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Item 1. Business

2026-03-17 compared with 2025-03-20 · 280 added, 282 removed, 1039 unchanged (35% of the section changed)

Therapeutics Inc., a Delaware corporation (together with our subsidiaries, “we,” “our,” “Abeona”

or the “Company”), is a clinical-stage biopharmaceutical company developing cell and gene therapies for life-threatening

Our lead clinical program is for prademagene zamikeracel (“pz-cel”), an autologous, cell-based gene therapy currently

in development for recessive dystrophic epidermolysis bullosa (“RDEB”).

Pz-cel has been granted Orphan Drug and Rare Pediatric

Disease (“RPD”) designations by the U.S.

Food and Drug Administration (“FDA”) and Orphan Drug Designation by

the European Medicines Agency (“EMA”).

plan to continue development of adeno-associated virus (“AAV”) based gene therapies designed to treat ophthalmic diseases

with high unmet need using the novel AIM™ capsids exclusively licensed from the University of North Carolina at Chapel Hill (“UNC”)

and developed internally through our AAV vector research programs.

Abeona’s novel, next-generation AAV capsids are being evaluated

to improve tropism profiles for a variety of devastating diseases.

or the “Company”), is a commercial-stage biopharmaceutical company developing cell and gene therapies for life-threatening

On April 28, 2025, the U.S.

Food and Drug Administration (“FDA”) approved ZEVASKYN ® (prademagene

zamikeracel) gene-modified cellular sheets, also known as ZEVASKYN ® , as the first and only autologous cell-based gene

therapy for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (“RDEB”),

a serious and debilitating genetic skin disease.

There is no cure for RDEB, and ZEVASKYN ® is the only FDA-approved product to treat RDEB wounds with

a single surgical application.

ZEVASKYN ® was granted Orphan Drug and Rare Pediatric Disease designations by the FDA

ZEVASKYN ® is manufactured at our current

Good Manufacturing Practices (“cGMP”) manufacturing facility in Cleveland, Ohio.

Treatments are available through ZEVASKYN ®

qualified treatment centers, a network of centers that are selected based on their expertise in cell and gene therapy and trained to administer

As of March 2026, we have activated 4 qualified treatment centers and are in discussions with additional centers

as we continue to expand the ZEVASKYN ® qualified treatment network.

The Company’s development portfolio also features adeno-associated virus (“AAV”)-based

gene therapies designed to treat ophthalmic diseases with high unmet need using novel AIM™ capsids.

Abeona’s novel AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases.

We partner with leading academic researchers, patient

advocacy organizations, caregivers and other biotechnology companies to develop and deliver therapies that address the underlying cause

of a broad spectrum of rare genetic diseases for which no effective treatment options exist today.

Mission and Strategy

is a clinical-stage biopharmaceutical company developing cell and gene therapies for life-threatening diseases.

Our lead clinical

program is pz-cel, autologous, COL7A1 gene-corrected epidermal sheets, an investigational product currently in development for RDEB.

In November 2022, we announced positive top-line data from the VIITAL™ study evaluating the efficacy, safety and tolerability

The VIITAL™ study met both its co-primary efficacy endpoints demonstrating statistically significant,

clinically meaningful improvements in wound healing and pain reduction in large chronic RDEB wounds.

In September 2023, we

submitted a Biologics License Application (“BLA”) for pz-cel to the FDA.

In November 2023, the FDA accepted and granted

priority review for our BLA for pz-cel, and subsequently, under the Prescription Drug User Fee Act (“PDUFA”), the FDA

set a target action date of May 25, 2024.

In April 2024, the FDA issued a Complete Response Letter (“CRL”) in response

The CRL noted that certain additional information needed to satisfy the Chemistry Manufacturing and Controls

(“CMC”) requirements of the pz-cel BLA must be satisfactorily resolved before the application can be approved.

did not identify any deficiencies related to the clinical efficacy or clinical safety data in the BLA, and the FDA did not request

any new clinical trials or clinical data to support the approval of pz-cel.

In August 2024, we completed a Type A Meeting with

the FDA to discuss the forthcoming resubmission of our BLA and in October 2024, we resubmitted our BLA.

The FDA notified the

Company in November 2024 that the BLA was accepted for review, with an assigned PDUFA target action date of April 29,

partner with leading academic researchers, patient advocacy organizations, caregivers and other biotechnology companies to develop therapies

that address the underlying cause of a broad spectrum of rare genetic diseases for which no effective treatment options exist today.

strategy consists of:

and Commercializing our Late-Stage Clinical Cell and Gene Therapy Programs with a Focus on Life-Threatening Diseases.

our cell and gene therapy expertise in research and development, we believe we are positioned to introduce efficacious and safe therapeutics

to transform the standard of care in devastating diseases and establish our leadership position in the field.

We intend to commercialize

our assets either by ourselves or through strategic partnerships, subject to FDA approval.

Commercializing

ZEVASKYN ® and Advancing and Commercializing our Cell and Gene Therapy Programs.

our cell and gene therapy expertise in research and development, we believe we are positioned to introduce efficacious and safe

therapeutics to transform the standard of care in devastating diseases and establish our leadership position in the field.

commercializing ZEVASKYN ® by ourselves and may develop future strategic partnerships for ZEVASKYN ® and

we intend to commercialize our other assets either by ourselves or through strategic partnerships, subject to FDA

Novel In-Vivo Gene Therapies Using AIM™ Capsid Technology.

