Diverse Products: The video discusses a wide range of products regulated by the FDA’s Center for Biologics Evaluation and Research (CBER) in the field of oncology. This includes CAR T-cells, various viral and non-viral vectors, genome-edited products, and cancer vaccines [01:16].

Preclinical Testing: The core of the presentation focuses on the preclinical testing program required for an IND submission [02:26]. Dr. Wang references the FDA’s 2013 guidance on this topic and emphasizes the importance of these studies for safety and efficacy [06:29].

Choosing Animal Models: A significant portion of the discussion is dedicated to selecting appropriate animal models for these studies. The chosen models must be able to show a biological response that is relevant to how the therapy is expected to work in humans [10:43].

Safety Assessments: The video provides a thorough review of the safety assessments required for different types of therapies [13:53]. This includes potential toxicities such as “on-target” and “off-target” effects, cytokine release syndrome, and neurotoxicity for genetically modified cells [15:35]. For gene therapies, concerns like biodistribution, persistence of the therapy in the body, and immune responses are addressed [24:16]. For genome-editing technologies, the risks of unintended genetic changes are a key focus [20:30].

Communication with the FDA: Dr. Wang highlights the importance of early and frequent communication with the FDA through programs like “INTERACT” to discuss preclinical development plans before a formal IND submission [10:58, 31:44].

Relevant Guidance Documents: The presentation concludes with a list of other relevant FDA guidance documents that can help sponsors in their development process [35:50].