Charles River Laboratories to Manufacture Plasmid DNA for Axovia

• Charles River Laboratories and Axovia Therapeutics have announced a collaboration for the manufacture of High Quality (HQ) gene of interest plasmid.

• The collaboration aims to support the development of Axovia’s gene therapies for ciliopathies, including Bardet-Biedl Syndrome (BBS).

• Axovia will leverage Charles River’s plasmid platform, eXpDNA, and expertise in plasmid DNA production.

Charles River Laboratories has announced a contract development and manufacturing organisation (CDMO) collaboration with Axovia Therapeutics Ltd. The collaboration will see Charles River manufacture High Quality (HQ) gene of interest plasmid to support the development of Axovia’s gene therapies for ciliopathies, including Bardet-Biedl Syndrome (BBS), a condition with limited treatment options and no cure.

Ciliopathies are diseases caused by cilia dysfunction, a group of complex disorders caused by genetic mutations that result in defective or dysfunctional cilia in many organs of the human body. Dysfunctional cilia can cause blindness, deafness, chronic respiratory infections, kidney disease, heart disease, infertility, obesity and diabetes. Over 20 ciliopathies have been identified, collectively affecting an estimated 1 in 1,000 people.

Axovia’s lead program, AXV101, is an adeno-associated virus (AAV9)-based gene therapy targeting retinal dystrophy associated with BBS. In preclinical studies for BBS, the novel gene therapy modified the underlying disease, rescuing vision loss by halting retinal degeneration, stopping BBS-induced weight gain and the development of obesity. Axovia has been granted FDA Orphan Drug and Rare Pediatric Disease Designations.

Axovia will leverage Charles River’s plasmid platform, eXpDNA, and expertise in plasmid DNA production, including HQ plasmid which combines key features of good manufacturing practice (GMP) manufacture with a rapid turnaround to accelerate time to clinic.

Victor Hernandez, Co-Founder and Chief Scientific Officer, Axovia, said, “This collaboration brings us closer to accomplishing our goal to develop potentially transformative therapies for ciliopathies. We are excited to leverage Charles River’s expertise in plasmid DNA production to help drive our work forward.”