Axovia partners with CDMO Viralgen to manufacture gene therapy for vision loss

Axovia Therapeutics has partnered with Spain-based CDMO Viralgen to advance the development and manufacture of an AAV9-based investigational gene therapy for patients with a rare disease that can cause vision loss and blindness.

AXV-101, Axovia’s lead program, is aimed at treating retinal dystrophy in patients with Bardet-Biedl Syndrome (BBS) caused by biallelic mutations in the BBS1 gene. The genetic disease appears in individuals carrying biallelic mutations in the BBS1 gene, leading to progressive vision loss and complete blindness.

AXV-101 uses the codon-optimized BBS1 AAV9 vector to decrease the vision loss caused by the genetic defects in the BBS1 gene.

Per the deal, the therapy, which is expected to enter clinical development in mid-2025, will be manufactured at Viralgen’s state-of-the-art facility in San Sebastian, Spain. Viralgen, which is subsidiary of AskBio, will use a proprietary AskBio Pro10 suspension manufacturing platform designed to accelerate clinical development and commercialization.