• Like bluebird bio, but focused on gene and cell therapies for a different portfolio of rare genetic diseases.
• Like a clinical-stage Sarepta Therapeutics, developing gene therapies for a distinct set of rare genetic conditions.

• EB-101: A gene-corrected cell therapy in Phase III clinical trial for recessive dystrophic epidermolysis bullosa.
• ABO-102: An AAV-based gene therapy for Sanfilippo syndrome type A.
• ABO-201: A gene therapy in development to treat CLN3 disease.
• ABO-401: A gene therapy in development for the treatment of cystic fibrosis.
• ABO-50X: A gene therapy in development for the treatment of genetic eye disorders.
• AIM vector platform programs: Initiatives for developing AAV-based gene therapies utilizing its proprietary vector platform.

Abeona Therapeutics Inc. (ABEO) is a clinical-stage biopharmaceutical company focused on developing gene and cell therapies for life-threatening rare genetic diseases. As of the provided information, the company's lead programs are in various stages of clinical trials (e.g., Phase III for EB-101) and are not yet approved for commercial sale.

Therefore, Abeona Therapeutics currently does not have major commercial customers for its products, and specific customer companies cannot be listed.

However, once its therapies receive regulatory approval and are commercialized, the company would primarily sell its highly specialized treatments to institutional customers within the healthcare system. These customer categories would typically include:

• Hospitals and Specialized Treatment Centers: Medical facilities equipped to administer complex gene and cell therapies to patients suffering from rare genetic diseases.
• Healthcare Distributors and Wholesalers: Companies that manage the logistics and distribution of pharmaceutical products from manufacturers to healthcare providers.
• Government Health Agencies and Large Insurance Providers: Entities, such as national health services or large insurance organizations, that may directly purchase or manage the procurement of high-cost specialized therapies for their covered populations.

Vishwas Seshadri, Ph.D., M.B.A. Chief Executive Officer, Director

Dr. Vishwas Seshadri joined Abeona in June 2021 as Head of Research & Clinical Development and was appointed President and Chief Executive Officer, and a member of the Company's Board of Directors, in October 2021. He brings over 20 years of experience in academia and various senior and executive leadership roles in the life sciences industry, overseeing product development, regulatory submissions, and commercialization for novel therapies, including personalized, autologous cell and gene therapies. Prior to Abeona, he served in roles of increasing responsibility at Celgene Corporation, a subsidiary of Bristol-Myers Squibb Company (BMS), where he focused on the development and commercialization of novel therapies in hematology and oncology, and notably led the team responsible for the launch of Breyanzi® (lisocabtagene maraleucel). He previously held the position of Head of Early-Stage Upstream Process Development for Biologics at Dr. Reddy's Laboratories. Dr. Seshadri holds a Ph.D. in Microbiology, Immunology & Molecular Biology and completed his post-doc in epigenetics at the University of Arizona. He also earned an M.B.A. in Finance and Healthcare from the Wharton School of the University of Pennsylvania.

Joseph Vazzano Chief Financial Officer

Joseph Vazzano joined Abeona as Chief Financial Officer in March 2022. Before joining Abeona, Mr. Vazzano served as Chief Financial Officer of Avenue Therapeutics, Inc. (Nasdaq: ATXI) from February 2019 to January 2022. Prior to that, he was Avenue's Vice President of Finance and Corporate Controller since August 2017. During his tenure at Avenue, Mr. Vazzano secured multiple equity financings and played a leadership role in signing a complex, two-stage acquisition for Avenue, which included future contingent value rights.

Mohamad Tabrizi, M.S., M.B.A. Chief Business Officer

Mr. Mohamad Tabrizi is an experienced executive, venture capitalist, and former investment banker with a 30-year career that combines scientific expertise with significant financial and corporate leadership. As a General Partner and Managing Director at Pandect Bioventures and Berkley Catalyst Fund, he was instrumental in the firms' investment activities and portfolio management, and also took on operational roles such as CEO for Azkarra Therapeutics and Interim CFO for Circle Pharma. His strategic financial insights were developed early in his career as SVP & CFO for ShangPharma Venture Innovation Center, where he contributed to completing 15 transactions and fostering valuable research collaborations. He also held executive corporate development roles at Nektar Therapeutics and Depomed, focusing on transactions including licensing, M&A, and royalty monetization. Mr. Tabrizi holds an M.B.A. from Cornell University, an M.S. in Biological Sciences from Stanford University, and a B.S. in Genetics from the University of California Davis.

