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Item 1. Business

2026-03-09 compared with 2025-03-05 · 395 added, 287 removed, 959 unchanged (42% of the section changed)

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Once the complex binds to the DNA sequence it is designed to recognize, the complex makes a specific cut in the DNA.

We believe we are the only human genome editing company with a platform that includes CRISPR/Cas9, CRISPR/Cas12a, engineered forms of both of these CRISPR systems, and foundational intellectual property for both of these CRISPR systems.

We believe we are the only human gene editing company with a platform that includes CRISPR/Cas9, CRISPR/Cas12a, engineered forms of both of these CRISPR systems, and foundational intellectual property for both of these CRISPR systems.

Because of the broad nature of this platform, we believe we can create gene editing molecules for more than 95% of the human genome.

We seek to be a leader in in vivo programmable gene editing, leveraging cutting edge gene editing technology to deliver therapies that simplify the usability for patients, minimize the burdens to patients and healthcare systems, and are meaningfully differentiated from the current standards of care, while also allowing a simpler, more scalable manufacturing process that can reduce costs to manufacture and administer the therapies relative to ex vivo treatments.

Our approach is focused on the in vivo use of functional upregulation, which aims to increase the expression of a normal gene copy and thereby increases its normal protein function to treat diseases caused by genetic mutations that eliminate or disrupt normal function.

We are able to upregulate by editing regulatory sequences upstream and downstream of a gene, which allows us to address those diseases where the gene knockdown approach, which aims to reduce the expression of one or more targeted genes, will not be effective.

Further, upregulation enables us to create treatments designed for most or all patients with the same disease, regardless of the specific genetic mutation causing the disease.

We previously demonstrated proof of concept of our functional upregulation strategy in our clinical trials of renizgamglogene autogedtemcel (“reni-cel”), an experimental ex vivo gene-edited medicine to treat sickle cell disease (“SCD”), a severe inherited blood disease that causes premature death, and transfusion-dependent beta thalassemia (“TDT”), the most severe form of beta-thalassemia, an inherited blood disorder characterized by severe anemia.

In our former reni-cel program, we used our indel CRISPR technology to upregulate the expression of the gamma globin gene, a functional homolog of the beta globin gene, through direct editing of the HBG1/2 promotor site using our highly specific and efficient proprietary engineered AsCas12a enzyme.

While we are no longer developing reni-cel, clinical data from our reni-cel program demonstrated that we can efficiently and specifically edit hematopoietic stem cells (“HSCs”), resulting in robust and clinically meaningful improvements for treated patients and a safety profile consistent with myeloablative busulfan conditioning and autologous HSC transplant.

We seek to be a leader in in vivo gene editing, leveraging cutting edge gene editing technology to deliver transformative therapies that simplify the usability for patients, minimize the burdens to patients and healthcare systems, and are meaningfully differentiated from the current standards of care, while also allowing a simple, scalable manufacturing process to reduce costs to manufacture and administer the therapies.

Our approach is focused on the in vivo use of functional upregulation, which aims to increase the expression of a normal gene copy and its normal protein function to treat diseases caused by genetic mutations that eliminate or disrupt normal function.

This approach edits non-coding regulatory sequences upstream and downstream of a gene and does not alter the sequence of the encoded protein, unlike other approaches, including gene knockdown, which aims to reduce the expression of one or more targeted genes by editing coding regions to knockdown a disease-causing protein, and gene correction, which aims to edit coding regions to correct a disease-causing protein.

We believe this approach will allow us to address those diseases where the gene knockdown approach has limited effectiveness and to create treatments designed for most or all patients with the same disease, regardless of the specific genetic mutation causing the disease.

We believe the ability to provide in vivo gene editing, in which the medicine is injected or infused into the patient to edit the cells inside their body, and functionally upregulate normal gene expression and normal protein function in the target tissues holds the potential to significantly expand the addressable therapeutic possibilities of CRISPR-based gene editing.

To that end, our preclinical efforts are also focused on the creation of a “plug ‘n play” lipid nanoparticle (“LNP”) platform to enable delivery of the gene editing cargo to multiple cells and tissues, including HSCs, the liver and other cells and tissues.

In October 2024, we shared our achievement of in vivo preclinical proof of concept of hematopoietic stem and progenitor cell (“HSPC”) editing and fetal hemoglobin (“HbF”) induction in humanized mice engrafted with human HSCs and lacking their own hematopoietic cells using a novel and proprietary targeted LNP (“tLNP”) formulation for non-liver, referred to as extrahepatic, tissue delivery.

Building on this achievement, in January 2025, we announced in vivo preclinical proof of concept of editing HSCs in non-human primates, a key step in developing a treatment for SCD and TDT.

We further announced in vivo delivery to two additional extrahepatic cell types in humanized mice using our proprietary lipid nanoparticle (“LNP”) targeting platform and in vivo editing of liver cells in non-human primates.

We expect to share additional in vivo preclinical proof of concept data in both HSCs and liver cells in 2025.

Preclinical Development

Leveraging our differentiated approach from our former reni-cel program, we are focused on editing HSCs through targeted delivery of our AsCas12a enzyme to our clinically validated HBG1 and HBG2 promotor site.

The myeloablative busulfan conditioning regimen for ex vivo HSC transplantation that is necessary for current gene editing therapies for SCD and TDT requires that patients undergo immunosuppression at the time of transplant and carries with it the long-term burdens of infertility and oncogenesis.

By editing HSCs directly in patients without transplantation, no myeloablative conditioning is required, thereby avoiding the adverse effects associated with current HSC transplantation protocols.

We believe such a product could be used in multiple types of healthcare settings with a much lower burden on patients and treatment sites, potentially enabling the treatment of larger patient populations around the world.

We are on track to declare a development candidate in HSCs for the in vivo treatment of SCD and TDT in mid-2025.

Our discovery and development efforts are also focused on in vivo gene editing medicines in liver cells and other tissues, with the goal of selecting therapeutic targets we believe have a significant probability of technical, clinical, regulatory, and commercial success.

We are on track to declare a development candidate for an in vivo gene editing medicine in liver cells for an undisclosed indication in mid-2025 and to establish and disclose an extrahepatic, non-HSC target cell type or tissue by the end of 2025.

In January 2025, we announced our strategic priorities through 2027, aimed to deliver transformative in vivo gene editing medicines for people living with serious diseases around the world.

Specifically, we aim to:

Launch clinical trials for multiple in vivo programs , including submitting at least one investigational new drug application or clinical trial application by mid-2026, beginning human trials by the second half of 2026, and initiating at least one late-stage clinical trial in the second half of 2027;

Achieve human in vivo proof of concept in at least one indication by the end of 2026 , validating our in vivo upregulation strategy in humans;

Expand the range of diseases addressable by in vivo gene upregulation , including announcing in vivo proof of concept in at least one additional tissue beyond HSCs and the liver by 2027, demonstrating the “plug ‘n play” potential of our proprietary extrahepatic LNP platform.

To that end, our preclinical efforts are also focused on the creation of a “plug ‘n play” lipid nanoparticle (“LNP”) platform to enable targeted delivery of in vivo gene editing medicines to multiple cells and tissues, including the liver, hematopoietic stem cells (“HSCs”), and other cells and tissues.

In September 2025, we announced the nomination of our lead in vivo development candidate, EDIT-401, an experimental, potential best-in-class, one-time therapy to significantly reduce LDL-cholesterol (“LDL-C”) through upregulation of the LDL receptor (“LDLR”).

EDIT-401 is designed to treat elevated levels of LDL-C, or hyperlipidemia, by directly editing the noncoding region of the LDLR gene to increase LDLR protein expression and reduce LDL-C levels.

This targeted approach has demonstrated an approximately 90% mean reduction of LDL-C in non-human primates (“NHPs”) in our preclinical studies with favorable tolerability data and supports the potential of EDIT-401 to deliver meaningful clinical outcomes for patients underserved by current lipid-lowering therapies.

We are on track to submit an investigational new drug application (“IND”) or foreign equivalent to conduct a clinical trial of EDIT-401 in patients with heterozygous familial hypercholesteremia (“HeFH”), a common inherited genetic disorder causing extremely high LDL-C, by mid-2026 with the expectation of achieving early human proof-of-concept data for EDIT-401 by the end of 2026.

We plan to complete enrolling the dose-finding portion of the first-in-human clinical trial with topline data results available in 2027.

We expect to present additional preclinical data for EDIT-401 by mid-2026.

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We also previously demonstrated proof-of-concept of our functional upregulation strategy in our former clinical trials of renizgamglogene autogedtemcel (“reni-cel”), an experimental ex vivo gene-edited medicine to treat sickle cell disease (“SCD”), a severe inherited blood disease that causes vaso-occlusive events and target organ damage, and often leads to premature death, and transfusion-dependent beta thalassemia (“TDT”), the most severe form of beta-thalassemia, an inherited blood disorder characterized by severe anemia.

Despite the robust and clinically meaningful improvements observed in these trials, we determined in December 2024 not to pursue commercialization for reni-cel in order to optimize our cost structure and accelerate our intent to achieve human proof of concept of our in vivo gene editing medicines.

Leveraging our differentiated approach from our former reni-cel program, we are developing an experimental in vivo gene-editing medicine to edit HSCs through targeted delivery of our AsCas12a enzyme to our clinically validated HBG1 and HBG2 promoter site.

Our in vivo discovery and development efforts further include other cells and tissues, with the goal of selecting therapeutic targets we believe have a significant probability of technical, clinical, regulatory, and commercial success.

We previously announced in vivo delivery to two additional cell types in humanized mice using our proprietary LNP targeting platform, demonstrating the “plug ‘n play” potential of our proprietary extrahepatic LNP platform.

Licensing and Business Development

We are pursuing the right combination of gene editing and targeted delivery tools through internal development and the in-licensing of complementary technologies to build our preclinical pipeline and accelerate the achievement of our goal of delivering lifesaving medicines to patients with previously untreatable diseases.

For example, in 2024 we secured a collaboration and license agreement accessing LNPs targeting the liver in support of our strategy to upregulate certain liver targets, including our first undisclosed liver upregulation target.

We are also evaluating technologies as part of a “plug ‘n play” in vivo gene editing LNP delivery platform designed to allow the rapid development of new medicines for extrahepatic tissues.

We are also leveraging our gene editing technology in areas outside of our core focus through partnerships and collaborations.

For example, in cellular therapy medicines, we are leveraging partnerships to progress engineered cell medicines to treat various cancers.

We are advancing alpha-beta T-cell experimental medicines for the treatment of solid and liquid tumors in collaboration with Bristol Myers Squibb Company (“BMS”) through its wholly owned subsidiary, Juno Therapeutics, Inc.

(“Juno Therapeutics”).

This collaboration, which leverages our Cas9 and AsCas12a platform technologies, has resulted in 14 total programs.

We are pursuing the right combination of gene editing and targeted delivery tools through internal development and the in-licensing of complementary technologies to build our preclinical pipeline, including our “plug ‘n play” in vivo gene editing LNP delivery platform, and accelerate the achievement of our goal of delivering lifesaving medicines to patients with previously untreatable diseases.

For example, we are party to a collaboration and license agreement accessing LNPs targeting the liver in support of our strategy to upregulate certain liver targets, including LDLR for our EDIT-401 program.

We are also leveraging our gene editing technology to drive the development of CRISPR-based medicines in therapeutic areas outside of our core focus through partnerships and collaborations.

For example, our collaboration with Bristol Myers Squibb Company (“BMS”) through its wholly owned subsidiary, Juno Therapeutics, Inc.

(“Juno Therapeutics”) is advancing alpha-beta T-cell experimental medicines for the treatment of solid tumors, liquid tumors, and autoimmune disease in collaboration.

This collaboration, which leverages our Cas9 and AsCas12a platform technologies, has resulted in 14 total programs, including BMS’ CD19 HD Allo CAR T program for the treatment of autoimmune disease currently in Phase I clinical development.

We are also party to a non-exclusive collaboration and licensing agreement with Immatics to combine gamma-delta T cell adoptive cell therapies and gene editing for the treatment of cancer.

In January 2023, we sold our preclinical multiplexed edited induced human pluripotent stem cell -derived natural killer (“iNK”) cell medicine for the treatment of solid tumors, as well as an additional iNK program under development and certain related manufacturing technologies, to Shoreline Biosciences, Inc., and granted Shoreline a non-exclusive license to our gene editing technology to develop allogeneic gene editing natural killer cell medicines.

In addition, we are leveraging our intellectual property portfolio to drive potential out-licensing opportunities that can provide non-dilutive capital or more broadly enable programs to more rapidly move towards commercialization and benefit patients.

In December 2023, we and Vertex Pharmaceuticals Incorporated (“Vertex”) entered into a license agreement, under which Vertex obtained a non-exclusive license for our Cas9 gene editing technology for ex vivo gene

editing medicines targeting the BCL11A gene in the fields of SCD and TDT, including Vertex’s CASGEVY TM (exagamglogene autotemcel).

In addition, we are leveraging our intellectual property portfolio to drive potential out-licensing opportunities that can provide non-dilutive capital.

In December 2023, we and Vertex Pharmaceuticals Incorporated (“Vertex”) entered into a license agreement (the “Vertex License Agreement”), under which Vertex obtained a non-exclusive license for our Cas9 gene editing technology for ex vivo gene editing medicines targeting the BCL11A gene in the fields of SCD and TDT, including Vertex’s CASGEVY TM (exagamglogene autotemcel).

We received a $50.0 million upfront cash payment in the fourth quarter of 2023 and the 2024 annual license fee of $10.0 million in the first quarter of 2024.

The license agreement further provides for the payment by Vertex of a potential additional $50.0 million contingent upfront payment and further future fixed and sales-based annual license fees, ranging from $10.0 million to $40.0 million annually, inclusive of certain sales-based annual license fee increases, through 2034.

The Vertex License Agreement further provides for the payment by Vertex of a potential additional $50.0 million contingent upfront payment and further future fixed and sales-based annual license fees, ranging from $5.0 million to $40.0 million annually, inclusive of certain sales-based annual license fee increases, through 2034.

We are required to pay The Broad Institute, Inc.

(“Broad”) and the President and Fellows of Harvard College (“Harvard”) a mid-double-digit percentage of amounts payable to us from Vertex under the license agreement as it relates to Cas9 technology licensed by us from Broad and Harvard.

In October 2024, we entered into an agreement (the “DRI Agreement”) with a wholly owned subsidiary of DRI Healthcare Trust (“DRI”) providing for an upfront cash payment by DRI to us of $57.0 million in exchange for the acquisition by DRI of up to 100% of certain of the annual license fees owed to us under the Vertex license agreement, which fees range from $5.0 million to $40.0 million per year (inclusive of certain sales-based annual license fees that may become due), and a mid-double-digit percentage of the $50.0 million contingent upfront payment, in each case after subtracting amounts owed by us to Broad and Harvard.

We have retained rights to our portions of certain other sales-based annual license fees and the contingent upfront payment that may become due under the license agreement with Vertex, and the amounts that correspond to our licensor obligations.

In August 2023, we entered into a license agreement with Vor Biopharma Inc.

(“Vor Bio”), providing Vor Bio a non-exclusive license for the development of ex vivo Cas9 gene edited HSC therapies for the treatment and/or prevention of hematological malignancies.

Under this agreement, we received an upfront payment and are eligible for future development, regulatory and commercial milestone payments, as well as royalties on medicines utilizing the related intellectual property.

(“Broad”) and the President and Fellows of Harvard College (“Harvard”) a mid-double-digit percentage of amounts payable to us from Vertex under the Vertex License Agreement as it relates to Cas9 technology licensed by us from Broad and Harvard.

Under the DRI Agreement, DRI is purchasing up to 100% of certain future fixed and sales-based annual license fees that the Company is entitled to receive under the Vertex License Agreement, which fees range from $5.0 million to $40.0 million per year, including increases based on sales.

