Abeona Therapeutics Inc.

Company overview

• Delaware corporation and commercial-stage biopharmaceutical company focused on cell and gene therapies for life-threatening diseases.
• Core focus:
  • Commercializing ZEVASKYN® (prademagene zamikeracel), an autologous cell-based gene therapy for wounds in recessive dystrophic epidermolysis bullosa (RDEB).
  • Developing AAV-based in‑vivo gene therapies using AIM™ capsids for ophthalmic and other indications.
• Go-to-market approach for ZEVASKYN:
  • Self-commercialization supported by a growing network of qualified treatment centers.
  • Potential future partnerships for ZEVASKYN and other assets.

ZEVASKYN (RDEB)

• FDA approval: April 28, 2025, as the first and only autologous cell-based gene therapy for treatment of wounds in adult and pediatric RDEB patients (single surgical application).
• Regulatory designations: RMAT, Breakthrough Therapy, Orphan Drug, Rare Pediatric Disease (FDA); Orphan designation in the EMA.
• Manufacturing: produced at Abeona’s cGMP facility in Cleveland, Ohio.
• Commercial network: 4 activated ZEVASKYN qualified treatment centers as of March 2026:
  • Ann & Robert H. Lurie Children’s Hospital of Chicago
  • Lucile Packard Children’s Hospital Stanford
  • Children’s Hospital Colorado
  • The University of Texas Medical Branch (UTMB), Galveston, Texas
• Coverage and reimbursement:
  • Broad insurance coverage from multiple national and regional commercial payors and CMS.
  • Medicaid coverage across all 50 states and Puerto Rico.
  • Permanent J‑code effective January 1, 2026.
• Commercial launch: first ZEVASKYN patient treated in Q4 2025.
• Product and manufacturing details:
  • ZEVASKYN is a multilayer cellular sheet composed of gene-corrected keratinocytes applied to wounds.
  • Retroviral vector LZRSE‑Col7A1 is used to deliver COL7A1.
  • AAV vectors are used for other vector production activities.

Pipeline and other programs

• ABO‑503 (XLRS): RS1 gene therapy using AIM™ capsid AAV204; preclinical efficacy in mouse models; pre‑IND feedback received; preclinical efficacy/toxicology work ongoing and delayed into 2026 to prioritize ZEVASKYN commercialization.
• ABO‑504 (Autosomal Recessive Stargardt Disease): dual AAV vector approach to reconstitute full‑length ABCA4; preclinical data demonstrate ABCA4 mRNA and protein expression in mouse retina; pre‑IND feedback received; IND submission planned.
• ABO‑505 (Autosomal Dominant Optic Atrophy, ADOA): retina‑targeted OPA1 expression using AAV204; preclinical data show retinal signaling and improved visual function in mutant mice; pre‑IND feedback received.
• AIM™ capsids and platforms:
  • UNC license covers AIM™ capsids for broad tissue targeting.
  • Abeona owns additional patent families for AAV capsids and multipartite delivery.
  • Ongoing development of capsids with improved tropism and potential for redosing.
  • Beacon agreement (July 2024) licensed AAV204 royalty-bearing rights for ophthalmology targets, including upfront, milestone payments and royalties.
• Rett syndrome and related IP: licenses from UNC and University of Edinburgh/University of Glasgow for MeCP2 therapy; some license agreements related to TSHA‑118 were terminated in 2026.
• Other licensing and collaborations:
  • Out‑licensed Sanfilippo syndrome type A (MPS IIIA) to Ultragenyx and Rett syndrome to Taysha Gene Therapies.
  • Licenses include development milestones, royalties, and potential back‑licensing provisions.

Manufacturing and operations

• Facility: Elisa Linton Center, 6555 Carnegie Avenue, Cleveland, Ohio — cGMP commercial/clinical‑scale manufacturing.
• ZEVASKYN manufacturing:
  • Autologous keratinocyte sheets produced from patient biopsy material.
  • Retroviral vector production (LZRSE‑Col7A1) performed at the Cleveland facility.
  • AAV vector manufacturing uses triple‑plasmid transient transfection in AAV‑293 cells with serum‑free, suspension‑based processes.
• Supply chain and quality:
  • Emphasis on chain of identity and custody for patient material.
  • Reliance on third‑party suppliers for certain materials.
  • No backup ZEVASKYN manufacturer qualified as of the reporting period.
• Post‑approval requirements: ongoing cGMP compliance, pharmacovigilance, and potential post‑approval commitments, with ongoing regulatory inspection risk.

Intellectual property and licenses

• ZEVASKYN (RDEB): Stanford University patent family (U.S. patents 12,110,504; 12,173,314; 12,385,010; EP 3400287 B1; JP patents) with expected expirations in early 2037, plus potential extensions and regional protections such as data and orphan exclusivity.
• AIM™ capsids: UNC‑exclusive license covering U.S. patents 10,532,110; 10,561,743; 11,491,242 and international patents (Australia, Israel, New Zealand, Russia, Japan); expirations extend into the 2030s and 2040s.
• Other IP: Rett syndrome licenses from UNC and University of Edinburgh/Glasgow; multipartite AAV delivery patents; new AAV capsid and ophthalmic treatment patents; patents covering dominant optic atrophy and XLR retinoschisis; expirations generally in the 2030s–2040s.
• Government rights and extensions: government‑owned/march‑in rights may apply to government‑funded innovations; patent term extensions and data exclusivity strategies are considered.

Corporate and financial highlights

• Employees: 226 full‑time employees as of December 31, 2025.
• Revenue and income:
  • Minimal revenue to date; no material product revenue reported in the period.
  • 2025 net income: $71.2 million, primarily from the gain on the sale of a Rare Pediatric Disease Priority Review Voucher (PRV).
  • Excluding the PRV gain, 2025 net loss: $(81.2) million.
• Financing and liquidity:
  • Accumulated deficit: $742.1 million through December 31, 2025.
  • 2025 PRV sale: gross proceeds of $155.0 million (completed June 2025 after regulatory clearance).
• Tax attributes:
  • As of 12/31/2025: U.S. federal NOL carryforwards $310.7 million; state NOL carryforwards $11.6 million; general business credits $5.2 million; state credits $0.1 million.
  • Section 382 ownership changes identified; certain NOLs are no longer realizable and valuation allowance adjustments have been made.
• Leases and facilities:
  • HQ and manufacturing: approximately 73,100 square feet leased in Cleveland, Ohio, through December 2030.
  • NYC office: approximately 10,400 square feet, lease expired September 2025.

Business model and market considerations

• Commercial model: direct commercialization of ZEVASKYN with potential strategic partnerships for other assets.
• Market footprint:
  • Four active ZEVASKYN treatment centers as of March 2026.
  • Payer coverage includes CMS and Medicaid programs nationwide.
• Product portfolio:
  • Single commercial product (ZEVASKYN) and ongoing preclinical and early clinical programs in gene therapy.
  • Pipeline emphasizes in‑vivo AAV approaches with AIM™ capsids and several ophthalmic disease programs.
• IP reliance: substantial dependence on licensed intellectual property and collaboration agreements; ongoing IP protection and potential term extensions are important to maintain competitive position.

Headquarters

• Office: 6555 Carnegie Ave, 4th Floor, Cleveland, OH 44103
• Phone: (646) 813‑4701