Charles River Laboratories International Inc. has announced a plasmid DNA contract development and manufacturing organization (CDMO) collaboration with Axovia Therapeutics Ltd. under which 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.
 
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.
 
“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,” said Victor Hernandez, Co-Founder and Chief Scientific Officer, Axovia.

Related Charles River News

Earlier this month, Charles River and Ship of Theseus, a therapeutics company, announced a GMP plasmid DNA CDMO agreement under which Ship of Theseus will leverage Charles River’s expertise to manufacture GMP plasmid DNA to serve as the active drug substance for its lead candidate.
 
Also, Durect Corporation entered into a co-marketing and collaboration agreement with Charles River for the ALZET Osmotic Pumps Portfolio and Associated Product Line in the U.S. and Canada.