Abeona Therapeutics Inc
NASDAQ:ABEO
Abeona Therapeutics Inc
Abeona Therapeutics, Inc. is a clinical stage biopharmaceutical company, which engages in the development of gene therapy for severe and life-threatening rare diseases. The company is headquartered in New York City, New York and currently employs 90 full-time employees. The Company’s lead clinical programs consist of EB-101, which is an autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa (RDEB), ABO-102, which is an adeno-associated virus (AAV)-based gene therapy for Sanfilippo syndrome type A (MPS IIIA), and ABO-101, which is an AAV-based gene therapy for Sanfilippo syndrome type B (MPS IIIB). The company is focused on to develop additional AAV-based gene therapies designed to treat ophthalmic and other diseases and AAV-based gene therapies using the AIM capsid platform that has a license from the University of North Carolina at Chapel Hill and internal AAV vector research programs.
Abeona Therapeutics, Inc. is a clinical stage biopharmaceutical company, which engages in the development of gene therapy for severe and life-threatening rare diseases. The company is headquartered in New York City, New York and currently employs 90 full-time employees. The Company’s lead clinical programs consist of EB-101, which is an autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa (RDEB), ABO-102, which is an adeno-associated virus (AAV)-based gene therapy for Sanfilippo syndrome type A (MPS IIIA), and ABO-101, which is an AAV-based gene therapy for Sanfilippo syndrome type B (MPS IIIB). The company is focused on to develop additional AAV-based gene therapies designed to treat ophthalmic and other diseases and AAV-based gene therapies using the AIM capsid platform that has a license from the University of North Carolina at Chapel Hill and internal AAV vector research programs.
Commercial Launch Progress: Abeona is seeing strong patient demand for ZEVASKYN, with the first patient treatment now expected in Q4 2025 after addressing a manufacturing assay issue.
QTC Network Expansion: The company has activated a third Qualified Treatment Center and is working to add more sites across the US.
Patient Pipeline: The number of identified eligible patients has more than doubled to around 30, with 12 product order forms already received.
Market Access: ZEVASKYN now has coverage policies from all major commercial payers and baseline Medicaid coverage across all 51 states and Puerto Rico.
Financial Position: Abeona ended Q3 2025 with $207.5 million in cash, expected to fund operations for over two years.
Profitability Outlook: Management reiterated that they expect to be profitable in the first half of 2026, with no significant change despite the delay.
Manufacturing Update: Release assay optimization caused a temporary pause, but biopsy collection has resumed and plant maintenance is scheduled for mid-December to early January.
Pipeline & Team: The AD0503 gene therapy program was selected for an FDA pilot, and a new SVP for Clinical Development was appointed.
