OT-004

OT-004 is an investigational gene therapy developed by Odylia Therapeutics, a nonprofit biotech organization, in partnership with PTC Therapeutics. It is designed to treat vision loss caused by mutations in the RPGRIP1 gene, which lead to RPGRIP1-associated retinal dystrophies such as Leber Congenital Amaurosis type 6 (LCA6), Cone-Rod Dystrophy 13 (CORD13), and juvenile Retinitis Pigmentosa. The therapy utilizes the Anc80 adeno-associated virus (AAV) vector—an ancestral AAV discovered at Massachusetts Eye and Ear—to deliver a functional copy of the RPGRIP1 gene to retinal photoreceptor cells. OT-004 has received Orphan Drug and Rare Pediatric Disease Designations from the FDA and is currently in late-stage preclinical development, with clinical trials anticipated to begin in 2025.

Other names

RPGRIP1 Gene TherapyRPGRIP-1 Gene TherapyRPGRIP 1 Gene TherapyAnc80-RPGRIP1Anc-80-RPGRIP1Anc 80-RPGRIP1