In addition, DRI is purchasing a mid-double-digit percentage of a $50.0 million contingent upfront payment that the Company may receive under the Vertex License Agreement.

All amounts above will be adjusted to exclude payments that the Company owes to Broad and Harvard.

The Company has retained rights to certain portions of certain other sales-based annual license fees and the contingent upfront payment that may become due under the Vertex License Agreement, and the amounts that correspond to our licensor obligations.

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Our Core Capability — Gene Editing

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Gene editing typically takes advantage of naturally occurring DNA repair mechanisms, including non-homologous end joining (“NHEJ”) and homology directed repair (“HDR”), to achieve its desired therapeutic outcome.

Edits that are repaired by NHEJ typically result in an insertion or deletion (indel) that can disrupt a regulatory sequence to functionally upregulate normal gene expression and normal protein function.

Edits that are repaired by NHEJ typically result in an insertion or deletion (an “indel”) that can disrupt a regulatory sequence to functionally upregulate normal gene expression and normal protein function.

Edits that are repaired by HDR, including targeted insertion, aim to correct or replace aberrant DNA sequences.

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The PAM sequence that is recognized by the DNA endonuclease creates a second layer of recognition in addition to the guide RNA.

We believe that CRISPR technology has three principal advantages for genome editing:

We believe that CRISPR technology has three principal advantages for gene editing:

• Rapid, comprehensive, and systematic identification of product candidates.

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This ability to target multiple DNA sequences expands the applicability of CRISPR technology and also creates the potential for self-regulating systems that control exposure to the editing machinery.

To address more than one

target, other gene editing technologies require the engineering, characterization, manufacture, and delivery of distinct nuclease proteins for each target.

To address more than one target, other gene editing technologies require the engineering, characterization, manufacture, and delivery of distinct nuclease proteins for each target.

• Ability to achieve a range of different types of edits.

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Thirdly, identifying Cas9 and Cas12a enzymes with different editing properties will expand the number of potential editing sites in the human genome.

The range of natural and engineered variants of Cas9 and Cas12a have significantly expanded the number of sites in the human genome that we can potentially target.

The range of natural and engineered

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variants of Cas9 and Cas12a have significantly expanded the number of sites in the human genome that we can potentially target.

As compared to the most commonly used, naturally occurring version of Cas9, from the bacterial species S.

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Our strategy is to leverage existing delivery technologies to target cell types of interest while developing next generation capabilities as warranted.

We have developed a variety of delivery approaches, including lipid nanoparticles for non-viral in vivo delivery, AAVs for in vivo viral delivery, and electroporation for efficient delivery to blood cells ex vivo for our former reni-cel program.

We have made substantial advances in the in vivo delivery of CRISPR systems to a number of cell types.

Using a novel tLNP platform and HSC targeting strategy, we have achieved in vivo HSPC editing at a level of 29% and HSC editing of approximately 40% after a single dose in a humanized mouse model.

Subsequently, our initial non-human primate study, which used one of our optimized tLNPs, resulted in delivery of the GFP reporter to to 75% of HSCs and achieved on average 17% HBG1/2 allelic editing after a single dose.

By substituting different targeting ligands conjugated to the tLNP, we expect our proprietary tLNP platform to enable specific targeting of different cell types.

In addition, using a separate LNP platform for liver delivery, in a non-human primate study, we achieved editing of approximately 65%, near the theoretical maximum liver editing of 70%, reflecting efficient editing of hepatocytes, with negligible editing in non-target tissues using our AsCas12a enzyme and a liver LNP.

To date, we have achieved in vivo delivery to two extra-hepatic, non-HSC cells in humanized mice.

Our “plug ‘n play” LNP platform is designed to enable targeted delivery of in vivo gene editing medicines to multiple cells and tissues, including the liver, HSCs, and other cells and tissues.

Our ability to deliver to the liver using this LNP platform enables us to develop new therapies by only reprogramming guide RNAs for specific liver targets.

For extrahepatic delivery, our LNP platform enables us to target multiple tissues through the substitution of different targeting ligands conjugated to the LNP.

For example, using the LNP platform for delivery to extrahepatic tissues, we achieved in vivo preclinical proof-of-concept editing of HSCs in NHPs and to date have also achieved in vivo delivery to two extrahepatic, non-HSC cells in humanized mice.

To optimize the specificity of our product candidates, there are a number of different aspects of the product configuration that we customize in addition to the sequence and quality of the guide RNA, including the length of the guide RNA, the type of Cas9 or Cas12a enzyme, including engineered forms, the delivery vector, including targeting moiety, and the duration of exposure, all of which contribute to overall specificity.

Our Gene Editing Medicine Programs

Our research and development efforts are focused on next generation in vivo medicines, including in vivo editing of HSCs, liver cells and other tissues.

Our research and development efforts are focused on next generation in vivo medicines, including in vivo editing of liver cells, HSCs and other tissues.

Our product development strategy is to target diseases where gene editing can be used to enable or enhance therapeutic outcomes for patients, while maximizing probability of technical, clinical, regulatory and commercial success.

We believe the therapeutic programs and delivery technologies we have chosen to pursue to date and those that are currently under development will demonstrate the depth and breadth of our ability to deploy our genome editing platform to develop differentiated, transformational medicines for previously untreatable diseases.

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editing platform to develop differentiated, transformational medicines for previously untreatable diseases.

The following summarizes our research programs and disease areas:

Cardiovascular

Our lead program, EDIT-401, is an experimental, potential best-in-class, one-time therapy designed to significantly reduce LDL-cholesterol (“LDL-C”) through the upregulation of the LDL receptor (“LDLR”).

Hyperlipidemia, or elevated levels of LDL-C, affects an estimated 70 million people in the United States, including approximately 1.2 million people who have HeFH.

Hyperlipidemia is a major causal factor for atherosclerotic cardiovascular disease (“ASCVD”), a serious condition driven by cholesterol-rich plaque accumulation in the arteries that can lead to severe events like heart attack and stroke.

ASCVD is the leading cause of death worldwide, and in the United States alone is projected to result in over $300 billion in related expenditures by 2035.

We estimate that approximately 15 million people in the United States suffer from ASCVD.

Lower LDL-C levels is well correlated to reduced risk of cardiovascular event.

The current standard of care for hyperlipidemia includes the use of statin therapy with additional treatment modalities to achieve specified targets for LDL-C reduction based on patient risk level, such as the inclusion of PCSK9 inhibitors.

This current standard of care has demonstrated a mean LDL-C reduction ranging from 40% to 60%, but can require multiple therapies and life-long administration, frequently resulting in non-adherence.

Non-adherence and treatment underutilization has resulted in approximately 75% of patients with established cardiovascular disease failing to achieve LDL-C targets, creating a significant unmet need across multiple at-risk segments of patients with hyperlipidemia.

EDIT-401 uses a differentiated upregulation strategy designed to significantly reduce LDL-C and treat hyperlipidemia.

Using our Cas9 gene editing nuclease and dual guide RNAs, EDIT-401 disrupts DNA sequences that inhibit expression of the LDLR gene, thereby increasing LDLR protein expression to reduce LDL-C levels.

In preclinical studies in NHPs, EDIT-401 achieved LDL-C reductions equal to or exceeding 90% within 48 hours of a single dose of EDIT-401.

EDIT-401 also achieved LDL-C reductions equal to or exceeding 90% in mice with high baseline LDL-C and reduced LDLR function.

The EDIT-401 LDL-C reductions were achieved with an at least six-fold mean increase in LDLR protein in the NHP liver, requiring only approximately 10-40% functional editing of LDLR alleles.

LDL-C reduction was maintained in mouse models in a three-month study, demonstrating the durability of effect.

EDIT-401 was well tolerated across all doses administered and no adverse clinical observations were noted.

While there were some transient increases in liver enzymes, these resolved within a week.

We are on track to submit an IND or foreign equivalent to conduct a clinical trial of EDIT-401 in patients with HeFH by mid-2026 with the expectation of achieving early human proof-of-concept data

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for EDIT-401 by the end of 2026.

We plan to complete enrolling the dose-finding portion of the first-in-human clinical trial with topline data results available in 2027.

We expect to present additional preclinical data for EDIT-401 by mid-2026.

Owing to the physiological relationship between LDL-C and Lp(a), and the observation that Lp(a) levels have been reduced with PCSK9 inhibitors, we are exploring the effects of EDIT-401, our LDLR upregulation program, on Lp(a) levels in pre-clinical species.

Hemoglobinopathies

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Sickle cell disease is an inherited life-threatening hematological disorder, which starts to cause serious complications in early childhood.

It affects millions of people worldwide, including approximately 100,000 people in the United States, and over 300,000 babies are born annually with SCD globally.

It affects millions of people worldwide, including approximately 100,000 people in the United States.

Patients suffering from SCD can experience severe anemia and sickling-induced blood vessel blockages resulting in unpredictable and severe attacks of acute pain, stroke, acute chest syndrome, liver disease, renal failure, and a shortened life span.

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TDT is characterized by severe anemia, hemolysis, and severe ineffective erythropoiesis, often requiring patients to undergo regular, lifelong blood transfusions for survival.

We are actively pursuing a distinct gene editing approach to treating these hemoglobinopathies.

We aim to use our AsCas12a gene editing nuclease to target the HBG1/2 promoter of the gamma-globin gene in human CD34+ cells and disrupt the binding site of the BCL11A, consistent with observed naturally occurring human mutations.

These mutations mimic the asymptomatic condition of hereditary persistence of fetal hemoglobin (“HPFH”) with high levels of HbF in red blood cells.

We are pursuing a distinct gene editing approach to treating these hemoglobinopathies.

We aim to use our AsCas12a gene editing nuclease to target the clinically validated HBG1/2 promoter site of the gamma-globin gene in human CD34+ cells and disrupt the binding site of the BCL11A protein, consistent with observed naturally occurring human mutations.

These mutations mimic the asymptomatic condition of hereditary persistence of fetal hemoglobin with high levels of fetal hemoglobin (“HbF”) in red blood cells.

HbF levels greater than 30% are associated with a significant reduction in, or the absence of, SCD symptoms, particularly vaso-occlusive events (“VOEs”).

By editing the HBG1/2 promoter in the gamma-globin gene, we seek to

generate protective changes that increase HbF production in a manner that is independent of erythropoietic stress, resulting in reduced sickling and VOEs in SCD patients, and resolving anemia and transfusion dependence in TDT patients.

We have focused our efforts on editing a site within the beta-globin locus, which includes the gamma-globin gene, that we believe has the potential to create superior expression of HbF since patients with elevated HbF levels have better clinical outcomes.

We believe that beta-globin expression can be impacted by increasing HbF and decreasing sickle globin.

In particular, our preclinical data shows that editing the HBG1/2 promotor of the gamma-globin gene in human CD34+ cells results in greater red blood cell production when compared to editing the BCL11A erythroid enhancer (“BC11Ae”) in the BCL11a gene.

Our preclinical studies thereby identified the potential that editing the BC11Ae locus might result in deleterious lineage skewing.

Our preclinical studies also demonstrated that editing the HBG1/2 promotor in human CD34+ cells results in greater HbF expression in erythroid lineage progeny when compared to editing the BCL11Ae.

Likewise, we believe our approach will reduce the sickle globin and, thereby not worsen alpha/beta globin chain imbalance as has been reported with lentiviral gene therapy in patients with SCD and alpha-thalassemia trait.

To get the high levels of beta-globin required for an efficacious therapy, there will be cells in the CD34+ population, which are cells that contain the long-term stem cells that repopulate the hematopoietic lineages, that carry more than twenty copies of the viral genome.

These random integration events have the potential to inadvertently activate or inactivate genes involved in cell function and tumorigenesis.

As such, we believe our approach to editing the beta-globin locus provides the highest likelihood of providing clinical benefit in patients while minimizing potential safety risks.

In addition to our selection of the HBG1/2 promoter as our target, we believe that our use of AsCas12a, our highly specific and efficient proprietary engineered CRISPR nuclease, potentially provides patients with better clinical outcomes than those gene editing medicines using a Cas9 enzyme.

AsCas12a has demonstrated superior characteristics that increase the efficiency of editing and significantly reduce off-target editing when compared to other CRISPR nucleases, including Cas9.

When combined with our comprehensive off-target assessment, which uses multiple statistically independent methods, including in silico, biochemical, and cellular, followed by a verification of candidate sites, our use of the high-specificity AsCas12a enzyme should provide high editing efficiency and low off-target editing.

Prior to the discontinuation of the clinical development of reni-cel, our experimental ex vivo gene editing medicine previously under investigation for the treatment of SCD and TDT, we investigated reni-cel in a single Phase 1/2/3 clinical trial, referred to as the RUBY trial, for the treatment of severe SCD and in a Phase 1/2 clinical trial for the treatment of TDT, referred to as the EdiTHAL trial.

We believe the RUBY and EdiTHAL clinical trials of reni-cel validated our distinct gene editing approach to treating hemoglobinopathies.

Reni-cel consists of patient-derived CD34+ HSPCs edited at the gamma globin gene ( HBG1 and HBG2 ) promoters by AsCas12a.

Despite the robust and clinically meaningful improvements observed in these trials, we determined in December 2024 not to pursue commercialization for reni-cel in order to optimize our cost structure and accelerate our intent to achieve in vivo human proof of concept in approximately two years.

Building on our experience with reni-cel, in October 2024, we announced that we achieved in vivo preclinical proof of concept of HBG1/2 editing in HSPC and HbF induction in humanized mice engrafted with human HSPCs and lacking their own hematopoietic cells.

Utilizing an HSC targeting strategy and a novel, Editas-proprietary tLNP for extrahepatic tissue delivery, we achieved an editing level of 29% in HSPCs and 40% in HSCs after a single dose.

Editing with our proprietary tLNP formulation resulted in the functional outcome of HbF induction, indicated by the presence of HbF expressing human red blood cells (on average 20%) that populate in the host by one month.

More recently, in January 2025, we announced new in vivo preclinical proof of concept data in non-human primates editing HSCs.

In this initial non-human primate study , we achieved delivery of the GFP reporter to 75% of HSCs and achieved on average 17% HBG1/2 allelic editing after a single dose, resulting in a mean editing of 17%, near the 20% predicted therapeutically relevant editing threshold based on allogeneic HSC transplant data.

We are currently evaluating further optimized tLNP formulations we expect to enable us to achieve therapeutic editing levels.

Liver and Other Cells/Tissues

We seek to additionally develop in vivo gene editing medicines targeted to liver cells and other cells/tissues.

In preclinical studies for an undisclosed liver target, we have achieved an editing level of approximately 65% in non-human primates, near the theoretical maximum liver editing level of 70%, with minimal non-target tissue editing using AsCas12a and a liver targeted LNP.

This editing resulted in robust serum biomarker reduction from the baseline of approximately 80%.

We believe our proprietary targeting LNP platform will enable specific targeting of different extrahepatic cell types by substitution of different targeting ligands conjugated to the LNP.

In preclinical studies, we have demonstrated in vivo proof of concept for this “plug n’ play” LNP delivery platform to two additional cell types beyond HSCs in humanized mice.

By editing the HBG1/2 promoter in the gamma-globin gene, we seek to generate protective changes that increase HbF production in a manner that is independent of erythropoietic stress, resulting in reduced sickling and VOEs in SCD patients, and resolving anemia and transfusion dependence in TDT patients.

Building on our experience with reni-cel, we have achieved in vivo preclinical proof-of-concept data of HSC editing in NHPs.

We intend to continue optimizing candidates for our HSC program, but plan to focus our resources on the advancement of our lead EDIT-401 program to human proof-of-concept.

Other Cells/Tissues

We seek to additionally develop in vivo gene editing medicines targeted to other cells and tissues.

