Abeona Therapeutics Announced That Its ABO-503 Gene Therapy For X-linked Retinoschisis Has Been Selected To Participate In FDA Rare Disease Endpoint Advancement Pilot Program
Benzinga Newsdesk 1 year ago
BiotechNewsHealth CareFDAGeneral
ABO-503 is composed of a functional human RS1 gene packaged in the novel AIM™ capsid AAV204. ABO-503 has shown preclinical efficacy following delivery to the retina in a mouse model of XLRS. Preclinical studies
- ABO-503 is composed of a functional human RS1 gene packaged in the novel AIM™ capsid AAV204. ABO-503 has shown preclinical efficacy following delivery to the retina in a mouse model of XLRS. Preclinical studies have demonstrated structural and functional improvements following robust RS1 expression throughout the retina, including improved cone photoreceptor density and overall photoreceptor cell survival, restoration of outer retina architecture by eliminating cysts characteristic of XLRS, and improvements in visual function as demonstrated by electroretinogram (ERG). Abeona anticipates completing IND-enabling studies in the second half of 2026.
- The FDA launched the RDEA Pilot Program to support the development of novel efficacy endpoints for rare disease treatments. Under the pilot program, between 2023 and 2027, the FDA will accept up to one RDEA proposal per quarter with a maximum of three proposals per year. To be considered, sponsors must submit a proposal detailing the data they plan to collect, the novelty of the endpoint, and its potential to establish substantial evidence of effectiveness.