ABEO US Equity☆

UNITED

STATES

SECURITIES

AND EXCHANGE COMMISSION

WASHINGTON,

DC 20549

FORM

10-K

(Mark One)

For the fiscal year ended December 31, 2025

Or

For the transition period from _______ to _______

Commission

file number 001-15771

ABEONA

THERAPEUTICS INC.

(Exact

name of registrant as specified in its charter)

6555

Carnegie Avenue, 4th

Floor

Cleveland,

OH44103

(Address

of principal executive offices, zip code)

(646)813-4701

(Registrant’s

telephone number, including area code)

Securities

registered pursuant to Section 12(b) of the Securities Exchange Act of 1934:

Title of each class Trading Symbol(s) Name of each exchange on which registered

Common Stock, $0.01 par value ABEO Nasdaq Capital Market

Indicate

by check mark if the registrant is a well-known seasoned issuer, as defined in Rule 405 of the Securities Act. Yes ☐ No ☒

Indicate

by check mark if the registrant is not required to file reports pursuant to Section 13 or Section 15(d) of the Act. Yes ☐ No ☒

Indicate

by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange

Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2)

has been subject to such filing requirements for the past 90 days. Yes ☒ No ☐

Indicate

by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule

405 of Regulation S-T (§ 232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant

was required to submit such files). Yes ☒ No ☐

Indicate

by check mark whether the Registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting

company, or an emerging growth company. See definitions of “large accelerated filer,” “accelerated filer,” “smaller

reporting company” and “emerging growth company” in Rule 12b-2 of the Act:

Large accelerated filer ☐ Accelerated filer ☐

Non-accelerated filer ☒ Smaller reporting company ☒

Emerging growth company ☐

If

an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying

with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐

Indicate

by check mark whether the registrant has filed a report on and attestation to its management’s assessment of the effectiveness

of its internal control over financial reporting under Section 404(b) of the Sarbanes-Oxley Act (15 U.S.C. 7262(b)) by the registered

public accounting firm that prepared or issued its audit report. Yes ☐ No ☒

If

securities are registered pursuant to Section 12(b) of the Act, indicate by check mark whether the financial statements of the registrant

included in the filing reflect the correction of an error to previously issued financial statements. Yes ☐ No ☒

Indicate

by check mark whether any of those error corrections are restatements that required a recovery analysis of incentive-based compensation

received by any of the registrant’s executive officers during the relevant recovery period pursuant to § 240.10D-1(b). Yes

☐ No ☒

Indicate

by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes ☐ No ☒

The

aggregate market value of the voting and non-voting common equity held by non-affiliates computed by reference to the average bid and

asked price of such common equity, as of June 30, 2025, was approximately $273,252,724.

The

number of shares outstanding of the registrant’s common stock as of March 11, 2026 was 57,049,023.

ABEONA

THERAPEUTICS INC.

Annual

Report on Form 10-K

Table

of Contents

Page

Part I

Item 1. Business 3

Item 1A. Risk Factors 27

Item 1B. Unresolved Staff Comments 55

Item 1C. Cybersecurity 55

Item 2. Properties 56

Item 3. Legal Proceedings 56

Item 4. Mine Safety Disclosures 56

Part II

Item 7A. Quantitative and Qualitative Disclosures About Market Risk 66

Item 8. Financial Statements and Supplementary Data 66

Item 9A. Controls and Procedures 66

Item 9B. Other Information 67

Item 9C. Disclosure Regarding Foreign Jurisdictions that Prevent Inspections 67

Part III

Item 10. Directors, Executive Officers and Corporate Governance 68

Item 11. Executive Compensation 68

Item 14. Principal Accounting Fees and Services 68

Part IV

Item 15. Exhibits, Financial Statement Schedules 69

Signatures 72

FORWARD-LOOKING

STATEMENTS

This

Form 10-K (including information incorporated by reference) contains statements that express management’s opinions, expectations,

beliefs, plans, objectives, assumptions or projections regarding future events or future results and therefore are, or may be deemed

to be, “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section

21E of the Securities Exchange Act of 1934, as amended. Words such as “expects,” “anticipates,” “intends,”

“plans,” “believes,” “could,” “would,” “seeks,” “estimates,”

and variations of such words and similar expressions, and the negatives thereof, are intended to identify such forward-looking statements.

