Limited Competition: Competing Revisions to Support Clinical Trials in Somatic Cell Genome Editing (U19 Clinical Trial Required)

The National Institutes of Health (NIH), through its Office of Strategic Coordination (Common Fund), has announced a limited competition Notice of Funding Opportunity (NOFO) titled "Competing Revisions to Support Clinical Trials in Somatic Cell Genome Editing" (U19 Clinical Trial Required). This NOFO is aimed at existing awardees of the NIH Somatic Cell Genome Editing (SCGE) Program under RFA-RM-22-015. Its purpose is to support the transition of therapeutic genome editing technologies developed in the U19 projects into first-in-human clinical trials. The program's ultimate goal is to accelerate the translation of these technologies into clinical applications for treating genetic diseases. The funding opportunity seeks to expand the scope of original SCGE U19 awards to include early-phase clinical trials. Supported activities include trial preparation, manufacturing of therapeutic materials, assay development, protocol preparation, participant recruitment, safety monitoring, and data analysis. Applications must demonstrate readiness for clinical trials, including pre-Investigational New Drug (IND) discussions with the FDA. Notably, the trials must focus on safety, tolerability, and pharmacokinetics without efficacy as the primary endpoint. NIH emphasizes incorporating health disparity populations into study designs and mandates the inclusion of a detailed community engagement plan. This NOFO will provide funds primarily through re-budgeting of existing awards but may allocate up to $2.6 million for successful applications, subject to availability. Up to two awards will be funded, with a project period of up to three years. Eligible applicants are limited to those currently funded under RFA-RM-22-015. Applications must adhere strictly to NIH guidelines, with conformance to submission instructions required to avoid delays or rejection. Review criteria for applications include the significance of the research, the investigators' expertise, the innovation and robustness of the proposed trial design, and the feasibility of milestones and timelines. The inclusion of diverse populations and engagement with stakeholders will also be critically evaluated. Applicants are encouraged to consult NIH SCGE staff during the application process to refine their project plans and milestone structures. Key deadlines include an open submission date starting January 14, 2025, and application due dates spanning May 29, 2025, to May 29, 2026, depending on the review cycle. No late submissions will be accepted. Awards are anticipated to commence between April 2026 and April 2027, following peer and advisory council reviews. For further assistance, applicants may contact designated NIH staff for scientific, peer review, and grants management inquiries. This NOFO represents an important step in translating genome editing technologies from preclinical research to clinical applications, with a strong emphasis on safety, regulatory compliance, and inclusivity.