ASGCT’s Q4 2024 Landscape Report Indicates Steady Rate of Growth
David Barrett, JD, the chief executive officer of ASGCT, broke down the highlights of the organization’s final report for 2024.
David Barrett, JD
Each quarter, the American Society of Gene & Cell Therapy (ASGCT) puts out its “Gene, Cell, & RNA Therapy Landscape Report”, which is intended to capture statistics related to the rapid evolution of the field. CGTLive® reached out to David Barrett, JD, the chief executive officer of ASGCT, following the publication of the report for quarter 4 (Q4) of 2024 to learn more about its highlights.
Barret pointed out the major FDA approvals that occurred in the field during the quarter and noted that the sector saw a steady rate of growth, an uptick in preclinical activity, and several billion-dollar acquisitions. He also discussed the broad reach of cell therapy and gene therapy and the diverse range of indications that are now being targeted with these modalities.
CGTLive: Can you give some background context about the Landscape Report and discuss what stand out as some of the major highlights from the Q4 Report?
David Barrett, JD: The Quarterly Landscape Report is a program that ASGCT runs with our partners at Citeline. What we do is we look at the developments in the cell and gene therapy and RNA therapy space, every quarter. We report on the number of new approvals, other catalysts in the space, and we also look at the funding space, particularly for new startup organizations and those that are typically coming out of academia and getting commercialized. That represents a big part of where ASGCT's membership is, and where it comes from, and what we're interested in.
We report this every quarter. For the last quarterly report, the fourth quarter of 2024, we had some pretty significant highlights that we were happy to put out in that quarterly report. The major highlights include the approval of Aucatzyl, which is an autologous CAR T-cell therapy for acute lymphocytic leukemia, the approval of Regencyte site, which is a hematopoietic stem cell therapy for certain types of immune disorders, and Tryngolza, which is an RNA therapy for lipoprotein lipase deficiency.
Over the quarter, we also saw the rate of growth across the entire field remain steady and actually uptick just a little bit in preclinical. We also saw the return of billion dollar deals to cell and gene therapy. We saw the acquisition of Kate Therapeutics by Novartis for about $1.1 billion and we saw the acquisition of Poseida Therapeutics by Roche for $1.5 billion—so a lot of big movement towards the end of 2024.
What can we learn about development trends and patient/therapy needs from the report?
We did see, as I mentioned, a slight increase in the total number of preclinical gene therapies over a somewhat slower third quarter. While there remains a focus on rare disease, and particularly rare cancers, gene therapies are also targeting other not so rare disorders. That includes ovarian cancers, head neck cancers, renal cancers. Also outside the cancer space, we're seeing additional development towards CAR-T therapies for lupus and also other therapies looking at very common diseases like cardiovascular disease. We are also seeing a trend of a return of early dealmaking and startup funding that is rising quarter over quarter, representing the fourth quarter in a row of increases in startup funding.
How would you summarize the big-picture implications that doctors and the broader healthcare community should take away from the report?
The big picture is 2 things. First, it's how broad the cell and gene therapy sector really is. It touches on almost every major category of disorder and almost every major biological system. Cell and gene therapies are being developed right now to treat such a broad variety of diseases that there's so much for other healthcare providers to learn from what's going on inside and what they can see within that Landscape Report. Then on the flip side of that, the other 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. But also there's everything that is behind those approvals. When we look deeper in that pipeline and we see hundreds of therapies in human clinical trial and more than 1,000 therapies that are in early stage trials, and then several thousand therapies that are under development right now—it's really exciting to see just how much work there is to do and work that's being done in the cell and gene therapy space. It's really rather exciting.
Is there anything not covered by the previous questions that you want to share with our audience?
We do this every quarter so we're very excited that we have new trends to announce this quarter, but looking back over the 3 previous quarters and even before that, it's a really great way to take a longitudinal view of just how far cell and gene therapy has come. So I would encourage anybody who reads the report to sort of go back in time and just watch the steady drum beat of approvals, growth, and development in cell and gene therapy. We're really happy that the quarterly report has gathered the audience that it has, and that people look to that report for news, and that they count on that report to stay connected to the field and understand what's going on more broadly. I'm particularly grateful to you and your readers that we get to tell you about the Landscape Report and have that resource available for the field. Thank you very much.
This transcript has been edited for clarity.
