Abeona Therapeutics® Announces ABO-503 Gene Therapy for X-linked Retinoschisis (XLRS) Selected by FDA for Rare Disease Endpoint Advancement (RDEA) Pilot Program

Core Insights - Abeona Therapeutics' ABO-503 gene therapy for X-linked retinoschisis (XLRS) has been selected for the FDA's Rare Disease Endpoint Advancement (RDEA) Pilot Program, which aims to enhance communication and collaboration with the FDA to expedite the development of rare disease therapies [1][2][3] Company Overview - Abeona Therapeutics Inc. is a commercial-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases, including the first autologous cell-based gene therapy for treating wounds in patients with recessive dystrophic epidermolysis bullosa [5] - The company operates a fully integrated cGMP manufacturing facility in Cleveland, Ohio, which is responsible for the commercial production of its therapies [5] Industry Context - The RDEA Pilot Program, launched by the FDA, supports the development of novel efficacy endpoints for rare disease treatments, allowing for up to three proposals per year from sponsors [3] - XLRS is a rare, monogenic retinal disease with an estimated incidence of 1 in 5,000 to 1 in 20,000 in males, affecting approximately 35,000 individuals in the U.S. and Europe combined, with no approved disease-modifying therapies currently available [4]