Brian Kevany, Ph.D. Chief Technical Officer & Chief Scientific Officer

Dr. Brian Kevany joined Abeona at the beginning of 2018 as a scientist in the product development department, bringing over 15 years of molecular biology and biochemistry research training. Prior to his time at Abeona, he managed a small biotechnology startup company that specialized in utilizing next-generation sequencing for high-throughput genotyping in both medical and agricultural applications.

Brendan O'Malley, J.D., Ph.D. General Counsel

Brendan O'Malley serves as the General Counsel for Abeona Therapeutics.

Here are the key risks to Abeona Therapeutics (ABEO):

1. Commercialization, Manufacturing, and Market Adoption Challenges for Zevaskyn: Despite the FDA approval of its lead product, Zevaskyn (EB-101, also referred to as PZIFERDEX), Abeona Therapeutics faces significant hurdles in successfully bringing this gene therapy to market. These include challenges in manufacturing scale-up while maintaining quality, as evidenced by a temporary facility shutdown mentioned in recent reports. The company must also navigate complex and systematic payer coverage across various insurers and Medicaid, along with the operational complexities of patient identification and coordinating a 3-4 month treatment cycle. Furthermore, as a curative therapy for a rare disease, Zevaskyn's market size is inherently limited, and it faces competition from existing and future gene therapy approaches. Slow adoption rates in key markets and difficulties in realizing its potential value outside the U.S. also pose commercialization risks.
2. Financial Distress and Funding Risk: Abeona Therapeutics' financial health presents a notable risk. The company's Altman Z-Score indicates it is in the distress zone, implying a potential risk of bankruptcy within the next two years. While the company has reported strong liquidity in some aspects, its P/S ratio is significantly high, reflecting current revenue challenges. As a clinical-stage biopharmaceutical company with a costly research and development pipeline, there is a continuous need for substantial capital, and the possibility of requiring additional dilutive financing if cash burn outpaces revenue growth is a significant concern for investors.
3. Clinical Development and Gene Therapy-Specific Risks for Pipeline Assets: While Zevaskyn is approved, Abeona Therapeutics continues to develop other gene therapies such as ABO-102, ABO-201, ABO-401, and ABO-50X for various rare genetic diseases. These pipeline assets are subject to the inherent risks of failure in clinical trials, which for new drug candidates can be as high as 90% even after entering Phase I. Gene and cell therapies, in particular, carry unique risks including potential immune responses, vector-related complications, challenges in precise cell targeting, and uncertainties regarding long-term safety and efficacy. Any negative results or setbacks in these earlier-stage pipeline programs could significantly impact the company's future prospects and valuation.

Abeona Therapeutics Inc. (ABEO) develops gene and cell therapies for life-threatening rare genetic diseases. The addressable markets for its main products or services are as follows:

• EB-101 for recessive dystrophic epidermolysis bullosa (RDEB): The market for Dystrophic Epidermolysis Bullosa (DEB) across the top seven major markets (US, EU4, UK, and Japan) was valued at approximately USD 589.9 million in 2024 and is projected to reach USD 2,128.8 million by 2035. The total Epidermolysis Bullosa market size in these seven major markets was approximately USD 1.7 billion in 2023. RDEB is considered the most critical type of Epidermolysis Bullosa and accounted for over 30% of prevalent cases in 2023. The United States alone represented the largest market size for DEB, at approximately USD 400 million in 2023.
• ABO-102 for Sanfilippo syndrome type A (MPS IIIA): The global market size for Mucopolysaccharidosis type IIIA (MPS IIIA) treatment was valued at US$ 242.6 million in 2024 and is expected to reach US$ 339.1 million by 2031. North America is expected to lead this market. Another estimate indicates the 7 major Sanfilippo Syndrome markets reached a value of USD 1.8 billion in 2024 and are expected to reach USD 2.7 billion by 2035. The global Sanfilippo syndrome market size is also projected to grow to $15.02 billion in 2030.
• ABO-201 to treat CLN3 disease (Batten disease): The global Batten Disease Treatment market size was approximately USD 40.2 million in 2023 and is predicted to grow to around USD 65.2 million by 2032. Another report estimates the global batten disease treatment market size to grow from USD 0.43 billion in 2023 to USD 0.72 billion by 2033. North America is expected to dominate this market, followed by Europe.
• ABO-401 for the treatment of cystic fibrosis (CF): The global cystic fibrosis therapeutics market size was valued at USD 11.81 billion in 2025 and is projected to reach around USD 44.72 billion by 2035. Another estimate indicates the global cystic fibrosis market was valued at USD 10.36 billion in 2023 and is expected to reach USD 56.72 billion by 2032. North America dominated the market in 2024.
• ABO-50X for the treatment of genetic eye disorders: The global inherited retinal disease market size was estimated at $11.56 billion in 2024 and is projected to reach $17.42 billion by 2029. The inherited retinal diseases treatment market is projected to grow from USD 12.6 billion in 2025 to USD 28.4 billion by 2035. North America was the largest regional market in 2024.