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our Leadership Position in Commercial-Scale Cell and Gene Therapy Manufacturing.

established current Good Manufacturing Practice (“cGMP”), clinical-scale manufacturing capabilities for engineered cell therapies

and AAV-based gene therapies in our state-of-the-art Cleveland, Ohio facility.

We believe that our manufacturing platform provides us

with distinct advantages, including flexibility, scale, reliability, and the potential for reduced development risk, reduced cost, and

established cGMP, commercial and clinical-scale manufacturing capabilities for engineered cell and gene therapies in our state-of-the-art Cleveland, Ohio facility.

We believe that our manufacturing platform provides us with

distinct advantages, including flexibility, scale, reliability, and the potential for reduced development risk, reduced cost, and

faster times to market.

We have focused on establishing internal CMC capabilities that drive value for our organization through process

development, assay development and manufacturing.

We have also deployed robust quality systems governing all aspects of product lifecycle

from preclinical through commercial stage.

We have focused on establishing internal Chemistry Manufacturing and Controls (“CMC”) capabilities that drive value for our organization through

process development, assay development and manufacturing.

We have also deployed robust quality systems governing all aspects of

product lifecycle from preclinical through commercial stage.

Additional Cell and Gene Therapy Franchises and Adjacencies through In-Licensing and Strategic Partnerships.

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engine and product candidates.

Next-Generation Cell and Gene Therapy

for the Treatment of RDEB

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and epidermal layers to one another.

a result of the genetic defect, RDEB patients have fragile skin, which can easily damage to produce open and blistering wounds, disfiguring

scars throughout the body, fused fingers and toes, limits in range of motion at joints (e.g., arms and legs), corneal abrasions, and

an abnormal narrowing of the esophagus.

Long-term RDEB patients can suffer from anemia, are at high risk of developing aggressive squamous

cell carcinomas, infections, and premature death.

The most severe patients are approximately 20 times more likely to die by 30 years

of age than the general population.

a result of the genetic defect, RDEB patients have fragile skin, which can easily damage to produce open and blistering wounds,

disfiguring scars throughout the body, fused fingers and toes, limits in range of motion at joints (e.g., arms and legs), corneal

abrasions, and an abnormal narrowing of the esophagus.

Long-term RDEB patients can suffer from anemia, infections and are at high

risk of developing aggressive squamous cell carcinomas, infections, and premature death.

The most severe patients are approximately

20 times more likely to die by 30 years of age than the general population.

to other rare diseases, the incidence and prevalence of RDEB are not well defined.

Incidence of 0.2 to 3.05 per million births and

prevalence of 0.14 to 1.35 per million people have been observed across different geographies, primarily estimated by limited

population analyses of clinical databases or registries (Eichstadt et al.;

Incidence of 0.2 to 3.05 per million births and prevalence

of 0.14 to 1.35 per million people have been observed across different geographies, primarily estimated by limited population analyses

of clinical databases or registries (Eichstadt et al.;

Clinical, Cosmetic and Investigational Dermatology, 2019).

Using genetic modeling of COL7A1 variants, Stanford University estimated the incidence of RDEB to be approximately 63 per

million births, and prevalence could be up to 3,850 patients in the U.S., whose wounds may benefit from COL7A1-mediated treatments

such as pz-cel.

Based on claims analysis, we estimate that approximately 750 moderate to severe RDEB patients in the U.S.

pz-cel eligible patients at the time of a potential pz-cel launch (Clearview Claims Analysis, 2024).

Using genetic modeling

of COL7A1 variants, Stanford University estimated the incidence of RDEB to be approximately 63 per million births, and prevalence could

be up to 3,850 patients in the U.S., whose wounds may benefit from COL7A1-mediated treatments such as ZEVASKYN ® .

on claims analysis, we estimate that approximately 750 moderate to severe RDEB patients in the U.S.

would be ZEVASKYN ®

eligible patients (Clearview Claims Analysis, 2024).

patients have active disease, with the majority of their wounds typically greater than 20 cm 2 in size (Stanford University;

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Nature Research, 2017).

our VIITAL TM phase 3 and phase 1/2a clinical trials, pz-cel was applied as a one-time surgical procedure onto RDEB wounds

and has shown up to 8 years of durable wound healing and associated pain reduction even in the tough large, chronic RDEB wounds.

Patients evaluated in the VIITAL TM phase 3 trial had large wounds (> 20cm 2 ) and, on average, had wounds

that remained open for 6.2 years, and in some cases up to 21 years, prior to pz-cel treatment.

Most RDEB patients have large and

chronic wounds that carry the highest burden, including the need for frequent lengthy dressing changes, pain, pruritus (itch), risk of

infection, and developing skin cancer.

our VIITAL TM phase 3 and phase 1/2a clinical trials, ZEVASKYN ® was applied as a one-time surgical

procedure onto RDEB wounds and has shown up to 12 years of durable wound healing and associated pain reduction even in the

tough-to-treat large, chronic RDEB wounds.

Patients evaluated in the VIITAL TM phase 3 trial had some of the worst wounds.

These wounds were large (> 20cm 2 ) and, on average, had remained open for 6.2 years, and in some

cases up to 21 years, prior to ZEVASKYN ® treatment.

