Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing novel gene therapies for life-threatening rare genetic diseases. The Company’s lead programs include ABO-102 (AAV-SGSH), an adeno-associated virus (AAV) based gene therapy for Sanfilippo syndrome type A (MPS IIIA) and EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB). It is also developing ABO-101 (AAV-NAGLU) for Sanfilippo syndrome type B (MPS IIIB), ABO-201 (AAV-CLN3) gene therapy for juvenile Batten disease (JNCL), ABO-202 (AAV-CLN1) for treatment of infantile Batten disease (INCL), EB-201 for epidermolysis bullosa, ABO-301 (AAV-FANCC) for Fanconi anemia disorder and ABO-302 using a novel CRISPR/Cas9-based gene editing approach to gene therapy for rare blood diseases. The Company also has a plasma-based protein therapy pipeline, including alpha-1 protease inhibitor (SDF Alpha) for inherited COPD, using its proprietary Salt Diafiltration ethanol-free process.
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