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Axevia

Lake Forest, Illinois, United Stateszoominfo.com ↗

Ownership: Private
Development stage: Other

Overview

Axovia Therapeutics develops AAV9-based gene therapies targeting rare genetic disorders caused by cilia dysfunction, with a lead program (AXV101) for Bardet-Biedl Syndrome type 1 (BBS1). AXV101 has received FDA Orphan Drug and Rare Pediatric Disease designations and will enter first-in-human studies in the UK in mid/late 2025. The company has a pipeline of up to six potential therapies for ciliopathies.

Therapeutic areas: PsychiatryOphthalmologyRare DiseasesEndocrinology
Modalities: Gene therapyAdeno-associated virus-based gene therapy
Industry: Biotech

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