We believe our proprietary targeting LNP platform can enable specific targeting of different extrahepatic cell types by substitution of different targeting ligands conjugated to the LNP.

We previously announced in vivo delivery to two additional cell types in humanized mice using our proprietary LNP targeting platform, demonstrating in vivo proof of concept for our “plug n’ play” LNP delivery platform .

Licensing and Business Development

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Through in-licensing of complementary technologies, we can expand our existing gene editing platform and further drive the development of our in vivo pipeline.

This was recently demonstrated with our entry into a collaboration and license agreement to access LNPs targeting the liver.

Using this in-licensed LNP, we achieved in vivo editing of hepatocytes in non-human primates, furthering our strategy to upregulate certain liver targets, including our first undisclosed liver upregulation target.

This was demonstrated with our entry into a collaboration and license agreement to access LNPs targeting the liver.

Using this in-licensed LNP, we achieved in vivo editing of hepatocytes in NHPs, furthering our strategy to upregulate certain liver targets, including LDLR for EDIT-401.

Further, as the exclusive licensee of Broad and Harvard’s Cas9 patent estates and Broad’s Cas12a patent estate for human medicines, we actively seek opportunities to out-license and partner our robust intellectual property portfolio to drive the development of CRISPR-based medicines in therapeutic areas outside of our core focus and to provide non-dilutive capital.

Examples of these licensing efforts include our non-exclusive license to Vor for the development of ex vivo Cas9 gene-edited HSC therapies for the treatment and/or prevention of hematological malignancies;

our non-exclusive license to Vertex for our Cas9 gene editing technology for ex vivo gene editing medicines targeting the BCL11A gene in the fields of SCD and TDT, including Vertex’s CASGEVY TM (exagamglogene autotemcel);

and our non-exclusive license to Immatics of our AsCas12a technology to advance gamma-delta T cell therapies for the treatment of cancer.

In our collaboration with BMS, we are researching and developing engineered alpha-beta T cell therapies to treat solid and liquid tumors leveraging our platform technologies, including Cas9 and AsCas12a.

Engineered T cells, including alpha-beta T cells, have shown encouraging clinical activity against multiple cancers, culminating in recent approvals of such therapies in the United States.

Examples of these licensing efforts include our non-exclusive license to Vertex for our Cas9 gene editing

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technology for ex vivo gene editing medicines targeting the BCL11A gene in the fields of SCD and TDT, including Vertex’s CASGEVY TM (exagamglogene autotemcel);

our non-exclusive license to Immatics of our AsCas12a technology to advance gamma-delta T cell therapies for the treatment of cancer;

and our collaboration with BMS.

Certain of our current collaborators and partners are expected to achieve clinical milestones in the next 12 to 18 months, which would result in milestone payments to us.

Our collaboration with BMS is advancing engineered alpha-beta T cell therapies to treat solid tumors, liquid tumors, and autoimmune disease leveraging our platform technologies, including Cas9 and AsCas12a.

For example, engineered T cells, including alpha-beta T cells, have shown encouraging clinical activity against multiple cancers, culminating in recent approvals of such therapies in the United States.

Because of these promising results, there is significant interest in the medical community in expanding the application of this technology across a broader range of cancers and patients.

We believe that our genome editing technology has the potential to improve multiple properties of these alpha-beta T cell therapies.

We believe that our gene editing technology has the potential to improve multiple properties of these alpha-beta T cell therapies.

Alpha-beta cells are part of the adaptive immune system and recognize tumors with endogenous alpha-beta T cell receptors or CARs or engineered T cell receptors (“eTCRs”).

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Through our collaboration with BMS, we have applied our Cas9 and AsCas12a platform technologies to multiple gene targets in order to improve the efficacy and safety of CAR/eTCR alpha-beta T cells directed against a range of tumor types.

In addition, we have optimized genome editing components and delivery methods compatible with engineered alpha-beta T cell manufacturing methods developed by BMS.

To date, this collaboration has resulted in 14 total programs.

In addition, we have optimized gene editing components and delivery methods compatible with engineered alpha-beta T cell manufacturing methods developed by BMS.

To date, this collaboration has resulted in 14 total programs, including BMS’ CD19 HD Allo CAR T program for the treatment of autoimmune disease currently in Phase I clinical development.

Our Collaborations and Licensing Strategy

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Under the BMS Collaboration Agreement, if BMS elects to opt-in with respect to a Research Program, it shall make a mid-six digit dollar payment to us and we shall amend the BMS License Agreement to include such Research Program by executing a licensed program addendum for such Research Program.

Following BMS ’ opt-in for each program we shall grant to BMS an exclusive (even as to us), royalty-bearing worldwide right and license under specified intellectual property rights to research, develop, manufacture commercialize or otherwise exploit the RNP Complexes in such Research Program to create products containing, incorporating, comprising or containing Alpha-beta T Cells and/or Other Derived T Cells, in each case modified using the RNP Complexes in such Research Program (each, a “ BMS Licensed Product”).

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Program to create products containing, incorporating, comprising or containing Alpha-beta T Cells and/or Other Derived T Cells, in each case modified using the RNP Complexes in such Research Program (each, a “ BMS Licensed Product”).

We are entitled to receive high single-digit to low double-digit percentage royalties on net sales made by BMS , its affiliates and sublicensees of any BMS Licensed Products, subject to reductions in certain circumstances.

We are also entitled to receive development milestones totaling up to $135.0 million in the aggregate upon achievement of certain clinical milestones and specified regulatory approvals and commercial milestone payments totaling up to $60.0 million in the aggregate for each of the first two BMS Licensed Products to achieve specified net sales milestones.

We have agreed during the term of the BMS Collaboration Agreement not to use (directly or indirectly), or license others to use, genome editing technology in connection with any research, development, manufacture, commercialization or other exploitation of any Alpha-beta T Cells or Other Derived T Cells.

We have agreed during the term of the BMS Collaboration Agreement not to use (directly or indirectly), or license others to use, gene editing technology in connection with any research, development, manufacture, commercialization or other exploitation of any Alpha-beta T Cells or Other Derived T Cells.

Our exclusivity obligation will not apply to activities related to (i) any identified RNP Complexes in a program for which BMS elects not to exercise its opt-in right, (ii) certain of our existing obligations to third parties, and (iii) certain existing programs of an acquiror of our company in a change of control.

We have agreed during the term of any licensed program addendum under the BMS License Agreement not to use (directly or indirectly), or license others to use, any genome editing technology that modulates or recognizes a gene target covered by such licensed program addendum for the conduct of any research, development, manufacture, commercialization or other exploitation with respect to any product that constitutes, incorporates, comprises or contains any Alpha-beta T Cell or Other Derived T Cells.

We have agreed during the term of any licensed program addendum under the BMS License Agreement not to use (directly or indirectly), or license others to use, any gene editing technology that modulates or recognizes a gene target covered by such licensed program addendum for the conduct of any research, development, manufacture, commercialization or other exploitation with respect to any product that constitutes, incorporates, comprises or contains any Alpha-beta T Cell or Other Derived T Cells.

The BMS Collaboration Agreement continues in effect until the later of expiration of the research term or expiration of the last to expire of BMS ’ right to opt-in with respect to any Research Program.

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We are a party to a number of license agreements under which we license patents, patent applications, and other intellectual property from third parties.

The licensed intellectual property covers, in part, CRISPR-related compositions of matter and their use for genome editing.

The licensed intellectual property covers, in part, CRISPR-related compositions of matter and their use for gene editing.

These licenses impose various diligence and financial payment obligations on us.

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converting the exclusive license to three specified targets to a non-exclusive license, subject to specified limitations;

revising certain provisions relating to the rights of Harvard and Broad to grant further licenses under specified circumstances to third parties that wish to develop and commercialize products that target a particular gene and that otherwise would fall within the scope of our exclusive license;

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otherwise would fall within the scope of our exclusive license;

and providing Harvard and Broad with certain rights to designate, and reserve all rights to, gene targets for which the designating institution has an interest in researching and developing products that would otherwise be covered by rights licensed to us.

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The March 2017 amendment to the Cas9-I License Agreement included a license to Rockefeller’s rights in such patents and patent applications.

The Harvard/Broad Cas9-I Patent Rights are directed, in part, to certain CRISPR/Cas9 compositions of matter and their use for genome editing and to certain CRISPR/Cas9 related delivery technologies.

The Harvard/Broad Cas9-I Patent Rights are directed, in part, to certain CRISPR/Cas9 compositions of matter and their use for gene editing and to certain CRISPR/Cas9 related delivery technologies.

Pursuant to the Cas9-I License Agreement, and as of December 31, 2025, we have certain rights under 85 U.S.

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In addition, certain rights granted to us under the Cas9-I License Agreement are further subject to a non-exclusive license to the Howard Hughes Medical Institute for research purposes.

Our exclusive license rights also are subject to rights retained by Broad, Harvard, MIT, and Rockefeller any third party to research, develop, make, have made, use, offer for sale, sell, have sold, import or otherwise exploit the Harvard/Broad Cas9-I Patent Rights and licensed products as research products or research tools, or for research purposes.

Our exclusive license rights also are subject to rights retained by Broad, Harvard, MIT, and Rockefeller for any third party to research, develop, make, have made, use, offer for sale, sell, have sold, import or otherwise exploit the Harvard/Broad Cas9-I Patent Rights and licensed products as research products or research tools, or for research purposes.

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We have the right to sublicense our licensed rights provided that the sublicense agreement must be in compliance and consistent with the terms of the Cas9-I License Agreement.

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In addition, any sublicense agreements must contain certain terms, including a provision requiring the sublicensee to indemnify Harvard, Broad, MIT, and Howard Hughes Medical Institute according to the same terms as are provided in the Cas9-I License Agreement and a statement that Broad, Harvard, MIT, and Howard Hughes Medical Institute are intended third party beneficiaries of the sublicense agreement for certain purposes.

Under the agreement, Harvard and Broad also retained rights to grant further licenses under specified circumstances to third parties, other than specified entities, that wish to develop and commercialize products that target a particular gene and that otherwise would fall within the scope of our exclusive license from Harvard and Broad.

Under the Cas9-I License Agreement, Harvard and Broad also retained rights to grant further licenses under specified circumstances to third parties, other than specified entities, that wish to develop and commercialize products that target a particular gene and that otherwise would fall within the scope of our exclusive license from Harvard and Broad.

If a third party requests a license under the Harvard/Broad Cas9-I Patent Rights for the development and commercialization of a product that would be subject to our exclusive license grant from Harvard and Broad under the Cas9-I License Agreement, Harvard and Broad may notify us of the request (the “Cas9-I Third Party Proposed Product Requests”).

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Patent and Trademark Office (“USPTO”), any opposition proceedings in the European Patent Organization, or any other inter partes or other post grant proceedings in these or other jurisdictions where we are seeking patent protection.

Therefore, we are obligated to reimburse Broad and/or Harvard for expenses associated with the interference and

opposition proceedings involving patents licensed to us under this agreement (described in more detail under Part I, Item 1A “Risk Factors—Risks Related to Our Intellectual Property—Some of Our In-Licensed Patents are Subject to Priority and Validity Disputes” of this Annual Report on Form 10-K).

Therefore, we are obligated to reimburse Broad and/or Harvard for expenses associated with the interference and opposition proceedings involving patents licensed to us under this agreement (described in more detail under Part I, Item 1A “Risk Factors—Risks Related to Our Intellectual Property—Some of Our In-Licensed Patents are Subject to Priority and Validity Disputes” of this Annual Report on Form 10-K).

Broad and Harvard are collectively entitled to receive clinical and regulatory milestone payments totaling up to $14.8 million in the aggregate per licensed product approved in the United States, the European Union (the “EU”) and Japan for the prevention or treatment of a human disease that afflicts at least a specified number of patients in the aggregate in the United States.

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Broad and Harvard are collectively entitled to receive clinical and regulatory milestone payments totaling up to $4.1 million in the aggregate per licensed product approved in the United States and at least one jurisdiction outside the United States for the prevention or treatment of a human disease that afflicts fewer than a specified number of patients in the aggregate in the United States or a specified number of patients per year in the United States, which we refer to as an ultra-orphan disease.

We are also obligated to make additional payments to Broad and Harvard, collectively, of up to an aggregate of $36.0 million upon the occurrence of certain sales milestones per licensed product for the prevention or treatment of an ultra-orphan disease.

We are also obligated to make additional payments

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to Broad and Harvard, collectively, of up to an aggregate of $36.0 million upon the occurrence of certain sales milestones per licensed product for the prevention or treatment of an ultra-orphan disease.

Broad and Harvard, collectively, are entitled to receive mid single-digit percentage royalties on net sales of licensed products for the prevention or treatment of human disease, and ranging from low single-digit to high single-digit percentage royalties on net sales of other licensed products and services, made by us, our affiliates, or our sublicensees.

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Nevertheless, we remain responsible for the cost of such interference or derivation proceeding.

We are responsible for the cost of the interference proceeding and appeal with respect to these patents and this patent application.

We are responsible for the cost of the interference proceeding and appeal with respect to the subject patents.

Broad and Harvard are required to maintain any application or patent within the Harvard/Broad Cas9-I Patent Rights so long as we meet our obligation to reimburse Broad and Harvard for expenses related to prosecution and there is a good faith basis for doing so.

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Broad and Harvard may terminate the agreement upon a specified period of notice in the event of our uncured material breach, such notice period varying depending on the nature of the breach.

Both Broad and Harvard may terminate the Cas9-I License Agreement immediately if we challenge

the enforceability, validity, or scope of any Harvard/Broad Cas9-I Patent Right or assist a third party to do so, or in the event of our bankruptcy or insolvency.

Both Broad and Harvard may terminate the Cas9-I License Agreement immediately if we challenge the enforceability, validity, or scope of any Harvard/Broad Cas9-I Patent Right or assist a third party to do so, or in the event of our bankruptcy or insolvency.

Neither Broad nor Harvard acting alone has the right to terminate the Cas9-I License Agreement.

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In December 2016, we entered into a license agreement with Broad, for specified patent rights (“Cpf1 Patent Rights”) related primarily to Cas12a compositions of matter and their use for gene editing, which was amended in January 2021 and February 2024 (as amended, the “Cpf1 License Agreement”).

Pursuant to the Cpf1 License Agreement, Broad, on behalf of itself, Harvard, MIT, Wageningen University (“Wageningen”), and the University of Tokyo (“Tokyo” and collectively with the other institutions, the “Cpf1 Institutions”) granted us an exclusive, worldwide, royalty-bearing, sublicensable license to the Cpf1 Patent Rights, to make, have made, use, have used, sell, offer for sale, have sold, export and import products solely in the field of the prevention or treatment of human disease using gene therapy, editing of genetic material, or targeting of genetic material, subject to certain limitations and retained rights (collectively, the “Exclusive Cpf1 Field”), as well as a non-exclusive, worldwide, royalty-bearing, sublicensable license to the Cpf1 Patent Rights for all other purposes, subject to certain limitations and retained rights.

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“Exclusive Cpf1 Field”), as well as a non-exclusive, worldwide, royalty-bearing, sublicensable license to the Cpf1 Patent Rights for all other purposes, subject to certain limitations and retained rights.

The licenses granted to us under the Cpf1 License Agreement exclude certain fields, including human germline modification;

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Pursuant to the Cpf1 License Agreement, and as of December 31, 2025, we have certain rights under 18 U.S.

patents, nine pending U.S.

patent applications, six European patents and related validations, six pending European patent applications, and other related patent applications in jurisdictions outside of the United States and Europe.

patents, eight pending U.S.

patent applications, six European patents and related validations, six pending European patent applications, and other related patents and patent applications in jurisdictions outside of the United States and Europe.

We are obligated to use commercially reasonable efforts to research, develop, and commercialize licensed products in the Exclusive Cpf1 Field.

