The Oncology Cell and Gene Therapy program focuses on clinical evaluations for, and helps to expedite development of, transformative cancer therapies based on cutting-edge technologies with curative potential. Examples include T-cells modified with chimeric antigen receptors (CAR-Ts) or with T-cell receptors with redirected specificity (TCR-Ts), and developed using technologies including gene-editing, e.g., clustered regularly interspaced short palindromic repeats (CRISPR) or transcription activator-like effector nucleases (TALEN); novel strategies in hematopoietic stem cell transplantation (HSCT); dendritic cells; adoptive T-cell therapies; tumor neoantigen-based personalized medicine (vaccine or cell therapy); natural killer cells; oncolytic bacteria and viruses; therapeutic cancer vaccines; therapies that modulate the microbiome; and combinations of these therapeutics with hematopoietic stem cell transplantation, checkpoint inhibitors, chemotherapies, radiation and other agents.

The program leverages the combined skills of FDA clinical oncology review staff and regulatory scientists from other disciplines to address unique challenges in the clinical trial design and development of such products. These may include potentially transformative products that are manufactured from the patient’s own cells (i.e., autologous products).  The clinical development of such autologous products has to consider the lag time required to manufacture the final product from the patient’s cells.  Other considerations in the development of cell and gene therapies include their unique pharmacodynamics and / or pharmacokinetics, development of biomarkers to determine which patients are eligible for any specific clinical trial, development of biomarkers to monitor the response to the therapy, the distinctive safety and activity profiles of live microorganisms and cell products that are capable of renewing and proliferating, and long-term follow-up of patients receiving genetically modified products.

As one of the programs within the Oncology Center of Excellence (OCE), the Oncology Cell and Gene Therapy program emphasizes emerging oncology science to achieve excellence in medical product regulation.  Using this scientific base, the Oncology Cell and Gene Therapy program collaborates with academia, industry, patient advocacy groups, professional societies, and other international regulators to advance the OCE mission to accelerate the development and streamline the evaluation process of safe and effective products for patients.