BBS1 Gene Therapy
Bardet-Biedl Syndrome (BBS1 mutation subset)
Pre-clinicalActive
Key Facts
Indication
Bardet-Biedl Syndrome (BBS1 mutation subset)
Phase
Pre-clinical
Status
Active
Company
About Axovia Therapeutics
Axovia Therapeutics is a private, preclinical-stage biotech founded by leading ciliopathy experts to address the significant unmet need in disorders like Bardet-Biedl Syndrome. The company is developing an AAV9-based gene therapy targeting the BBS1 gene mutation, which accounts for approximately 40% of BBS cases, with the dual aims of preventing retinal degeneration and reversing obesity. Operating from the Cambridge biotech hub, Axovia is positioned to leverage advanced genetic medicine platforms to create first-in-class therapies for these rare diseases with no approved treatments.
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