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If a third party requests a license under the Cpf1 Patent Rights for the development and commercialization of a product that would be subject to our exclusive license grant from Broad (a “Cpf1 Third Party Proposed Product Request”), Broad may notify us of such request.

A Cpf1 Third Party Proposed Product Request must be accompanied by a research, development and commercialization plan reasonably satisfactory to Broad, including evidence that the third party has, or reasonably expects to have, access to any necessary

intellectual property and funding.

Broad may not grant a Cpf1 Third Party Proposed Product Request (i) if we, directly or through any of our affiliates, sublicensees, or collaborators are researching, developing, or commercializing a product directed to the same gene target that is the subject of the Cpf1Third Party Proposed Product Request (“Cpf1 Licensee Product”) and we can demonstrate such ongoing efforts to Broad’s reasonable satisfaction, or (ii) if we, directly or through any of our affiliates or sublicensees, wish to do so either alone or with a collaboration partner, and we can demonstrate to Broad’s reasonable satisfaction that we are interested in researching, developing, and commercializing a Cpf1 Licensee Product, that we have a commercially reasonable research, development, and commercialization plan to do so, and we commence and continue reasonable commercial efforts under such plan.

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The Cpf1 License Agreement also provides Broad with the right, subject to certain limitations, to designate gene targets for which Broad, whether alone or together with a Cpf1 Institution, affiliate or third party, has an interest in researching and developing products that would otherwise be covered by rights licensed to us under the Cpf1 License Agreement.

Broad may not so designate any gene target for which we, directly or through any of our affiliates, sublicensees, or collaborators, are researching, developing, or commercializing a product, or for which we can demonstrate to Broad’s reasonable satisfaction that we are interested in researching, developing, and commercializing a product, that we have a commercially reasonable research, development, and commercialization plan to do so, and we commence and continue reasonable commercial efforts under such plan.

Broad may not so designate any gene target for which we, directly or through any of our affiliates,

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sublicensees, or collaborators, are researching, developing, or commercializing a product, or for which we can demonstrate to Broad’s reasonable satisfaction that we are interested in researching, developing, and commercializing a product, that we have a commercially reasonable research, development, and commercialization plan to do so, and we commence and continue reasonable commercial efforts under such plan.

If we, directly or through any of our affiliates, sublicensees, or collaborators, are not researching, developing, or commercializing a product directed toward the gene target designated by Broad and are not able to develop and implement a plan reasonably satisfactory to Broad, Broad is entitled to reserve all rights under the Cpf1 License Agreement, including the right to grant exclusive or non-exclusive licenses to third parties, to develop and commercialize products directed to such gene target, our license with respect to such gene target will terminate, and we will not be entitled under the Cpf1 License Agreement to develop and commercialize products directed to such gene target.

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If we sublicense any of the Cpf1 Patent Rights to a third party, Broad and Wageningen, collectively, had the right to receive high single-digit to low double-digit percentages of the sublicense income, which percentage decreased to a high single-digit percentage in 2022 for sublicenses executed thereafter.

Under the Cpf1 License Agreement, Broad and Wageningen are also entitled, collectively, to receive success payments in the event our market capitalization reaches specified thresholds ascending from a high nine digit dollar amount to $10.0 billion (“Market Cap Success Payments”) or sale of our company for consideration in excess of those thresholds,

(“Company Sale Success Payments,” which with the Market Cap Success Payments, the “Success Payments”).

Market Cap Success Payments are payable by us in cash, in shares of our common stock, with such shares being valued for such purpose at the closing price of our common stock as reported the Nasdaq Stock Market for the trading day immediately preceding the date of such payment if our common stock was then listed on the Nasdaq Stock Market, or in the form of promissory notes (the “Promissory Notes”).

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In 2017, two Market Cap Success Payments of $5.0 million each became due and payable and we issued Promissory Notes in such amounts, which we fully settled by issuing shares of our common stock in 2017 and 2018.

In December 2020, an additional Market Cap Success Payment of $15.0 million became due and payable, which we settled through the issuance of shares of our common stock in January 2021.

In December 2020, an additional Market Cap Success Payment of $15.0 million

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became due and payable, which we settled through the issuance of shares of our common stock in January 2021.

The remaining Success Payments that may be paid to Broad and Wageningen range from a low-eight digit dollar amount to a mid-eight digit dollar amount, and collectively will not exceed, in aggregate, $100.0 million, which maximum would be payable only if we achieve a market capitalization threshold of $10.0 billion and have at least one product candidate covered by a claim of a patent right licensed to us under either the Cpf1 License Agreement or the Cas9-I License Agreement that is or was the subject of a clinical trial pursuant to development efforts by us or any of our affiliates or sublicensees.

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Other Broad Agreements

In addition to the Cas9-I License Agreement and the Cpf1 License Agreement, in December 2016, we entered into a license agreement with Broad for certain Cas9 compositions of matter and their use for gene editing, which was amended

in January 2021 and February 2024 (as amended, the “Cas9-II Agreement”), and, in June 2018, we entered into a Sponsored Research Agreement with Broad providing for Broad to conduct research useful or relevant to genome editing in the field of genomic medicines for the prevention of treatment of human diseases with funding from us, which was amended in January 2021 (as amended, the “Sponsored Research Agreement”).

Under the Cas9-II Agreement and the Sponsored Research Agreement, we have potential obligations with respect to success payments, which are described in Note 8 to the Notes to Consolidated Financial Statements included in Part II, Item 8 “Financial Statements and Supplementary Data” of this Annual Report on Form 10-K.

In addition to the Cas9-I License Agreement and the Cpf1 License Agreement, in December 2016, we entered into a license agreement with Broad for certain patent rights covering Cas9 compositions of matter and their use for gene editing, which was amended in January 2021 and February 2024 (as amended, the “Cas9-II Agreement”), and, in June 2018, we entered into a Sponsored Research Agreement with Broad providing for Broad to conduct research useful or relevant to gene editing in the field of genomic medicines for the prevention of treatment of human diseases with funding from us, which was amended in January 2021 (as amended, the “Sponsored Research Agreement”).

Intellectual Property

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We seek to protect our proprietary position by, among other things, exclusively licensing and filing U.S.

and certain foreign patent applications related to our platform technology, existing and planned programs, and improvements that are important to the development of our business, where patent protection is available.

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We also rely on trade secrets, know-how, continuing technological innovation, and confidential information to develop and maintain our proprietary position and protect aspects of our business that are not amenable to, or that we do not consider appropriate for, patent protection.

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We also seek to preserve the integrity and confidentiality of our data and trade secrets by maintaining physical security of our premises and physical and electronic security of our information technology systems.

Our in-licensed patents and patent applications cover various aspects of our genome editing platform technology, including CRISPR systems that employ Cas9 including S.

Our in-licensed patents and patent applications cover various aspects of our gene editing platform technology, including CRISPR systems that employ Cas9 including S.

aureus Cas9, high-fidelity Cas9 nucleases and Cas9 PAM variants, self-inactivating forms of Cas9, Cas9 nickases, CRISPR systems that employ Cas12a including Cas12a nickases and other variants and self-inactivating forms of Cas12a, and also CRISPR systems that employ viral vectors for delivery, single guide RNAs, or modified guide RNAs, including guide nucleic acids containing both DNA and RNA components.

We also have filed patent applications and have in-licensed rights to filed patent applications directed to each of the four components of our genome editing platform technology.

We also have filed patent applications and have in-licensed rights to filed patent applications directed to each of the four components of our gene editing platform technology.

We intend to pursue, when possible, additional patent protection, including composition of matter, method of use, and process claims, directed to each component of our platform technology.

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non-provisional patent applications, 23 European patents and related validations, 59 pending European patent applications, 14 pending U.S.

provisional patent

applications, eight pending Patent Cooperation Treaty (“PCT”) patent applications, and other related patent applications in jurisdictions outside the United States and Europe that are related to our CRISPR technology and which include claims directed to our genome editing platform, including our directed editing component, as well as composition of matter and method of use claims for our therapeutic programs.

provisional patent applications, eight pending Patent Cooperation Treaty (“PCT”) patent applications, and other related patents and patent applications in jurisdictions outside the United States and Europe that are related to our CRISPR technology and which include claims directed to our gene editing platform, including our directed editing component, as well as composition of matter and method of use claims for our therapeutic programs.

Four of these U.S.

patents, one of these European patents and their U.S., European and foreign counterpart applications are co-owned with Broad and Iowa and we have obtained an exclusive license to such co-ownership rights from these third parties in the field of prevention or treatment of human disease using gene therapy or genome editing.

In addition, six of these issued U.S.

patents and 11 of these pending U.S.

In addition, seven of these issued U.S.

patents and seven of these pending U.S.

non-provisional patent applications are co-owned with certain of our collaborators because they encompass inventions developed under our collaborations.

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As of December 31, 2025, we in-licensed 153 U.S.

patents, 65 European patents and related validations, and 403 pending patent applications, including 84 pending U.S.

non-provisional patent applications, 55 pending European patent applications, and other related patents and patent applications in jurisdictions outside the United States and Europe that are related to our CRISPR technology collectively from various universities and institutions.

patents, 68 European patents and related validations, and approximately 350 pending patent applications, including 85 pending U.S.

non-provisional patent applications, 55 pending

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European patent applications, and other related patents and patent applications in jurisdictions outside the United States and Europe that are related to our CRISPR technology collectively from various universities and institutions.

The patents and patent applications outside of the United States and Europe are held primarily in Canada, China, Japan, and Australia, although some of our in-licensed patent families were filed in a larger number of countries.

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aureus Cas9, high-fidelity Cas9 nucleases, Cas9 PAM variants and self-inactivating forms of Cas9, CRISPR systems that employ Cas12a including Cas12a nickases and other variants and self-inactivating forms of Cas12a, and also CRISPR systems that employ viral vectors for delivery, single guide RNAs, or modified guide RNAs.

Our current in-licensed U.S.

patents, if the appropriate maintenance fees are paid, are expected to expire between 2033 and 2037, excluding any additional term for patent term adjustments or patent term extensions.

Our current U.S.

patents in-licensed from the various universities and institutions, if the appropriate maintenance fees are paid, are expected to expire between 2033 and 2037, excluding any additional term for patent term adjustments or patent term extensions.

If issued as U.S.

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For more information regarding these license agreements, please see the section titled “Business —Intellectual Property Licenses” of Part I, Item 1 of this Annual Report on Form 10-K.

As of December 31, 2024, our registered trademark portfolio consisted of registrations in the United States for EDITAS, EDITAS in Stylized Letters and the Infinity Logo, registrations in Australia, China, the EU, Japan, Switzerland and the United Kingdom (the “UK”) for EDITAS, registrations in Australia, China, the EU, Japan, Switzerland and the UK for the Infinity Logo, registrations in the EU and the UK for UDITAS, registrations in Australia, China, the EU, Japan, Switzerland and the UK for SLEEK, and registrations in Australia, China, the EU and the UK for the Double Helix Design.

The biotechnology and pharmaceutical industries, including in the gene therapy, genome editing and cell therapy fields, are characterized by rapidly advancing technologies, intense competition, and a strong emphasis on intellectual property and proprietary products.

As of December 31, 2025, our registered trademark portfolio consisted of registrations in the United States for EDITAS, EDITAS in Stylized Letters, the Infinity Logo, UDITAS, SLEEK, the Double Helix Design, and PIONEERING THE POSSIBLE, registrations in Australia, China, the EU, Japan, Switzerland and the United Kingdom (the “UK”) for EDITAS, registrations in Australia, China, the EU, Japan, Switzerland and the UK for the Infinity Logo, registrations in the EU and the UK for UDITAS, registrations in Australia, China, the EU, Japan, Switzerland and the UK for SLEEK, and registrations in Australia, China, the EU and the UK for the Double Helix Design.

The biotechnology and pharmaceutical industries, including in the gene therapy, gene editing and cell therapy fields, are characterized by rapidly advancing technologies, intense competition, and a strong emphasis on intellectual property and proprietary products.

While we believe that our technology, development experience, and scientific knowledge provide us with competitive advantages, we face potential competition from many different sources, including major pharmaceutical, specialty pharmaceutical, biotechnology companies, governmental agencies, and public and private research institutions.

Any product candidates that we successfully develop and commercialize may compete with existing therapies and new therapies may become available in the future.

We compete in the segments of the pharmaceutical, biotechnology, and other related markets that utilize technologies encompassing genomic medicines to create therapies, including genome editing and gene therapy.

There are additional companies that are working to develop therapies in areas related to our research programs.

For hemoglobinopathies, these companies include Beam Therapeutics, bluebird bio, CRISPR Therapeutics, Ensoma, Kamau Therapeutics, Orna Therapeutics, Sanofi, Scribe Therapeutics, Tessera Therapeutics, Vertex, and Yoltech Therapeutics.

For example, in late 2023 and early 2024, the U.S.

Food and Drug Administration (“FDA”) approved Vertex’s CASGEVY TM (exagamglogene autotemcel), a Cas9 genome-edited cell medicine, for the treatment of SCD and TDT.

The FDA also approved in late 2023 bluebird bio’s LYFGENIA TM (lovotibeglogene autotemcel), a cell-based gene therapy, for the treatment of SCD.

Our platform and product focus is the development of therapies using CRISPR technology specifically for genome editing.

Other companies developing CRISPR Cas9 or Cas12a technology or therapies using CRISPR Cas9 or Cas12a technology include AvenCell Therapeutics, Caribou Biosciences, Cellistic, CRISPR Therapeutics, EdiGene, eGenesis, ERS Genomics, Excision Biotherapeutics, Inscripta, Intellia Therapeutics, Kamau Therapeutics, Sigma-Aldrich, ToolGen, and Yoltech Therapeutics.

For hyperlipidemia, these companies include AccurEdit Therapeutics, CRISPR Therapeutics, Eli Lilly , EmendoBio, nChroma Bio, Scribe Therapeutics, Tune Therapeutics, and Yoltech Therapeutics, For hemoglobinopathies, these companies include Beam Therapeutics, BRL Medicine, CRISPR Therapeutics, Ensoma, Genetix Biotherapeutics (formerly bluebird bio), Kamau Therapeutics, Orna Therapeutics, Reforgene Medicine, Sanofi, Scribe Therapeutics, Tessera Therapeutics, Vertex, and Yoltech Therapeutics.

Our platform and product focus is the development of therapies using CRISPR technology specifically for gene editing.

Other companies developing CRISPR Cas9 or Cas12a technology or therapies using CRISPR Cas9 or Cas12a technology include AccurEdit Therapeutics, Arsenal Biosciences, AvenCell Therapeutics, Caribou Biosciences, Cellistic, Century Therapeutics, CRISPR Therapeutics, EdiGene, eGenesis, ERS Genomics, Excision Biotherapeutics, Fate

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Therapeutics, Inscripta, Intellia Therapeutics, Kamau Therapeutics, Sarepta Therapeutics, Sigma-Aldrich, ToolGen, Vittoria Biotherapeutics, and Yoltech Therapeutics.

In addition, there have been and may continue to be discoveries of new CRISPR-based gene editing technologies.

There are additional companies developing therapies using related CRISPR genome editing technologies, including other CRISPR nucleases, base editing, prime editing and gene writing.

These companies include Amber Bio, Arbor Biotechnologies, Beam Therapeutics, Eligo Biosciences, Emendo Biotherapeutics, Ensoma, Epic Bio, Integra Therapeutics, KSQ Therapeutics, Locus Biosciences, Mammoth Biosciences, Metagenomi, nChroma Bio, Poseida Therapeutics, Prime Medicine, Profluent Bio, Scribe Therapeutics, Tessera Therapeutics, Tune Therapeutics, and Verve Therapeutics.

There are additional companies developing therapies using related CRISPR gene editing technologies, including other CRISPR nucleases, base editing, prime editing and gene writing.

