Quarter Century Update: Platform Approaches and Industry Investment

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 discussed current trends and challenges in advancing cell and gene therapies. Phippard noted that research focus tends to be cyclical—shifting between cell types and now emphasizing platform approaches that can streamline development and approval by reusing proven delivery mechanisms. She points out that such strategies could make therapies for rare diseases more viable by grouping similar conditions under a shared platform. Brentjens agreed, but highlighted the difficulties of securing industry investment, as companies prefer derisked projects that are already proven in academia. He also stressed that while many new approaches—like allogeneic or in vivo gene transfer—are exciting, key technical and immunologic issues remain. For him, perfecting the core “engine” of autologous T-cell therapy to effectively treat solid tumors should remain the main priority before branching out to broader innovations.