AAVAnc80-RPGRIP1

AAVAnc80-RPGRIP1 (OT-004) is a preclinical gene therapy candidate developed by Odylia Therapeutics for the treatment of RPGRIP1-associated retinal dystrophies, including Leber Congenital Amaurosis Type 6 (LCA6), cone-rod dystrophy 13 (CORD13), and juvenile retinitis pigmentosa. The therapy utilizes the Anc80 synthetic adeno-associated virus (AAV) vector, which is designed for enhanced transduction efficiency and broader retinal distribution compared to naturally occurring AAV serotypes. By delivering a functional copy of the RPGRIP1 gene, the therapy aims to restore the production of the RPGRIP1 protein, a critical component of the photoreceptor connecting cilium. The program has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA and was previously licensed to PTC Therapeutics before returning to Odylia's internal pipeline.

Other names

AAVAnc80-RPGRIP1AAVAnc-80-RPGRIP1AAVAnc 80-RPGRIP1OT-004OT004OT 004