Successful gene therapies necessitate meticulous design to ensure effective delivery of nucleic acids to targeted cells and tissues, employing either viral or non-viral vectors.

Viral vector-based gene therapies, especially those utilizing adeno-associated viruses (AAV), are gaining traction despite facing several development challenges.

Different viral vectors, such as AAV, exhibit distinct properties that influence their efficiency, tissue specificity, and immunogenicity; for instance, AAV12 is particularly effective for targeting salivary gland cells.

To overcome development challenges, scientists can conduct additional in vitro or ex vivo testing, which is more cost-effective than animal models, facilitating better optimization of gene therapies.

Charles River offers a comprehensive range of products and services designed to assist researchers in optimizing the design and development of viral gene therapies.

Traditional treatments typically focus on alleviating symptoms rather than addressing the underlying genetic issues, which gene therapies aim to rectify by modifying patients' cells in vivo.

Genetics significantly contributes to the onset and progression of a wide array of diseases, ranging from rare genetic disorders to various forms of cancer.

As of August 2023, the FDA has approved eight gene therapies, with numerous others currently in different stages of preclinical and clinical development.

Researchers often encounter high costs when moving from in vitro to in vivo studies, which can impede the development process due to limited resources for testing multiple therapy versions.

Their offerings include off-the-shelf plasmid and viral vector solutions, AAV and lentivirus packaging plasmids, and custom plasmid design options tailored to specific research needs.

Additionally, Charles River provides viral vector packaging services, supplying ready-to-use recombinant AAV and lentiviral particles suitable for various project scales, from small-scale testing to large clinical trials.