd meetings and quarterly on-site events to strengthen our culture and find different ways to interact.

Some of these events include contributing to the Alameda Food Bank and participating in San Francisco Bay clean-up events.

We offer “fun runs,” yoga in a hybrid format, and have local gym facilities available for our Berkeley employees.

As we continue to grow, we will find more opportunities to connect our teams, taking into account our different functions and locations, while focusing on building a culture that is driven by our mission to develop innovative, transformative therapies through novel genome editing for patients with devastating diseases.

We hold bi-monthly herd meetings in a hybrid on-site/videoconferencing format and periodically have all-employee on-site events to strengthen our culture, such as participating in San Francisco Bay clean-up events.

We strive to build a culture that is driven by our mission to develop innovative, transformative therapies through novel genome editing for patients with devastating diseases.

We regularly conduct engagement surveys and use the feedback to enhance our workplace culture.

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We have established a culture advisory group comprised of cross-functional representatives from various geographic locations.

Based on feedback from focus groups and the culture advisory group, along with the results of employee surveys, we have focused on highlighting how our daily work connects to our corporate values, connecting teams to other functions, and helping employees understand how decisions that impact them or their roles are made.

Survey results demonstrate that employees feel more connected and are committed and excited to come to work, increasing overall engagement.

Based on feedback from our culture advisory group, along with the results of employee surveys, we have focused on highlighting how our daily work connects to our corporate values, connecting teams to other functions, helping employees understand how decisions that impact them or their roles are made, ensuring leadership accessibility, and engaging our employees across multiple communication channels.

We expect all employees to observe the highest levels of business ethics while delivering the highest levels of performance.

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Our employees are talented and passionate people who are committed to making a difference in our community and beyond.

As a company, we actively participate in outreach efforts to increase opportunities for underrepresented groups, including hosting and providing volunteers for science, technology, engineering, and mathematics (“STEM”) programs at local elementary, junior high, and high schools as well as community colleges and universities.

Many of our employees speak at local schools about careers in biotechnology and we have hosted students at our facility to engage them in aspects of biotechnology to which they may not have been previously exposed.

We look for opportunities to foster the growth of future scientists and a love of science.

We provide each of our employees with eight hours of paid volunteer time each year, which can be used for participating in school activities, voter registrations, environmental activities, and the like.

We are environmentally conscious.

With this in mind, we strive to mitigate our impact on the environment where possible and pursue innovative ways to grow our business while minimizing our environmental footprint.

The City of Berkeley requires companies with 10 or more employees to have a commuter benefits program in place, and we offer pre-tax commuter benefits to ride public transportation, which is connected to our facility through various free shuttle services.

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Additionally, we provide bicycle vouchers to employees who bike to work and have bike repair tools on site as well as bike storage areas, and our employees have access to electric vehicle charging stations.

Our facility is equipped with water stations that filter water to discourage the use of plastic bottles.

All refuse generated at our facility is sorted among recycle, compost, and landfill.

We have moved to electronic documentation and files in most functions.

We strive to mitigate our impact on the environment where possible and pursue innovative ways to minimize our environmental footprint.

The City of Berkeley requires companies with 10 or more employees to have a commuter benefits program in place, and we offer pre-tax commuter benefits to ride public transportation.

Additionally, we provide bicycle vouchers to employees who bike to work and have bike repair tools on site as well as bike storage areas.

Our employees have access to electric vehicle charging stations.

Workforce Health and Safety

Workplace safety is a priority for us.

To maintain a safe and healthy workplace, we have implemented Environment, Health, and Safety (“EHS”) initiatives that focuses on key risk mitigation programs to identify, assess, and correct hazards.

To maintain a safe and healthy workplace, we have implemented Environment, Health, and Safety (“EHS”) initiatives that focus on key risk mitigation programs to identify, assess, and correct hazards.

We also have a safety training program that is designed for employees to be assigned the appropriate training to understand how to safely perform their duties.

Information Available on the Internet

Investors and others should note that we announce material information to our investors using our investor relations website (https://cariboubio.com/investors), our filings with the U.S.

Investors and others should note that we announce material information to our investors using our investor relations website (cariboubio.com), our filings with the U.S.

Securities and Exchange Commission (“SEC”), press releases, public conference calls, and webcasts.

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Our Annual Reports on Form 10-K, Quarterly Reports on Form 10-Q, Current Reports on Form 8-K, including exhibits, proxy and information statements and amendments to those reports filed or furnished pursuant to Sections 13(a) and 15(d) of the Exchange Act, are available on our website free of charge as soon as reasonably practicable after we electronically file the material with, or furnish it to, the SEC.

The materials we file with or furnish to the SEC are also available at http://www.sec.gov.

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The materials we file with or furnish to the SEC are also available at www.sec.gov.

Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.

What changed 10-K

Item 1. Business