Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company that develops cell and gene therapies for serious rare diseases, including recessive dystrophic epidermolysis bullosa (RDEB), ophthalmic disorders, and neurological conditions such as Sanfilippo Syndrome Type A (MPS IIIA), Rett Syndrome, and Infantile Batten Disease (CLN1 Disease). The company offers ZEVASKYN (prademagene zamikeracel; pz-cel or EB-101), an FDA-approved autologous, gene-corrected epidermal cell sheet therapy indicated for treating wounds in adult and pediatric patients with RDEB following a single application; adeno-associated virus (AAV)-based gene therapies including preclinical programs ABO-503 for X-Linked Retinoschisis (XLRS), ABO-504 for Stargardt Disease, and ABO-505 for Autosomal Dominant Optic Atrophy (ADOA); and partnered programs such as UX111 for MPS IIIA with Ultragenyx Pharmaceutical Inc. and TSHA-102 for Rett Syndrome and TSHA-118 for CLN1 Disease with Taysha Gene Therapies Inc. Founded in 1974 and headquartered in Cleveland, Ohio, Abeona Therapeutics operates primarily in the United States with a focus on rare genetic diseases affecting pediatric and adult patients. Recent developments include the U.S. FDA approval of ZEVASKYN on April 28, 2025, based on the Phase 3 VIITAL study demonstrating significant wound healing and pain reduction; commercial launch preparations through Qualified Treatment Centers beginning in the third quarter of 2025; entry into a definitive agreement on May 12, 2025, to sell its Rare Pediatric Disease Priority Review Voucher for $155 million, which closed on June 27, 2025, boosting cash reserves to approximately $225 million as of June 30, 2025; and a $75 million public offering priced in May 2024 to support biologics license application resubmission and operations.