Viralgen and Axovia Partner to Manufacture Gene Therapy for Retinal Dystrophy in Bardet-Biedl Syndrome Patients

Viralgen and Axovia Therapeutics are partnering to develop and manufacture a gene therapy for the treatment of retinal dystrophy in patients with Bardet-Biedl Syndrome (BBS) caused by biallelic mutations in the BBS1 gene. This will offer patients access to an investigational gene therapy that uses the codon-optimized BBS1 AAV9 vector to halt vision loss.¹

BBS is a rare genetic disorder that occurs in one in 250,000 patients around the world, with an estimated 3,000 of those patients being in the United States and Canada.² Patients suffer from progressive vision loss, sometimes resulting in complete blindness. Obesity, reduced kidney function, hypogonadism, kidney malformations/malfunctions, and polydactyly (extra digits of the hands or feet) are more notable symptoms of BBS.² Typical treatment options, besides gene therapy, include stem cell and suppressor therapy.

Axovia cites Viralgen’s experience with producing AAV therapies as a key factor in the partnership and says that it expects to use the CDMO’s facility and its Pro10 cell line and manufacturing platform to manufacture the gene therapy.

"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," Axovia co-founder and chief scientific officer Dr. Victor Hernandez said in the release. "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," Jimmy Vanhove, CEO of Viralgen, added.

Earlier this month, the company also announced a partnership with Vallabh/Minikel Lab and Deverman Lab, both part of the Broad Institute of MIT and Harvard, to develop and manufacture a gene therapy for prion disease—a progressive neurodegenerative disorder with an urgent unmet need.³ This therapy also uses Viralgen’s AAV manufacturing capabilities and Pro10™ cell line.

Sonia Vallabh, co-leader of the prion disease initiative at the Broad Institute, said in the release announcing the partnership, "We're incredibly lucky to be doing this work at this exact moment in time, because prion disease is a whole brain disease, we've faced two major challenges from the beginning: finding a molecular machine that can target our gene and delivering that machine to enough neurons in the adult human brain to make a difference. It is just now becoming imaginable that, with the right team, we could piece together answers to these two big questions in our lifetime. We're so fortunate to have the chance to try."

Sources

Viralgen and Axovia partner to manufacture an investigational gene therapy for retinal dystrophy in Bardet-Biedl Syndrome (BBS) patients. Viralgen. January 22, 2025. https://www.prnewswire.com/news-releases/viralgen-and-axovia-partner-to-manufacture-an-investigational-gene-therapy-for-retinal-dystrophy-in-bardet-biedl-syndrome-bbs-patients-302357167.html

What is BBS? Bardet Biedl Syndrome Foundation. 2022. Accessed January 22, 2025. https://www.bardetbiedl.org/what-is-bbs

Viralgen partners with Vallabh/Minikel Lab and Deverman Lab at the Broad Institute to advance gene therapy manufacturing hope for prion disease. January 8, 2025. Accessed January 22, 2025. https://www.prnewswire.com/news-releases/viralgen-partners-with-vallabhminikel-lab-and-deverman-lab-at-the-broad-institute-to-advance-gene-therapy-manufacturing-hope-for-prion-disease-302344759.html