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About Axovia Therapeutics

Written by: Editor
Published on: 23 May 2024

Axovia TherapeuticsAxovia Therapeutics is leading the development of therapies that address the genetic causes of blindness and obesity which are driven by ciliopathies. Ciliopathies are a group of more than 40 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 the first half of 2025 based on robust preclinical data, scaled manufacturing and established patient registries. 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. Axovia is backed by ALSA Ventures and was formed following decades of work on ciliopathies at University College London by co-founders Professor Phil Beales and Dr. Victor Hernandez.