Cellular Therapy

The complexities of cellular therapy programs present scientific and technical challenges. They are highly customized and vary greatly based on product attributes. Now, because of tremendous promise to cure many types of diseases, there is a lot of pressure to move these programs through regulatory approval faster than ever. It is therefore crucial to find a single partner of choice who offers access to a deep scientific and regulatory network and integrates the various elements of your program to achieve your milestones and make it to market on time.

We can help you navigate challenges with your cellular therapy programs.
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Cells under a microscope on black background

With over 70 years of experience, Charles River has a unique and comprehensive portfolio to support the development and execution of cellular therapy programs from animal model selection to discovery and safety evaluations through to clinical and CMC testing support. Our facilities in the United States, Canada, Britain, Finland, Netherlands, and Germany form a global scientific network, allowing us to provide our clients with flexible, comprehensive solutions to maximize resources and optimize results.

Careful choice of a species is a must for cellular therapy programs, one that is both appropriate for proof of concept studies and pharmacologically sensitive that will serve as a progressive host for the specific therapeutic in development in consideration of the intended clinical route of administration. Multiple Charles River locations offer immunodeficient rodent models, large animal models, neonates, surgically altered models, and tumor/syngeneic models for both pilot and safety assessment studies for a range of therapeutic areas including but not limited to inflammation, bone disease, neuroscience research, and metabolic disease. Our surgeons have vast experience in surgical cell implantation methods which mimic clinical routes of administration (e.g., injection into the brain or spinal cord using stereotaxic devices or intraocular instillations).

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Critical elements of a preclinical program strategy for a cellular therapy product include a well-characterized product, clinically relevant and robust animal models that allow for characterization of exposure and effects expected in humans, and reliable and sensitive methods of cell detection. These tools are used in characterization, proof of concept, pilot and definitive biodistribution studies, as well as toxicity evaluations. While using available guidance and objectives from traditional toxicology programs in planning safety studies, 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 regulatory reviewers at the Center for Biologics Evaluation (CBER) and other regulatory authorities to ensure program success.

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Our scientific experts, including a former FDA reviewing pharmacologist at the Centers for Drug Evaluation and Biologics Evaluation (CDER & CBER), have a deep understanding of regulatory expectations to help design the best strategy to advance a cellular therapy program. Having this scientific support is particularly crucial for cellular therapy, as reviewers at CBER/FDA highly encourage pre-IND meetings from the Office of Cell, Gene, and Tissue Therapy at the start of each program to discuss current standards of practice in relation to the specific cellular therapy being developed. Not only can they assist with IND filing, but they can also help to identify clinical trial risk mitigation steps, establish clinical biomarkers to address translation gaps, and prepare for BLA filing.

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In order to develop a cellular therapy program that meets regulatory expectations, the cell source, type, and degree of manipulation must be considered to establish well characterized cell product manufacturing practices and controls that assure product quality and consistency, essential to safety studies. Charles River’s characterization capabilities include identity and composition, viability and stability as well as purity and sterility. To properly identify the cells, a series of translation biomarkers must be developed, including morphology, surface, and genetic markers–qualified and optimized for the cell target that reveal pharmacologic actions that are then used for lot release and serve as tools for evaluating in vivo safety study results.

In addition, all manufacturers of cellular therapy products are required to assess the ability of the manufacturing process to generate a product safe for human use. Therefore, a viral clearance study is performed to evaluate key steps of the manufacturing process to ensure that it is effective at removing or inactivating viruses. Our scientists have extensive experience in the design and performance of viral clearance studies and we take a customized approach in the selection of process steps and model viruses, scaling-down of purification processes, and subsequent design of study protocols to ensure a successful program is established and reported to meet timelines.

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