Partnership Supports Strategic Expansion into Rare Disease Gene Therapy Space

Comprehensive AAV gene therapy manufacturing collaboration to support early phase trials

With our gene therapy expertise and offerings continuing to support therapeutic advancements for rare diseases, our contract development and manufacturing organization (CDMO) team has entered an agreement to reinforce a leading biopharmaceutical company's drug development pipeline in the Asia-Pacific (APAC) region.

As they enter preclinical development with multiple gene therapy candidates targeting rare diseases, the company has announced the intention to file an Investigational New Drug (IND) application to conduct a Phase I clinical trial in 2025.

Supporting a comprehensive ophthalmic gene therapy program through this strategic partnership, the Company will leverage our industry-leading CDMO capabilities, including established production platforms and dedicated centers of excellence, supplying Good Manufacturing Practice (GMP)-grade plasmid DNA and AAV vectors.

A Commitment to Rare Disease Development

Across the global organization, our integrated teams have deep expertise in the discovery, development, and manufacturingof drugs to treat a myriad of rare diseases. In fact, in 2023 alone, we worked on over 80% of the rare disease drugs approved by the US Food and Drug Administration.

Earlier this year we also announced strategic CDMO manufacturing collaborations with organizations including FOXG1 Research Foundationand AAVantgarde, dedicated to providing therapeutic treatments for rare disease patients.

We are thrilled about this latest collaboration as the Company enters the gene therapy space. Our teams are already working closely together to manufacture, test, and release the drug product in support of clinical studies, validation, and conformance lots to support the upcoming IND application."

Kerstin Dolph, CSVP, Global Manufacturing, Charles River

For more information on our integrated plasmid DNA, viral vector, and cell therapy development and manufacturing platforms and services, please visit Cell and Gene Therapy CDMO Solutions.