Such “forward-looking statements” speak only as of the date made and are not guarantees of future performance and involve

certain risks, uncertainties, estimates, and assumptions by management that are difficult to predict. Various factors, some of which

are beyond the Company’s control, could cause actual results to differ materially from those expressed in, or implied by, such

forward-looking statements. In addition, we disclaim any obligation to update any forward-looking statements to reflect events or circumstances

after the date of this report, except as may otherwise be required by the federal securities laws.

Forward-looking

statements necessarily involve risks and uncertainties, and our actual results could differ materially from those anticipated in forward-looking

statements due to a number of factors. These statements include statements about: our ability to successfully commercialize ZEVASKYN®and generate future revenue; our plans to continue development of AAV-based gene therapies designed to treat ophthalmic diseases;

our pipeline of product candidates, including the achievement of or expected timing, progress and results of clinical development, clinical

trials and potential regulatory approvals; our dependence upon our third-party customers and vendors and their compliance with applicable

regulations; our estimates regarding expenses, capital requirements, and needs for additional financing; our intellectual property position

and our ability to obtain, maintain and enforce intellectual property protection and exclusivity for our proprietary assets; our estimates

regarding the size of the potential markets for ZEVASKYN®and our product candidates, the strength of our commercialization

strategies and our ability to serve and supply those markets; and future economic conditions or performance.

Important

factors that could affect performance and cause results to differ materially from management’s expectations are described in the

sections entitled “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results

of Operations” in this Form 10-K. These factors include: our ability to maintain existing and obtain additional regulatory approvals

of ZEVASKYN®and any future product candidates; our ability to successfully commercialize and market ZEVASKYN®and any future product candidates, if approved, and the timing of any commercialization and marketing efforts; our ability to manufacture

sufficient batches of ZEVASKYN® to meet demand; our ability to activate additional qualified treatment centers to administer

ZEVASKYN® on patients; our ability to access our existing at-the-market sale agreement; our ability to access additional

financial resources and/or our financial flexibility to reduce operating expenses if required; our ability to obtain additional equity

funding from current or new stockholders; the potential impact of unpredicted changes in the structure and/or administration of the United

States government or its agencies; our ability to out-license technology and/or other assets, deferring and/or eliminating planned expenditures,

restructuring operations and/or reducing headcount, and sales of assets; the dilutive effect that raising additional funds by selling

additional equity securities would have on the relative equity ownership of our existing investors, including under our existing at-the-market

sale agreement; the outcome of any interactions with the FDA or other regulatory agencies relating to any of our products or product

candidates; our ability to continue to secure and maintain regulatory designations for our product candidates; our ability to develop

manufacturing capabilities compliant with current good manufacturing practices for our product candidates; our ability to manufacture

cell and gene therapy products and produce an adequate product supply to support clinical trials and potentially future commercialization;

the rate and degree of market acceptance of our product candidates for any indication once approved; our ability to meet our obligations

contained in license agreements to which we are party; and macroeconomic uncertainty resulting from changes to U.S. trade policy, including

current or future tariffs or other trade restrictions.

This

Form 10-K includes our trademarks, trade names and service marks, such as “ZEVASKYN®” and “AIMTM,”

which are protected under applicable intellectual property laws and are the property of Abeona Therapeutics Inc. or its subsidiaries.

Solely for convenience, trademarks, trade names and service marks referred to in this report appear without the ® and TM symbols,

but such references are not intended to indicate, in any way, that we will not assert, to the fullest extent under applicable law, our

rights or the right of the applicable licensor to these trademarks, trade names and service marks. We do not intend our use or display

of other parties’ trademarks, trade names or service marks to imply, and such use or display should not be construed to imply,

a relationship with, or endorsement or sponsorship of us by, these other parties.

PART

I

ITEM

1. BUSINESS

Business

Abeona

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

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

diseases. 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

ZEVASKYN®. 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 AIMTM 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.

Our

Mission and Strategy

Our

strategy consists of:

Commercializing

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

Through

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 are

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

approval.

Developing

Novel In-Vivo Gene Therapies Using AIMTM Capsid Technology.

We

are researching and developing AAV-based gene therapies using novel AAV capsids both derived from the licensed AIMTM Capsid Technology

Platform and invented by the Company. We plan to continue to develop chimeric AAV capsids capable of improved tissue targeting for various

indications and that can potentially evade immunity to wild-type AAV vectors.

Leveraging

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

We

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 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.

