Charles River Laboratories International, Inc. and AAVantgarde announced a contract development and manufacturing organization (CDMO) agreement to produce Good Manufacturing Practice- (GMP) plasmid DNA. AAVantgarde, a clinical-stage biotechnology company with two proprietary adeno-associated viral (AAV) vector platforms for large gene delivery and developing products to treat inherited retinal diseases, 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 their AAV-intein platform, which has demonstrated a very 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. Combined with Charles River’s legacy testing capabilities, developers can leverage a comprehensive “concept to cure” advanced therapies solution.
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.
SOURCE: Businesswire