Most RDEB patients have large and chronic wounds that carry the

highest burden, including the need for frequent lengthy dressing changes, pain, pruritus (itch), risk of infection, and developing

Management of RDEB

of care in RDEB wound management currently consists of lengthy and labor-intensive supportive care to limit contamination and infection,

RDEB wound management currently consists of lengthy and labor-intensive supportive care to limit contamination and infection,

and reduction in mechanical forces that produce new blisters.

Care usually includes treatment of new blisters by lancing and draining.

Wounds are then dressed with non-adherent material, covered with padding for stability and protection, and secured with an elastic

wrap for integrity.

In a cost analysis conducted by Debra of America, based on 3,274 patient health insurance claims from private

insurance, the annual cost of care for dystrophic epidermolysis bullosa (DEB) was found to be 465% greater than the annual cost to the

healthcare system from all people.

The cost of wound care supplies could be as high as $996,000 per year.

Wounds are then dressed with non-adherent material, covered with padding for stability and protection, and secured with an elastic wrap

for integrity.

In a cost analysis conducted by Debra of America, based on 3,274 patient health insurance claims from private insurance,

the annual cost of care for dystrophic epidermolysis bullosa (DEB) was found to be 465% greater than the annual cost to the healthcare

system from all people and a substantial share of this burden stems from ongoing wound-care needs.

For many patients, these wound-care expenses

represent a major, persistent financial strain on both families and the healthcare system, reflecting the chronic and resource-intensive

nature of RDEB management.

patients also have periodic surgeries to relieve disease related issues such as narrowing of their esophagus, fusing of fingers, and

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associated with Junctional (JEB) and DEB, respectively.

RDEB patients continue to seek durable treatments for addressing their

wounds in the current treatment landscape.

is an investigational product comprised of autologous epidermal sheets in which a functioning COL7A1 gene is inserted into a patient’s

own skin cells (keratinocytes) using a retrovirus.

The keratinocytes are then grown into credit card-sized sheets and surgically applied

to the patient to restore Type VII collagen expression and skin function.

from a completed Phase 1/2a study that enrolled seven patients with large and chronic RDEB wounds at Stanford University showed that

pz-cel was well-tolerated and resulted in significant and durable wound healing (Siprashvili, Z., et al., 2016), with up to eight years

of follow-up (So.

Y, Nazaraoff, et al., Orphanet Journal Rare Disease 2022).

patients continue to seek durable treatments for addressing their wounds in the current treatment landscape.

Program History

is a commercial product comprised of autologous epidermal gene-modified sheets in which a functioning COL7A1 gene is inserted into a

patient’s own skin cells (keratinocytes) using a retrovirus vector.

The gene-modified keratinocytes are then grown into credit

card-sized sheets and surgically applied to the patient to restore Type VII collagen expression and skin function.

from a completed Phase 1/2a study that enrolled seven patients and treated 38 large and chronic RDEB wounds at Stanford University

showed that ZEVASKYN ® was well-tolerated and resulted in significant and durable wound healing (Siprashvili, Z., et

al., 2016), with up to eight years of follow-up after a single surgical application (So.

Y, Nazaraoff, et al., Orphanet Journal Rare

Disease 2022).

To date, there have been no reported serious adverse events.

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The pivotal phase 3 VIITAL™ study evaluated

the efficacy, safety, and tolerability of pz-cel in 43 large chronic wound pairs in 11 subjects with RDEB.

The large chronic wounds

randomized and treated in VIITAL™ measured greater than 20 cm 2 of surface area and had remained open for a minimum

of six months and a maximum of 21 years (mean 6.2 years).

The co-primary endpoints of the study were assessed at the six-month timepoint for:

(1) the proportion of RDEB

wound sites with greater than or equal to 50% healing from baseline, comparing randomized treated with matched untreated (control)

wound sites, as determined by direct investigator assessment;

and (2) pain reduction associated with

wound dressing change assessed by the mean differences in scores of the Wong-Baker FACES ® Pain Rating Scale between

randomized treated and matched untreated (control) wounds.

the efficacy, safety, and tolerability of ZEVASKYN ® in 43 large chronic wound pairs in 11 subjects with RDEB.

large chronic wounds randomized and treated in VIITAL™ measured greater than 20 cm 2 of surface area and had

remained open for a minimum of six months and a maximum of 21 years (mean 6.2 years).

The co-primary endpoints of the study were

assessed at the six-month timepoint for:

(1) the proportion of RDEB wound sites with greater than or equal to 50% healing from

baseline, comparing randomized treated with matched untreated (control) wound sites, as determined by direct investigator

and (2) patient-reported pain reduction associated with wound dressing change assessed by the mean differences in scores

of the Wong-Baker FACES ® Pain Rating Scale between randomized treated and matched untreated (control)

VIITAL™ study met both co-primary efficacy endpoints demonstrating statistically significant, clinically meaningful

improvements in wound healing and pain reduction in large chronic RDEB wounds.

Pz-cel was shown to be well-tolerated with no serious

treatment-related adverse events observed, consistent with past clinical experience.

There were no deaths or instances of positive

replication-competent retrovirus results, and no systemic immunologic responses were reported during the study, as well as no

squamous cell carcinoma at treatment sites after application of pz-cel.

Two subjects reported at least one serious adverse event

unrelated to pz-cel.

Four subjects reported related treatment emergent adverse events, including procedural pain, muscle spasms and

Infections unrelated to pz-cel were observed in eight patients.

September 2023, we submitted a BLA for pz-cel to the FDA.