Establishing

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

We

seek to be the partner of choice in cell and gene therapy treatments and have closely collaborated with leading academic institutions,

key opinion leaders, patient foundations, and industry partners to accelerate research and development, understand the needs of patients

and their families, and generate novel intellectual property.

Maintaining

and Growing our IP Portfolio.

We

seek patent rights for various aspects of our programs, including vector engineering and construct design, our production process, and

all features of our clinical products, including compositions of matter and methods of manufacture, administration, and delivery. We expect

to continue to expand our intellectual property portfolio by aggressively seeking patent rights for promising aspects of our product

engine and product candidates.

ZEVASKYN®

for the Treatment of RDEB

Disease

Overview

RDEB

belongs to a broad group of genetic skin disorders known as epidermolysis bullosa. Patients with RDEB have a defect in the COL7A1 gene,

resulting in the inability to produce Type VII collagen, which plays a vital role in skin functioning by anchoring the skin’s dermal

and epidermal layers to one another.

As

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.

Similar

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.; 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 ZEVASKYN®. Based

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).

RDEB

patients have active disease, with the majority of their wounds typically greater than 20 cm2 in size (Stanford University;

Solis, D., et al., 2017). In 2020, a survey of RDEB patients reported that approximately 60% have active wounds covering greater than

30% of their bodies (Bruckner et al.; Orphanet Journal of Rare Diseases, 2020). Wounds covering up to approximately 80% of body surface

area have been recorded in some EB patients (Hirsch et al.; Nature Research, 2017).

In

our VIITALTM 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 VIITALTM phase 3 trial had some of the worst wounds.

These wounds were large (> 20cm2) 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

skin cancer.

Current

Management of RDEB

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 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.

RDEB

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

corneal abrasions.

In

2023, Vyjuvek® and Filsuvez® were approved by the FDA for treatment of wounds associated with DEB and wounds

associated with Junctional (JEB) and DEB, respectively.

RDEB

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

Our

Program History

ZEVASKYN®

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.

Results

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.

In

November 2022, we announced positive topline data from our VIITALTM study. The pivotal phase 3 VIITALTM study evaluated

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

large chronic wounds randomized and treated in VIITALTM measured greater than 20 cm2 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) 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)

wounds.

The

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

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

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.

On

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.

Among

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

preventing FDA approval of another product deemed to be the same as the approved product for the same indication, waiver of application

fees, and tax credits for qualified clinical testing expenses conducted after orphan designation is received. A sponsor who receives

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

by the FDA. 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

of the PRV.

We

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 VIITALTM study participants was manufactured at our Cleveland facility.

Commercial

Operations

Our

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®.

Treatment

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.

We

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

the U.S. 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.

Developing

Next-Generation Cell and Gene Therapy

ABO-503

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

Disease

Overview and Program Overview

XLRS

is a rare, monogenic retinal disease that results in the irreversible loss of photoreceptor cells and severe visual impairment. XLRS

is caused by mutations in the RS1 protein, which is normally secreted by retinal photoreceptors and bipolar neurons and functions to

mediate cell-cell adhesion. XLRS is characterized by abnormal splitting of the layers of the retina, resulting in poor visual acuity,

which can progress to legal blindness. The incidence of XLRS is estimated to be between 1 in 5,000 and 1 in 20,000 in males, with an

estimated prevalence of 35,000 in the United States and Europe combined. There are currently no disease modifying therapies approved

for XLRS, but because the genetics of the disease are well understood, early intervention via gene therapy has significant potential

to reverse or stabilize disease progression at early stages and prevent vision loss.

ABO-503,

composed of a functional human RS1 packaged in the novel AIMTM capsid AAV204, has shown preclinical efficacy following delivery

to the retina in a mouse model of XLRS. Preclinical studies have demonstrated robust RS1 expression in the retina, improved cone

photoreceptor density and overall photoreceptor cell survival, as well as a restoration of outer retina architecture. Results of

these studies were presented at the American Society of Gene and Cell Therapy (“ASGCT”) Annual Meeting in May 2023. A

pre-IND meeting for ABO-503 was conducted with the FDA in April 2023 and provided Abeona with comprehensive feedback to support a

future IND submission. Due to focus on ZEVASKYN® commercialization efforts, animal

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

ABO-504

for the Treatment of Stargardt Disease

Disease

Overview and Program Overview

Autosomal

recessive Stargardt disease, the most common form of juvenile macular degeneration with estimated incidence of 1 in 8,000 to 10,000 people,

causes vision loss in children and young adults. The most common form of Stargardt disease is caused by mutations in the ABCA4 gene,

which prevent removal of toxic compounds from photoreceptor cells that results in photoreceptor cell death and progressive vision loss.

