First human trial launches for gene therapy targeting rare blindness disorder
Disease control
Not yet recruiting
This early-stage trial tests a single dose of AXV-101, a gene therapy, in 12 children aged 4 to 17 with Bardet-Biedl syndrome type 1 (BBS1) who have vision loss. The main goal is to see if the treatment is safe and tolerable, and to find the right dose. Participants will have eye…
Phase: EARLY_PHASE1 • Sponsor: Axovia Therapeutics • Aim: Disease control
Last updated May 17, 2026 16:35 UTC