The Field of Cell Therapy and Gene Therapy Continues to Grow and Diversify

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 this field of medicine. Following the publication of the most recent report, for quarter 3 (Q3) of 2024, CGTLive® reached out to David Barrett, JD, the chief executive officer of ASGCT, to learn more about its highlights.

Barret gave some background about the quarterly report and then went over the latest report's key findings. Notably, he touched on the approval of Adaptimmune Therapeutics’ cell therapy afamitresgene autoleucel (afami-cel, marketed as Tecelra) for the treatment of synovial sarcoma and data pointing to the continued steady growth of the field as a whole.

CGTLive: Can you give some background context about the Landscape Report and what it is?

David Barrett, JD: The landscape report is a report that we produce every quarter where we relay what has happened over the course of the past quarter in the field of cell and gene therapy. Some of the things that we track are the number of approvals—both in the United States and outside of the United States—we track the clinical pipeline, so we're looking at the clinical trials in cell and gene therapy. We're also looking at not just what we might think of as more traditional gene therapies—gene edition, gene replacement, gene editing, and genetically modified cell therapies—but we're also looking at RNA mediated therapies as well as vaccines, and we are also looking at nongenetically modified cell therapies and reporting on that entire pipeline.

In addition to the total number of trials and total number of clinical programs that that we're aware of through the research, we are also reporting on what some of those targets are. So we're looking at the the most common diseases that are being targeted by these therapies; we're looking at as much information as we have on what the therapeutic type or therapeutic modality is. So we report on gene therapies that are delivered via viral vector versus gene therapies that are in a CAR-T versus other therapeutic modalities like RNA or nongenetically modified cell therapies.

What stand out as some of the major highlights or key items from the Q3 Landscape Report?

One of the big key takeaways was the approval of to Tecelra, which was approved during the quarter. One of the other things that we noted was the consistent trend of an increasing proportion of trials that are targeting nononcology indications. What I what I mean to say there is the the split of newly-initiated trials that are focusing on cancer versus focusing on noncancer targets is shifting slightly towards noncancer targets, which we think shows a maturation in the space and it shows one of the things that we that we thought would happen as the field evolves—which had been focused on cancer in many spaces—to an expansion of indications that are targeted through gene therapy. Now they are still primarily all rare disease, but that shift, I think, is showing some of the exciting changes that are in the space. What we are seeing is an increase in the proportion of disease targets that are nononcology-related. We are not seeing a reduction in oncology-related drugs and trials, but an increase in nononcology-related trials and new programs.

The other thing that we've seen that we think is a really interesting and positive trend in cell and gene therapy is that while the volume remains somewhat flat, the startup financing has rebounded, so we're seeing that number increase. When we look over the course of the last several quarters, we see the total number of new programs that are being spun out and funded has stayed relatively stable. So in Q3 of 2023 there were 17 programs. In Q3 of 2024 there were 19 programs. While we saw a dip in Q4 of 2023, subsequent dips in Q1 of 2024 and Q2 of 2024, overall, the number of new programs that are being funded is finding some stability, and we are starting to see an increase in the amount of money that is being invested into launching those programs.

What can we learn about development trends and patient/therapy needs from the report?

One of the things that we are seeing is a continuation of trials that are moving through phase 1 into phase 2 and phase 3. So while the total number of new programs is increasing slightly, we're continuing to see that increase in clinical trials. So these trials are moving forward, mostly successfully. In Q3 of 2024, there were 35 phase 3 trials in cell and gene therapy, and 289 phase 2 trials, as well as 318 phase 1 trials. What we are seeing now is a continuation of the robust pipeline that we've been witnessing for the past several years. Despite some fluctuations in overall market performance, what remains is the steady drumbeat of cell therapies and gene therapies moving through the clinical pipeline. I think we will continue to see that trend for quite some time into the future. So for patients and caregivers, we are seeing the continuation of approvals in gene therapy, and we will likely see the continuation, hopefully, of successful trials and more approvals in the space.

This transcript has been edited for clarity.

REFERENCE
1. Gene, Cell, & RNA Therapy Landscape Report, Q3 2024 Quarterly Data Report. Presentation. ASGCT and Citeline. Accessed November 19, 2024. https://www.asgct.org/global/documents/asgct-citeline-q3-2024-report.aspx