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

Viralgen and Axovia Therapeutics have announced a strategic collaboration to develop and manufacture an AAV9-based investigational gene therapy targeting retinal dystrophy in patients with Bardet-Biedl Syndrome (BBS). This partnership aims to provide patients with an innovative therapy designed to prevent photoreceptor cell death, halt retinal degeneration, and preserve vision.

Advancing Gene Therapy for BBS-Related Retinal Dystrophy

Through this collaboration, Axovia will leverage Viralgen’s expertise in AAV manufacturing to ensure the scalable production of AXV-101, Axovia’s lead gene therapy candidate for BBS1-related retinal dystrophy. The therapy is expected to enter clinical development in mid-2025.

Leadership Perspectives

Victor Hernandez, Ph.D., co-founder and Chief Scientific Officer of Axovia Therapeutics, emphasized the importance of this partnership:

“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, 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.”

Leveraging Viralgen’s AAV Manufacturing Expertise

The companies will use a codon-optimized BBS1 AAV9 vector to target genetic defects in the BBS1 gene, aiming to slow or prevent vision loss in affected patients. The therapy will be manufactured at Viralgen’s state-of-the-art facility, utilizing its proprietary Pro10™ cell line and advanced AAV manufacturing platform to ensure high-quality vector production.

Jimmy Vanhove, CEO of Viralgen, highlighted the company’s commitment to supporting Axovia’s mission:

"We are delighted to partner with Axovia and bring in our AAV manufacturing expertise. We are committed to supplying quality vectors and ensuring timely delivery, with the goal of providing a disease-modifying treatment for patients affected by BBS1-related retinal dystrophy."

Conclusion

This partnership between Viralgen and Axovia Therapeutics represents a major step forward in gene therapy for inherited retinal diseases. With AXV-101 set to enter clinical development in 2025, this collaboration brings new hope to patients with Bardet-Biedl Syndrome, offering the potential to halt disease progression and preserve vision.