Ciliopathies are a group of more than 40 rare inherited genetic diseases linked to more than 950 genes that impact the function of cilia. These microscopic finger-like organelles protrude from most cells in the body.

The lead program AXV101, an AAV9-based gene therapy targeting retinal dystrophy associated with Bardet-Biedl Syndrome (BBS) in patients carrying biallelic mutations in the BBS1 gene. It is designed to halt photoreceptor cell death and retinal degeneration.