Chris Spivey is director of Industry Relations and Strategic Partnership, MJH Life Sciences.

Chris Spivey

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.

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.

Chris Spivey is the editorial director of 

Articles

Government provides a spoonful of sugar, and genuine leadership, for good medicines.

FDA Gets Its Arms Around Advanced Therapy Medicinal Products Early

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If the Alliance for mRNA Medicines (AMM) tested the waters in June 2023 at the BIO Conference in Boston, then they engaged in a full-fledged triathlon in September, in the heat and humidity of Washington, DC. It is refreshing to relate that government has continued its early support of this technology (a relationship between the US Army Medical Research Institute of Infectious Diseases, Novartis, and the Biomedical Advanced Research and Development Authority). A documentary feature explaining this background debuts this month.

The potential for mRNA medicines extends beyond vaccines into myriad other indications, but also represents efficiencies in pharmaceutical manufacturing, workforce education strengthening, and for high paying jobs. With the possibility of viewing its somewhat straightforward manufacturing as a single “platform” for regulatory approvals, this then could have the salutary effect of freeing up FDA staff, through this a potential new form of consolidated consanguinity management and approval. Time will tell.

AMM’s diverse membership was represented in Washington, DC by organizations such as Acuitas Therapeutics, Adjuvant Partners, Akron Bio, Arcturus Therapeutics, BioNTech, CSL Behring, Danaher Corp, The Johns Hopkins University, Leavitt Partners, Maravai Life Sciences, MJH Life Sciences, Precision Medicine Group, Replicate Bioscience, Precision Medicine Group, and Texas A&M Engineering. Their representatives enjoyed a free-flowing back and forth dialogue on optimizing the opportunity RNA presents the world, with Peter Marks, director of FDA’s Center for Biologics Evaluation and Research; leadership from Cancer Moonshot and White House Office of Science and Technology Policy; The Center for Medicare and Medicaid Innovation; the Office of Senator Gary Peters (MI) Chair, Senate Homeland Security and Governmental Affairs; the office of Bill Cassidy Ranking Member of the Senate HELP Committee; the office of Brett Guthrie, Chair of the House Energy and Commerce Committee, Health Subcommittee; the office of Senator Casey, Chairman of the Senate HELP Subcommittee on Children and Families; among other governmental groups.

It’s surprising that AMM, a group scheduled to officially launch in Berlin in October, was granted access to such a range of influential decision makers. Further, AMM was requested to not only assist in convening expert panelists but also offer advice for ongoing health systems and organizations re-shaping. A testament to the talent and knowledge of the AMM members, who transferred a strong sense of competence, knowledge, and trust.

When referring to this article, please cite it as Spivey, C. Washington, DC Houses mRNA Leadership. 

If the Alliance for mRNA Medicines (AMM) tested the waters in June 2023 at the BIO Conference in Boston, then they engaged in a full-fledged triathlon in September, in the heat and humidity of Washington, DC.

Washington, DC Houses mRNA Leadership

In this excerpt from a recent episode of the 

, Chris Spivey, editorial director, hosts a panel on advances in mRNA. In this clip, Chris, Kate Broderick, and Tom Madden discuss how extensive research is being conducted around various gene editing techniques and what that means for the future of mRNA. Other topics include epigenetic approaches to modulate gene expression, the expression of monoclonal antibodies, and protein replacement treatments.

For the complete discussion, check out the full podcast episode 

, chief innovation officer at Maravai LifeSciences has extensive experience and expertise in leading and liaising with multi-disciplinary groups from discovery and R&D to engineering and clinical teams. She brings strong and broad scientific expertise to TriLink Biotechnologies, which covers multiple areas, including gene delivery, medical devices, gene therapies for the treatment of various infectious diseases, cancer immunotherapies and vaccine development. Additionally, she has extensive experience with non-viral delivery systems for a wide range of vaccine targets and cancer immunotherapies.

, president & CEO of Acuitas Therapeutics, is a world-renowned expert in the area of nanotechnology. Dr. Madden co-founded Acuitas Therapeutics in February 2009 and has guided the company into its position as a global leader through the development and application of lipid nanoparticle (LNP) technology. Acuitas Therapeutics partners with leading pharmaceutical and biotechnology companies and prestigious academic institutions around the world, providing its proprietary LNP delivery technology to enable new drugs based on nucleic acid therapeutics.

 presents the Drug Solutions podcast, where the editors will chat with industry experts from across the pharmaceutical and biopharmaceutical supply chain. Join us as experts share insights into your biggest questions—from the technologies, to strategies, to regulations related to the development and manufacture of drug products.

Videos

Extensive research on various gene editing techniques could inform the future of mRNA therapies.

Nascent Science is Likely the Future for mRNA

Chris Spivey, editorial director, hosts a wide ranging discussion on the COVID-19 pandemic response, future government communications, and healthcare funding for mRNA with experts Dr. Kate Broderick, Chief Innovation Officer, Maravai LifeSciences and Dr. Tom Madden

President & CEO at Acuitas Therapeutics. 

Highlights include supply chain modalities, ensuring a fully trained manufacturing workforce, combining delivery with gene editing innovations, epigenetic approaches to modulate gene expression moving toward the clinic. We conclude with the huge potential for RNA expression of monoclonal antibodies and protein replacement applications.

Podcasts

An mRNA conversation about the recent past, current hot topics, and probable future.

Drug Solutions Podcast: mRNA Past, Present, and Future

In this excerpt from the most recent episode of the 

, Chris Spivey, editorial director, hosts a panel on advances in mRNA. There is promise of personalized cancer vaccines through the inherent nimbleness of mRNA platform. From a manufacturers point of view, how can we best to automate, miniaturize and speed up performance?

Experts discuss the promise of personalized cancer vaccines through the inherent nimbleness of mRNA platform.

Miniaturing Personalized Cancer Vaccines Through mRNA

Highlights from a wide ranging discussion on COVID-19 pandemic response, future government communications, and healthcare funding for mRNA with experts Dr. Kate Broderick, Chief Innovation Officer, Maravai LifeSciences, and Dr. Tom Madden, President & CEO, Acuitas Therapeutics.

Topics include supply chain modalities, ensuring a fully trained manufacturing workforce, combining delivery with gene editing innovations, epigenetic approaches to modulate gene expression moving toward the clinic. We conclude with the huge potential for RNA expression of monoclonal antibodies and protein replacement applications.

Catch the full podcast episode featuring Broderick and Madden when it goes live at the 

Dr. Kate Broderick is Chief Innovation Officer at at TriLink Biotechnologies, a Maravai LifeSciences company. Prior to joining TriLink, Kate spent 15 years with Inovio Pharmaceuticals where she assumed positions of increasing responsibility, starting as Scientist, to her most recent position as Senior Vice President, R&D. She received her B.Sc. in Genetics and her Ph.D. in Molecular Genetics from the University of Glasgow.

Dr. Tom Madden is President & CEO at Acuitas Therapeutics. A world-renowned expert in the area of nanotechnology. Dr. Madden co-founded Acuitas Therapeutics in February 2009 and has guided the company into its position as a global leader through the development and application of lipid nanoparticle (LNP) technology. Dr. Madden obtained his BSc. and Ph.D. in Biochemistry from the University of London, U.K.

Highlights from a wide ranging discussion on COVID-19 pandemic response from Kate Broderick, Chief Innovation Officer at Maravai LifeSciences, and Tom Madden, President & CEO at Acuitas Therapeutics.