Abeona Therapeutics

Abeona Therapeutics Inc., a clinical-stage biopharmaceutical company, develops gene and cell therapies for life-threatening rare genetic diseases. Its lead program is EB-101, an autologous, gene-corrected cell therapy that is in Phase III clinical trial for recessive dystrophic epidermolysis bullosa. 

The company also develops ABO-102, an adeno-associated virus (AAV)-based gene therapy for Sanfilippo syndrome type A; ABO-201 to treat CLN3 disease; ABO-401 for the treatment of cystic fibrosis; and ABO-50X for the treatment of genetic eye disorders. In addition, it is developing AAV-based gene therapy through its AIM vector platform programs. 

The company was formerly known as PlasmaTech Biopharmaceuticals, Inc. and changed its name to Abeona Therapeutics Inc. in June 2015. Abeona Therapeutics Inc. was incorporated in 1974 and is headquartered in New York, New York.

Founded in 2013, headquartered in Cleveland, Ohio. Abeona pioneered cutting-edge technology bringing innovative gene and cell therapies to patients with a diverse range of rare genetic diseases. Rare monogenic diseases affect the blood, bone marrow, nervous system, and other organ systems. Abeona has developed a platform of gene and cell therapies including ABO-102, ABO-101, and EB-101.

Abeona's lead product, ABO-102, is a gene therapy designed to treat Sanfilippo Syndrome Type A, an inherited lysosomal storage disorder that leads to progressive neurodegeneration in children. Abeona has been granted Orphan Drug Designation for ABO-102 by the US Food and Drug Administration (FDA). Abeona is also developing ABO-101, a gene therapy designed to treat MPS IIIA, an inherited lysosomal storage disorder that primarily affects the central nervous system.

Abeona has partnered with universities and other biotechnology companies to advance its research initiatives and has also acquired exclusive licenses to various technologies and intellectual property. In 2015, the company acquired a portfolio of gene therapy technologies from the University of North Carolina at Chapel Hill.

Abeona’s mission is to develop and provide novel gene therapies to improve patients’ lives and make gene therapy accessible to more individuals.

Abeona’s vision is to be the global leader in developing and commercializing gene therapies that provide revolutionary treatments for life-threatening rare genetic diseases.