There are currently no FDA approved treatments available, and to date, development of investigational gene modifying therapies has remained

challenging in part due to the large size of the ABCA4 gene, which exceeds the encapsidation capacity of a single AAV capsid.

Abeona’s

internal research and development team developed ABO-504, which is designed to efficiently reconstitute the full-length ABCA4 gene by

implementing a dual AAV vector strategy using the Cre-LoxP recombinase system. Abeona previously reported preclinical data demonstrating

the ability of the dual AAV vector system to produce full length ABCA4 protein in cell culture. Recent proof-of-concept studies, presented

at the 2023 ASGCT Annual Meeting, have extended these findings by showing expression of ABCA4 mRNA and full-length ABCA4 protein in the

retina of subretinally dosed abca4-/- knockout mice, at levels similar to endogenous ABCA4 in wild-type animals. A pre-IND meeting for

ABO-504 was conducted with the FDA in June 2023 and provided Abeona with comprehensive feedback to support a future IND submission.

ABO-505

for the Treatment of Autosomal Dominant Optic Atrophy (“ADOA”)

Disease

Overview and Program Overview

ADOA,

a form of hereditary vision loss associated with retinal ganglion cell (“RGC”) death, is predominantly caused by mutations

in the Opa1 gene. Opa1, a dynamin-related GTPase, acts to stabilize the inner mitochondrial membrane and acts in mitochondrial fusion

and inner membrane remodeling. 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. ADOA affects approximately 1 in 30,000 people

worldwide. Currently, there is no approved treatment for people living with ADOA.

ABO-505

is designed to express a functional copy of human Opa1 in the retina following para-retinal injection. ABO-505 aims to take advantage

of the robust optic nerve and RGC transduction ability of AAV204 to deliver its genetic payload to the cells most affected by ADOA. Preclinical

studies have confirmed expression of Opa1 in both cell culture and the retinas of dosed wild-type and disease model animals. Initial

efficacy results suggest an improvement in retinal signaling to the brain and improved visual acuity in treated mutant mice. These studies

were presented at the ASGCT Annual Meeting in May 2023.

Gene

Therapy Treatments anchored in AIMTM Vector Platform

In

2016, we licensed a library of novel AAV capsids from UNC. The AIMTM vector system is a platform of AAV capsids capable of widespread

central nervous system gene transfer and can be used to confer high transduction efficiency for various therapeutic indications. In partnership

with academic institutions, our own scientific research teams have identified capsids within the AIMTM capsid library showing strong

potential to successfully target and reach the central nervous system (including the retina) as well, lung, muscle, liver, and other

tissues. Based on continuing research by Abeona and our research partners, we have observed improvements in gene delivery to specific

tissues compared to currently available AAV technology. We believe AIMTM vectors also have the potential for redosing subjects who

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 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.

Strategic

Licensing Agreements

We

have out-licensed certain clinical and research programs, including for the treatment of Sanfilippo syndrome type A (MPS IIIA) to

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

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.

Leveraging

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

We

have established a cGMP manufacturing facility, the Elisa Linton Center located in Cleveland, Ohio at 6555 Carnegie Avenue, which enables

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

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.

We

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.

Our

AAV vector manufacturing process uses the triple plasmid transient transfection method. We insert (“transfect”) many copies

of three DNA plasmids encoding the specific therapeutic gene sequence, or transgene, the capsid coding sequence, and helper sequences

into AAV-293 cells using a serum-free, suspension-based bioreactor vector production technology. During an incubation period following

transfection, each cell produces AAV vectors through biosynthesis using the cells’ natural machinery. At the end of the incubation

period, the newly generated AAV vectors are harvested, filtered, and purified in a multi-step process.

We

have established and maintained strong and collaborative relationships with third-party companies specializing in the testing of cell

and gene therapy material to complement our process and assay development needs.

