Pipeline
We may be science-led, but we’re people-focused
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 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.
| Disease | Program | 2024 | 2025 | 2026 | 2027 | ||
|---|---|---|---|---|---|---|---|
| Ciliopathies | |||||||
| Disease: BBS1 | Program: AXV101 (SR) | Stage: Pre-Clinical | |||||
| Disease: BBS1 | Program: AXV102 (ICV) | Stage: Pre-Clinical | |||||
| Disease: MC4R | Program: AXV201 (ICV) | Stage: Discovery | |||||
- Discovery
- Pre-Clinical
- First in Human
AVX-101
To learn more about the study, sponsored by Axovia Therapeutics, and eligibility criteria for participation, please visit https://www.isrctn.com/ISRCTN96250868. The study is not currently enrolling.