Quarter Century Update: Where are the Biggest Bottlenecks Today?

This is the second part of a Special Report with Deborah Phippard, PhD, and Renier Brentjens, MD, PhD. For the first part, click here.

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 focus on the major bottlenecks facing cell and gene therapies today—chiefly cost and patient access. Phippard notes that while CAR T-cell therapies show remarkable promise, many doctors still avoid recommending them because of complexity and limited availability, meaning few patients benefit despite strong clinical results. Brentjens agrees, emphasizing that high costs limit both access for patients and progress in developing next-generation treatments. He compared the situation to early flat-screen TVs—expensive at first but likely to become more affordable over time—and highlighted the funding gap between preclinical research and early-stage clinical trials as a major barrier to innovation.