Axovia Therapeutics and Bardet-Biedl Syndrome (BBS)

Current Development

Axovia Therapeutics is working on a treatment for Bardet-Biedl Syndrome (BBS) called AXV-101, a gene therapy for people with mutations in the BBS1 gene. The first clinical trial of AXV-101 is now recruiting. It will investigate whether the treatment is safe and whether it can slow down retinal degeneration, a common issue in BBS.

The trial is open to children and young people aged 4 to 17 with BBS1 mutations and retinal degeneration. In England, it is taking place at Moorfields Eye Hospital in London.

Find out more about this trial: AXV-101 BBS1 gene therapy trial (ISRCTN96250868)

Future Plans

Following the retinal study, Axovia plans to explore treatments for other BBS-related symptoms, including hyperphagia and obesity, with trials for this aspect expected to start in 2026-27.

Key resources:

Axovia Therapeutics, Pipeline (August 2024). https://axoviatherapeutics.com/about-our-pipeline/

ISRCTN registry, AXV-101 trial in BBS1 (ISRCTN96250868). https://www.isrctn.com/ISRCTN96250868

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