In November 2023, the FDA accepted and granted priority review for our BLA

for pz-cel, and subsequently, under the Prescription Drug User Fee Act (“PDUFA”), the FDA set a target action date of May

In April 2024, the FDA issued a Complete Response Letter (“CRL”) in response to the BLA.

The CRL noted that certain

additional information needed to satisfy the Chemistry Manufacturing and Controls (“CMC”) requirements of the pz-cel BLA

must be satisfactorily resolved before the application can be approved.

The CRL did not identify any deficiencies related to the clinical

efficacy or clinical safety data in the BLA, and the FDA did not request any new clinical trials or clinical data to support the approval

In August 2024, we completed a Type A Meeting with the FDA to discuss our forthcoming resubmission of our BLA and in October 2024, we resubmitted our BLA.

The FDA notified the Company in November 2024 that the BLA was accepted for review, with an assigned

PDUFA target action date of April 29, 2025.

Pz-cel has been granted Regenerative Medicine Advanced Therapy (“RMAT”), Breakthrough

Therapy, Orphan Drug and RPD designations by the by the FDA as well as Orphan Drug designation by the EMA.

ZEVASKYN ® was shown to be

well-tolerated with no serious treatment-related adverse events observed, consistent with past clinical experience.

There were no

deaths or no instances of positive replication-competent retrovirus, no systemic immunologic responses were reported

during the study, as well as no squamous cell carcinoma at treatment sites after application of ZEVASKYN ® .

subjects reported at least one serious adverse event unrelated to ZEVASKYN ® .

Four subjects reported related treatment

emergent adverse events, including procedural pain, muscle spasms and pruritis.

Infections unrelated to ZEVASKYN ®

were observed in eight patients.

April 28, 2025, the FDA approved ZEVASKYN ® as the first and only autologous cell-based gene therapy for the treatment

of wounds in adult and pediatric patients with RDEB.

ZEVASKYN ® has been granted Regenerative Medicine Advanced Therapy

(“RMAT”), Breakthrough Therapy, Orphan Drug and RPD designations by the FDA as well as Orphan Drug designation by the EMA.

the potential benefits of Orphan Drug designation are a potential seven years of market exclusivity following FDA approval, potentially

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an approval for a BLA with RPD designation may qualify for a Priority Review Voucher (“PRV”), subject to final determination

A PRV may be used to receive an expedited review of a subsequent marketing application for a different product or sold to another

have continued to prepare our current Good Manufacturing Practices (“cGMP”) facility in Cleveland, Ohio for

manufacturing commercial grade pz-cel drug product to support our planned commercial launch of pz-cel, if approved.

drug product for all our VIITAL™ study participants has been manufactured at our Cleveland facility.

As part of our commercial

planning, we continue to engage with stakeholders across the healthcare system, including private payors that cover the majority of

RDEB lives, and healthcare providers to better understand market access and potential pricing for pz-cel.

We are also in discussions

with 5 to 7 epidermolysis bullosa centers of excellence to onboard them as pz-cel Qualified Treatment Centers (“QTC”)

following potential FDA approval.

A PRV may be used to receive an expedited review of a subsequent marketing application for a different product or sold to

another company.

We received a PRV upon ZEVASKYN ® ’s approval, and on May 9, 2025, we entered into a definitive asset

purchase agreement that transferred the PRV to a third party.

The PRV sale was completed in June 2025 following early termination of

the applicable waiting period for U.S.

antitrust review of the transaction.

We received gross proceeds of $155.0 million from the sale

have prepared our current cGMP facility in Cleveland, Ohio for manufacturing commercial

grade ZEVASKYN ® drug product to support our commercial launch of ZEVASKYN ® .

ZEVASKYN ® study

drug product for all our VIITAL™ study participants was manufactured at our Cleveland facility.

commercialization strategy centers on establishing and expanding a network of qualified treatment centers with the clinical expertise

and infrastructure required to administer our therapy.

As of March 2026, we had activated four qualified treatment centers.

These centers

were selected based on their expertise in areas such as cell and gene therapy and have undergone specialized training to administer ZEVASKYN ® .

involves obtaining a biopsy from the patient and shipping the biopsied cells to our manufacturing facility, where the patient specific

product is manufactured as multilayer cellular sheets containing gene-corrected keratinocytes.

Following testing, the product is then

shipped back to the qualified treatment center where the patient receives treatment.

treated our first ZEVASKYN ® patient in the fourth quarter of 2025.

As part of commercial launch efforts, we continue

to engage with multiple stakeholders across the healthcare system, including leading EB hospital institutions, private and public health

insurers, as well as the patient and physician community.

To date, we have activated four qualified treatment centers that now can identify and treat patients with ZEVASKYN ® .

These qualified treatment centers are geographically dispersed across

and include Ann & Robert H.

Lurie Children’s Hospital of Chicago, Lucile Packard Children’s Hospital Stanford,

Children’s Hospital Colorado, and The University of Texas Medical Branch (UTMB) in Galveston, Texas.

We have secured broad insurance coverage

for ZEVASKYN ® from multiple national and regional commercial insurers as well as from the CMS (Centers for Medicare and

Medicaid Services).

ZEVASKYN ® has coverage from all Medicaid programs across 50 US states and Puerto Rico.

Effective January

1, 2026, CMS also has issued a permanent J-code for ZEVASKYN ® that we expect will simplify claims and reimbursement processing

between qualified treatment centers and all payer types.

Next-Generation Cell and Gene Therapy

for the treatment of X-linked Retinoschisis (“XLRS”)

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future IND submission.

