Reducing Costs of Goods in Cell and Gene Therapy

This is the ninth part of a transcript of a Special Report with Deborah Phippard, PhD, and Renier Brentjens, MD, PhD. For the eighth 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. 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 transcript of the ninth episode, Phippard shared her thoughts with Brentjens on how the cost of goods may be reduced for cell and gene therapy products. She noted that the adoption of gene therapy approaches for major common conditions, like diabetes and obesity, may compel companies to find cost-effective ways to fund gene therapy technologies. She also touched on the need for more patient and general public education with regard to gene therapy and the need for more standardized protocols in the field. Overall, she emphasized that the field is "wide open" for possibilities.

CGTLive: Is there anything we didn't discuss yet that you want to bring attention to?

Deborah Phippard, PhD: Yeah, I suppose thoughts about reducing costs of goods. It's not necessarily my expertise, and probably not Renier's (I don't know how much you have to actually think about doing that). We touched on allogeneic. Any allogeneic therapy is going to reduce cost. As an immunologist, I find that very difficult to get my head around how you could make that work. There's just some very interesting developments in the field. Something that absolutely fascinates me, is I know there are companies working on gene therapy for GLP-1, which just really blows my mind to think about. We're currently injecting GLP-1 agonists. There are multiple companies working on how to up regulate your GLP-1 permanently in the context of diabetes, but you can imagine that moving into obesity.

This is going to sound—I don't know how this is going to sound—but I sort of feel like obesity and funding, in some ways, can be easier than these rare diseases. You know, some types of cancer, the market is so huge, pharma is so driven, that they have to think about those larger markets. I wonder, when you sort of asked us, where do we see the next 25 years?—I wonder if something will come out of left field because of the types of research that are sort of going on over there with obesity, and how they're thinking about modulating those pathways. Because if they work something out with gene therapy delivery for the masses, and we can pull that back in and adapt it—that, to me, is sort of an interesting way of thinking about what might make our field take a giant leap forward.

And there's still a lot of patient education to be done. I talk to my lay friends and misconceptions [come up] about what we mean by gene therapy and the concept of "oh, my babies will be affected"—No, we're not modulating the germ line. These types of things—there's still a lot of education that we need to do in this space. I think people that have life-threatening diseases are potentially more open to novel therapies than maybe the general public, but I just see so many different ways that we could come up with better standardized protocols. Renier made a very valid point that because there's no real standardized protocol, because there's still all these unknowns, different doctors are thinking about, "well, I've got this way of doing it. Maybe I'll try this way." I can just see that all these different things have to come together, that we're just at that huge spread open field with different ways of administering the drugs, different platforms, all these different ways of thinking about manufacturing your drug. It's wide open.

This transcript has been edited for clarity.