4D Molecular Therapeutics Announces First Patient Dosed in Phase 1/2 Clinical Trial of 4D-125 by Intravitreal Injection for the Treatment of X-Linked Retinitis Pigmentosa

News | 08. 19. 2020

4D Molecular Therapeutics

EMERYVILLE, CA -- 4D Molecular Therapeutics (4DMT), a clinical-stage leader in the development of precision-guided AAV gene medicines based on directed evolution, announced that the first patient has been dosed in the Phase 1/2 clinical trial of 4D-125 for X-Linked Retinitis Pigmentosa (XLRP), a blinding and currently untreatable inherited retinal disease. 4D-125 is an AAV gene therapy with an optimized and proprietary vector designed to deliver a functional copy of the RPGR gene to photoreceptors in the retina. This vector enables both administration by intravitreal injection, a safe and routine clinical route, plus the potential to treat broader and earlier-stage patient populations as compared to subretinal approaches. 4D-125 is a 4DMT-declared ophthalmology therapeutic candidate under 4DMT’s collaboration with Roche, where Roche has an option to take an exclusive world-wide license prior to initiation of a pivotal trial.