Viralgen and Axovia Therapeutics are joining forces to advance the development and manufacture of an AAV9-based investigational gene therapy aimed at treating retinal dystrophy in patients with Bardet-Biedl Syndrome (BBS) caused by biallelic mutations in the BBS1 gene. This partnership will offer patients access to an investigational treatment that has the potential to stop photoreceptor cell death and retinal degeneration, and halt vision loss.
Bardet-Biedl Syndrome (BBS) is a genetic condition that causes multi-system disorders, with obesity and progressive retinal dystrophy being among the most debilitating symptoms. This disease appears in individuals carrying biallelic mutations in the BBS1 gene, leading to progressive vision loss and complete blindness, among other manifestations.
Through this collaboration, Viralgen and Axovia will work together to bring this investigational gene therapy to patients, which uses the codon-optimized BBS1 AAV9 vector to decrease the vision loss caused by the genetic defects in the BBS1 gene. This investigational therapy will be manufactured at Viralgen's facility, leveraging the company's expertise in AAV and using the Pro10 cell line and manufacturing platform.
This partnership will combine Axovia's expertise in gene therapy development with Viralgen's AAV manufacturing capabilities. Both companies aim to provide an effective gene therapy solution to address the pressing medical needs of patients suffering from BBS1-related retinal dystrophy.
