Driving Your Gene and Cell Therapy Research

Gene and Cell therapies require scientists to rethink previous drug development models and consider a path from source material to the clinic that is unique to each program. Conducting hundreds of gene and cell therapy programs, Charles River offers the scientific and regulatory expertise to ensure you are asking the right questions to drive your research down the right path along with the products and services you need to get to market. From source material, safety and efficacy testing to process optimization, and final product testing, we can provide the support and continuity necessary for success. ¬¬¬

• Product characterization
• Safety and Efficacy
• Laboratory Sciences
• CMC Process Development
• Scientific and Regulatory Advisory Services

High-quality Starting Material
The quality and consistency of the primary source material is directly related to the quality and efficacy of the final cell-based product. HemaCare, a Charles River company, is the global leader in providing human-derived biological products and apheresis collection services, directly enabling customers to advance autologous and allogeneic cell therapies. With access to the largest registry of healthy reliable and recallable donors, the cell-based starting materials are delivered with exceptional purity, viability, and quality to ensure consistency and reproducibility.

In Vitro/ in Vivo Efficacy and Safety
Novel in vitro models aid lead optimization and can provide insight into potential toxicities utilizing primary cell-based assays, whereas clinically relevant and robust animal models allow for characterization of exposure and effects in humans. Reliable and sensitive methods of cell/gene/vector detection are critical elements in developing advanced therapies. These tools are used in characterization, proof of concept, pilot and definitive biodistribution studies, as well as toxicity evaluations. Each program is unique therefore we advise taking a multidisciplinary approach and partnering with us to build a team of our toxicology experts, immunologists, discovery scientists, and regulatory advisors who will work closely with you and the regulatory authorities to ensure program success.

Laboratory Sciences
Charles River supports advance therapies through every stage of drug development with decades of experience in PK/PD, molecular biology, immunogenicity and bioanalytical assays across multiple platforms and state-of-the-art technologies. With comprehensive gene expression, immunochemistry, flow cytometry, chromatographic, molecular genomics, histopathology and chromatographic services to support nonclinical programs and clinical trials our dedicated team will help select the most relevant technology based on your specific program needs.

CMC Process Development and Scale-up
To develop a gene and cell therapy program that meets regulatory expectations the GMP release and characterization testing follows the basic tenants for biologics. The cell source, type, and degree of manipulation must be considered to establish well characterized cell product manufacturing practices and controls. Charles River’s characterization capabilities include cGMP cell banking, cell bank characterization and release testing, NGS and Sanger Sequencing, potency testing, identity, composition, mycoplasma, endotoxin, viability and stability as well as purity and sterility assessments. To properly identify the cells, a series of translation biomarkers must be developed, including morphology, surface, and genetic markers that reveal pharmacologic actions that are then used for lot release followed by viral clearance. Collectively we can provide end to end documentation that assures the safety and efficacy of your product.

Scientific Advisory Services
Advanced medicines require discovery and development strategies that go beyond traditional approaches, many times into uncharted territory. Cell and gene therapy, gene editing, CAR Ts, and second generation oligonucleotides, require scientists to rethink previous drug development models and consider a path from target validation to the clinic that is unique to each program. Our Scientific Advisory Services group is comprised of scientists with diverse backgrounds in nonclinical development and can assist with program design, study conduct, data interpretation, and regulatory filing to help drive your gene and cell therapy research.