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.