IND-enabling Studies for Platform Clinical Trials of Genome Editing in Multiple Diseases (U01 Clinical Trial Not Allowed) | RFA RM 24 001

Opportunity Information: Apply for RFA RM 24 001

This NIH funding opportunity (RFA-RM-24-001) supports IND-enabling studies for a new in vivo genome editing therapeutic platform that can be advanced toward multiple disease indications in a coordinated way. The core idea is that applicants should develop a single platform made up of both the genome editor and its delivery system, then apply that same editor, the same delivery vehicle, and the same route of administration to at least two distinct disease targets. By keeping those key elements constant across indications, NIH is testing whether a platform approach can reduce duplicated effort, clarify what evidence can be shared across programs, and generally streamline the regulatory path when moving from one indication to the next. A major emphasis is not only generating the data needed for an Investigational New Drug (IND) application, but also capturing and sharing the regulatory lessons and supporting documentation so other researchers can learn what worked and what regulators required.

The award mechanism is a U01 cooperative agreement, which usually means NIH staff will have substantial scientific or programmatic involvement compared with a standard research grant. The activity area is health (CFDA 93.310). The notice specifies "Clinical Trial Not Allowed," so the work is expected to stay in the preclinical/IND-enabling phase rather than enrolling human participants. In practice, this kind of project commonly includes activities like establishing or optimizing the genome editing construct and delivery formulation, demonstrating proof-of-concept editing in relevant models, performing biodistribution and persistence studies, characterizing on-target and off-target effects, evaluating immunogenicity risks where relevant, developing assays and release methods, and conducting toxicology and other safety studies aligned with regulatory expectations for IND submission. Because the focus is a platform, a strong application would typically justify why the platform is broadly applicable, explain how the two (or more) indications are selected, and spell out what data can be shared across indications versus what must be indication-specific.

NIH also frames this opportunity as an effort to explore and document the regulatory efficiencies of a platform model. That means applicants should be prepared to think beyond the science and into the documentation and quality systems that make IND packages credible: how manufacturing and controls information (CMC) will be generated, what standardized assays and comparability approaches will be used, how study designs map to FDA expectations, and how decisions will be recorded in a way that can be disseminated to the broader community. The opportunity explicitly mentions dissemination of regulatory information and supporting documentation, signaling that the program values producing reusable templates, rationales, and evidence packages that other platform developers can adapt when pursuing additional indications.

Eligibility is broad and includes many types of U.S.-based organizations: state, county, and city governments; special districts; independent school districts; public and private institutions of higher education; federally recognized tribal governments; tribal organizations that are not federally recognized; public housing authorities/Indian housing authorities; nonprofits (with or without 501(c)(3) status); for-profit organizations (other than small businesses); and small businesses. The notice also highlights several categories often called out in NIH eligibility language, including Alaska Native and Native Hawaiian Serving Institutions, AANAPISIs, Hispanic-serving Institutions, HBCUs, Tribally Controlled Colleges and Universities, eligible federal agencies, faith-based or community-based organizations, regional organizations, and U.S. territories or possessions. Foreign organizations are not eligible to apply, and non-U.S. components of U.S. organizations are not eligible to apply; however, foreign components, as defined in the NIH Grants Policy Statement, are allowed, which typically means certain discrete parts of the project may be carried out abroad if they meet NIH policy requirements and are well-justified.

Key administrative details provided include an original application due date of 2024-02-22 and an award ceiling of $2,000,000. The sponsor is the National Institutes of Health, and the instrument type is a cooperative agreement, reinforcing that NIH expects an active partnership in shaping milestones, deliverables, and program direction. Overall, the opportunity is designed for teams ready to bring an in vivo genome editing platform to the brink of first-in-human readiness across multiple diseases, while also generating clear, shareable regulatory roadmaps that can help turn genome editing from one-off programs into more efficient, repeatable development pipelines.

• The National Institutes of Health in the health sector is offering a public funding opportunity titled "IND-enabling Studies for Platform Clinical Trials of Genome Editing in Multiple Diseases (U01 Clinical Trial Not Allowed)" and is now available to receive applicants.
• Interested and eligible applicants and submit their applications by referencing the CFDA number(s): 93.310.
• This funding opportunity was created on 2023-11-01.
• Applicants must submit their applications by 2024-02-22. (Agency may still review applications by suitable applicants for the remaining/unused allocated funding in 2026.)
• Each selected applicant is eligible to receive up to $2,000,000.00 in funding.
• Eligible applicants include: State governments, County governments, City or township governments, Special district governments, Independent school districts, Public and State controlled institutions of higher education, Native American tribal governments (Federally recognized), Public housing authorities/Indian housing authorities, Native American tribal organizations (other than Federally recognized tribal governments), Nonprofits having a 501 (c) (3) status with the IRS, other than institutions of higher education, Nonprofits that do not have a 501 (c) (3) status with the IRS, other than institutions of higher education, Private institutions of higher education, For-profit organizations other than small businesses, Small businesses, Others.

Apply for RFA RM 24 001

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