Abeona Therapeutics
Abeona Therapeutics is a commercial-stage biopharmaceutical company developing cell and gene therapies, with its FDA-approved ZEVASKYN (prademagene zamikeracel) being the first and only gene therapy for recessive dystrophic epidermolysis bullosa (RDEB) wounds, priced at $6.2M per two-cycle treatment.
- Company typePublic
- Founded1989
- HeadquartersNew York, United States
- Headcount101–250
- GTM typeB2B
- OfferingHardware or Manufacturing
What Abeona Therapeutics does
Abeona Therapeutics Inc. (NASDAQ: ABEO) is a commercial-stage biopharmaceutical company headquartered in Cleveland, Ohio that develops cell and gene therapies for serious rare diseases. Its lead and only commercial product is ZEVASKYN (prademagene zamikeracel), which received FDA approval on April 28, 2025 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 severe genetic skin disease caused by a defect in the COL7A1 gene. The therapy uses gene-modified cellular sheets applied to chronic wounds and is priced at $6.2 million for two treatment cycles.
ZEVASKYN is manufactured at Abeona's fully integrated cGMP cell and gene therapy facility in Cleveland and administered exclusively through a Qualified Treatment Center network that includes Lucile Packard Children's Hospital Stanford, NewYork-Presbyterian/Columbia, Children's Hospital of Philadelphia, and the University of Texas Medical Branch. As of Q1 2026, six sites were activated, three patients had been treated, and 95% of commercially insured U.S. lives were covered under payer policies. Revenue is recognized on a per-treatment basis, and the company benefited from a permanent CMS J-code established effective January 1, 2026.
Abeona's development pipeline is anchored by ABO-701 (PSMA-SIR-T), an engineered T-cell therapy for advanced prostate cancer licensed from Angeles Therapeutics (Reverence Enterprises) in March 2026, and ABO-503, an AAV gene therapy for X-linked retinoschisis selected for the FDA Rare Disease Endpoint Advancement Pilot Program in October 2025. The company has historically supplemented product revenue with licensing and royalty streams from out-licensed assets, including tiered royalties (mid-single-digits to 8%) plus up to $30 million in commercial milestones from Ultragenyx for ABO-102 (Sanfilippo syndrome type A), and earned a one-time $155 million gain in 2025 from the sale of the Rare Pediatric Disease Priority Review Voucher granted alongside ZEVASKYN's approval.
Abeona Therapeutics firmographics
Firmographics- Name
- Abeona Therapeutics
- Legal name
- Abeona Therapeutics Inc.
- Website
- https://abeonatherapeutics.com
- Company type
- Public
- Founded year
- 1989
- Operating status
- Operating
- Headcount range
- 101–250 employees
- Short description
- Abeona Therapeutics is a commercial-stage biopharmaceutical company developing cell and gene therapies, with its FDA-approved ZEVASKYN (prademagene zamikeracel) being the first and only gene therapy for recessive dystrophic epidermolysis bullosa (RDEB) wounds, priced at $6.2M per two-cycle treatment.
- Ownership category
- akta.pro rank
Abeona Therapeutics industry classification
Industry- Product category
- Biopharmaceutical Gene Therapy
- NAICS
- Biological Product (except Diagnostic) Manufacturing (325414), Research and Development in Biotechnology (except Nanobiotechnology) (541714)
- SIC
- Biological Products, (No Disgnostic Substances) (2836)
- akta.pro primary industry
- Genetic & Genomic Rare Disease Therapeutics (HLAIAIAA)
- akta.pro secondary industry
- Cell Therapy Manufacturing Services (GMP, autologous/allogeneic) (HLAAAKAC)
Keywords
Where Abeona Therapeutics is headquartered
LocationHeadquarters
- HQ city
- New York
- HQ country
- United States
- HQ region
- North America
Offices2 records
Markets served
Abeona Therapeutics business model
Business model- GTM type
- B2B
- Offering type
- Hardware or Manufacturing
- Cost components
- Technology or R&D, Personnel, Operations, Infrastructure, Marketing or Sales
Revenue model
- ZEVASKYN Product Sales: Revenue generated from sales of ZEVASKYN gene therapy product to qualified treatment centers. ZEVASKYN is sold as a single treatment application for RDEB wounds.
- Licensing and Milestone Revenue: Revenue from out-licensing technology including AAV capsids to third parties, with potential event-based milestones up to $24M and sales-based milestones up to $45M, plus royalties.
- Ultragenyx Royalty Stream: Tiered royalties from mid-single-digits to 8% on net sales of ABO-102 (Sanfilippo syndrome type A), plus up to $30M in commercial milestone payments.
