Abeona Therapeutics, Inc. is a clinical stage biopharmaceutical company, which engages in the development of gene therapy for severe and life-threatening rare diseases. The company is headquartered in Cleveland, Ohio and currently employs 136 full-time employees. The firm's ZEVASKYN (prademagene zamikeracel) is an autologous cell-based gene therapy for the treatment of wounds in adults and pediatric patients with recessive dystrophic epidermolysis bullosa. Its fully integrated cell and gene therapy cGMP manufacturing facility in Cleveland, Ohio serves as the manufacturing site for ZEVASKYN commercial production. Its development portfolio features adeno-associated virus (AAV)-based gene therapies for ophthalmic diseases with high unmet medical need. Its AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases. Its pipeline programs include ABO-504 (Stargardt disease), ABO-503 (X-linked retinoschisis), ABO-505 (autosomal dominant optic atrophy), UX111 (Sanfilippo Syndrome Type A (MPS IIIA)), TSHA-102 (Rett Syndrome), and TSHA-118 (Infantile Batten Disease (CLN1 Disease)).
