Abeona Therapeutics, Inc. develops and delivers gene therapy and plasma-based products for rare diseases. The Company’s programs include AB0-101 (AAV9 NAGLU) and ABO-102 (scAAV9 SGSH), adeno-associated virus (AAV)-based gene therapies for Sanfilippo syndrome (MPS IIIB and IIIA). It is developing ABO-201 (scAAV9 CLN3) gene therapy for juvenile Batten disease (JBD); and ABO-301 (AAV LK19 FANCC) for Fanconi anemia (FA) disorder using a novel CRISPR/Cas9-based gene editing approach to gene therapy program for rare blood diseases. In addition, the Company is also developing rare plasma protein therapies including SDF Alpha (alpha-1 protease inhibitor) for inherited COPD using its SDF (Salt Diafiltration) ethanol-free process.
