Quarter Century Update: What Lessons Should Guide the Next Generation of Developers and Regulators?

The past 25 years, from 2000 to 2025, have been an unprecedented and rapid period of development of the field of cell and gene therapy. A number of critical breakthroughs have occurred in this time, and the technologies have gone from preclinical musings to realities of standard clinical practice, at least in a few key indications like hematologic malignancies and neuromuscular disease. To get a perspective on how far we've come, and how far we have yet to go, CGTLive® reached out to Deborah Phippard, PhD, the chief scientific officer of Precision for Medicine, and Renier Brentjens, MD, PhD, the chair of the department of medicine and the deputy director at Roswell Park Comprehensive Cancer Center, to hold a Special Report discussion on the topic entitled: "Quarter Century Update: What’s Holding up Progress in Development? Where Have We Seen the Most?"

In this episode, Phippard and Brentjens shared advice for young scientists entering the cell and gene therapy field, emphasizing perseverance, adaptability, and scientific rigor. Phippard encouraged researchers to “stay the course,” collaborate across disciplines, engage with regulators early, and recognize that success often depends on navigating complex, multifactorial challenges—from delivery methods to FDA requirements. Brentjens echoed her points, reflecting on how far the field has come during his time in it and urged new scientists to celebrate small victories while questioning established assumptions. He warned against treating current norms—like the perceived role of cell persistence or necessity of preconditioning chemotherapy—as immutable truths, reminding listeners that many past “rules” were later proved wrong. Both stressed the importance of curiosity, flexibility, and persistence in a young field still defining its own principles.