FDA Issues Final Guidance for Designing Early-Stage Gene Therapy Toxicity Studies

Clinical trials to study early-phase toxicity of cellular and gene therapy products have distinctive features that may pose greater risk to subjects, the FDA said in a final guidance published June 11.

“Guidance for Industry: Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products” provides recommendations on early-stage clinical trials—the studies to assess the safety, tolerability, or feasibility of administering investigational products—on cellular and gene therapy (CGT) products. The FDA said early adverse experiences with these products—including multi-organ failure, death of a subject who received a gene therapy product and tumors that developed in the ...