Structuring Partnerships Among Academia, Biotech, and Pharma

This is the fourth part of a transcript of a Special Report with Deborah Phippard, PhD, and Renier Brentjens, MD, PhD. For the third 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 fourth episode, Phippard and Brentjens shared their thoughts on how the different entities that contribute to cell and gene therapy research and development can better collaborate. Brentjens noted that academia and industry have moved closer during his time working in the field, characterizing it as a positive development, but stated that the process is still ongoing. Phippard agreed and highlighted the increase in partnerships between biotech startups and large pharmaceutical companies. She concluded by emphasizing that the path forward for the field's future will not be easy, despite the promise it shows.

CGTLive: How can partnerships between academia, biotech, and pharma be better structured to help move therapies faster to patients?

Renier Brentjens, MD, PhD: That is a great question, and that question was probably anathema 25 years ago when I started working in the laboratory. Industry and academia were very separated. That has changed for the better, significantly, over my career as a scientist. But better is not the same as good and it's to a degree understandable. I think we're very close, which means that my suspicion is that certainly Big Pharma is not the place where something like a CAR T-cell would have come out of because it was a massively big risk and cost a lot of money to get to where it is.

That having been said, if you approach Big Pharma, certainly over the last 5 years, when there's been some cooling down of enthusiasm for these cell therapies, and say, "Look, the mice get better"—You know, if you want to license it, and to get it into into first-in-human, that bridge has not been built yet. So it's really up to the academic to find the resources and to find institutions or CROs that can actually run that phase 1 trial for them, and that has its own challenges. Fortunately, I've been very lucky to be at places where we can meet that challenge.

But once the technology is licensed, I've personally had a very good experience with industry, having been a scientific cofounder for Juno. We were able to treat, and cure in many cases, patients that we otherwise would not have been able to do so without that industry venture capitalist financing. So it's not a good guy/bad guy thing. If we could just meet a little closer in the middle, where some of these therapies could be done in patients, we might be further along in the technology. When, in fact, you look at what is now FDA-approved, the design of the CAR we and others published 20-plus years ago—the second generation 28 4-1BB CAR, that's what's currently FDA approved—and if you stack all the papers that have been published on all these new approaches and all these new designs, it becomes a pretty high stack of papers and manuscripts, but none of it's actually yet crossed over to FDA approval, and only a handful of them have crossed over to industry and phase 1 clinical trials. And I think that if that relationship between industry and academia could be brought just a little closer together, I think that we would be further ahead with this technology than we currently are.

Deborah Phippard, PhD: That's a good point about academia and industry. I see a little more about small pharmas, small biotechs, and then the big guys. Precision does a lot of work in the gene therapy space and a lot of the companies that were startups or biotechs now have either Big Pharma who own them or Big Pharma who are partnered with them. I think some of the early success in gene therapy—not saying we don't have a long way to go—I think it did increase the appetite for some of those pharmas to put some money behind it.

So I do see some progress, but 100% agree that this has not been swift. I mean, these were very, very disruptive technologies. And it does take a long time to change the paradigm. We were just talking the other day about how long monoclonal antibodies have been around. I mean, they were equally earth shattering, but they've been around 25-plus years now. But at the time, that was such a huge shock to how we thought about therapy. Now, they're sort of standard of care, basically. But I think we're in that same sort of cycle with cell and gene that there's all this potential, but pulling everybody together, the risk appetite—it's happening—I hope the next 25 years go a little faster than the last 25 years. We're on a reasonable path, but there's going to be challenges. People don't necessarily want to come out and say it, but you see deaths on these studies, not everybody gets better. I think that was a valid point that was just made. These are not foolproof. The path is not going to be strewn with rose petals. There's a lot that has to be worked out, but the promise is there, so we're all committed to making this happen. But it's not a simple, straightforward clinical development path by any means.

This transcript has been edited for clarity.