Abeona Therapeutics Incについて 企業名

Abeona Therapeutics Inc. is a commercial-stage biopharmaceutical company developing cell and gene therapies for serious diseases. The Company'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)).