Meet the California Institute Pushing Stem Cell & Gene Therapy Research: Part 3

Welcome back to our interview series with Jon Thomas, President and CEO of the California Institute for Regenerative Medicine (CIRM). CIRM has been funding stem cell and gene therapy research for 20 years. In part 1, we looked at the story behind CIRM. Part 2 focused on the wider regenerative medicine field. Now, we bring the conversation back to the institute, with a specific focus on funding.

What does CIRM look for when funding projects?
 

We have regular funding rounds. We accept applications for clinical trials monthly, and for translational and basic research two to three times a year. When we receive grant applications, we rely on peer reviewers from a pool of around 200 to 300 experts in stem cell and gene therapy. These reviewers evaluate the applications primarily on two components. First and foremost is the science itself, which includes looking at the researchers or, if it’s a company, assessing the management team and their track record. The peer reviewers score these applications, and a select few are recommended to the CIRM board for funding. The board then makes the final decision on which projects to fund.

The second component, which is unique to CIRM, is our focus on diversity, equity, and inclusion (DEI). We require applicants to address DEI, whether it’s the diversity of their research team or strategies for including underserved communities in clinical trials. This aspect is also rated in the peer review process, and several of our patient advocate board members participate in the review to specifically evaluate the DEI component of each application.

For anyone interested in applying, the best advice I can give is to reach out to CIRM. We have science officers who manage all our grants and work closely with applicants to guide them through the process. Unlike many funding entities, we’re highly participatory. We want to do everything we can to help applicants succeed. There’s a lot of give-and-take before submission to ensure the application has the best chance of success. Although our science officers aren’t involved in the peer review itself, they know what a successful application looks like and can provide valuable guidance.

Additionally, for clinical trials and increasingly for translational projects, we offer advisory panels that meet regularly with funded PIs. These panels include external experts and internal CIRM staff, who review the project's progress and offer recommendations to improve the likelihood of success. We also perform milestone analyses to ensure PIs are on track to move to the next stage of their approved funding.

One other thing I need to stress, which applies to any applicant, is the California Nexus. Applicants don’t need to be based in California, but there must be a component of the project taking place in the state. For instance, if you’re a company based in Boston and you’re running a principal clinical trial site at UCLA, the California-associated part of your project is eligible for funding, even though headquarters costs outside of California aren’t covered. We encourage companies to consider how they can establish some California presence to meet this eligibility requirement!

At the end of the day, we’re focused on finding the best science and the best projects with the potential to make a real impact, and we encourage all applicants to explore how they can qualify under our criteria.

How do you encourage collaboration between academia and industry?
 

One of our big focuses is encouraging academic researchers to collaborate with industry partners. Ultimately, many of these projects require substantial funding to get through clinical trials, let alone commercialization. If we can highlight promising academic research, it might attract interest from big pharma, which would be a win-win.

In the early days of the field, there was limited industry involvement, so the bulk of our funding went to academic institutions. Back then, maybe 90 percent of applicants were academic, with only a few companies in the mix. As the field has grown, more biotech and startup companies have emerged. Now, our funding is closer to a 50-50 split between academia and industry. This trend will likely continue as the field becomes more advanced and companies take on a greater role.

Most of our industry funding goes to smaller biotech firms. Big pharma has deep pockets and isn’t our typical applicant. Many larger pharmaceutical companies are still on the sidelines, watching to see how the field progresses. They often look to funders like CIRM to de-risk projects by supporting them through phase I and II. Big pharma is more inclined to get involved later, although some are beginning to invest a bit earlier.

We see our role as providing critical early funding that makes these projects more appealing to larger players as they mature.

What lies ahead for CIRM?
 

CIRM originally started with $3 billion in funding in 2004. Another $5.5 billion was added in 2020. Currently, we have $3.8 billion left to deploy. Although that sounds substantial, it goes quickly so we’ve been deciding how to prioritize how we’ll allocate the remaining funds. Depending on a number of variables, that money could last another 8 to 10 years.

To guide that deployment, CIRM underwent a data-driven months long reprioritization earlier this year. That effort led to a “Strategic Allocation Framework” which identified six goals going forward and a number of recommendations per goal. These goals and recommendations have given rise to 13 new or amended concept plans for a variety of programs. The CIRM team is busy developing these concept plans, which will be rolled out in the coming months.

One of the great things about CIRM as a funding agency is that we’re incredibly nimble and able to adapt quickly to evolving technologies. Let me share an example. In March 2020, when the first news of the pandemic was emerging, I called our president and said, “We don’t have much funding left, but there’s a global push to address this crisis, and we need to be part of that effort.”

Less than a week later, we had prepared a request for applications specifically for COVID-19-related research. Over the next four months, we held 10 board and subcommittee meetings and seven peer review sessions. By the end, we awarded 17 grants for COVID-19 projects in relatively record time.

As we look to the future, we know that technology will continue to evolve. We’re ready to adapt and incorporate these changes into our funding strategy. It’s part of what makes CIRM such a unique operation. We have an exceptional internal team, a highly supportive board, and strong relationships with patient advocates, advocacy groups, disease foundations, and all other stakeholders in the field. Everything we do centers around the patient, and we make sure their voices are heard. Patient advocates were instrumental in helping us get measures passed and spreading the word about CIRM, and hold 12 of CIRM’s 35 board seats.

The entire ecosystem we’ve built is thriving, and there’s a lot of work ahead, but we’re optimistic that, when the history of this remarkable era in biomedical research is written, CIRM will get a prominent chapter. I’m proud of what we have accomplished so far and there’s still more to come.