After decades of helping people with ciliopathies to manage their medical conditions without dedicated treatments, Professor Phil Beales and Dr. Victor Hernandez co-founded Axovia to develop treatments to address the disease burden for this group of disorders.
While there are more than 25 genes linked to Bardet-Biedl Syndrome (BBS), we are developing our lead program for the 40% of people with the disorder who carry two mutations in the BBS1 gene. Our novel gene therapy utilizes an adeno-associated virus (AAV9) to deliver a functional copy of the faulty BBS gene in key tissues as we seek to prevent blindness and reverse obesity.
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