Gene therapy injection aims to save sight in rare genetic disease
Disease control
Not yet recruiting
This early-stage trial tests a single injection of AXV-101 gene therapy into one eye of 12 children and teens (ages 4-17) with Bardet-Biedl syndrome type 1, a rare genetic condition that causes progressive vision loss. The main goal is to check safety and find the right dose, whi…
Phase: EARLY_PHASE1 • Sponsor: Axovia Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 12:02 UTC