Writing An IND Module 3 For Cell And Gene Therapy Products: Considerations And Challenges

By Lara Stevanato, PhD, Ergomed Associate Director, Regulatory Affairs

Preparing Module 3 of an Investigational New Drug (IND) application is one of the most complex steps in advancing cell and gene therapy (CGT) products into clinical development. As part of the electronic common technical document (eCTD), Module 3 contains the Chemistry, Manufacturing, and Control (CMC) information that demonstrates product safety, quality, purity, identity, and potency. For CGT products, this requires careful alignment with FDA and ICH regulations, including 21 CFR Parts 210, 211, 600, and 610, while addressing unique challenges such as raw material control, process reproducibility, comparability after manufacturing changes, and the development of validated potency assays.

The amount of CMC detail expected in an IND depends on the phase of development—early trials require sufficient data to ensure safety and suitability, while later phases demand robust product characterization, validated analytical methods, stability data, and well-defined specifications. Failure to provide adequate CMC content can result in a clinical hold, delaying trial initiation.

This whitepaper outlines the critical considerations for CGT IND Module 3, including banking systems, donor eligibility and screening, analytical procedure validation, cGMP compliance, and stability programs. Drawing on Ergomed’s regulatory expertise, it emphasizes structured CMC strategies that evolve alongside clinical progress, ensuring regulatory readiness, minimizing delays, and supporting the safe advancement of innovative therapies.