Abeona Therapeutics, Inc., formerly PlasmaTech Biopharmaceuticals, Inc., is focused on developing and delivering gene therapy and plasma-based products for severe and rare diseases. The Company's lead program includes adeno-associated virus (AAV)-based gene therapy for Sanfilippo syndrome (MPS IIIA and IIIB). Its product pipeline includes ABO-101 (sc AAV9 NAGLU), which is used for the treatment of MPS III B; ABO-102 (sc AAV9 SGSH), which is used for the treatment of MPS III A; ABO-201 (sc AAV9 CLN3), which is used for the treatment of Juvenile Batten Disease (JBD); ABO-301 (AAV LK19 FANCC), which is used for the treatment of Fanconi Anemia (FA), and clustered, regularly interspaced short palindromic repeats/CRISPR associated protein 9 (CRISPR/Cas9) gene editing, which is used for treatment of other rare blood diseases. The Company offers two platforms: Salt Diafiltration (SDF) Process and Polymer Hydrogel Technology (PHT).