Pricing tiers
| Model | Billing | Price |
|---|---|---|
| One time/ perpetual license | Pay-as-you-go | ZEVASKYN gene therapy treatment - $6.2 million for two treatment cycles |
Go-to-market motion1 record
Distribution channels1 record
Marketing channels4 records
Abeona Therapeutics product offering
Product offeringCore offering
Abeona Therapeutics is a commercial-stage biopharmaceutical company that develops and commercializes cell and gene therapies. Its flagship product, ZEVASKYN (prademagene zamikeracel), is the first and only FDA-approved autologous cell-based gene therapy for treating wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB). The company also maintains a development pipeline including PSMA-SIR-T (ABO-701) for prostate cancer and ABO-503 for X-linked retinoschisis, and operates a fully integrated cGMP manufacturing facility in Cleveland, Ohio.
Product overview
Abeona Therapeutics is a commercial-stage biopharmaceutical company with ZEVASKYN (prademagene zamikeracel) as its FDA-approved flagship product—the first and only autologous cell-based gene therapy for recessive dystrophic epidermolysis bullosa (RDEB). The company operates a fully integrated cGMP manufacturing facility in Cleveland, Ohio. Its development pipeline includes ABO-701 (PSMA-SIR-T), an engineered T-cell therapy for prostate cancer, and ABO-503 for X-linked retinoschisis.
Differentiator
Problem solved
Functional benefit
Brands
- ZEVASKYN (prademagene zamikeracel): FDA-approved gene therapy for recessive dystrophic epidermolysis bullosa (RDEB), the first and only autologous cell-based gene therapy for this condition.
- Abeona Assist
- PSMA-SIR-T (ABO-701)
- AIM capsids
Products and services
- ZEVASKYN (prademagene zamikeracel) FDA-approved autologous cell-based gene therapy for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB). Uses gene-modified cellular sheets to promote wound healing with a single application. Administered through Qualified Treatment Centers.
- ABO-701 (PSMA-SIR-T) Engineered T-cell therapy targeting Prostate-Specific Membrane Antigen (PSMA) for advanced prostate cancer. Features Synthetic Immune Receptor (SIR-T) technology designed to overcome limitations of CAR and TCR approaches in solid tumors.
- ABO-503 Gene therapy for X-linked retinoschisis (XLRS), a genetic eye disorder causing retinal damage and vision loss. Selected for FDA's Rare Disease Endpoint Advancement (RDEA) Pilot Program.
Quantifiable outcome
- ZEVASKYN received FDA approval on April 28, 2025 as the first and only autologous cell-based gene therapy for RDEB wounds
- +3 more outcomes
Companies that use Abeona Therapeutics
Customer profileNamed customers3 records
Segments1 record
Ideal customer profiles2 records
Abeona Therapeutics technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature3 records
Abeona Therapeutics partnerships and signals
Strategic signalPartnerships
Five partnerships are on record, tiered core and minor.
- Reverence Enterprises, LLC (Angeles Therapeutics subsidiary)coreLicense and Joint Development Agreement for PSMA-SIR-T (ABO-701) engineered T-cell therapy targeting PSMA-positive prostate cancer. Abeona obtained exclusive license to develop and commercialize the therapy. Abeona agreed to file an IND and conduct a Phase 1 Study. Total potential deal value includes $7M upfront and up to $1M in milestone payments.
- Ultragenyx Pharmaceutical Inc.coreExclusive license agreement for AAV gene therapy ABO-102 (Sanfilippo syndrome type A/MPS IIIA). Ultragenyx assumed responsibility for development, manufacturing, and commercialization worldwide. Abeona eligible for tiered royalties from mid-single-digits to 8% on net sales plus up to $30M commercial milestones.
- Taysha Gene TherapiesminorSublicense agreements for CLN1 disease gene therapy (ABO-202/TSHA-118) and Rett Syndrome gene therapy. Taysha received worldwide exclusive rights. Agreements were jointly terminated on February 25, 2026.
- The Board of Trustees of Leland Stanford Junior UniversitycoreLicensing agreements for EB-101 (LZRSE-Col7A1 Engineered Autologous Epidermal Sheets) and EB-201 (AAV DJ COL7A1) for RDEB gene therapy. Abeona pays upfront licensing fees, annual maintenance fees, milestone payments, and low single-digit royalties on net sales.
- University of North Carolina at Chapel HillcoreLicensed international patent family covering novel AAV capsids (AIM capsids) for gene therapy delivery. Also licensed rights to CLN1 disease (infantile Batten disease) and Rett Syndrome programs. Abeona sublicensed some rights to Taysha Gene Therapies.
Scale indicators9 records
Recent moves6 records
Expansion highlights6 records
Abeona Therapeutics competitors and assessment
Company assessmentDirect peers
- Krystal Biotech: Commercial-stage gene therapy company with Vyjuvek, the first FDA-approved topical gene therapy for dystrophic epidermolysis bullosa (DEB) — the closest direct competitor to Abeona's ZEVASKYN in the same DEB indication, with a similar ultra-rare disease, single-application treatment model.
- Sarepta Therapeutics: Commercial-stage gene therapy company with Elevidys for Duchenne muscular dystrophy and a pipeline of AAV-based therapies for rare neuromuscular diseases — closely comparable business model of approved genetic medicines targeting rare diseases with high-price, single-application treatment economics.
