Axovia Therapeutics is focused on developing innovative therapies for genetic disorders associated with ciliopathies, particularly Bardet-Biedl Syndrome (BBS). Ciliopathies are a group of rare inherited diseases that affect the function of cilia, which are essential for various cellular processes. Axovia's lead program, AXV-101, targets individuals with BBS who have mutations in the BBS1 gene. The company employs a novel gene therapy approach using adeno-associated virus (AAV9) to deliver a functional copy of the faulty gene to key tissues, aiming to prevent blindness and reverse obesity. This therapy is backed by robust preclinical data and is set to enter clinical trials in mid-2025.

In addition to its lead program, Axovia is also developing a second program targeting the most common genetic cause of obesity, with plans to initiate clinical testing in 2026. The AAV platform used by Axovia is notable for its safety profile, as it does not replicate like disease-causing viruses and has been utilized in other successful therapies. The company’s commitment to addressing the unmet medical needs of patients with ciliopathies is underscored by its focus on modifying the underlying disease mechanisms, with the potential to significantly improve the quality of life for affected individuals.

United Kingdom

Axovia Therapeutics is dedicated to the research and development of novel AAV gene therapies that target key aspects of ciliopathies, addressing the devastating impact of the disease head-on for patients, their families and caregivers.

Axovia is currently developing therapies for patients with mutations in the BBS1 gene which manifests as Bardet Biedl Syndrome. Our initial goal is to halt the progression of BBS by targeting the most debilitating aspects of the neurological, metabolic and retinal dysfunctions.

Axovia Therapeutics

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