These companies include Amber Bio, Arbor Biotechnologies, Aurora Therapeutics, Beam Therapeutics, Eligo Biosciences, Eli Lilly, Emendo Biotherapeutics, Ensoma, Epicrispr Biotechnologies, Integra Therapeutics, KSQ Therapeutics, Locus Biosciences, Mammoth Biosciences, Metagenomi, Modalis Therapeutics, nChroma Bio, Prime Medicine, Profluent Bio, Roche, Scribe Therapeutics, Tessera Therapeutics, and Tune Therapeutics.

There are also companies developing therapies using transcription activator-like effector nucleases, meganucleases, Mega-TALs and zinc finger nucleases.

These companies include 2Seventy Bio, Allogene Therapeutics, bluebird bio, Cellectis, Precision Biosciences, and Sangamo Therapeutics.

In addition to competition from other genome editing therapies, gene therapies or cell medicine therapies, any products that we may develop may also face competition from other types of therapies, such as small molecule, antibody, protein, oligonucleotide, or ribonucleic acid therapies.

For hemoglobinopathies, these companies include Acceleron Pharma, Agios Pharmaceuticals, Novartis Pharmaceuticals, Pfizer, and Vertex.

In addition, many of our current or potential competitors, either alone or with their collaboration partners, may have greater financial resources and expertise in research and development, manufacturing, preclinical testing, conducting clinical trials, and marketing approved products than we do.

These companies include Allogene Therapeutics, BMS, Cellectis, Genetix Biotherapeutics, Precision Biosciences, and Sangamo Therapeutics.

In addition to competition from other gene editing therapies, gene therapies or cell medicine therapies, any products that we may develop may also face competition from other types of therapies, such as small molecule, antibody, protein, oligonucleotide, or ribonucleic acid therapies.

For hyperlipidemia, these companies include Amgen, Arrowhead Therapeutics, LIB Therapeutics, Merck, NewAmsterdam Pharma, Novartis, Regeneron Pharmaceuticals, and Wave Life Sciences.

In addition, statin medications are widely available from numerous pharmaceutical manufacturers and are offered in both branded and generic forms.

Mergers and acquisitions in the pharmaceutical, biotechnology, and gene therapy industries may result in even more resources being concentrated among a smaller number of our competitors.

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Our commercial opportunity could be reduced or eliminated if our competitors develop and commercialize products that are safer, more effective, have fewer or less severe side effects, are more convenient or are less expensive than any products that we may develop.

Our competitors also may obtain FDA or other regulatory approval for their products more rapidly than we may obtain approval for ours, which could result in our competitors establishing a strong market position before we are able to enter the market.

Our competitors also may obtain U.S.

Food and Drug Administration (“FDA”) or other regulatory approval for their products more rapidly than we may obtain approval for ours, which could result in our competitors establishing a strong market position before we are able to enter the market.

The key competitive factors affecting the success of all of our programs are likely to be their efficacy, safety, convenience, and availability of reimbursement.

If our current programs are approved for the indications for which we are currently planning clinical trials, they may compete with other products currently under development, including genome editing and gene therapy products.

Competition with other related products currently under development may include competition for clinical trial sites, patient recruitment, and product sales.

Manufacturing

We currently perform some manufacturing activities such as the production of RNP Complexes for our various internal programs and may also agree to produce RNP Complexes for our partners.

These activities are performed on site at our existing facilities or at current good manufacturing practice-compliant space leased by us.

We have limited manufacturing operations and do not own or operate any substantial manufacturing facilities for large-scale production of

our program materials.

At the appropriate time in the product development of each product candidate, we will determine whether to expand our internal manufacturing capabilities or rely on third parties to manufacture commercial quantities of any products that we may successfully develop and have approved.

The use of contracted manufacturing and reliance on collaboration partners is relatively cost-efficient and has eliminated the need for substantial direct investment in manufacturing facilities and additional staff.

Although we rely on contract manufacturers for certain materials, we have personnel with manufacturing experience to oversee our contract manufacturers.

We contract with third parties for the manufacturing of materials for preclinical studies and our planned clinical trials.

We have no manufacturing operations and do not own or operate any manufacturing facilities for large-scale production of our program materials.

At the appropriate time in the product development of each product candidate, we will determine whether to invest in internal manufacturing capabilities or rely on third parties to manufacture late-stage clinical or commercial quantities of any products that we may successfully develop and have approved.

The use of contracted manufacturing and reliance on collaboration partners is relatively cost-efficient and has eliminated the need for direct investment in manufacturing facilities and additional staff.

Although we rely on contract manufacturers, we have personnel with manufacturing experience to oversee our contract manufacturers.

We expect third-party manufacturers to be capable of providing sufficient quantities of our program materials to meet anticipated needs for preclinical studies and clinical trials.

To meet our projected needs for commercial manufacturing, third parties with whom we currently work might need to increase their scale of production or we will need to secure alternate suppliers.

We believe that there are alternate sources of supply that can satisfy our preclinical, clinical, and commercial requirements, although we cannot be certain that identifying and establishing relationships with such sources, if necessary, would not result in significant delay or material additional costs.

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Commercialization

We currently intend to build the commercial infrastructure in the United States necessary to effectively support the commercialization of our programs, if and when we first believe a regulatory approval of a product candidate under one of our programs in a particular geographic market appears probable.

We will evaluate whether to build the commercial infrastructure in the United States necessary to effectively support the commercialization of our programs, if and when we first believe a regulatory approval of a product candidate under one of our programs in a particular geographic market appears probable.

In Europe and elsewhere outside of the United States, where appropriate, we may elect in the future to utilize strategic partners, distributors, or contract sales forces to assist in the commercialization of our products.

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Data, the size of the development programs, the size of the target market, the size of a commercial infrastructure, and manufacturing needs may all influence our strategies in the United States, Europe, and the rest of the world.

The commercial infrastructure for orphan products typically consists of a targeted, specialty sales force that calls on a limited and focused group of physicians supported by sales management, medical liaisons, internal sales support, an internal marketing group, and distribution support.

Additional capabilities important to the orphan marketplace include the management of key accounts such as managed care organizations, group purchasing organizations, specialty pharmacies, and government accounts.

To develop the appropriate commercial infrastructure, we will have to invest significant amounts of financial and management resources, some of which will be committed prior to any confirmation that any product candidate we may develop will be approved.

Government Regulation and Licensure of Products

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• performance of adequate and well-controlled human clinical trials to establish the safety, potency, and purity of the product candidate for each proposed indication, in accordance with current Good Clinical Practices (“GCP”);

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• preparation and submission to the FDA of a Biologic License Application (“BLA”) for a biologic product requesting marketing for one or more proposed indications, including submission of detailed information on the manufacture and composition of the product in clinical development and proposed labelling;

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These studies are generally referred to as IND-enabling studies.

The conduct of the preclinical tests and formulation of the compounds for testing must comply with federal regulations and requirements, including GLP regulations and standards and the U.S.

Department of Agriculture’s Animal Welfare Act, if applicable.

The conduct of the preclinical tests and formulation of the compounds for testing must comply with federal regulations and requirements, including GLP regulations.

With passage of the FDA’s Modernization Act 2.0 in December 2022, Congress eliminated provisions in both the FDCA and the PHSA that required animal testing in support of an NDA or BLA.

While animal testing may still be conducted, the FDA was authorized to rely on alternative non-clinical tests, including cell-based assays, microphysiological systems, or bioprinted or computer models.

In April 2025, the FDA released a roadmap to replace animal testing in preclinical safety studies with scientifically validated new approach methodologies, such as organ-on-a-chip systems and computational modeling, which are referred to as in silica models, as well as advanced in vitro assays.

The results of the preclinical tests, together with manufacturing information and analytical data, are submitted to the FDA as part of an IND application.

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Expanded access, sometimes called “compassionate use,” is the use of investigational products outside of clinical trials to treat patients with serious or immediately life-threatening diseases or conditions when there are no comparable or satisfactory alternative treatment options.

The rules and regulations related to expanded access are intended to improve access to investigational products for patients who may benefit from investigational therapies.

The rules and regulations related to expanded access are intended to improve

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access to investigational products for patients who may benefit from investigational therapies.

FDA regulations allow access to investigational products under an IND by the company or the treating physician for treatment purposes on a case-by-case basis for:

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or 15 days after the investigational drug or biologic receives designation as a Breakthrough Therapy, Fast Track product, or regenerative medicine advanced therapy.

In October 2025, the FDA issued final guidance further clarifying the statutory and regulatory requirements governing expanded access.

In addition, on May 30, 2018, the Right to Try Act, was signed into law.

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

A sponsor who wishes to conduct a clinical trial outside the United States 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, all FDA IND requirements must be met unless waived.

When a foreign clinical trial is not conducted under an IND, the sponsor must ensure that the trial complies with certain regulatory requirements of the FDA in order to use the trial as support for an IND or application for marketing approval.

Specifically, the FDA requires that such trials be conducted in accordance

with GCP, including review and approval by an independent ethics committee and informed consent from subjects.

The GCP requirements encompass both ethical and data integrity standards for clinical trials.

The FDA’s regulations are intended to help ensure the protection of human subjects enrolled in non-IND foreign clinical trials, as well as the quality and integrity of the resulting data.

They further help ensure that non-IND foreign trials are conducted in a manner comparable to that required for clinical trials in the United States.

Further, each clinical trial must be reviewed and approved by an IRB either centrally or individually at each institution at which the clinical trial will be conducted.

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The FDA will typically inspect one or more clinical sites to assure compliance with GCP and the integrity of the clinical data submitted.

In December 2025, the FDA released final guidance outlining its processes and practices applicable to bioresearch monitoring inspections.

Additionally, some clinical trials are overseen by an independent group of qualified experts organized by the clinical trial sponsor, known as a data monitoring committee (“DMC”).

This group may recommend continuation of the study as planned, changes in study conduct, or cessation of the study at designated check points based on certain available data from the study to which only the DMC has access.

This group may recommend continuation of the

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study as planned, changes in study conduct, or cessation of the study at designated check points based on certain available data from the study to which only the DMC has access.

Finally, research activities involving infectious agents, hazardous chemicals, recombinant DNA, and genetically altered organisms and agents may be subject to review and approval of an Institutional Biosafety Committee in accordance with NIH Guidelines for Research Involving Recombinant or Synthetic Nucleic Acid Molecules.

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such Phase 3 studies are referred to as “pivotal.”

In February 2026, the Commissioner of FDA and the Director of Center for Biologics Evaluation and Research published an editorial in the New England Journal of Medicine in which they declared that, in most cases, the new default requirement for FDA approval of a new product will be one adequate and well-controlled pivotal clinical trial plus confirmatory evidence, rather than two pivotal clinical trials.

In determining whether to rely on one trial, the FDA will focus on the single trial’s quality, including magnitude of effect, appropriateness of control arms, endpoint selection, statistical power, blinding, handling of missing data, biological plausibility and alignment with intermediate biomarkers.

The FDA has long had authority to approve new products on the basis of one trial plus confirmatory evidence and, in recent years, the agency has exercised that authority with respect to certain types of products.

The FDA now takes the position that this will be the new official default standard for most product candidates.

At this point, it is unclear how this new policy will be implemented by the FDA and how, if at all, it will affect our clinical development programs.

In some cases, the FDA may approve a BLA for a product candidate but require the sponsor to conduct additional clinical trials to further assess the product candidate’s safety and effectiveness after approval.

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These plans are meant to encourage the enrollment of more diverse patient populations in late-stage clinical trials of FDA-regulated products.

Specifically, actions plans must include the sponsor’s goals for enrollment, the underlying rationale for those goals, and an explanation of how the sponsor intends to meet them.

In addition to these requirements, the legislation directs the FDA to issue new guidance on DAPs.

In June 2024, as mandated by FDORA, the FDA issued draft guidance outlining the general requirements for DAPs.

Unlike most guidance documents issued by the FDA, the DAP guidance when finalized will have the force of law because FDORA specifically dictates that the form and manner for submission of DAPs are specified in FDA guidance.

On January 27, 2025, in response to an Executive Order issued by President Trump on January 21, 2025, on Diversity, Equity and Inclusion programs, the FDA removed this draft guidance from its website.

This action raises questions about the applicability of statutory obligations to submit DAPs and the agency’s current thinking on best practices for clinical development.

In June 2023, the FDA issued draft guidance with updated recommendations for GCPs aimed at modernizing the design and conduct of clinical trials.

On January 27, 2025, in response to an executive order issued by President Trump on January 21, 2025, relating to Diversity, Equity and Inclusion programs, the FDA removed the draft DAP guidance from its website.

Subsequently, in July 2025, pursuant to a court order, the FDA restored the draft DAP guidance to its website with a statement that “information on this page may be modified and/or removed in the future subject to the terms of the court’s order and implemented consistent with applicable law.” Accordingly, in light of these ongoing actions, there is considerable uncertainty surrounding the draft DAP guidance and how the FDA will consider diversity action plans in connection with its review of BLAs.

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In September 2025, the FDA issued final guidance with updated recommendations for GCPs aimed at modernizing the design and conduct of clinical trials.

The updates are intended to help pave the way for more efficient clinical trials to facilitate the development of medical products.

The draft guidance is adopted from the International Council for Harmonisation’s updated E6(R3) draft guideline that was developed to enable the incorporation of rapidly developing technological and methodological innovations into the clinical trial enterprise.

In addition, the FDA issued draft guidance outlining recommendations for the implementation of decentralized clinical trials.

The final guidance is adopted from the International Council for Harmonisation’s updated E6(R3) final guideline that was developed to enable the incorporation of rapidly developing technological and methodological innovations into the clinical trial enterprise.

In September 2024, the FDA issued final guidance outlining recommendations for the implementation of decentralized clinical trials.

In October 2025, the FDA issued final guidance that focuses on patient-focused drug development.

The guidance outlines how stakeholders, such as patients, caregivers, researchers and medical product developers, can submit patient experience data in support of the development and approval of drug products.

To that end, the guidance provides an overview of clinical outcome assessments (“COAs”) in clinical trials, and the role that COAs may play in evaluating the clinical benefit of a medical product.

Sponsors of clinical trials are required to register and disclose certain clinical trial information on a public registry (clinicaltrials.gov) maintained by the NIH.

The NIH’s Final Rule on registration and reporting requirements for clinical trials became effective in 2017, and both NIH and the FDA have signaled the government’s willingness to begin enforcing those requirements against non-compliant clinical trial sponsors.

Although the FDA has historically not enforced these reporting requirements due to Department of Health and Human Services’ (“HHS”) long delay in issuing final implementing regulations, FDA has as of December 2024 issued six notices of non-compliance, thereby signaling the government’s willingness to begin enforcing these requirements against non-compliant clinical trial sponsors.

Although the FDA has historically not enforced these reporting requirements due to Department of Health and Human Services’ (“HHS”) long delay in issuing final implementing regulations, FDA has as of January 2026 issued eight notices of non-compliance, thereby signaling the government’s willingness to begin enforcing these requirements against non-compliant clinical trial sponsors.

While these notices of non-compliance did not result in civil monetary penalties, the failure to submit clinical trial information to clinicaltrials.gov is a prohibited act under the FDCA with violations subject to potential civil monetary penalties of up to $10,000 for each day the violation continues.

Violations may also result in injunctions and/or criminal prosecution or disqualification from federal grants.

Clinical Trials Outside the United States in Support of FDA Approval

In connection with our clinical development program, we may have trial sites outside the United States from time to time.

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

Specifically, the trials must be conducted in accordance with GCP, including undergoing review and receiving approval by an independent ethics committee (“IEC”) and seeking and receiving informed consent from subjects.

GCP requirements encompass both ethical and data integrity standards for clinical studies.

The FDA’s regulations are intended to help ensure the protection of human subjects enrolled in non-IND foreign clinical trials, as well as the quality and integrity of the resulting data.

