GA001

GA001 is an investigational gene therapy developed by Jianda Kyushu (Beijing) Biotechnology for the treatment of retinitis pigmentosa (RP). It utilizes the AAV2.7m8 capsid, a synthetic adeno-associated virus variant engineered for enhanced retinal penetration following intravitreal injection, allowing it to reach the outer retina more effectively than standard AAV2 vectors. GA001 is designed as a mutation-independent (broad-spectrum) therapy, aiming to preserve or restore visual function in RP patients regardless of their specific genetic mutation. It is currently being evaluated in Phase 1 clinical trials in China to assess its safety, tolerability, and preliminary efficacy in adult patients over a 52-week period.