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 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.

Link:
Cell and Gene Therapy Services | Charles River

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