IND-enabling efficacy and toxicology animal studies are scheduled to be completed by the end of 2025.

Additionally,

cGMP manufacturing of clinical grade material has been initiated at a third-party vendor in anticipation of first in human trials being

initiated in 2026.

Due to focus on ZEVASKYN ® commercialization efforts, animal

efficacy and toxicology studies and cGMP manufacturing of clinical grade material has been postponed to 2026.

for the Treatment of Stargardt Disease

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and inner membrane remodeling.

Mutant phenotypes present with a progressive loss of RGCs that results in optic nerve degeneration and

Mutant phenotypes present with a progressive loss of RGCs that result in optic nerve degeneration and

legal blindness with a loss of visual acuity, optic disc pallor, and color vision deficits.

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previously received certain AAV gene therapy or subjects who have pre-existing antibodies to naturally occurring AAV serotypes.

In July 2024, we entered into a non-exclusive agreement

with Beacon Therapeutics (“Beacon”) under which Beacon will evaluate Abeona’s patented AAV204 capsid for the development

and commercialization of potential gene therapies for select ophthalmology indications.

Following a 12-month evaluation period, Beacon

will have the option to take a worldwide, non-exclusive license to use AAV204 in connection with up to five gene or disease targets.

will also have the right to use AAV204 for up to four additional nominated gene or disease targets subject to certain conditions.

receive an upfront payment upon Beacon’s exercise of its option to license AAV204, with additional payments upon the achievement

of certain development, regulatory, and sales milestones, along with tiered royalties on worldwide net sales for licensed products incorporating

July 2024, we entered into a non-exclusive agreement with Beacon Therapeutics (“Beacon”) under which Beacon will evaluate

Abeona’s patented AAV204 capsid for the development and commercialization of potential gene therapies for select ophthalmology

Following a 12-month evaluation period, Beacon exercised its option to take a worldwide, non-exclusive license to use AAV204

in connection with up to five gene or disease targets.

Beacon will also have the right to use AAV204 for up to four additional nominated

gene or disease targets subject to certain conditions.

We received an upfront payment upon Beacon’s exercise of its option to license

AAV204, with additional payments upon the achievement of certain development, regulatory, and sales milestones, along with tiered royalties

on worldwide net sales for licensed products incorporating AAV204.

Licensing Agreements

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Ultragenyx Pharmaceutical Inc.

(“Ultragenyx”), and for CLN1 disease (infantile Batten disease) and Rett syndrome to

Taysha Gene Therapies, Inc.

Under the terms of our agreement with Ultragenyx, we are eligible to receive

payments based on the achievement of certain sales milestones and royalties on net sales.

On February 18, 2025, Ultragenyx reported

that its BLA for its MPS IIIA product, UX111 (ABO-102), had been accepted for review by the FDA, with a PDUFA date of August 18, 2025.

our agreements with Taysha, we are eligible to receive payments based on certain clinical, regulatory, and sales milestones and

royalties on net sales.

(“Ultragenyx”)) and Rett syndrome to Taysha Gene Therapies, Inc.

Under the terms of our agreement with Ultragenyx, we are eligible to receive payments based on the achievement of certain sales

milestones and royalties on net sales.

Under our agreements with Taysha, we are eligible to receive payments based on certain

clinical, regulatory, and sales milestones and royalties on net sales.

On February 25, 2026, the Company, UNC and Taysha jointly

terminated both the license agreement between Abeona and UNC and the corresponding sublicense agreement between

Abeona and Taysha relating to Taysha’s development program for TSHA-118 for CLN1 disease.

Leadership Position in Commercial-Scale Cell and Gene-Therapy Manufacturing

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us to enhance supply chain control, establish tighter quality control testing, increase supply capacity, reduce production costs and

gain manufacturing efficiency for clinical trials related to our product candidates and ensure commercial demand is met in the event

our therapies receive marketing approval.

Our facility is led by a team of highly skilled production, process/assay development and quality

control scientists with expertise in cell and gene therapy, particularly in cell culture, upstream manufacturing, downstream purification,

assay development and wet lab techniques.

have over 16,000+ square foot manufacturing space in Cleveland, Ohio.

The first phase, completed in 2018, was a 6,000 square foot state-of-the-art

cGMP production facility for the manufacturing of cell and gene therapies.

The facility is designed to initially manufacture clinical

drug products with intent of manufacturing commercial grade cGMP drug product.

The second phase, completed in 2019, was the completion

of an additional 8,000 square feet of state-of-the-art laboratory space to support our expanding quality control, process development,

and assay development teams.

The second phase also included nearly 2,000 square feet of cGMP Inventory Control space.

October 18, 2024, we signed a lease for 16,566 square feet of office space at 6700 Euclid Avenue, Cleveland, Ohio.

The lease commenced

on January 1, 2025 and the lease term matches the term for our existing 6555 Carnegie Avenue facility.

The additional space at the 6700

Euclid Avenue facility will allow us to convert office space at the 6555 Carnegie Avenue facility into additional manufacturing space

to increase pz-cel manufacturing capacity.

have advanced our in-house manufacturing capabilities for pz-cel.

The product is manufactured as multilayer cellular sheets containing

gene-corrected keratinocytes that is fastened to a petrolatum gauze backing with surgical titanium ligating clips.

Engineered keratinocyte

sheets expressing functional Type VII collagen are applied over wound areas, providing immediate wound coverage and allowing for long-term

wound healing.

