Quarter Century Update: What Were the Most Significant Inflection Points or Breakthroughs in the Field?

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 first episode of the Special Report, Phippard and Brentjens give their respective thoughts on what have been the major inflection points and breakthroughs in cell and gene therapy during the past 25 years. Phippard highlighted key moments such as the pioneering treatment of Emily Whitehead with the first chimeric antigen receptor (CAR) T-cell therapy and recent advances in gene therapy for Huntington disease and sickle cell disease. Brentjens agreed, emphasizing how CAR T-cell therapy transformed once “pie-in-the-sky” ideas into real cures for leukemia. He adds that industry involvement—particularly partnerships like the one between Novartis and the University of Pennsylvania—was crucial in scaling these therapies from boutique lab work to a global field now supported by hundreds of companies.