Charles River Laboratories International Inc. and AAVantgarde have entered a contract development and manufacturing organization (CDMO) agreement to produce Good Manufacturing Practice- (GMP) plasmid DNA.

AAVantgarde, a clinical-stage biotechnology company, will leverage Charles River’s expertise in manufacturing GMP plasmid DNA.

AAVantgarde has two proprietary AAV-based large gene delivery platforms, both of which aim to enable efficient delivery of large genes to tissue and cells in vivo. Within this collaboration, Charles River will develop the plasmid DNA for AAVantgarde’s Stargardt’s disease program (AAVB-039), using its AAV-intein platform, which has demonstrated an efficient recombination to deliver therapeutically meaningful protein levels.

Stargardt’s is the most prevalent inherited macular dystrophy and is an autosomal recessive genetic disorder due to mutations in the ABCA4 gene characterized by progressive loss of central vision starting from childhood or adolescence, leading to profound vision loss. It is the most common form of inherited juvenile macular degeneration representing a very high unmet need as there are currently no therapies available for a disease that affects approximately 1:6,500 people.

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.

Through this collaboration, AAVantgarde will have access to Charles River’s GMP plasmid DNA CDMO center of excellence based in Keele, United Kingdom, which will lead the collaboration and additional evaluation of off-the-shelf Rep/Cap and pHelper plasmid products in addition to GMP manufacturing services to support therapeutic development.

“Charles River is thrilled to produce GMP plasmid DNA to help advance AAVantgarde’s platform for the treatment of Stargardt’s disease – a condition in which there is high unmet need. Our team brings more than two decades of CDMO expertise, and we look forward to leveraging these capabilities to make a difference for patients,” said Kerstin Dolph, Corporate SVP, Global Manufacturing, Charles River.

Nina Kotsopoulou, PhD, Chief Technical Officer, AAVantgarde, added, “By collaborating with Charles River, we are one step closer to beginning clinical trials to ensure our therapeutic product for Stargardt’s Disease is safe and efficacious for patients. We trust the team’s decades of success developing, producing, and reliably delivering plasmid DNA and look forward to expanding treatment options for this patient population.”