Expected drivers of future revenue growth for Abeona Therapeutics (ABEO) over the next 2-3 years include:

1. Commercial Launch and Ramping Sales of ZEVASKYN (EB-101): Abeona Therapeutics received FDA approval for pz-cel (EB-101), now marketed as ZEVASKYN, in April 2025 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). The company anticipates ZEVASKYN to be available at qualified treatment centers in the third quarter of 2025. Abeona is actively scaling the launch to meet strong and growing patient demand and remains committed to its 2026 launch goals for this gene-corrected cell therapy. This direct commercialization of its lead product is expected to be the most significant immediate revenue driver.
2. Expansion of ZEVASKYN's Market Access and Treatment Network: The company has established broad market access for ZEVASKYN, securing positive coverage decisions from major commercial payers and all 51 state Medicaid programs. The permanent CMS J-code for ZEVASKYN, which became effective in January 2026, is expected to further enhance payer coverage and facilitate broader adoption. Abeona is also strategically expanding its network of qualified treatment centers to increase patient reach and access to the therapy.
3. Potential Future Milestone and Royalty Payments from Licensed Assets: Although Abeona no longer directly develops ABO-102 for Sanfilippo syndrome type A, it licensed the global rights to Ultragenyx Pharmaceutical Inc. in May 2022. Under this agreement, Abeona is eligible to receive tiered royalties of up to 10% on net sales and commercial milestone payments following ABO-102's regulatory approval. The successful development and eventual commercialization of ABO-102 by Ultragenyx could provide a recurring revenue stream for Abeona.
4. Advancement of Gene Therapy Pipeline, notably ABO-503: Abeona continues to progress its pipeline of gene therapies for other rare genetic diseases. Notably, the gene therapy program for X-linked retinoschisis, ABO-503, has been selected for the FDA Rare Disease Endpoint Advancement pilot program. This selection could potentially accelerate its development and bring it closer to potential regulatory approval and commercialization, representing an additional driver of revenue growth in the medium to long term.

Share Issuance

• Abeona Therapeutics raised approximately $17.5 million in aggregate gross proceeds from an underwritten public offering of common stock in December 2021.
• In November 2022, the company completed a private placement financing, generating approximately $35 million in gross proceeds from the sale of common stock, pre-funded warrants, and accompanying warrants.
• From late 2025 into early 2026, Abeona granted inducement equity awards, consisting of restricted stock awards, to new non-executive employees. For example, 28,676 restricted shares were granted on January 31, 2026, 79,584 restricted shares on December 30, 2025, and 45,685 restricted shares on November 30, 2025.

Inbound Investments

• Abeona Therapeutics completed the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) for $155 million on June 27, 2025, following the FDA approval of ZEVASKYN™.
• Strategic collaborations, such as the partnership with Ultragenyx for UX-111, could generate up to $30 million in milestone payments over time, along with ongoing royalties.
• The company is also partnered with Taysha Gene Therapies on programs TSHA-102 and TSHA-118, which are expected to generate a combination of clinical, regulatory, and sales-based milestones and royalties.

Capital Expenditures

• Abeona operates a dedicated cGMP facility in Cleveland for patient-specific gene therapy production, with a current capacity of six patients per month and planned expansion to ten patients monthly in 2026.
• Research and development (R&D) spending for Q3 2025 was $4.2 million, with some costs being capitalized into inventory and reclassified to selling, general, and administrative (SG&A) expenses following FDA approval of ZEVASKYN.