A key component to the pz-cel drug product manufacturing process is the retroviral vector, which delivers the functional

copy of the Collagen VII Alpha 1 cDNA to the patient’s own cells.

We manufacture the LZRSE-Col7A1 retroviral vector at our Cleveland

gain manufacturing for ZEVASKYN ® .

Our facility is led by a team of highly skilled production, process/assay development,

and quality control scientists with expertise in cell and gene therapy, particularly in cell culture, upstream manufacturing, downstream

purification, assay development and wet lab techniques.

have advanced our in-house manufacturing capabilities for ZEVASKYN ® .

The product is manufactured as multilayer cellular

sheets containing gene-corrected keratinocytes that is fastened to a petrolatum gauze backing with surgical titanium ligating clips.

Engineered keratinocyte sheets expressing functional Type VII collagen are applied over wound areas, providing immediate wound coverage

and allowing wound healing.

A key component to the ZEVASKYN ® drug product manufacturing process is the retroviral

vector, which delivers the functional copy of the Collagen VII Alpha 1 cDNA to the patient’s own cells.

We manufacture the LZRSE-Col7A1

gamma retroviral vector at our Cleveland facility.

AAV vector manufacturing process uses the triple plasmid transient transfection method.

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have made significant investments in developing optimized manufacturing processes and believe that our processes and methods developed

to date provide a comprehensive manufacturing process for pz-cel and AAV-based vector therapies, including:

scale to support commercial manufacturing requirements for pz-cel

related to biopsy, cell collection, storage and transportation as part of manufacturing for pz-cel

related to product release testing for pz-cel

to date provide a comprehensive manufacturing process for ZEVASKYN ® and AAV-based vector therapies, including:

scale to support commercial manufacturing requirements for ZEVASKYN ® ;

related to biopsy, cell collection, storage and transportation as part of manufacturing for ZEVASKYN ® ;

related to product release testing for ZEVASKYN ® ;

related to the manufacture and release testing of retroviral vector;

transportation and packaging processes and materials for finished pz-cel product

transportation and packaging processes and materials for finished ZEVASKYN ® product;

AAV vector manufacturing processes and techniques that produce a highly purified product candidate;

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believe that these investments will enable us to develop best-in-class, next-generation cell and gene therapy products.

As we look to

commercialize pz-cel (subject to FDA approval), we have filed our BLA to support commercial manufacturing of pz-cel from our Cleveland

Strong Intellectual Property Protection

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Dystrophic Epidermolysis Bullosa

support our EB franchise, we licensed a patent family from Stanford University covering pz-cel and its use in the treatment of RDEB.

Patents covering our investigational pz-cel product have been granted in the United States (U.S.

12,110,504 and 12,173,314),

by the European Patent Office (EP3400287B1) and in other geographical regions and are expected to expire in early 2037.

Patent applications

remain pending in the United States which, if granted, would be expected to expire in 2037.

support our EB franchise, we licensed a patent family from Stanford University covering ZEVASKYN ® and its use in the

treatment of RDEB.

Patents covering our investigational ZEVASKYN ® product have been granted in the United States

and 12,385,010), by the European Patent Office (EP3400287B1), by the Japan Patent Office

(JP7159048, JP7555380), and in other geographical regions, and are expected to expire in early 2037.

Patent applications remain

pending in the United States which, if granted, would be expected to expire in 2037.

A patent covering the packaging and transport

system for pz-cel has been granted in the United States (U.S.

12,144,340) and is expected to expire in mid-2040.

may also rely on the additional protection afforded by data exclusivity (currently 12 years for biologics like pz-cel), other market

exclusivity such as orphan drug exclusivity (currently seven years), and patent term extensions, where applicable.

system for ZEVASKYN ® has been granted in the United States (U.S.

12,144,340) and is expected to expire in

may also rely on the additional protection afforded by data exclusivity (currently 12 years for biologics like ZEVASKYN ® ),

other market exclusivity such as orphan drug exclusivity (currently seven years), and patent term extensions, where applicable.

have an exclusive license to an international patent family from The University of North Carolina at Chapel Hill (“UNC”)

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Patent”), was issued to UNC on January 14, 2020.

The ‘110 Patent is entitled to 352 days of patent term adjustment, making

its projected expiration date November 6, 2036.

The ‘110 Patent is entitled to 352 days of patent term adjustment and will

not expire before November 6, 2036.

The second U.S.

patent in this patent family, U.S.

10,561,743 (the “‘743

Patent”), was issued to UNC on February 18, 2020.

The ‘743 Patent is expected to expire on November 20, 2035.

patent in this patent family, U.S.

10,561,743 (the “‘743 Patent”),

was issued to UNC on February 18, 2020.

The ‘743 Patent will not expire before November 20, 2035.

patent in this patent

11,491,242 (the “‘242 Patent”) issued on November 8, 2022.

Patent is entitled to 429 days of patent term adjustment and will not expire before January 22, 2037.

Patents have also been granted

in Australia (AU2015349759B2), Israel (IL252072), and Russia (RU2727015).

We have exclusive rights to these patents under our license

The ‘242 Patent is entitled

to 429 days of patent term adjustment and will not expire before January 22, 2037.

Patents have also been granted in Australia (AU2015349759

and AU2022201540), Israel (IL252072), New Zealand (NZ731673), and Russia (RU2727015).

We have exclusive rights to these patents under

our license with UNC.

also own a second patent family directed to certain AAV capsids and have filed national stage applications in the United States, Europe

and other geographical regions.

