FDA Gets Its Arms Around Advanced Therapy Medicinal Products Early

Peter Marks, MD, PhD, director, Center for Biologics Evaluation and Research, FDA

It is common knowledge that messenger RNA (mRNA) advances saved lives and limited the extent of the COVID-19 pandemic. Far less recognized, even in the science and technology community, is the decade-long nurture and support governmental organizations provided the mRNA ecosystem and the remarkable range of therapeutic advances that mRNA (and mRNA coupled to other platforms) could solve for, going forward.

On Aug. 23, 2023, the latter was made explicit when, as part of the Biden administration moonshot initiative, the Advanced Research Projects Agency for Health (ARPA-H) announced the launch of “Curing the Uncurable via RNA-Encoded Immunogene Tuning” (CUREIT) project. This effort, led by a team at Emory University in Atlanta, Ga., with up to $24 million in new funding, has the goal of developing “… generalizable mRNA platforms that can be harnessed to train the immune system to more effectively fight cancer, … other applications could extend to autoimmune disorders, transplants, and infectious diseases” (1).

As mRNA and RNA approaches surge in investment dollars and drug candidates moving into clinical trials, the Alliance for mRNA Medicines (AMM), initiated its “DC Day” meetings with a discussion with Peter Marks, MD, PhD, director, Center for Biologics Evaluation and Research, FDA. Top of mind for Marks was the possibility that public and political resistance to advanced therapies could be an obstacle to therapeutic efficacy, saying “We don't want the benefits of mRNA technology wasted due to poor uptake. We need to help people understand how well-established this technology is, and how it is not altering the human genome.”

On the side of production expedience, economics, and workforce efficiencies, Marks avers that, “mRNA vaccines have already shown tremendous capabilities which demonstrate a strong value proposition,” compared to more traditional approaches.

In relation to why there was the need for a new advocacy group, such as AMM, Marks says that while recognizing “When a group such as [AMM] is formed, it floats all boats,” he particularly emphasizes, “it’s very helpful to have a single unified voice to streamline engagement of expert working groups, build consensus, and relay authoritative guidance, fundamental facts (and constraints), to both the general public and also the pertinent governmental agencies. I look forward to regular working discussions with AMM—and other participants—in this field.”

Excellent and exhaustive communication was a hallmark for vaccine strategy during the past pandemic. Taking hard-won lessons forward, groups such as AMM will doubtless play a central role in coordination and consolidation for future efforts. Government agencies such as FDA, the Biomedical Advanced Research and Development Authority, the Office of Science and Technology Policy, and the US Army Medical Research Institute of Infectious Diseases, deserve to take a bow for having helped establish the necessary pre-conditions to allow pharmaceutical sciences to move smoothly between discovery, development, and deployment stages. As for the next generation of life saving medicines, AMM and partners should provide that clear voice to this shared vision for innovative mRNA and RNA therapies.

Reference

1. The White House, As Part of President Biden’s Unity Agenda, Biden Cancer Moonshot Announces Launch of ARPA-H’s CUREIT Project Led by Emory University to Develop New Tools to Strengthen the Immune System and Save Lives. Press Release, Aug. 23, 2023.

About the author

Chris Spivey is the editorial director of BioPharm International®.