They further help ensure that non-IND foreign trials are conducted in a manner comparable to that required for IND trials.

The acceptance by the FDA of trial data from clinical trials conducted outside the United States in support of U.S.

approval may be subject to certain conditions or may not be accepted at all.

In cases where data from foreign clinical trials are intended to serve as the sole basis for marketing approval in the United States, the FDA will generally not approve the application on the basis of foreign data alone unless (i) the data are applicable to the U.S.

population and U.S.

medical practice;

(ii) the trials were performed by clinical investigators of recognized competence and pursuant to GCP regulations;

and (iii) the data may be considered valid without the need for an on-site inspection by the FDA, or if the FDA considers such inspection to be necessary, the FDA is able to validate the data through an on-site inspection or other appropriate means.

In December 2025, in the context of negotiations involving reauthorization of PDUFA, the FDA proposed cutting fees for companies conducting clinical development programs in the United States, rather than abroad.

It is unclear whether and how this proposal will be adopted and finalized.

In addition, even where the foreign trial data are not intended to serve as the sole basis for approval, the FDA will not accept the data as support for an application for marketing approval unless the trial is well-designed and well-conducted in accordance with GCP requirements and the FDA is able to validate the data from the trial through an onsite inspection if deemed necessary.

Many foreign regulatory authorities have similar approval requirements.

In addition, such foreign trials are subject to the applicable local laws of the foreign jurisdictions where the trials are conducted.

Interactions with the FDA During the Clinical Development Program

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Following the clearance of an IND and the commencement of clinical trials, a sponsor is given the opportunity to meet with the FDA at certain points in the clinical development program.

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A Type C meeting is any meeting other than a Type A or Type B meeting regarding the development and review of a product.

A type D meeting is focused on a narrow set of issues and should not require input from more than 3 disciplines or Divisions.

A Type D meeting is focused on a narrow set of issues and should not require input from more than three disciplines or Divisions.

Finally, INTERACT meetings are intended for novel products and development programs that present unique challenges in the early development of an investigational product.

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Those plans must contain an outline of the proposed pediatric study or studies the sponsor plans to conduct, including study objectives and design, any deferral or waiver requests, and other information required by regulation.

The sponsor, the FDA, and the FDA’s internal review

committee must then review the information submitted, consult with each other, and agree upon a final plan.

The sponsor, the FDA, and the FDA’s internal review committee must then review the information submitted, consult with each other, and agree upon a final plan.

The FDA or the sponsor may request an amendment to the plan at any time.

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Within the CBER, the review of gene therapy and related products is consolidated in the Office of Cellular Therapeutic Products, and the FDA has established the Cellular, Tissue and Gene Therapies Advisory Committee to advise CBER on its reviews.

The NIH, including its Novel and Exceptional Technology Research Advisory Committee (“NExTRAC”), also advises the FDA on gene therapy issues and other issues related to emerging biotechnologies.

The NIH, including its Novel and Exceptional Technology Research Advisory Committee (“NExTRAC”), also

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advises the FDA on gene therapy issues and other issues related to emerging biotechnologies.

The FDA and the NIH have published guidance documents with respect to the development and submission of gene therapy protocols.

The FDA has issued numerous guidance documents regarding gene therapies, including a draft guidance issued in November 2024 to address frequently asked questions surrounding the development of cellular and gene therapy products.

The FDA has issued numerous guidance documents regarding gene therapies.

Although the FDA has indicated that these and other guidance documents it previously issued are not legally binding, compliance with them is likely necessary to gain approval for any gene therapy product candidate.

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the chemistry, manufacturing, and control information that should be included in an IND application;

the proper design of tests to measure product potency in support of an IND or BLA application;

the proper design of tests to measure product potency in support of an IND or 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 high.

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The PREVENT Pandemics Act, which was enacted in December 2022, clarifies that foreign drug manufacturing establishments are subject to registration and listing requirements even if a drug or biologic undergoes further manufacture, preparation, propagation, compounding, or processing at a separate establishment outside the United States prior to being imported or offered for import into the United States.

In May 2025, the FDA disclosed plans to expand its use of unannounced inspections of foreign manufacturing facilities that produce drugs and biologics distributed in the United States.

Subsequently, in August 2025, the FDA introduced a “PreCheck” program with the intention of supporting companies as they build new facilities in the United States.

The PreCheck program provides manufacturers with more frequent FDA communication at critical development stages, including facility design, construction, and pre-production.

These FDA initiatives flow from an Executive Order issued by President Trump on May 5, 2025, calling for actions to reduce regulatory barriers to pharmaceutical manufacturing in the United States.

Submission and Filing of a BLA

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The BLA must contain extensive manufacturing information and detailed information on the composition of the product and proposed labeling as well as payment of a user fee.

Under federal law, the submission of most BLAs is subject to an application user fee, which for federal fiscal year 2025 is approximately $4.3 million for an application requiring clinical data.

The sponsor of a licensed BLA is also subject to an annual program fee, which for fiscal year 2025 is more than $403,889.

Under federal law, the submission of most BLAs is subject

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to an application user fee, which for federal fiscal year 2026 is approximately $4.7 million for an application requiring clinical data.

The sponsor of a licensed BLA is also subject to an annual program fee, which for fiscal year 2026 is $442,213.

Certain exceptions and waivers are available for some of these fees, such as an exception from the application fee for products with orphan designation and a waiver for certain small businesses.

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In the event that the FDA determines that an application does not satisfy this standard, it will issue a Refuse to File (“RTF”) determination to the sponsor.

In October 2025, the FDA issued internal guidance clarifying that “materially incomplete or inadequately organized” applications that would not permit timely, efficient and complete review will be the subject of an RTF.

The internal guidance also provides that the agency will issue an RTF for an application that relies on a single adequate and well-controlled investigation to support approval if prior communications with the FDA determined the need for more than one clinical study and any justification for a single investigation is inadequate.

On the other hand, once the submission has been accepted for filing, the FDA begins an in-depth review of the application.

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Additionally, before approving a BLA, the FDA will typically inspect one or more clinical sites to assure compliance with GCP.

With passage of FDORA, Congress clarified the FDA’s authority to conduct inspections by

expressly permitting inspection of facilities involved in the preparation, conduct, or analysis of clinical and non-clinical studies submitted to the FDA as well as other persons holding study records or involved in the study process.

With passage of FDORA, Congress clarified the FDA’s authority to conduct inspections by expressly permitting inspection of facilities involved in the preparation, conduct, or analysis of clinical and non-clinical studies submitted to the FDA as well as other persons holding study records or involved in the study process.

Moreover, the FDA will review a sponsor’s financial relationship with the principal investigators who conducted the clinical trials in support of the NDA.

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

The potency of a product is required to be supported by substantial evidence.

The FDA has previously interpreted this evidentiary standard to require at least two adequate and well-controlled clinical investigations to establish

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effectiveness of a new product.

In December 2025, however, the FDA signaled that it is considering only requiring one clinical study for approval of certain products.

The FDA indicated at such time that it may issue guidance regarding this change through a press release or other means;

the FDA has not yet issued such guidance.

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 non-clinical and clinical trial sites to assure compliance with GCPs, the FDA may issue an approval letter or a complete response letter (“CRL”).

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The FDA will not approve an application until issues identified in the CRL have been addressed.

While CRLs were previously treated by the FDA as confidential and were only disclosed in action packages for approved products, the agency announced in September 2025 that it will now release CRLs promptly after they are issued to sponsors.

Since that announcement, the FDA has posted a number of CRLs on its website.

If the FDA approves a new product, it may limit the approved indications for use of the product.

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To qualify for the Breakthrough Therapy program, product candidates must be intended to treat a serious or life-threatening condition and preliminary clinical evidence must indicate that such product candidates may demonstrate substantial improvement on one or more clinically significant endpoints over existing therapies.

The FDA will seek to ensure the sponsor of a Breakthrough Therapy product candidate receives intensive guidance on an efficient development program, intensive involvement of senior managers and experienced staff on a proactive, collaborative and cross-disciplinary review and rolling review.

The FDA will seek to ensure the sponsor of a Breakthrough Therapy product candidate

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receives intensive guidance on an efficient development program, intensive involvement of senior managers and experienced staff on a proactive, collaborative and cross-disciplinary review and rolling review.

• Priority review.

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The benefits of a regenerative advanced therapy designation include early interactions with the FDA to expedite development and review, benefits available to Breakthrough Therapies, potential eligibility for priority review and accelerated approval based on surrogate or intermediate endpoints.

• Commissioner’s National Priority Voucher Program .

On June 17, 2025, the FDA announced the creation of a new voucher program to expedite the development and approval of new drug products.

Vouchers issued under the new program, which is known as the Commissioner’s National Priority Voucher (“CNPV”) Program, may reportedly be redeemed by sponsors to shorten the review time of an NDA from approximately 10-12 months to 1-2 months.

The FDA has indicated that the new CNPV process will convene experts from the FDA’s offices for a team-based review rather than using the standard review system of a drug application being sent to numerous FDA offices.

Clinical information will be reviewed by a multidisciplinary team of physicians and scientists who will pre-review the submitted information and convene for a one-day meeting.

Vouchers under this program will reportedly be given to companies aligned with U.S.

national priorities.

As with the FDA’s other programs for expediting review and approval of new drug products, there is no guarantee it would result in approval of our marketing applications or that such approval, if granted, would be on an expedited basis.

• Rare Disease Evidence Principles .

In September 2025, the FDA introduced a framework intended to streamline the approval of new therapies for ultrarare diseases.

The Rare Disease Evidence Principles (“RDEP”) is intended to allow sponsors to rely on a single-arm trial in support of approval of drugs and biologics that treat rare diseases with very small patient populations and where the disease is linked to a known genetic defect and characterized by progressive functional deterioration leading to disability or death in a short period of time.

The targeted diseases should also lack adequate alternative therapies.

Accelerated Approval

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Further, FDORA requires the agency to publish on its website “the rationale for why a post-approval study is not appropriate or necessary” whenever it decides not to require such a study upon granting accelerated approval.

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In March 2023, the FDA issued draft guidance that outlines its current thinking and approach to accelerated approval.

The agency indicated that the accelerated approval pathway is commonly used for approval of oncology drugs due to the serious and life-threatening nature of cancer.

Although single-arm trials have been commonly used to support

accelerated approval, a randomized controlled trial is the preferred approach as it provides a more robust efficacy and safety assessment and allows for direct comparisons to an available therapy.

Although single-arm trials have been commonly used to support accelerated approval, a randomized controlled trial is the preferred approach as it provides a more robust efficacy and safety assessment and allows for direct comparisons to an available therapy.

Subsequently, in December 2024 and January 2025, the FDA issued additional draft guidances relating to accelerated approval.

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After approval, a drug product generally may not be promoted for uses that are not approved by the FDA, as reflected in the product’s prescribing information.

In September 2021, the FDA published final regulations which describe the types of evidence that the agency will consider in determining the intended use of a drug or biologic.

In September 2021, the FDA published

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final regulations which describe the types of evidence that the agency will consider in determining the intended use of a drug or biologic.

On September 9, 2025, President Trump issued a Memorandum directing HHS to “ensure transparency and accuracy in direct-to-consumer (“DTC”) prescription drug advertising, including by increasing the amount of information regarding any risks associated with the use of any such prescription drug required to be provided in prescription drug advertisements.” To that end, the FDA announced that it is initiating a rulemaking process “to eliminate the ‘adequate provision’ loophole that allows pharmaceutical advertisements to hide safety information by placing it in another format or location.” In this context, the FDA declared that it will no longer tolerate what it characterized as “deceptive practices” in prescription drug advertising and that the agency would “aggressively deploy” its available enforcement tools, with “heightened scrutiny” of fair balance and disclosures in social media promotions.

The FDA also issued a generic “notice letter” directing companies to “remove any noncompliant advertising and bring all promotional communications into compliance.”

It may be permissible, under very specific, narrow conditions, for a manufacturer to engage in nonpromotional, non-misleading communication regarding off-label information, such as distributing scientific or medical journal information.

Moreover, with passage of the Pre-Approval Information Exchange Act (the “PIE Act”) in December 2022, sponsors of products that have not been approved may proactively communicate to payors certain information about products in development to help expedite patient access upon product approval.

Moreover, with passage of the Pre-Approval Information Exchange Act in December 2022, sponsors of products that have not been approved may proactively communicate to payors certain information about products in development to help expedite patient access upon product approval.

In addition, in January 2025, the FDA published final guidance outlining the agency’s non-binding policies governing the distribution of scientific information on unapproved uses to healthcare providers.

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Orphan drug exclusivity means that the FDA may not approve another sponsor’s marketing application for the same product for the same indication for seven years, except in certain limited circumstances.

In particular, the concept of what constitutes the “same drug” for purposes of orphan drug exclusivity remains in flux in the context of gene therapies, and the FDA has recently issued guidance indicating it would consider two gene therapy products for the same indication to be different, thus each eligible for orphan drug exclusivity, if they express different transgenes or have or use different vectors, so long as those differences are not “minor .” The FDA will determine whether two vectors from the same viral class are the same on a case-by-case basis and may consider additional key features in assessing sameness.

In particular, the concept of what constitutes the “same drug” for purposes of orphan drug

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exclusivity remains in flux in the context of gene therapies, and the FDA has recently issued guidance indicating it would consider two gene therapy products for the same indication to be different, thus each eligible for orphan drug exclusivity, if they express different transgenes or have or use different vectors, so long as those differences are not “minor .” The FDA will determine whether two vectors from the same viral class are the same on a case-by-case basis and may consider additional key features in assessing sameness.

If a product designated as an orphan drug ultimately receives marketing approval for an indication broader than what was designated in its orphan drug application, it may not be entitled to exclusivity.

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The FDA cannot, however, approve the same product made by another manufacturer for the same indication during the market exclusivity period unless it has the consent of the sponsor or the sponsor is unable to provide sufficient quantities.

In September 2021, the Court of Appeals for the 11th Circuit held that, for the purpose of determining the scope of market exclusivity, the term “same disease or condition” in the statute means the designated “rare disease or condition” and could not be interpreted by the FDA to mean the “indication or use.” Thus, the court concluded, orphan drug exclusivity applies to the entire designated disease or condition rather than the “indication or use.” Although there have been legislative proposals to overrule this decision, they have not been enacted into law.

On January 23, 2023, the FDA announced that, in matters beyond the scope of that court order, the FDA will continue to apply its existing regulations tying orphan-drug exclusivity to the uses or indications for which the orphan drug was approved.

The FDA and Congress may further reevaluate the Orphan Drug Act and its regulations and policies.

This may be particularly true in light of a decision from the Court of Appeals for the 11th Circuit in September 2021.

In Catalyst Pharms, Inc.

Becerra (“Catalyst”), the Court held that, for the purpose of determining the scope of orphan drug exclusivity, the term “same disease or condition” in the statute means the designated “rare disease or condition” and could not be interpreted by the FDA to mean the “indication or use.” On January 23, 2023, the FDA announced that, in matters beyond the scope of the Catalyst court order, the FDA will continue to apply its existing regulations tying orphan-drug exclusivity to the uses or indications for which the orphan drug is approved.

More recently however, on February 14, 2025, a federal district court in Washington, D.C.

fully embraced the reasoning of the Catalyst decision in another decision challenging the scope of orphan drug exclusivity.

On April 17, 2025, the FDA appealed this decision to the U.S.

Court of Appeals for the D.C.

The implications of this decision, and its impact on the FDA’s implementation of the Orphan Drug Act, are unclear at this point.

Pediatric Exclusivity

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In December 2022, Congress clarified through FDORA that the FDA may approve multiple first interchangeable biosimilar biological products so long as the products are all approved on the first day on which such a product is approved as interchangeable with the reference product.

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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.