We

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 ZEVASKYN® and AAV-based vector therapies, including:

● processes related to product release testing for ZEVASKYN®;

● processes related to the manufacture and release testing of retroviral vector;

● AAV serum-free suspension technology that is readily scalable;

We

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

Maintain

Strong Intellectual Property Protection

We

strive to protect our commercially important proprietary technology, inventions, and know-how, including by seeking, maintaining, and

defending patent rights, both for inventions developed internally and for inventions licensed from third parties. We also rely on trade

secrets and know-how relating to our proprietary technology platforms, continuing technological innovation, and in-licensing opportunities

to develop, strengthen and maintain our position in the field of cell and gene therapy. We may also rely on the additional protections

afforded by data exclusivity (currently 12 years for biologics), other market exclusivities such as orphan drug exclusivity, and patent

term extensions, where applicable.

Our

success may depend in part on our ability to obtain and maintain patents and other protections for commercially important technology,

inventions, and know-how related to our business; defend and enforce our patents; preserve the confidentiality of our trade secrets;

and operate without infringing the valid enforceable patents and other intellectual property rights of third parties. Our ability to

stop third parties from making, having made, using, selling, offering to sell, or importing our products may depend on the extent to

which we have rights under valid and enforceable licenses, patents, or trade secrets that cover these activities. In some cases, these

rights may need to be enforced by third-party licensors. With respect to both licensed and company-owned intellectual property, we may

not be granted patents with respect to any of our pending patent applications or with respect to any patent applications filed by us

in the future, nor can we be sure that any of our existing patents or any patents that may be granted to us in the future will be commercially

useful in protecting our commercial products and methods of manufacturing the same.

We

are actively seeking U.S. and international patent protection, together with our licensors, for a variety of technologies, including

AAV capsids, AAV-based biological products, methods of designing novel AAV constructs, compositions and methods for treating diseases

of interest, including RDEB, and methods for manufacturing, packaging, and transporting our product candidates. We also intend to seek

patent protection or rely upon trade secret rights to protect other technologies that may be used to discover and validate targets and

that may be used to identify and develop novel biological products. We seek protection, in part, through confidentiality and proprietary

information agreements. We are a party to various license agreements that give us rights to use specific technologies in our research

and development, and future commercialization.

Licensed

Technologies and Intellectual Property

1. Recessive Dystrophic Epidermolysis Bullosa

To

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

(U.S. Patent Nos. 12,110,504; 12,173,314; 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 ZEVASKYN® has been granted in the United States (U.S. Patent No. 12,144,340) and is expected to expire in

mid-2040.

We

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.

2. AIMTM Capsids

We

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

covering novel AAV capsids (“AIMTM capsids”) that may potentially be used to deliver a wide variety of therapeutic transgenes

to human cells to treat genetic diseases. National stage applications directed to the AIMTM capsids have been filed in the United

States, Europe, and other geographical regions. The first U.S. patent in this patent family, U.S. Patent No. 10,532,110 (the “‘110

Patent”), was issued to UNC on January 14, 2020. 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. Patent No. 10,561,743 (the “‘743 Patent”),

was issued to UNC on February 18, 2020. The ‘743 Patent will not expire before November 20, 2035. A third U.S. patent in this patent

family, U.S. Patent No. 11,491,242 (the “‘242 Patent”) issued on November 8, 2022. 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.

We

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. U.S. Patent No. 12,454,701 (the “‘701 Patent”), was issued on October 28, 2025. The ‘701

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).

3. Rett Syndrome

We

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

Edinburgh”) and The University Court of the University of Glasgow (“U. Glasgow”) relating to gene therapy for the treatment

of Rett Syndrome. The patent family licensed from UNC at Chapel Hill is directed to viral genomes designed to regulate expression of

the MeCP2 gene, which is mutated in patients with Rett Syndrome. This patent family has pending applications in the United States, Europe

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

U.S. Patent No. 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. U.S. Patent No. 11,969,479 was issued

to U. 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.

4. Multipartite AAV Delivery of Large Transgenes

We

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. provisional

application in the third patent family. Patents issuing from the provisional application are not expected to expire before 2046.

We

own a patent family directed to (i) novel AAV capsid proteins and (ii) treating ophthalmic diseases via para-retinal administration of

AAV vectors 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 2042.

6. Treatment of Dominant Optic Atrophy and X-linked Retinoschisis

We

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.

We

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

U.S.

