From Conservative Treatments to Disease-Transformative Gene Therapies

To date, 22 BBS genes have been identified and ~85% of BBS patients have mutations in any one of these genes. Our team is committed to developing transformative gene therapies that will greatly improve the quality of life of BBS patients and their caregivers.

Our Gene Therapies

Axovia is currently developing therapies for patients with mutations in the BBS1 gene. The ultimate goal of these treatments is to help better manage and to cure the most debilitating aspects of the disease, blindness, metabolic and neurocognitive impairment.

Our Technology

Using the latest Adeno-Associated Virus (AAV) technology, we are developing gene replacement therapies to treat the root cause of the disease. AAV has been shown to be safe and effective in preclinical and clinical settings.

Axovia Milestones

To develop the preclinical evidence for a viable gene therapy, the UCL team received funding from a number of sources including Apollo Therapeutics. With support from Jaguar Gene Therapy, Axovia Therapeutics was formed to advance these first-in-human therapies to clinic.