Viralgen and Axovia Therapeutics are set to form a partnership to progress the development and manufacturing of an adeno-associated virus vector serotype 9 (AAV9)-based gene therapy for retinal dystrophy in individuals with Bardet-Biedl Syndrome (BBS) due to BBS1 gene mutations.

The collaboration seeks to prevent vision loss in BBS patients.

The investigational gene therapy utilises a codon-optimised BBS1 AAV9 vector tailored to slow down vision loss caused by genetic defects.

Viralgen will manufacture the therapy at its facility, employing its expertise in AAV and the Pro10 cell line and manufacturing platform.

Combined with Axovia's gene therapy development knowledge, the company seeks swift delivery.

Axovia co-founder and chief scientific officer Dr Victor Hernandez stated: “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 [contract development and managing organisation].

“Our lead programme, AXV-101, which is being developed to address retinal dystrophy associated with 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.”

BBS leads to multi-system impairments, including obesity and progressive retinal dystrophy.

Viralgen, established in 2017 as AskBio’s independently operated subsidiary, is part of the Bayer Group.

The company’s facilities in San Sebastián, Spain, feature a commercial facility with three modules, each housing three current good manufacturing practices suites with capacities exceeding 2,000L.