Charles River to Manufacture Plasmids for AAVantgarde

Charles River Laboratories International, Inc. (NYSE: CRL) and AAVantgarde have entered into a contract development and manufacturing organization (CDMO) agreement to produce Good Manufacturing Practice (GMP) plasmid DNA. AAVantgarde, a clinical-stage biotechnology firm specializing in adeno-associated viral (AAV) vector platforms for large gene delivery, intends to utilize Charles River’s expertise in producing GMP plasmid DNA for their products aimed at treating inherited retinal diseases.

AAVantgarde boasts two proprietary AAV-based platforms designed for efficient large gene delivery to tissues and cells within the body. Through their collaboration, Charles River will develop the plasmid DNA necessary for AAVantgarde’s Stargardt’s disease program (AAVB-039) using the AAV-intein platform. This platform has shown high efficiency in recombination, delivering therapeutically significant protein levels.

Stargardt’s disease is the most prevalent form of inherited macular dystrophy, an autosomal recessive genetic disorder caused by mutations in the ABCA4 gene. Characterized by the progressive loss of central vision beginning in childhood or adolescence, it leads to severe vision impairment. Affecting approximately 1 in 6,500 individuals, it represents a significant unmet medical need as there are currently no available treatments.

In recent years, Charles River has expanded its cell and gene therapy portfolio to address the increasing demand for plasmid DNA, viral vectors, and cell therapy services. By combining these capabilities with its established testing services, Charles River offers a comprehensive solution from concept to cure for advanced therapies.

This collaboration allows AAVantgarde to access Charles River’s GMP plasmid DNA CDMO center of excellence located in Keele, United Kingdom. This center will spearhead the collaboration and further assess off-the-shelf Rep/Cap and pHelper plasmid products, alongside providing GMP manufacturing services to support therapeutic development.

Approved Quotes:

According to Kerstin Dolph, Corporate Senior Vice President of Global Manufacturing at Charles River, "Charles River is thrilled to produce GMP plasmid DNA to help advance AAVantgarde’s platform for the treatment of Stargardt’s disease – a condition with a high unmet need. Our team brings over two decades of CDMO expertise, and we are excited to leverage these capabilities to make a difference for patients."

Nina Kotsopoulou, PhD, Chief Technical Officer at AAVantgarde, expressed, "By collaborating with Charles River, we are one step closer to initiating clinical trials to ensure our therapeutic product for Stargardt’s Disease is safe and effective for patients. We trust in the team's decades of experience in developing, producing, and reliably delivering plasmid DNA and look forward to broadening treatment options for this patient population."

About Charles River:

Charles River provides essential products and services to pharmaceutical and biotechnology companies, government agencies, and leading academic institutions worldwide, helping to accelerate their research and drug development processes. The company's employees are dedicated to meeting client needs to expedite the discovery, early-stage development, and safe manufacture of new therapies for patients who need them.

About AAVantgarde:

AAVantgarde is an international biotechnology company headquartered in Italy, in the clinical stage, specializing in adeno-associated viral (AAV) vector platforms to overcome the gene therapy cargo capacity limitations of AAV vectors. Founded by Professor Alberto Auricchio at TIGEM (Telethon Institute of Genetics and Medicine) in Naples, Italy, and Telethon Foundation, AAVantgarde aims to initially validate its platforms in the clinic for two inherited retinal diseases with significant unmet needs.

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