The chief executive officer of ASGCT broke down the highlights of the organization’s final report for 2024.
“[One] thing that I see is how much road there is in front of us in cell and gene therapy. We're still at the point where every single approval feels like a big landmark—because it is. Most of these approvals are either a first-in-class type of treatment for a disorder that already has a treatment or they are expansions of treatments for diseases for which there are no current therapies. So we're seeing a lot of big landmarks every quarter over quarter with the new approvals.”
The field of cell and gene therapy is rapidly evolving, with new FDA approvals, the introduction of new modalities, the initiation of hundreds of new clinical trials and preclinical programs, the launch of startup biotech companies, the financial/investment climate, and other factors all continuously shaping the landscape. In order to help capture and analyze these shifts, the American Society of Gene & Cell Therapy (ASGCT) puts out its quarterly “Gene, Cell, & RNA Therapy Landscape Report”, which covers traditional gene therapies and genetically modified cell therapies like chimeric antigen receptor T-cell (CAR-T) therapies, in addition to unengineered cell therapies, gene-editing therapies, RNA therapies, and RNA vaccines.
Following the publication of the Landscape Report for 2024’s fourth quarter, CGTLive® sat down with David Barrett, JD, the chief executive officer of ASGCT, to discuss the key takeaways. He noted that major approvals included an autologous CAR T-cell therapy for acute lymphocytic leukemia, a hematopoietic stem cell therapy for certain immune disorders, and an RNA therapy for lipoprotein lipase deficiency. In addition, he pointed out that the sector saw a steady rate of growth, an uptick in preclinical activity, and billion-dollar acquisitions by Novartis and Roche. He also touched on how gene therapies are continuing to expand beyond rare cancers and other rare diseases into more common diseases, such as cardiovascular conditions. Barrett also emphasized the field’s broad impact and spoke about the landmark nature of many of its major happenings.