Cell and Gene Therapy Services & Products

• Highly characterized, reliable donor pool for allogeneic cell therapy development, robust apheresis collection network, RUO, clinical grade, GMP-compliant cellular materials
• Isolated immune cells and custom antigen-specific T cells
• Engineered antibodies for cell therapy discovery
• Target engineering and model development with CRISPR and RNAi technology and expertise

• In vivo stem cell engraftment and biodistribution studies
• In vivo pharmacology models and assays
• In vivo imaging, nuclear imaging (PET, SPECT), MRI-guided gene delivery
• Research models supporting human cell engraftment/transfer
• Engineered antibody libraries for in vitro and in vivo CAR-T Testing
• Stem cell platforms
• Gene editing and screening ASOs, CRISPR/Cas9, RNAi, Bioinformatics services

• Non-clinical research to IND and laboratory support
• In vivo and analytical support
• Surgery, specialized administration and dosing routes
• DART
• Specialty models and endpoints
• Pathology
• In situ hybridization (ISH) and immunohistochemistry (IHC)
• Single combination non-GLP and GLP pharmacology and toxicology studies
• Method development, validation, and sample analysis (early screening through nonclinical)
• Clinical support

• Biosafety (in vivo/in vitro), potency, and impurity testing
• Electron microscopy and adeno-associated virus (AAV) capsid characterization
• Drug substance/product release testing
• Cell line characterization
• Viral clearance studies
• Stability studies
• Endotoxin detection instruments and services
• Rapid sterility testing
• Same day microbial identification services
• Method development, validation, clinical sample analysis

• Cell therapy CDMO services
• Plasmid DNA CDMO services
• Viral vector CDMO services
  • Molecular cloning services
• Viral vector packaging services for research discovery
• Plasmid and viral vector products
• Cell bank manufacturing
• Virus bank manufacturing

• Customized and expedited programs for rare and orphan disease indications
• GMP compliance
• Enabling robust program design and data interpretation
• Cell sourcing guidance to build donor pools

Cell therapy research is the process of developing and testing whole cells that can be delivered to a patient to treat or cure a disease. Bone marrow transplants are one of the earliest cell therapies developed where bone marrow cell from a healthy person are used to repopulate the bone marrow of patients with some cancers. Newer cell therapies are cells that may be harvested from altered to target a disease or may be cells that are produced in a laboratory. The cell therapy is infused or transplanted into the patient as a treatment for a disease.

Genes are responsible for creating the proteins that control cellular processes. In many diseases, genetic instructions to code for the protein are missing or defective. Gene therapy research is the process by which scientists develop and test a DNA or RNA molecule that could be delivered back to the patient to treat the disease. Scientists first identify the impacted gene and the nature of the defect in patient cells. Depending on the nature of the defect, a gene therapy can be developed that is a proper copy of the gene or suppresses the defective copy of the gene from being used by the cell. The gene “therapy” is then put inside of a vector or a nanoparticle that can target the therapy to the proper cells when it is administered to the patient. Once delivered to the patient, the gene therapy provides genetic instructions for that can be used by the cell to correct or minimize the impact of the defect.

Researchers work with contract research organizations, like Charles River, by using gene and cell therapy products and services to prove efficacy and safety through various study types prior to submitting to agency for approval for first-in-human clinical trials. During and after approval of human trials, researchers continue to work with contract research organizations for clinical trial support, extended nonclinical safety studies and manufacturing support. Additional steps and studies are taken following clinical trials to ensure safety in the manufacturing stages before market release.

Cell and gene therapy research is considered advanced therapeutic medical products or tissue products. As such, the development of these therapies is directed by a specific set of regulations and guidance that must be followed. Additionally, the development of these therapies is often not as straightforward as a chemical or biological drug material and require customized cell and gene therapy products and services to meet the milestones in the development of these therapies.

Cell and gene therapies have to potential to cure some diseases after a single dose. Cell therapies can replace cells that have either been lost or have a dysfunction that causes the disease. Gene therapies can provide proper copies of genetic material that is missing or damaged or can be developed to suppress the expression of mutated genes that cause the disease. By permanently correcting the loss of function or dysfunction that caused the disease, both cell and gene therapy research has the potential to be curative treatment for some diseases.

• You can review some regulatory guidelines for ensuring the safety of your cell and gene therapy products:
  • Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs)
  • Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus During Product Manufacture and Patient Follow-Up
  • USP <1071>, Rapid Microbial Tests for Release of Sterile Short-Life Products: A Risk-Based Approach
  • European Pharmacopoeia Chapter 2.6.27, Microbiological Examination of Cell-Based Preparations
  • EudraLex: Governing Medicinal Products in the European Union. Volume 4. GMP. Guidelines on Good Manufacturing Practice Specific to Advanced Therapy Medicinal Products