Washington, D.C., November 13, 2025 — The Alliance for mRNA Medicines (AMM) released the following statement after senior leaders with the U.S. Food and Drug Administration (FDA) announced a new regulatory pathway—the Plausible Mechanism Pathway—for personalized gene editing therapies:
“The announcement by senior leaders at the FDA of a new regulatory pathway designed to accelerate the development and approval of bespoke gene therapies is a potential game-changer for patients and the research community. It recognizes that the future of medicine will be more personalized and precisely tailored to each patient’s specific disease and needs.
“In the announcement, FDA Commissioner Marty Makary and Dr. Vinay Prasad cite the case of Baby KJ, who made history as the first patient to receive a personalized gene-editing therapy for a rare metabolic disease.
“Baby KJ was treated with an mRNA-encoded base editor targeting the specific mutation responsible for his condition — a remarkable example of precision medicine in action. We’re proud that AMM members Danaher, Aldevron, Integrated DNA Technologies, and Acuitas Therapeutics were all part of Baby KJ’s team and helped advance this historic innovation.
“This breakthrough underscores how mRNA technology is transforming the future of medicine. It’s also a call to action: the federal government must continue to invest in mRNA research – a critical step toward realizing the full potential of these life-changing therapies for other countless patients like Baby KJ. As Commissioner Makary and Dr. Prasad write: ‘For patients and families, there is no time to wait.’”
Alliance for mRNA Medicines (AMM)
The Alliance for mRNA Medicines (AMM) is the leading global organization dedicated to advancing and advocating for mRNA and next generation encoding RNA therapeutics and vaccines for the benefit of patients, public health, and society. Our mission is to propel the future of mRNA medicine, improve patients’ lives, and advance scientific knowledge by convening and empowering mRNA industry leaders, innovators, scientists, and other key stakeholders. Learn more at https://mrnamedicines.org
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