- uniQure: Clinical-stage gene therapy company developing AAV-based therapies for rare diseases including hemophilia B and Huntington's disease — a directly comparable AAV gene therapy peer in the rare disease space with similar technology foundation and pre-commercial/early-commercial stage.
- bluebird bio: Gene therapy company commercializing Zynteglo for beta-thalassemia and Skysona for cerebral adrenoleukodystrophy — comparable as a developer of approved, ultra-rare disease gene therapies with similar pricing and treatment-center delivery model, though currently undergoing strategic restructuring.
- Solid Biosciences: Clinical-stage genetic medicines company developing AAV gene therapies for Duchenne muscular dystrophy and other rare neuromuscular diseases — comparable as a small-cap rare-disease gene therapy developer with similar technology platform and pipeline-stage economics.
- Taysha Gene Therapies: Clinical-stage gene therapy company focused on rare monogenic diseases of the central nervous system — a directly comparable rare-disease AAV gene therapy peer and former sublicensee of Abeona's CLN1 and Rett Syndrome programs (jointly terminated Feb 2026).
Broad incumbents
- BioMarin Pharmaceutical: Established rare disease biopharma with Roctavian (AAV gene therapy for hemophilia A) and a broader portfolio of rare genetic disease therapeutics — comparable in the rare-disease gene therapy commercial model but with substantially greater scale and a more diversified portfolio.
- Ultragenyx Pharmaceutical: Commercial-stage rare disease biopharma with multiple approved products across genetic diseases — directly comparable as Abeona's exclusive licensee for ABO-102 (Sanfilippo A) and operates in the same ultra-rare genetic disease commercial space with substantially greater scale.
- Vertex Pharmaceuticals: Large-cap biopharma with Casgevy, the first FDA-approved CRISPR/Cas9 gene-edited cell therapy for sickle cell disease and beta-thalassemia — comparable as a leader in approved genetic medicines targeting rare diseases, with substantially greater scale and a broader commercial and pipeline portfolio.
Emerging players
- Sangamo Therapeutics: Clinical-stage gene therapy and genome editing company developing AAV and zinc finger nuclease-based therapies for rare genetic diseases and neurology — comparable as a smaller rare-disease gene therapy peer with platform-level technology overlap, though Sangamo is broader in editing modalities.
Market position
Strengths5 records
Weaknesses5 records
Competitive moat4 records
Key risks7 records
Key highlights7 records
Customer concentration
Abeona Therapeutics social profiles
Digital presenceAbeona Therapeutics compliance and trust
Trust signalCompliance2 records
Abeona Therapeutics financial estimates
Financial estimateRevenue estimate
Valuation estimate
Abeona Therapeutics leadership team
Management profileNumber of profiles
Profiles8 records
Abeona Therapeutics funding detail
Funding detailFunding overview
Funding rounds13 records
Investors15 records
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
Abeona Therapeutics M&A and investment
M&A and investmentM&A1 record
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about Abeona Therapeutics
What does Abeona Therapeutics do?
Abeona Therapeutics is a commercial-stage biopharmaceutical company that develops and commercializes cell and gene therapies. Its flagship product, ZEVASKYN (prademagene zamikeracel), is the first and only FDA-approved autologous cell-based gene therapy for treating wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB). The company also maintains a development pipeline including PSMA-SIR-T (ABO-701) for prostate cancer and ABO-503 for X-linked retinoschisis, and operates a fully integrated cGMP manufacturing facility in Cleveland, Ohio.
Is Abeona Therapeutics a public or private company?
Abeona Therapeutics is a public company. It is classified as public and is currently operating.
When was Abeona Therapeutics founded?
Abeona Therapeutics was founded in 1989. It employs 101 to 250 people.
Where is Abeona Therapeutics based?
Abeona Therapeutics is headquartered in New York, United States, in the North America region.
How does Abeona Therapeutics make money?
Three revenue lines are on record. ZEVASKYN Product Sales are the primary driver. The others are licensing and Milestone Revenue and ultragenyx Royalty Stream.
Who are Abeona Therapeutics's main competitors?
Direct peers on record are Krystal Biotech, Sarepta Therapeutics, uniQure, bluebird bio, Solid Biosciences and Taysha Gene Therapies. Broad incumbents are BioMarin Pharmaceutical, Ultragenyx Pharmaceutical and Vertex Pharmaceuticals. Sangamo Therapeutics is listed as an emerging player.
Does Abeona Therapeutics have an API?
No public API is recorded for Abeona Therapeutics.
What industry is Abeona Therapeutics in?
Abeona Therapeutics's product category is Biopharmaceutical Gene Therapy. Its primary akta.pro industry code is HLAIAIAA, Genetic & Genomic Rare Disease Therapeutics, with a secondary code of HLAAAKAC, Cell Therapy Manufacturing Services (GMP, autologous/allogeneic). Its NAICS code is 325414 and its SIC code is 2836.