Biologic Products Development Process

In

the United States, the FDA regulates biologic products including gene therapy products under the Federal Food, Drug, and Cosmetic Act

(“FDCA”), the Public Health Service Act (“PHSA”), and regulations implementing these laws. The FDCA, PHSA and

their corresponding regulations govern, among other things, the testing, manufacturing, safety, efficacy, labeling, packaging, storage,

record keeping, distribution, advertising, and promotion of biologic products. Applications to the FDA are required before conducting

human clinical testing of biologic products. FDA approval also must be obtained before marketing of biologic products. Gene therapy studies

may also need to comply with the National Institutes of Health (“NIH”) Guidelines for Research Involving Recombinant or Synthetic

Nucleic Acid Molecules (“NIH Guidelines”), which includes additional requirements, such as the review and approval of the

study by an Institutional Biosafety Committee.

Within

the FDA, the Center for Biologics Evaluation and Research (“CBER”) regulates gene therapy products. Within CBER, the review

of gene therapy and related products is consolidated in the Office of Tissues and Advanced Therapies (“OTAT”) and the FDA

has established the Cellular, Tissue and Gene Therapies Advisory Committee (“CTGTAC”), a panel of medical and scientific

experts and consumer representatives, to advise CBER on its reviews. The FDA has issued a growing body of guidance documents on CMC,

clinical investigations and other areas of gene therapy development, all of which are intended to facilitate the industry’s development

of gene therapy products.

The

process required by the FDA before a biologic product candidate may be marketed in the United States generally involves the following:

Before

testing any biologic product candidate on humans, including a gene therapy product candidate, the product candidate must undergo preclinical

testing. Preclinical tests, also referred to as nonclinical studies, include laboratory evaluations of product chemistry, toxicity, and

formulation, as well as in vivo studies to assess the potential safety and activity of the product candidate. The conduct of the preclinical

tests must comply with federal regulations and requirements including GLPs.

If

a gene therapy trial is conducted at, or sponsored by, institutions receiving NIH funding for recombinant DNA research, the study must

also comply with the NIH Guidelines. Compliance with the NIH Guidelines is mandatory for investigators at institutions receiving NIH

funds for research involving recombinant DNA. However, many companies and other institutions, not otherwise subject to the NIH Guidelines,

voluntarily follow them.

The

clinical trial sponsor must submit the results of the preclinical tests, together with manufacturing information, analytical data, any

available clinical data or literature and a proposed clinical protocol, to the FDA as part of the IND. Some preclinical testing may continue

even after the IND is submitted. The IND automatically becomes effective 30 days after receipt by the FDA, unless the FDA places the

clinical trial on a clinical hold. In such a case, the IND sponsor and the FDA must resolve any outstanding concerns before the clinical

trial can begin. The FDA also may impose clinical holds on a biologic product candidate at any time before or during clinical trials

due to safety concerns or non-compliance. If the FDA imposes a clinical hold, trials may not commence or recommence without FDA authorization

and then only under terms authorized by the FDA.

Human

clinical trials under an IND

Clinical

trials involve the administration of the biologic product candidate to healthy volunteers or patients under the supervision of qualified

investigators, which generally are physicians not employed by, or under the control of, the trial sponsor. Investigators must also provide

certain information to the clinical trial sponsors to allow the sponsors to make certain financial disclosures to the FDA. Clinical trials

are conducted under protocols detailing, among other things, the objectives of the clinical trial, dosing procedures, subject selection

and exclusion criteria and the parameters to be used to monitor subject safety, including stopping rules that assure a clinical trial

will be stopped if certain adverse events should occur. Each protocol and any amendments to the protocol must be submitted to the FDA

as part of the IND. Clinical trials must be conducted and monitored in accordance with the FDA’s regulations comprising the GCP

requirements, including the requirement that all research subjects provide informed consent.

Further,

each clinical trial must be reviewed and approved by an IRB at or servicing each institution at which the clinical trial will be conducted.

An IRB is charged with protecting the welfare and rights of trial participants and considers items such as whether the risks to individuals

participating in the clinical trials are minimized and are reasonable in relation to anticipated benefits. The IRB also approves communications

to study subjects before a study commences at that site and the form and content of the informed consent that must be signed by each

clinical trial subject, or his or her legal representative, and must monitor the clinical trial until completed. Clinical trials involving

recombinant DNA also must be reviewed by an institutional biosafety committee (“IBC”), a local institutional committee that

reviews and oversees basic and clinical research that utilizes recombinant DNA at that institution. The IBC assesses the safety of the

research and identifies any potential risk to public health or the environment.

Information

about certain clinical trials, including a description of the study and study results, must be submitted within specific timeframes to