Patents issuing from these applications would not be expected to expire before 2039.

Disease (Infantile Batten Disease)

have also licensed from UNC rights to two patent families directed to treating CLN1 disease (also known as infantile Batten disease).

The first patent family is directed to optimized CLN1 genes and expression cassettes for use in treating CLN1 disease, which has applications

pending in the United States, Europe, and other geographical regions.

patent in the first patent family, U.S.

(the “‘435 Patent”), was issued to UNC on November 22, 2022.

The ‘435 Patent is entitled to 578 days of patent

term adjustment, making its projected expiration date January 12, 2039.

The second patent family is directed to treating CLN1 disease

using a combination of intrathecal and intravenous administrations, and has applications pending in the United States, Europe and other

geographical regions.

Patents issuing from applications in the second patent family would have a 20-year expiration date of no earlier

We have entered into agreements exclusively sublicensing these two CLN1 patent families to Taysha Gene Therapies, Inc.

12,454,701 (the “‘701 Patent”), was issued on October 28, 2025.

patent is entitled to 1179 days of patent term adjustment and will not expire before February 25, 2043.

A patent has also been granted

in Japan (JP7590968).

have licensed rights to one patent family from UNC and two patent families from The University Court of the University of Edinburgh (“U.

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Patents issuing from these applications would have a 20-year expiration date of no earlier than 2039.

12,311,034 was issued to UNC on May 27, 2025 in this family.

The patent families licensed from U.

Edinburgh and U.

Glasgow are directed to expression cassettes for MeCP2 polypeptides and to synthetic

MeCP2 polypeptides.

The patent family directed to MeCP2 expression cassettes has pending applications in the United States, Europe and

other geographical regions.

The patent family directed to synthetic MeCP2 polypeptides has pending applications in the United States

and other geographical regions.

Patents issuing from applications in the Edinburgh patent families would have a 20-year expiration date

of no earlier than 2038.

In October 2020, we entered into an agreement exclusively sublicensing these UNC and University of Edinburgh

patent rights to Taysha Gene Therapies, Inc.

are directed to expression cassettes for MeCP2 polypeptides and to synthetic MeCP2 polypeptides.

The patent family directed to MeCP2

expression cassettes has pending applications in the United States, Europe and other geographical regions.

The patent family directed

to synthetic MeCP2 polypeptides has pending applications in the United States and other geographical regions.

Patents issuing from applications

in the Edinburgh patent families would have a 20-year expiration date of no earlier than 2038.

11,969,479 was issued

Edinburgh and U.

Glasgow in in this patent family on April 20, 2024.

In October 2020, we entered into an agreement exclusively

sublicensing these UNC and University of Edinburgh patent rights to Taysha Gene Therapies, Inc.

AAV Delivery of Large Transgenes

own a patent family directed to multipartite delivery of large transgenes using AAV vectors and have filed national stage applications

in the United States, Europe and other geographical regions.

Patents issuing from these applications would not be expected to expire

also own a pending U.S.

application (U.S.

2022/0090129 A1) directed to multipartite AAV delivery and its use for treating

Stargardt disease.

own three patent families directed to multipartite delivery of large transgenes using AAV vectors.

For two of these patent families we

have filed national stage applications in the United States, Europe and other geographical regions.

Patents issuing from these applications

are not expected to expire before 2041 for the first patent family, or before 2044 for the second patent family.

A European patent application

in the first patent family (EP4182467) is allowed and will be validated in European states in 2026.

We have also filed a U.S.

application in the third patent family.

Patents issuing from the provisional application are not expected to expire before 2046.

AAV Capsids and Ophthalmic Disease Treatment via Para-retinal AAV Administration

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Patents issuing

from these applications would not be expected to expire before 2042.

from these applications are not expected to expire before 2042.

of Dominant Optic Atrophy and X-linked Retinoschisis

own a pending PCT application (PCT/US2023/065877) directed to compositions and methods for treating dominant optic atrophy and X-linked

retinoschisis.

Patents issuing from future national stage applications of this PCT application would not be expected to expire before

own a patent family directed to compositions and methods for treating dominant optic atrophy and X-linked retinoschisis and have filed

national stage applications in the United States, Europe, and other geographical regions.

Patents issuing from these applications are

not expected to expire before 2043.

expect to explore in due course strategies to support patent term extensions for all of our patent portfolios.

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The FDA will not approve an application unless it determines that the manufacturing processes and facilities comply

with cGMP requirements and are adequate to assure consistent production of the product within required specification.

with cGMP requirements and are adequate to ensure consistent production of the product within required specification.

with clinical trials, companies usually complete additional preclinical studies and must also develop additional information about the

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carefully when making decisions.

During the product approval process, the FDA also will determine whether a REMS is necessary to assure

During the product approval process, the FDA also will determine whether a REMS is necessary to ensure

the safe use of the product candidate.

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Both the PDMA and state laws limit the distribution of prescription

biopharmaceutical product.

biopharmaceutical products.

Certain reporting related to samples is also required.

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FDA for such data.

The data do not need to show the product to be effective in the pediatric population studied;

The data does not need to show the product to be effective in the pediatric population studied;

rather, if the clinical

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disease products where the product is intended to treat serious or life-threatening diseases that primarily affect individuals up to

To qualify, the product must contain no active ingredient (including any ester or salt of the active ingredient) that has been

previously approved by the FDA.