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At the same time, since passage of the BPCIA, many states have passed laws or amendments to laws, which address pharmacy practices involving biosimilar products.

In October 2025, the FDA issued draft guidance which proposes to eliminate the need for sponsors of biosimilar products to conduct comparative human clinical efficacy studies, allowing them to rely instead on analytical testing to demonstrate product differences from a reference product.

Patent Term Restoration and Extension

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Patent term restoration cannot be used to extend the remaining term of a patent past a total of 14 years from the product’s approval date.

Only one patent applicable to an approved product is eligible for

the extension, and the application for the extension must be submitted prior to the expiration of the patent in question.

Only one patent applicable to an approved product is eligible for the extension, and the application for the extension must be submitted prior to the expiration of the patent in question.

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

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The 2020 guidance expands on the policy statement in the 2014 guidance by recommending that companion diagnostic developers consider a number of factors when determining whether their test could be developed, or the labeling for approved companion diagnostics could be revised through a supplement, to support a broader labeling claim such as use with a specific group of oncology therapeutic products (rather than listing an individual therapeutic product(s)).

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Under the FDCA, in vitro diagnostics, including companion diagnostics, are regulated as medical devices.

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Device manufacturers must also establish registration and device listings with the FDA.

A medical device manufacturer’s manufacturing processes and those of its suppliers are required to comply with the applicable portions of the Quality System Regulation, which covers the methods and documentation of the design, testing, production, processes, controls, quality assurance, labeling,

packaging and shipping of medical devices.

Domestic facility records and manufacturing processes are subject to periodic unscheduled inspections by the FDA.

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In order to market any product outside of the United States, a company must also comply with numerous and varying regulatory requirements of other countries and jurisdictions regarding quality, safety and efficacy and governing, among other things, clinical trials, marketing authorization, commercial sales and distribution of products.

Whether or not it obtains FDA approval for a product, a sponsor will need to obtain the necessary approvals by the comparable foreign regulatory authorities before it can commence clinical trials or marketing of the product in those countries or jurisdictions.

Whether or not it obtains FDA approval for a product, a sponsor will need to obtain the necessary approvals by the comparable regulatory authorities of foreign countries or economic areas, such as the 27-member EU, before it can commence clinical trials or marketing of the product in those countries or jurisdictions.

The process governing approval of medicinal products in the EU generally follows the same lines as in the United States.

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Clinical Trial Approval

On January 31, 2022, the new Clinical Trials Regulation (EU) No 536/2014 became effective in the EU and replaced the prior Clinical Trials Directive 2001/20/EC.

On January 31, 2022, the new Clinical Trials Regulation (EU) No 536/2014 (“CTR”) became effective in the EU and replaced the prior Clinical Trials Directive 2001/20/EC.

The new regulation aims at simplifying and streamlining the authorization, conduct and transparency of clinical trials in the EU.

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The submission will be made through the Clinical Trials Information System, a new clinical trials portal overseen by the European Medicines Agency (“EMA”) and available to clinical trial sponsors, competent authorities of the EU Member States and the public.

As of January 31, 2025, all clinical trials in the EU (including those which are ongoing) are subject to the CTR.

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Beyond streamlining the process, the new regulation includes a single set of documents to be prepared and submitted for the application as well as simplified reporting procedures for clinical trial sponsors, and a harmonized procedure for the assessment of applications for clinical trials, which is divided in two parts.

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The role of the relevant ethics committees in the assessment procedure will continue to be governed by the national law of the concerned EU Member State.

However, overall related timelines will be defined by the Clinical Trials Regulation (“CTR”).

However, overall related timelines will be defined by the CTR.

The new regulation did not change the preexisting requirement that a sponsor must obtain prior approval from the competent national authority of the EU Member State in which the clinical trial is to be conducted.

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Furthermore, the sponsor may only start a clinical trial at a specific study site after the applicable ethics committee has issued a favorable opinion.

The CTR foresees a three-year transition period.

The extent to which ongoing and new clinical trials will be governed by the CTR varies.

Clinical trials for which an application was submitted (i) prior to January 31, 2022 under the Clinical Trials Directive, or (ii) between January 31, 2022 and January 31, 2023 and for which the sponsor has opted for the application of the Clinical Trials Directive, remain governed by said Directive until January 31, 2025.

After this date, all clinical trials (including those which are ongoing) will become subject to the provisions of the CTR.

Parties conducting certain clinical studies must, as in the U.S., post clinical trial information in the EU at the EudraCT website:

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Pursuant to Regulation (EC) No.

726/2004, the centralized procedure is compulsory for specific products, including for medicines produced by certain biotechnological processes, products designated as orphan medicinal products, advanced therapy products and products with a new active substance indicated for the treatment of certain diseases, including products for the treatment of cancer.

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of certain diseases, including products for the treatment of cancer.

For products with a new active substance indicated for the treatment of other diseases and products that are highly innovative or for which a centralized process is in the interest of patients, the centralized procedure may be optional.

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Under these procedures, before granting the marketing authorization, the EMA or the competent authorities of the member states make an assessment of the risk-benefit balance of the product on the basis of scientific criteria concerning its quality, safety, and efficacy.

Except conditional marketing authorizations, marketing authorizations have an initial duration of five years.

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conditional marketing authorizations, marketing authorizations have an initial duration of five years.

After these five years, the authorization may be renewed on the basis of a reevaluation of the risk-benefit balance.

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The European Parliament requested several amendments in April 2024.

At this time, the proposed revisions remain to be agreed and adopted by the European Parliament and European Council and the proposals may therefore be substantially revised before adoption, which is not anticipated before early 2026.

The revisions may, however, have a significant impact on the pharmaceutical industry in the long term, if and when adopted.

On December 11.

2025, the European Parliament and Council reached a provisional political agreement on the legislation which is expected to be adopted by mid-2026.

Key changes include updating regulatory data exclusivity to a new system with eight years data exclusivity and reduced market exclusivity period to one year which can be extended if specific conditions are fulfilled, adding launch/supply obligations, incentivizing antibiotic innovation with transferable vouchers, and streamlining approval procedures in the EU.

If the legislation is finalized in line with the provisional political agreement it will have a profound impact on the pharmaceutical industry.

Periods of Authorization and Renewals

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Following approval, the holder of the marketing authorization is required to comply with a range of requirements applicable to the manufacturing, marketing, promotion and sale of the medicinal product.

These include compliance with the EU’s stringent pharmacovigilance or safety reporting rules, pursuant to which post-authorization studies and additional monitoring obligations can be imposed.

These include compliance with the EU’s stringent pharmacovigilance or safety reporting rules, pursuant to which post-authorization studies and additional

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monitoring obligations can be imposed.

In addition, the manufacturing of authorized products, for which a separate manufacturer’s license is mandatory, must also be conducted in strict compliance with the EMA’s GMP requirements and comparable requirements of other regulatory bodies in the EU, which mandate the methods, facilities, and controls used in manufacturing, processing and packing of drugs to assure their safety and identity.

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Reimbursement and Pricing of Prescription Pharmaceuticals

The EU/European Economic Area (“EEA”) applies harmonized regulatory rules for medicinal products, for the approval process and requirements governing the conduct of clinical trials, and for the regulatory approval of medicinal products.

However, pricing and reimbursement for medicinal products varies greatly between countries and jurisdictions and can involve additional testing for health technology assessments.

In the EU, similar political, economic and regulatory developments to those in the United States may affect our ability to profitably commercialize our product candidates, if approved.

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In these countries, pricing negotiations with governmental authorities can take considerable time after the receipt of marketing approval for a product.

To obtain reimbursement or

pricing approval in some countries, pharmaceutical firms may be required to conduct a clinical trial that compares the cost-effectiveness of the product to other available therapies.

To obtain reimbursement or pricing approval in some countries, pharmaceutical firms may be required to conduct a clinical trial that compares the cost-effectiveness of the product to other available therapies.

Orphan Drug Designation and Exclusivity

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The rules and requirements for obtaining an SPC are similar to those in the United States.

An SPC may extend the term of a patent for up to five years after its originally scheduled expiration date and can provide up to a maximum of fifteen years of marketing exclusivity for a drug.

An SPC may extend the term of a patent for up to five years after its originally scheduled expiration date and can provide up to a maximum of fifteen years of

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marketing exclusivity for a drug.

These periods can be extended for six additional months if pediatric exclusivity is obtained, which is described in detail below.

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Brexit and the Regulatory Framework in the United Kingdom

The UK’s withdrawal from the EU, commonly referred to as Brexit, took place on January 31, 2020.

The EU and the UK reached an agreement on their new partnership in the Trade and Cooperation Agreement, which entered into force on May 1, 2021.

As of January 1, 2021, the Medicines and Healthcare Products Regulatory Agency (the “MHRA”) became responsible for supervising medicines and medical devices in Great Britain, comprising England, Scotland and Wales under domestic law, whereas Northern Ireland continues to be subject to EU rules under the Northern Ireland Protocol, as amended by the so called Windsor Framework agreed in February 2023.

As of January 1, 2025, the changes introduced by the Windsor Framework resulted in the MHRA being responsible for approving all medicinal products destined for the UK market (Great Britain and Northern Ireland), and the EMA will no longer have any role in approving medicinal products destined for Northern Ireland.

As of January 1, 2025, the Medicines and Healthcare Products Regulatory Agency (the “MHRA”), is responsible for approving all medicinal products destined for the UK market (Great Britain and Northern Ireland), and the EMA will no longer have any role in approving medicinal products destined for Northern Ireland.

The MHRA relies on the Human Medicines Regulations 2012 (SI 2012/1916) (as amended) (the “HMR”), as the basis for regulating medicines.

The HMR has incorporated into the domestic law the body of EU law instruments governing medicinal products that pre-existed prior to the UK’s withdrawal from the EU.

The HMR has incorporated into domestic law the body of EU law instruments governing medicinal products that pre-existed prior to the United Kingdom’s withdrawal from the EU.

On April 28, 2025,the UK Parliament adopted amendments to improve and strengthen the UK’s clinical trials regulatory regime;

they will take effect on April 28, 2026.

These changes were needed since the current UK requirements are based upon the now-repealed EU Clinical Trials Directive (2001/20/EC), which has been replaced by the European Clinical Trials Regulation (Regulation EU No 536/2014).

Since the UK left the EU prior to the date on which the EU CTR took effect, the UK legal framework did not benefit from the same revisions as occurred at EU level.

As of January 1, 2024, a new international recognition procedure (“IRP”) applies which intends to facilitate approval of pharmaceutical products in the UK.

The IRP is open to applicants that have already received an authorization

for the same product from one of the MHRA’s specified Reference Regulators (“RRs”).

The RRs notably include EMA and regulators in the EEA member states for approvals in the EU centralized procedure and mutual recognition procedure as well as the FDA (for product approvals granted in the U.S.).The RR assessment must have undergone a full and standalone review.

The IRP is open to applicants that have already received an authorization for the same product from one of the MHRA’s specified Reference Regulators (“RRs”).

The RR assessment must have undergone a full and standalone review.

RR assessments based on reliance or recognition cannot be used to support an IRP application.

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Many countries outside of the United States maintain rigorous laws governing the privacy and security of personal information.

The collection, use, disclosure, transfer, or other processing of personal data, including personal health data, regarding individuals who are located in the European Economic Area (“EEA”), and the processing of personal data that takes place in the EEA, is subject to the General Data Protection Regulation (“GDPR”), which became effective on May 25, 2018.

The collection, use, disclosure, transfer, or other processing of personal data, including personal health data, regarding individuals who are located in the EEA, and the processing of personal data that takes place in the EEA, is subject to the General Data Protection Regulation (“GDPR”), which became effective on May 25, 2018.

The GDPR is wide-ranging in scope and imposes numerous requirements on companies that process personal data, and it imposes heightened requirements on companies that process health and other sensitive data, such as requiring in many situations that a company obtain the consent of the individuals to whom the sensitive personal data relate before processing such data.

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Compliance with the GDPR is a rigorous and time-intensive process that may increase the cost of doing business or require companies to change their business practices to ensure full compliance.

In July 2020, the Court of Justice of the European Union (the “CJEU”) invalidated the EU-U.S.

Privacy Shield framework, one of the mechanisms used to legitimize the transfer of personal data from the EEA to the United States.

The CJEU decision also drew into question the long-term viability of an alternative means of data transfer, the standard contractual clauses, for transfers of personal data from the EEA to the United States.

Following the withdrawal of the U.K.

from the EU, the U.K.

Data Protection Act 2018 applies to the processing of personal data that takes place in the U.K.

and includes parallel obligations to those set forth by GDPR.

Following the July 2020 Court of Justice of the European Union judgment invalidating the so-called EU-U.S.

Privacy Shield, the European Commission adopted an adequacy decision for the EU-U.S.

Data Privacy Framework in July 2023.

This adequacy decision permits U.S.

companies who self-certify under the EU-U.S.

Data Privacy Framework to rely on it as a valid data transfer mechanism for data transfers from the European Union to the United States.

However, some privacy advocacy groups have already suggested that they will be challenging the EU-U.S.

Data Privacy Framework, and there is currently one pending litigation against the EU-U.S.

Data Privacy Framework before the Court of Justice of the European Union (the “CJEU”), C-703/25 P – Latombe v Commission.

If these challenges are successful, they may not only impact the EU-U.S.

Data Privacy Framework, but also further limit the viability of the so-called standard contractual clauses and other data transfer mechanisms.

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The European Commission decided in June 2021 that the level of data protection in the UK is “essentially adequate” for purposes of data transfer from the EU to the UK.

On December 19, 2025, the European Commission renewed this decision until December 27, 2031.

The UK and the U.S.

have also agreed to a U.S.- UK “Data Bridge,” which functions similarly to the EU-U.S.

Data Privacy Framework and provides an additional legal mechanism for companies to transfer personal data from the UK to the U.S.

Switzerland has also taken an adequacy decision in relation to the Swiss-U.S.

Data Privacy Framework (which functions similarly to the EU-U.S.

Data Privacy Framework and the U.S.-UK Data Bridge in relation to data transfers from Switzerland to the U.S.).

Additionally, in October 2022, President Biden signed an executive order to implement the EU-U.S.

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Pricing of prescription pharmaceuticals is subject to governmental control in many countries.

Pricing negotiations with governmental authorities can extend well beyond the receipt of regulatory marketing approval for a product and may require us to conduct a clinical trial that compares the cost effectiveness of any product candidates we may develop to other available therapies.

Pricing negotiations with governmental authorities can extend well beyond the receipt of regulatory marketing approval for a

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product and may require us to conduct a clinical trial that compares the cost effectiveness of any product candidates we may develop to other available therapies.

The conduct of such a clinical trial could be expensive and result in delays in our commercialization efforts.

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Restrictions under applicable federal and state healthcare laws and regulations, include the following:

kind, to induce or reward either the referral of an individual for, or the purchase, order or recommendation of, any good or service, for which payment may be made, in whole or in part, under a federal healthcare program such as Medicare and Medicaid;

federal Anti-Kickback Statute, which prohibits, among other things, persons and entities from knowingly and willfully soliciting, offering, paying, receiving, or providing remuneration, directly or indirectly, in cash or in kind, to induce or reward either the referral of an individual for, or the purchase, order or recommendation of, any good or service, for which payment may be made, in whole or in part, under a federal healthcare program such as Medicare and Medicaid;

• the federal civil and criminal false claims laws, including the civil False Claims Act, and civil monetary penalties laws, which prohibit individuals or entities from, among other things, knowingly presenting, or causing to be presented, to the federal government, claims for payment that are false, fictitious, or fraudulent or knowingly making, using, or causing to made or used a false record or statement to avoid, decrease, or conceal an obligation to pay money to the federal government;

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• HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act, and their respective implementing regulations, including the Final Omnibus Rule published in January 2013, which impose obligations, including mandatory contractual terms, with respect to safeguarding the privacy, security, and transmission of individually identifiable health information;

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• the federal false statements statute, which prohibits knowingly and willfully falsifying, concealing or covering up a material fact or making any materially false statement in connection with the delivery of or payment for health care benefits, items or services;

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For example, with enactment of the Tax Cuts and Jobs Act of 2017, which was signed by President Trump on December 22, 2017, Congress repealed the “individual mandate.” The repeal of this provision, which requires most Americans to carry a minimal level of health insurance, became effective in 2019.

