About

We are developing new therapies to treat diseases caused by cilia dysfunction

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.

Pipeline

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 safety data, 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 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.

Disease Program 2024 2025 2026 2027
Ciliopathies
Disease: BBS1 Program: AXV101 (SR) Stage: Pre-Clinical
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Disease: BBS1 Program: AXV102 (ICV) Stage: Pre-Clinical
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Disease: Undisclosed Ciliopathy Program: AXV201 (ICV) Stage: Discovery
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  • Discovery
  • Pre-Clinical
  • First in Human