From The Editor | August 31, 2023

Regulating the CGT Workflow To Improve Patient Safety


By Erin Harris, Editor-In-Chief, Cell & Gene
Follow Me On Twitter @ErinHarris_1


In case you missed it, our most recent Cell & Gene Live, Regulating the CGT Workflow To Improve Patient Safety, featuring expert panelists Nimi Chhina, Ph.D., J.D. Executive Director, Head of Global R&D and Regulatory Policy Global Regulatory Affairs, Worldwide Research & Development at BioMarin Pharmaceutical, and Bob Pietrusko, Pharm.D., Chief Regulatory & Quality Officer at Vor Biopharma, is available on demand in its entirety.

As advancements in technology, science, and workflows continue to open up potential within the cell and gene therapy space, issues around scale, policy development, pre- and post-approval monitoring just to name a select few impact the entire landscape. That’s why we focused the 60-minute presentation on regulating the late-stage comparability of the CGT manufacturing process. But current workflows are time consuming and present challenges to auditing and quality control. And while global regulators demand tight control over the products, one of the common goals shared among researchers, clinicians, industry, and regulators is to improve the workflow to translate these therapies into safe and effective options to improve patients’ lives.

We began the hour by addressing CGT biotechs’ biggest hurdles around late-stage comparability and how these challenges affect patient safety. Regarding partnerships with CDMOs specifically, we talked through how the current financial climate and increasing CDMO capacity affects companies and their decisions regarding manufacturing.

Policy Development
The FDA has issued guidance to support development of cell and gene therapy products. There is guidance development based on commitments under PDUFA VII and CBER guidance agenda. Both Chhina and Pietrusko detailed how to best to approach and support policy development in the field. We also covered post-approval monitoring, as the FDA recently held a workshop on post-approval safety and efficacy data collection for cell and gene therapy products. As such, we discussed the key considerations for post-approval monitoring, safety and efficacy data collection, as well as the long-term follow-up for cell and gene therapy products. We covered class considerations for cell and gene therapy products that industry and regulators alike may collectively consider to drive policy development. Chhina and Pietrusko also shared their thoughts on the use of registries and other sources of real-world data collected in clinical settings to support cell and gene therapy products development and post-approval data collection.

The Patient Voice

The patient perspective plays a critical role in the CGT space, and patients who are included as active partners in research deliver immeasurable value. As such, we talked through the considerations for use of patient experience data and incorporation of patient voice in cell and gene therapy product development and regulatory review and decision-making.

This is just a summary of what we covered during the hour, but you must tune in to truly absorb the incredible insight Chhina and Pietrusko shared. Tune in to Regulating the CGT Workflow To Improve Patient Safety at your convenience and email me with questions or topics you’d like us to cover in more detail. We have another can’t-miss Cell & Gene Live coming up, The Future of Viral Vector Technologies, which will take place on September 26, 2023 at 11 a.m. EST. Our featured panelists include Candel Therapeutics’ Dave Maheu and REGENXBIO’s Curran Simpson. Registration is free. Hope to see you there.