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Axovia Therapeutics, Viralgen Partner on Bardet-Biedl Syndrome Gene Therapy

NEW YORK – Axovia Therapeutics and Viralgen on Wednesday said they have entered into a partnership to advance development and manufacturing for AXV-101, a gene therapy designed to treat retinal dystrophy in patients with Bardet-Biedl syndrome (BBS).

BBS is a rare and inherited disorder that causes a variety of symptoms, often including vision loss as tissue in the retina progressively deteriorates. It can be caused by mutations in more than a dozen genes.

Within this partnership, London-based Axovia, a biotech company that develops gene therapies, and San Sebastián, Spain-based Viralgen, a contract development and manufacturing organization, will focus on developing a treatment for BBS caused by biallelic mutations in the BBS1 gene. AXV-101, Axovia's lead candidate, uses an adeno-associated virus 9 vector to deliver a codon-optimized version of BBS1 that's designed to reduce vision loss by stopping photoreceptor cell death and retinal degeneration.

The gene therapy will be manufactured by Viralgen using its Pro10 cell line and manufacturing platform, which it licensed from Bayer's AskBio. Viralgen is a subsidiary of AskBio.

Axovia Cofounder and CSO Victor Hernandez said in a statement that he anticipates AXV-101 will enter clinical development in mid-2025.