Platform Clinical Trials of Genome Editors in Multiple Diseases (UG3/UH3, Clinical Trial Required) | RFA RM 22 016

Opportunity Information: Apply for RFA RM 22 016

The National Institutes of Health (NIH) grant opportunity titled "Platform Clinical Trials of Genome Editors in Multiple Diseases (UG3/UH3, Clinical Trial Required)" (Funding Opportunity Number RFA-RM-22-016) supports the development and execution of a clinical trial that tests a genome editing approach across more than one disease area. The defining feature is a platform-style trial design: applicants must propose a single genome editor used in at least two different diseases, while keeping the route of administration and the delivery system the same across the diseases. In practical terms, the opportunity is aimed at accelerating clinical translation by leveraging a shared editing tool and delivery strategy, so that lessons learned, manufacturing, safety monitoring, and operational infrastructure can be reused rather than rebuilt separately for each condition.

This funding mechanism uses a cooperative agreement and falls under the health activity category (CFDA 93.310). A cooperative agreement typically means NIH will have substantial scientific and programmatic involvement during the project, which often includes collaboration on milestones, study conduct expectations, data and safety oversight planning, and other aspects common to complex, high-risk clinical research such as first-in-human or early-stage genome editing trials. The FOA explicitly requires a clinical trial, so the application needs to be built around an actual interventional study in humans rather than only preclinical development or observational work.

The scientific and operational scope is intentionally constrained to emphasize platform efficiency and comparability. The same genome editor must be used across the included diseases, and the method of getting that editor into the body (route of administration) and the technology used to carry it (delivery system) must also be identical. This requirement pushes applicants to think carefully about disease selection and trial architecture, since the chosen diseases must be compatible with the same delivery route and delivery vehicle, and the editor must plausibly address pathogenic mechanisms in each disease. The "novel genome editing clinical trial" language indicates that NIH is looking for proposals that move beyond incremental repackaging of existing trials, with a clear rationale for innovation, feasibility, and how the platform approach improves speed, safety learning, or generalizability across conditions.

Eligibility is broad and includes many types of domestic organizations. Eligible applicants include state, county, city or township governments; special district governments; independent school districts; public and state-controlled institutions of higher education; private 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 with and without 501(c)(3) status (other than institutions of higher education); for-profit organizations other than small businesses; and small businesses. The FOA also highlights additional eligible categories such as Alaska Native and Native Hawaiian Serving Institutions, Asian American Native American Pacific Islander Serving Institutions (AANAPISISs), Hispanic-serving Institutions, Historically Black Colleges and Universities (HBCUs), Tribally Controlled Colleges and Universities (TCCUs), eligible federal agencies, faith-based or community-based organizations, regional organizations, and U.S. territories or possessions. These inclusions signal NIH interest in widening participation and encouraging applications from diverse institutional settings, including organizations that may have strong community ties or unique patient access relevant to rare or underserved diseases.

Foreign eligibility is more limited and nuanced. Non-domestic (non-U.S.) entities and foreign institutions are not eligible to apply as the primary applicant organization. However, non-domestic components of U.S. organizations are eligible to apply, and foreign components, as defined in the NIH Grants Policy Statement, are allowed. In effect, a U.S.-based applicant can include certain foreign collaborations or sites/components when justified, but a foreign institution cannot serve as the main applicant.

Key administrative details included in the source data are that the opportunity was created on April 11, 2022, and the original closing date was November 10, 2022. The award ceiling and expected number of awards are not specified in the provided listing, which usually means applicants would need to consult the full FOA text for budget limits, project period details, and the planned award count. Overall, the opportunity is aimed at building a shared clinical testing framework for genome editing that can be efficiently extended across multiple diseases while maintaining consistency in the editor, delivery system, and administration route, thereby strengthening cross-disease learning and potentially speeding clinical development.

• The National Institutes of Health in the health sector is offering a public funding opportunity titled "Platform Clinical Trials of Genome Editors in Multiple Diseases (UG3/UH3, Clinical Trial Required)" 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 2022-04-11.
• Applicants must submit their applications by 2022-11-10. (Agency may still review applications by suitable applicants for the remaining/unused allocated funding in 2026.)
• 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 22 016

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