Viralgen and Axovia partner to manufacture an investigational gene therapy for retinal dystrophy in Bardet-Biedl Syndrome (BBS) patients

Collaborative effort will bring hope to BBS patients suffering from retinal degeneration through this investigational gene therapy.

SAN SEBASTIÁN, Spain and LONDON, Jan. 22, 2025 /PRNewswire/ -- 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 innovative 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 needed 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 state-of-the-art facility, leveraging the company's expertise in AAV and using the Pro10™ cell line and manufacturing platform.

"As Axovia advances its pipeline of potential therapies addressing the genetic causes of blindness towards the clinic, 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," said Co-Founder and Chief Scientific Officer, Dr. Victor Hernandez. "Our lead program, AXV-101, which is being developed to address retinal dystrophy associated with Bardet-Biedl Syndrome (BBS) is expected to enter clinical development in mid-2025, and we believe this partnership will ensure fast and efficient development as we seek to advance this therapy toward patients as soon as possible."

"We are delighted to partner with Axovia and bring in our AAV manufacturing expertise. 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" said Jimmy Vanhove, CEO of Viralgen.

This partnership will combine Axovia's expertise in gene therapy development with Viralgen's world-class AAV manufacturing capabilities, ensuring that patients with Bardet-Biedl Syndrome may receive investigational treatment as swiftly as possible. Both companies aim to provide an effective gene therapy solution to address the pressing medical needs of patients suffering from BBS1-related retinal dystrophy. This collaboration marks a step forward in delivering disease modifying treatment to a patient population that has had few options until now.

About Viralgen

Viralgen is a fully integrated Contract Development and Manufacturing Organization (CDMO) founded in 2017 as an independently operated subsidiary of AskBio Inc., part of the Bayer AG group. Viralgen was created to support development through large-scale commercial production of current Good Manufacturing Practices (cGMP) AAV (adeno-associated virus) for cell and gene therapies. Through the AskBio licensed proprietary Pro10™ suspension manufacturing platform, Viralgen delivers industry-leading titers for all AAV serotypes, optimizing speed to market and cost-of-goods to accelerate clinical development and commercialization.

The company's state-of-the-art facilities based in San Sebastian, Spain include a commercial facility consisting of three modules, each having three cGMP suites with capacities of >2,000 L, one of which has received the cGMP certification by the Spanish Agency for Medicines and Medical Devices (AEMPS) as part of the EMA for the manufacture of human medicinal products, investigational medicinal products and sterile or biological active substances. The recently inaugurated MSAT laboratory provides advanced services for transitioning programs from clinical to commercial manufacturing. Leveraging the proven expertise in AAVs, Viralgen has recently launched several development services focused on construct optimization, process optimization and right-sized scaling for customers needing additional support, in addition to the established research, process development, formulation and filling activities.

For more information, visit viralgen.com.

SOURCE Viralgen