Preclinical Pharmacology Services to Support Evaluation of Novel Cell & Gene Therapies
Speaker: Leila Kokabee, Ph.D,
Senior Scientist II & Study Director, Preclinical Pharmacology in Biocytogen Boston
Cell and gene therapies aim to treat diseases through the restoration or alteration of certain sets of cells, or by using cells to carry a therapy through the body. While cell therapies typically use engineered CAR-T or CAR-NK cells to target tumor cells for destruction, gene therapies aim to treat diseases by replacing, inactivating or introducing genes into the cell, either inside the body (in vivo) or outside of the body (ex vivo).
To investigate and predict safety, toxicity and efficacy of novel cell and gene therapies, preclinical studies need to be conducted, which involves in vivo evaluation as well as profiling changes in immune cell subtypes in response to the treatment, using tools like flow cytometry and other in vitro assays. Here, we provided an overview of the types of preclinical studies that can be performed and the models that support such assays, including immunodeficient models (B-NDG mice) and humanized models, to assess efficacy, safety, and reveal immune related mechanisms of such therapeutic interventions.
