Viralgen and Axovia Partner to Advance Gene Therapy for Bardet-Biedl Syndrome

  • Viralgen and Axovia Therapeutics are partnering to develop and manufacture an investigational AAV9-based gene therapy for Bardet-Biedl Syndrome (BBS).
  • The therapy, expected to enter clinical development by mid-2025, aims to halt retinal degeneration and vision loss in patients with BBS caused by BBS1 gene mutations.

Viralgen and Axovia Therapeutics have announced a collaboration to advance the development and production of an investigational gene therapy targeting Bardet-Biedl Syndrome (BBS), a genetic condition causing progressive vision loss and multi-system disorders. The partnership aims to address the needs of patients suffering from retinal dystrophy caused by biallelic mutations in the BBS1 gene.

The therapy, using a codon-optimised BBS1 AAV9 vector, is designed to prevent photoreceptor cell death and retinal degeneration, thereby halting vision loss in affected individuals. Axovia’s lead programme, AXV-101, is expected to enter clinical trials by mid-2025. Viralgen will manufacture the therapy at its cutting-edge facility, employing its Pro10™ cell line and AAV manufacturing platform.

Dr. Victor Hernandez, Co-Founder and Chief Scientific Officer of Axovia, highlighted the importance of this collaboration: “As Axovia advances its pipeline of potential therapies addressing the genetic causes of blindness, we want to ensure we have appropriately scaled AAV manufacturing to support this effort, and are very happy to have partnered with Viralgen as our CDMO.”

Jimmy Vanhove, CEO of Viralgen, commented, “We are committed to supplying quality vectors and timely supply with the goal of providing disease-modifying treatment for patients affected by BBS1-related retinal dystrophy.”

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