Abeona Therapeutics Expands Manufacturing Capacity Following FDA Approval of First Gene Therapy for Rare Skin Disorder
Key Insights
Abeona Therapeutics received FDA approval in April for ZEVASKYN (search), the first autologous cell-based gene therapy for recessive dystrophic epidermolysis bullosa (search) (RDEB (search)), a rare genetic skin disorder.
The company is investing $2.85 million to expand its Cleveland manufacturing facilities with a new 7,587-square-foot laboratory space to support commercial production and development of additional gene therapies.
Following the therapy approval, Abeona sold its Priority Review Voucher for $155 million, providing sufficient cash for over two years of operations and positioning the company for profitability by early 2026.
Abeona Therapeutics Inc. is undertaking a significant $2.85 million expansion of its Cleveland manufacturing facilities following FDA approval of ZEVASKYN (search), the first and only autologous cell-based gene therapy for treating recessive dystrophic epidermolysis bullosa (search) (RDEB (search)), a rare genetic skin disorder that causes extremely fragile skin and painful blistering.
FDA Breakthrough Drives Commercial Expansion
The publicly traded biopharmaceutical company (Nasdaq: ABEO) received FDA approval for ZEVASKYN (search) (prademagene zamikeracel) in April 2024, marking a significant milestone for patients with RDEB (search), a genetic disorder that leads to blisters and open sores from minor injuries or friction. The therapy represents the first treatment option specifically designed for this devastating condition affecting both adults and pediatric patients.
Following the regulatory approval, Abeona has filed permits with the Cleveland Building Department to fit-out a 7,587-square-foot laboratory space on the second floor of Building One at Midtown Tech Park, located at 6700 Euclid Avenue. This expansion complements the company's existing fully integrated cell and gene therapy Current Good Manufacturing Practices facility in Building Three, which serves as the manufacturing site for ZEVASKYN (search) commercial production.
Strong Financial Position Supports Growth
The FDA approval triggered significant financial benefits for Abeona through the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $155 million. Chief Financial Officer Joe Vazzano stated that the proceeds provide "sufficient cash for more than two years of operating expenses without the need for capital infusion and not accounting for ZEVASKYN (search) sales."
With ZEVASKYN (search) expected to become available to patients beginning in the third quarter of 2025, the company anticipates achieving profitability by early 2026. This timeline reflects the transition from a development-stage company to a commercial operation generating revenue from its breakthrough therapy.
Expanding Gene Therapy Pipeline
Beyond ZEVASKYN (search), Abeona is developing additional treatments including adeno-associated virus (search) (AAV (search))-based gene therapies for ophthalmic diseases with high unmet medical need. The expanded manufacturing capacity will support the development and potential commercial production of these pipeline candidates.
The company, founded in 1974 as Access Pharmaceuticals Inc., transformed into Abeona Therapeutics in 2015 to focus specifically on gene and cell therapies for serious diseases. The current expansion represents the latest growth phase for the company at Midtown Tech Park, following a previous 12,000-square-foot expansion five years ago.
Cleveland's Growing Biotech Hub
Abeona's expansion contributes to Cleveland's growing reputation as a biotechnology hub, with nearly 200 health-technology and high-technology employers located in the city's Health Tech Corridor, which runs along Cedar and Chester avenues from University Circle to the Campus District. Fifty of these firms are located in Midtown proper, according to MidTown Cleveland, highlighting the area's concentration of life sciences companies.