Viralgen and Axovia Therapeutics partner to manufacture gene therapy for Bardet-Biedl Syndrome

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Key Takeaways

Viralgen and Axovia are developing an AAV9-based gene therapy for retinal dystrophy in Bardet-Biedl Syndrome (BBS) patients.
The therapy aims to stop photoreceptor cell death and vision loss using a codon-optimized BBS1 AAV9 vector.
Axovia's lead program, AXV-101, is expected to enter clinical development by mid-2025.
Viralgen will manufacture the therapy using its Pro10 cell line and platform, ensuring efficient development and timely supply.

The companies will work together by using the codon-optimized BBS1 AAV9 vector to minimize the vision loss caused by the genetic defects in the BBS1 gene.

(Image Credit: AdobeStock/neirfy)

Viralgen and Axovia Therapeutics are partnering 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). The collaboration will give patients access to a dynamic investigational treatment with the potential to stop photoreceptor cell death and retinal degeneration, as well as halt vision loss.

“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,” Victor Hernandez. co-founder and chief scientific officer, said. “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.”

The two companies will work together by using the codon-optimized BBS1 AAV9 vector to minimize the vision loss caused by the genetic defects in the BBS1 gene. The therapy will also be manufactured at Viralgen’s facility, capitalizing on the company’s expertise in AAV and using the Pro10 cell line and manufacturing platform.

“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,” Jimmy Vanhove, CEO of Viralgen, said.

Reference:

Viralgen and Axovia Partner to Manufacture an Investigational Gene Therapy for Retinal Dystrophy in Bardet-Biedl Syndrome (BBS) patients. Viralgen. Accessed January 27, 2025. Viralgen and Axovia Partner to Manufacture an Investigational Gene Therapy for Retinal Dystrophy in Bardet-Biedl Syndrome (BBS) Patients