Axovia Therapeutics

Axovia Therapeutics is a biotechnology company developing adeno-associated viral gene therapies for inherited ciliopathies and genetic obesity.

Its lead candidate delivers a functional BBS1 gene into retinal cells to investigate treatment of vision loss associated with Bardet-Biedl syndrome. A first-in-human, early Phase 1 dose-escalation trial began in June 2026 and is recruiting participants.

Additional preclinical programmes investigate central nervous system delivery for hyperphagia and obesity associated with BBS1 and MC4R mutations.