Axovia teams with Viralgen to manufacture BBS gene therapyAxovia teams with Viralgen to manufacture BBS gene therapy

Contract development manufacturing organization (CDMO) Viralgen and biopharmaceutical developer Axovia Therapeutics have partnered to develop and manufacture AXV-101, an investigational gene therapy for treating retinal dystrophy in patients with Bardet-Biedl Syndrome (BBS).

According to Axovia CEO Victor Hernandez, the therapy is expected to enter clinical development in mid-2025. “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 [adeno-associated virus] manufacturing to support this effort and are very happy to have partnered with Viralgen as our CDMO.”

Viralgen will make the drug at its medicinal products and gene therapies manufacturing facility in San Sebastian, Spain using AskBio’s Pro10 cell line and manufacturing platform, which is a mammalian suspension technology designed to produce AAV vectors. According to Viralgen, the Pro10 cells “have been optimized for  recombinant AAV production and successfully scaled up to 2000 liters, demonstrating consistent and reproducible cell growth rates.”

BBS is a rare genetic disease that can cause a number of physiological symptoms including blindness due to retinal degeneration. According to a presentation by Hernandez in May 2024, AXV-101 safely and effectively stopped retinal degeneration in preclinical animal models.

“These data suggest that our BBS1 novel gene therapy can modify the underlying disease of BBS, including rescuing vision loss by halting retinal degeneration but also supports our development efforts to correct the genetic cause of hyperphagia and morbid obesity that is a hallmark of BBS mutations. 

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

For Viralgen, the dawn of 2025 has been an eventful one. Earlier this month, the CDMO partnered with university laboratories based in the Broad Institute of Massachusetts Institute of Technology (MIT) and Harvard University to develop and manufacture gene therapies to treat prion disease, a fatal and currently untreatable neurodegenerative disorder.