Groundbreaking eye treatment trial offers hope for rare genetic vision loss in children
Disease control
Not yet recruiting
This first-in-human study aims to test the safety and find the right dose of a new treatment called AXV-101 for children aged 4-17 with Bardet-Biedl syndrome type 1 (BBS1) who have early retinal degeneration. Researchers will give a single dose to one eye and closely monitor both…
Phase: EARLY_PHASE1 • Sponsor: Axovia Therapeutics • Aim: Disease control
Last updated Mar 31, 2026 12:11 UTC