About

Axovia Therapeutics is leading the development of therapies that address the genetic forms of blindness and obesity associated with ciliopathies. Ciliopathies are a group of more than 55 rare inherited genetic diseases linked to more than 950 genes that impact the function of cilia which are critical for protein transport and cellular signaling.

The company is positioned to initiate clinical studies for its lead program for Bardet-Biedl Syndrome (BBS), AXV-101, in mid-2025 based on robust preclinical efficacy and toxicological data, established scaled manufacturing and strong patient community engagement and a leading comprehensive patient registry. The initial subretinal study is designed to halt photoreceptor cell death and prevent further retinal degeneration and the CNS delivery program, which will begin in 2026, will seek to address hyperphagia and obesity. AXV-101 has achieved U.S. Food and Drug Administration Orphan Drug Designation and Rare Pediatric Disease Designation. The company is developing its second program, AXV-201, for genetic obesity caused by MC4R mutations.