Navigating The Complexities Of Cell And Gene Therapies With Regulatory And CMC Expertise

The Cell and Gene Therapy (CGT) drug development lifecycle poses complexities for sponsors as standards and protocols differ from more traditional therapy areas. Chemistry, manufacturing, and controls (CMC) challenges are particularly poignant due to factors such as shorter shelf life, variability in starting and ancillary materials, manufacturing and logistical complexities, and cost.

This case study examines a biopharmaceutical company focused on developing novel autologous T cell therapies that had promising findings targeting a rare-disease tumor type. During IND submission, the FDA indicated that there was insufficient data within the drug master file. Review the submission process and CMC expertise which helped the biopharmaceutical company avoid a clinical hold and advance to phase 1 clinical trials.