Quarter Century Update: What Does the Field Need Most Right Now?

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 told Brentjens her thoughts on the progress and promise of cell and gene therapy, emphasizing how major clinical successes—like recent advances in Huntington disease and sickle cell disease—energize the field by showing what’s possible. She highlighted the potential of gene therapy to address rare diseases, noting that while each is uncommon, together they represent a significant global health burden. Phippard also looked ahead to the possibility of applying these technologies to more common conditions, such as cardiomyopathies, where gene therapy could offer true cures rather than symptom management. She envisions continued progress through precision medicine—understanding and targeting the genetic drivers of disease—but acknowledged that cost and complexity remain significant barriers.