In June 2021, the U.S.

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June 2021, the U.S.

Supreme Court dismissed an action seeking to strike down the ACA after finding that the plaintiffs do not have standing to challenge the constitutionality of the ACA.

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That regulation was challenged in a lawsuit by the Pharmaceutical Research and Manufacturers of America (“PhRMA”) but the case was dismissed by a federal district court in February 2023 after the court found that PhRMA did not have standing to sue the HHS.

Seven states (Colorado, Florida, Maine, New Hampshire, New Mexico, Texas and Vermont) have passed laws allowing for the importation of products from Canada.

North Dakota and Virginia have passed legislation establishing workgroups to examine the impact of a state importation program.

As of May 2024, five states (Colorado, Florida, Maine, New Hampshire and New Mexico) had submitted Section 804 Importation Program proposals to the FDA.

Several states have passed laws allowing for the importation of products from Canada.

On January 5, 2023, the FDA approved Florida’s plan for Canadian product importation.

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The state will also need to relabel the products and perform quality testing of the products to meet FDA standards.

On May 21, 2025, the FDA announced that it would offer individual states the opportunity to submit a draft proposal for pre-review and meet with the agency to obtain initial feedback from FDA prior to formally submitting their SIP proposal.

The intent of these meetings is to assist states in developing their proposals by further clarifying requirements, enhancing the quality of proposals submitted to the agency and ultimately shortening the review timeline.

On August 16, 2022, the Inflation Reduction Act of 2022 (“IRA”) was signed into law by President Biden.

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and replaces the Part D coverage gap discount program with a new discounting program (beginning in 2025).

The IRA permits the

Secretary of the HHS to implement many of these provisions through guidance, as opposed to regulation, for the initial years.

The IRA permits the Secretary of the HHS to implement many of these provisions through guidance, as opposed to regulation, for the initial years.

Specifically, with respect to price negotiations, Congress authorized Medicare to negotiate lower prices for certain costly single-source drug and biologic products that do not have competing generics or biosimilars and are reimbursed under Medicare Part B and Part D.

The CMS may negotiate prices for ten high-cost drugs paid for by Medicare Part D starting in 2026, followed by 15 Part D drugs in 2027, 15 Part B or Part D drugs in 2028, and 20 Part B or Part D drugs in 2029 and beyond.

This provision applies to drug products that have been approved for at least 9 years and biologics that have been licensed for 13 years, but it does not apply to drugs and biologics that have been approved for a single rare disease or condition.

This provision applies to drug products that have been approved for at least nine years and biologics that have been licensed for 13 years Drugs and biologics that have been approved for a single rare disease or condition were originally categorically excluded from price negotiation.

With passage of the One Big Beautiful Bill Act on July 3, 2025, which was signed into law on July 4, 2025, Congress extended this exemption to drugs and biologics with multiple orphan drug designations.

Further, the legislation subjects drug manufacturers to civil monetary penalties and a potential excise tax for failing to comply with the legislation by offering a price that is not equal to or less than the negotiated “maximum fair price” under the law or for taking price increases that exceed inflation.

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While there had been some questions about the Trump Administration’s position on this program, the CMS issued a public statement on January 29, 2025, declaring that lowering the cost of prescription drugs is a top priority of the new administration and the CMS is committed to considering opportunities to bring greater transparency in the negotiation program.

The second cycle of negotiations with participating drug companies will occur during 2025, and any negotiated prices for this second set of drugs will be effective starting January 1, 2027.

On June 6, 2023, Merck filed a lawsuit against the HHS and the CMS asserting that, among other things, the IRA’s Drug Price Negotiation Program for Medicare constitutes an uncompensated taking in violation of the Fifth Amendment of the Constitution.

Subsequently, a number of other parties, including the U.S.

Chamber of Commerce (“Chamber”), Bristol Myers Squibb Company, the Pharmaceutical Research and Manufacturers of America, Astellas, Novo Nordisk, Janssen Pharmaceuticals, Novartis, AstraZeneca and Boehringer Ingelheim, also filed lawsuits in various courts with similar constitutional claims against the HHS and the CMS.

The HHS has generally won the substantive disputes in these cases, and various federal district court judges have expressed skepticism regarding the merits of the legal arguments being pursued by the pharmaceutical industry.

Certain of these cases are now on appeal and, on October 30, 2024, the Court of Appeals for the Third Circuit heard oral argument in three of these cases.

The second cycle of

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negotiations with participating drug companies will occur during 2025, and any negotiated prices for this second set of drugs will be effective starting January 1, 2027.

On June 6, 2023, Merck & Co., Inc., filed a lawsuit against HHS and CMS asserting that, among other things, the IRA’s Drug Price Negotiation Program for Medicare constitutes an uncompensated taking in violation of the Fifth Amendment of the U.S.

Constitution.

Subsequently, other parties, including the U.S.

Chamber of Commerce, or Chamber of Commerce, Bristol Myers Squibb Company, the PhRMA, Astellas Pharma US, Inc., Novo Nordisk Inc., Janssen Pharmaceuticals, Inc., Novartis Pharmaceutical Corporation, AstraZeneca L.P.

and Boehringer Ingelheim Pharmaceuticals, Inc.

also filed lawsuits in various courts with similar constitutional claims against HHS and CMS.

HHS has generally won the substantive disputes in these cases or succeeded in getting claims dismissed for lack of standing.

Most of these cases are now on appeal.

On October 30, 2024, the U.S.

Court of Appeals for the Third Circuit heard oral arguments in three of these cases.

In April 2025, the U.S.

Court of Appeals for the Second Circuit and the U.S.

Court of Appeals for the Third Circuit heard arguments in an additional three cases.

On May 8, 2025, the U.S.

Court of Appeals for the Third Circuit rejected AstraZeneca L.P.’s challenge to the Medicare price negotiation program, finding that the program did not violate the company’s due process rights under the Constitution since there is no protected property interest in selling goods to Medicare beneficiaries at a price higher than what the government is willing to pay in reimbursement.

Litigation involving these and other provisions of the IRA will continue with unpredictable and uncertain results.

The Trump Administration has taken a number of actions to reduce the costs of pharmaceutical products.

For example, on April 15, 2025, President Trump issued an Executive Order which directs HHS to take steps to reduce the prices of pharmaceutical products.

Such measures include streamlining the state drug importation program and modifying provisions of the 340B program.

Further, on May 12, 2025, President Trump issued an additional Executive Order calling on pharmaceutical manufacturers to voluntarily reduce the prices of medicines in the United States.

The Order provides that if such actions do not lower the costs of pharmaceuticals, the Secretary of HHS would pursue other actions, including proposing a rulemaking that imposes Most-Favored Nation (“MFN”) pricing in the United States.

Thereafter, on July 31, 2025, the President issued letters to 17 pharmaceutical companies reiterating the requirements of the May 12, 2025, Executive Order and demanding that such companies extend MFN pricing to Medicaid patients, guarantee MFN pricing for newly-launched drug products, return increased revenues abroad to patients in the U.S.

and provide for direct purchasing at MFN pricing.

Since that time, virtually all of these pharmaceutical companies have entered into agreements with the administration to provide for lower prices on certain pharmaceuticals.

At the state level, individual states are increasingly aggressive in passing legislation and implementing regulations designed to control pharmaceutical and biological 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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These laws are evolving and may increase both our obligations and our regulatory risks in the future.

In the health care industry generally, under HIPAA, the

HHS has issued regulations to protect the privacy and security of protected health information (“PHI”) used or disclosed by covered entities including certain healthcare providers, health plans and healthcare clearinghouses.

In the health care industry generally, under HIPAA, the HHS has issued regulations to protect the privacy and security of protected health information (“PHI”) used or disclosed by covered entities including certain healthcare providers, health plans and healthcare clearinghouses.

HIPAA also regulates standardization of data content, codes and formats used in healthcare transactions and standardization of identifiers for health plans and providers.

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HIPAA may apply to us in certain circumstances and may also apply to our business partners in ways that may impact our relationships with them.

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Our clinical trials will be regulated by the Common Rule, which also includes specific privacy-related provisions.

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The provisions in the CPRA may apply to some of our business activities.

In addition to California, at least eighteen other states have passed comprehensive privacy laws similar to the CCPA and CPRA.

These laws are either in effect or will go into effect sometime before the end of 2026.

In addition to California, several other states have passed comprehensive privacy laws similar to the CCPA and CPRA that are either in effect or will go into effect sometime before the end of 2026.

Like the CCPA and CPRA, these laws create obligations related to the processing of personal information, as well as special obligations for the processing of “sensitive” data, which includes health data in some cases.

Some of the provisions of these laws may apply to our business activities.

There are also states that are strongly considering or have already passed comprehensive privacy laws during the 2024 legislative sessions that will go into effect in 2025 and beyond.

There are also states that are strongly considering additional laws that will go into effect that could go into effect in 2026 and beyond.

Other states will be considering similar laws in the future, and Congress has also been debating passing a federal privacy law.

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For example, the State of Washington passed the My Health My Data Act in 2023 which specifically regulated health information that is not otherwise regulated by the HIPAA rules, and the law also has a private right of action, which further increases the relevant compliance risk.

Connecticut and Nevada have also passed similar laws regulating consumer health data, and more states are considering such legislation in 2024.

Connecticut and Nevada have also passed similar laws regulating consumer health data, and more states are considering such legislation.

These laws may impact our business activities, including our identification of research subjects, relationships with business partners and ultimately the marketing and distribution of our products.

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The heightening compliance environment and the need to build and maintain robust and secure systems to comply with different privacy compliance and/or reporting requirements in multiple jurisdictions could increase the possibility that a healthcare company may fail to comply fully with one or more of these requirements.

diminished profits and future earnings, additional reporting requirements and/or oversight if we become subject to a consent decree or similar agreement to resolve allegations of non-compliance with these laws, and the curtailment or restructuring of our operations, any of which could adversely affect our ability to operate our business and our results of operations.

If our operations are found to be in violation of any of the privacy or data security laws or regulations described above that are applicable to us, or any other laws that apply to us, we may be subject to penalties, including potentially significant criminal, civil and administrative penalties, damages, fines, contractual damages, reputational harm, diminished profits and future earnings, additional reporting requirements and/or oversight if we become subject to a consent decree or similar agreement to resolve allegations of non-compliance with these laws, and the curtailment or restructuring of our operations, any of which could adversely affect our ability to operate our business and our results of operations.

To the extent that any product candidates we may develop, once approved, are sold in a foreign country, we may be subject to similar foreign laws.

Additional Regulations

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In addition to the foregoing, state, and federal laws regarding environmental protection and hazardous substances, including the Occupational Safety and Health Act, the Resource Conservation and Recovery Act, and the Toxic Substances Control Act, affect our business.

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As of February 1, 2026, we had 87 full-time employees, including 26 employees with M.D.

In December 2024, we announced a reduction in our employee workforce by approximately 180 positions (the “Reduction”) in connection with the decision to discontinue clinical development of reni-cel.

The Reduction commenced in December 2024, and we expect it to be substantially complete by the end of June 2025.

Following the Reduction, we expect to have approximately 110 full-time employees, including approximately 30 employees with M.D.

Approximately 80 of the 110 employees will be primarily engaged in research and development activities.

Approximately 66 of our full-time employees are primarily engaged in research and development activities.

None of our employees are represented by a labor union or covered by a collective bargaining agreement.

At Editas, we seek to unlock the full potential of gene editing technology, and we recognize that our success is driven by our dynamic, passionate and diverse team.

In recognition of the importance of our employees to the success of our mission, we aim to create a best-in-class people experience driven by performance excellence.

In 2024, we continued to build on the culture roadmap we set in 2023, focusing on three key areas.

First, we sought to strengthen our next generation of leaders through a formal development series focused on deepening enterprise leadership capability and building our executive succession pipeline.

Second, we conducted extensive workforce planning to ensure that our workforce capabilities and mastery aligned with our corporate strategy, and we engaged with functional leadership on approaches to develop and execute on an enterprise talent strategy.

Third, we sought to expand on the ways in which we engage with our employee community.

To that end, we created the Editas Medicine Culture Committee, which is comprised of a diverse group of employees from varying levels across all functions of our organization.

This committee is tasked with the goal of creating an exceptional workplace experience that enables employees to work effectively, efficiently and enjoyably with speed to achieving our corporate mission.

The committee is structured into six teams:

Learning and Development, Belonging, Inclusion, Diversity, and Equity, Events, Performance and Recognition, Advocacy and Community, and Decision Making, which meet periodically to review and develop executable plans for the continued progress of our cultural mission.

At Editas, we seek to translate the power and potential of CRISPR gene editing into a robust pipeline of medicines for people living with serious diseases around the world.

We recognize that our success is driven by our courageous, collaborative, and passionate employees, who celebrate diversity and together foster a workplace that values inclusivity, where all voices are heard and respected.

We seek to be an employer of choice that empowers our employees to drive innovation to develop transformative medicines for people living with serious diseases around the world.

Our Core Values

We work together with integrity, guided by our distinct culture, to develop transformative medicines for people living with serious diseases around the world.

We work together with integrity, guided by our distinct culture.

At the center of our culture are our core values, which guide and define the behaviors that make our culture unique and enable us to bring our best selves forward to achieve our mission of translating the promise of gene editing into a broad class of differentiated, transformational medicines for previously untreatable diseases:

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As of December 31, 2025, 48% of our full-time employees were women and 37% of our senior management (director level and above) were women.

Following the Reduction, we expect our full-time employee workforce to be comprised of approximately 53% women and our senior management to be approximately 44% women.

As of December 31, 2025, 44% of our full-time employees identify as racially/ethnically diverse and 37% of our senior management identify as racially/ethnically diverse.

Following the Reduction, we expect our full-time employee workforce to be comprised approximately 35% of individuals who identify as racially/ethnically diverse and approximately 26% of our senior management to be individuals who identify as racially/ethnically diverse.

Recruitment, Retention and Development

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We believe a key component of recruiting, retaining and motivating our employees is our total compensation package.

For this reason, we provide employees with competitive compensation, including market-competitive salary and equity awards, along with competitive benefits packages, including medical, dental, vision and life insurance, an employee stock purchase plan, flexible spending accounts, short- and long-term disability and matching contributions to a 401(k) tax-deferred savings plan.

We also provide annual cash incentive bonus opportunities that are tied to both company performance and individual performance to foster a pay-for-performance culture.

For this reason, we provide employees with competitive compensation, including market-competitive salary and equity awards, along with competitive benefits packages, including medical, dental, vision and life insurance, flexible spending accounts, short- and long-term disability and matching contributions to a 401(k) tax-deferred savings plan.

We also provide annual cash incentive bonus opportunities that are tied to both company performance and

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individual performance to foster a pay-for-performance culture.

We regularly benchmark these total rewards against our industry peers to ensure we remain competitive and attractive to potential new hires.

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Available Information

We maintain an internet website at www.editasmedicine.com and make available free of charge through our website our Annual Reports on Form 10-K, Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and amendments to those reports filed or furnished pursuant to Sections 13(a) and 15(d) of the Exchange Act of 1934, or the Exchange Act.

We make these reports available through our website as soon as reasonably practicable after we electronically file such reports with, or furnish such reports to, the U.S.

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Our website address is included in this Annual Report on Form 10-K as an inactive technical reference only.

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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