Charles River Laboratories International, Inc. (NYSE: CRL) today announced a plasmid DNA contract development and manufacturing organization (CDMO) collaboration with Axovia Therapeutics Ltd. Charles River will 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, which is 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.

Plasmid DNA Manufacturing Services

Axovia will leverage Charles River’s established plasmid platform, eXpDNA™, and premier 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.

In recent years, Charles River has significantly broadened its cell and gene therapy portfolio to simplify complex supply chains and meet the growing demand for plasmid DNA, viral vector, and cell therapy services. Combined with the Company’s legacy testing capabilities, Charles River offers a comprehensive “concept-to-cure” advanced therapies solution.