Develops gene and cell therapies for severe genetic diseases, focusing on rare diseases affecting children.
Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical firm specializing in the development of gene and cell therapies aimed at addressing life-threatening rare genetic diseases. Established in 1974 and based in New York, New York, the company is dedicated to advancing innovative treatments that target severe genetic disorders. Among its flagship initiatives is EB-101, an autologous cell therapy currently in Phase III clinical trials for recessive dystrophic epidermolysis bullosa, a debilitating skin condition.
In addition to EB-101, Abeona Therapeutics is actively progressing several other promising therapies. These include ABO-102, a gene therapy utilizing adeno-associated virus (AAV) technology designed to treat Sanfilippo syndrome type A, and ABO-201 for CLN3 disease. The company's diverse pipeline also encompasses ABO-401, focusing on cystic fibrosis treatment, and ABO-50X, targeting genetic eye disorders, all developed using their AIM vector platform.
Formerly known as PlasmaTech Biopharmaceuticals, Inc., Abeona Therapeutics rebranded in June 2015 to reflect its renewed focus on cutting-edge genetic therapies. With a rich history in biopharmaceutical innovation and a commitment to advancing treatments for underserved patient populations, Abeona Therapeutics continues to drive forward in its mission to revolutionize the treatment landscape for rare genetic diseases.