The application must also meet other qualifying criteria, including eligibility for FDA priority review.

If the necessary qualifying criteria are met, upon a sponsor’s request and product approval, the FDA may award a priority review

This voucher may be transferred and may be redeemed to receive priority review of a subsequent marketing application for a different

To qualify, the product must contain no active ingredient (including any ester or salt of the active ingredient) that has

been previously approved by the FDA.

The application must also meet other qualifying criteria, including eligibility for FDA

priority review.

If the necessary qualifying criteria are met, upon a sponsor’s request and product approval, the FDA may

award a priority review voucher.

This voucher may be transferred and may be redeemed to receive priority review of a subsequent

marketing application for a different product.

Use of a priority review voucher is subject to an FDA user fee.

As these vouchers are transferable, sponsors may sell these

vouchers for substantial sums of money.

Vouchers may, however, be revoked by the FDA under certain circumstances and sponsors of approved

rare pediatric disease products must submit certain reports to the FDA.

To take advantage of the benefits of this program, the product

must be designated by the FDA for a rare pediatric disease no later than December 20, 2024 (extended from September 30, 2024 under the

Continuing Appropriations and Extensions Act, 2025, signed into law by President Biden on September 26, 2024), and approved no later

than September 30, 2026, unless the law is reauthorized by Congress.

As these vouchers are

transferable, sponsors may sell these vouchers for substantial sums of money.

Vouchers may, however, be revoked by the FDA under

certain circumstances and sponsors of approved rare pediatric disease products must submit certain reports to the FDA.

advantage of the benefits of this program, the product must be designated by the FDA for a rare pediatric disease no later than

September 30, 2029.

Rare Pediatric Disease Priority Review Voucher program has been subject to periodic statutory sunset provisions and extensions.

the current statutory sunset provisions, the FDA may only award a rare pediatric disease priority review voucher if the NDA for the product

is approved before September 30, 2029.

After September 30, 2029, the FDA may not award any rare pediatric disease priority review vouchers,

unless Congress extends the program further.

regulation outside of the United States

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public disclosure requirements in relation to clinical trial information.

the European Union there are also broadly equivalent regimes for the other issues addressed in relation to US regulation including cGMP

the European Union there are also broadly equivalent regimes for the other issues addressed in relation to U.S.

regulation including cGMP

requirements, accelerated access (generally through so-called Conditional Marketing Authorizations), pediatric requirements and incentives

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uncertainty exists as to the coverage and reimbursement status of any products for which we may obtain regulatory approval.

United States, sales of any product candidates for which regulatory approval for commercial sale is obtained will depend in part on

the availability of coverage and adequate reimbursement from third-party payors.

Third-party payors include government authorities

and health programs in the United States such as Medicare and Medicaid, managed care providers, private health insurers and other

organizations.

These third-party payors are increasingly reducing reimbursements for medical products and services.

The process for

determining whether a payor will provide coverage for a drug product may be separate from the process for setting the reimbursement

rate that the payor will pay for the drug product and/or application procedure.

Third-party payors may limit coverage to specific drug products on an approved

list, or formulary, which might not include all FDA-approved drugs for a particular indication.

Additionally, the containment of

healthcare costs has become a priority of federal and state governments, and the prices of drugs have been a focus in this effort.

government, state legislatures and foreign governments have shown significant interest in implementing cost-containment

programs, including price controls, required disclosures of pricing and sensitive cost data, requirement for payment of manufacturer

rebates and negotiation of supplemental rebates, restrictions on reimbursement and requirements for substitution of generic

Coverage policies and third-party reimbursement rates may change at any time.

Even if favorable coverage and reimbursement

status is attained for one or more products for which we receive regulatory approval, less favorable coverage policies and

reimbursement rates may be implemented in the future.

In the United

States, sales of any product candidates for which regulatory approval for commercial sale is obtained will depend in part on the availability

of coverage and adequate reimbursement from third-party payors.

Third-party payors include government authorities and health programs

in the United States such as Medicare and Medicaid, managed care providers, private health insurers and other organizations.

These third-party

payors are increasingly reducing reimbursements for medical products and services.

The process for determining whether a payor will provide

coverage for a drug product may be separate from the process for setting the reimbursement rate that the payor will pay for the drug

product and/or application procedure.

Third-party payors may limit coverage to specific drug products on an approved list, or formulary,

which might not include all FDA-approved drugs for a particular indication.

Additionally, the containment of healthcare costs has become

a priority of federal and state governments, and the prices of drugs have been a focus in this effort.

government, state legislatures

and foreign governments have shown significant interest in implementing cost-containment programs, including price controls, required

disclosures of pricing and sensitive cost data, requirement for payment of manufacturer rebates and negotiation of supplemental rebates,

restrictions on reimbursement and requirements for substitution of generic products.

Coverage policies and third-party reimbursement

rates may change at any time.

Even if favorable coverage and reimbursement status is attained for one or more products for which we receive

regulatory approval, less favorable coverage policies and reimbursement rates may be implemented in the future.

the EU, pricing and reimbursement schemes vary widely from country to country.

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Capital Resources

a clinical-stage biopharmaceutical company developing cell and gene therapies for life-threatening diseases, we seek to attract, hire,

a commercial-stage biopharmaceutical company developing cell and gene therapies for life-threatening diseases, we seek to attract, hire,

develop and retain qualified and highly skilled personnel with experience in areas such as research and development and manufacturing

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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