IND-enabling Studies of Somatic Genome Editing Therapeutic Leads (U19, Clinical Trial Not allowed) | RFA RM 22 015

Opportunity Information: Apply for RFA RM 22 015

The NIH opportunity titled "IND-enabling Studies of Somatic Genome Editing Therapeutic Leads (U19, Clinical Trial Not allowed)" (RFA-RM-22-015) is a cooperative agreement designed to move promising somatic genome editing therapies closer to the point where an Investigational New Drug (IND) application can be submitted to the U.S. Food and Drug Administration. It sits within the NIH Common Fund Somatic Cell Genome Editing (SCGE) Phase II Program, which is meant to accelerate the practical development of genome editing approaches that can be translated into real therapeutic candidates. The emphasis here is on team-based, milestone-oriented work that goes beyond early discovery and into the kind of rigorous optimization, characterization, and preclinical evidence generation that regulators expect before first-in-human testing can be proposed.

This FOA specifically supports U19 "multi-project" efforts, meaning applicants are expected to organize a coordinated set of projects under a single program that functions like an integrated development team rather than a collection of loosely related studies. Because the mechanism is a cooperative agreement, NIH staff are expected to have substantial involvement during the project period, typically through coordinated planning, oversight, and progress tracking. The overall intent is to de-risk genome editing therapeutic leads by pushing them through key IND-enabling steps, such as refining the editing system, improving delivery to the intended cell or tissue, building a stronger efficacy case in relevant models, and developing the types of data packages that would support regulatory interactions and eventual IND submission. Despite this translational focus, the FOA explicitly does not allow clinical trials under this award, so the funded work should remain preclinical and centered on development and IND-enabling studies rather than human testing.

A central expectation is that the proposed genome editing therapeutic leads already have a strong biological rationale and credible proof-of-concept data, generated in vitro and/or in vivo, in model systems relevant to the targeted cell or tissue type. In other words, the program is not looking for speculative concepts; it is looking for leads that have cleared an initial bar for plausibility and early performance, and now need the kind of systematic optimization and validation that turns a promising tool into a plausible therapeutic product. The FOA also highlights adaptability: the approach should be potentially generalizable within a given cell or tissue context, such that it could be adapted to address different pathogenic variants affecting the same target cell or tissue type. This reflects an interest in platform-like therapeutic strategies where delivery, editing modality, and tissue targeting can be reused and tailored across multiple variant targets within the same biological setting.

The awarding agency is the National Institutes of Health, and the activity category is health (CFDA 93.310). The instrument is a cooperative agreement, signaling a collaborative governance structure and an expectation of close coordination with the SCGE program goals. The original closing date listed for the opportunity was July 19, 2022, and the FOA was created on April 11, 2022. While the listing does not provide an award ceiling or expected number of awards in the supplied text, the U19 structure and IND-enabling scope generally imply relatively complex, resource-intensive programs that bring together multiple specialties, such as genome editing engineering, delivery and formulation, relevant disease biology, preclinical model development, and translational/regulatory planning.

Eligibility is broad across U.S.-based organizational types and includes state, county, city/township, and special district governments; independent school districts; public and state-controlled institutions of higher education; private institutions of higher education; federally recognized Native American tribal governments; tribal organizations other than federally recognized governments; public housing authorities/Indian housing authorities; 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 explicitly calls out additional eligible applicant categories such as Alaska Native and Native Hawaiian Serving Institutions, AANAPISISs, Hispanic-serving Institutions, Historically Black Colleges and Universities, Tribally Controlled Colleges and Universities, faith-based or community-based organizations, regional organizations, eligible federal agencies, and U.S. territories or possessions, reinforcing NIH's intent to include a diverse range of institutions capable of contributing to translational genome editing development.

On the foreign eligibility side, the rules are specific: non-domestic (non-U.S.) entities and non-domestic (non-U.S.) institutions are not eligible to apply as the primary applicant organization. However, non-domestic components of U.S. organizations may participate, and foreign components (as defined by the NIH Grants Policy Statement) are allowed. Practically, that means a U.S. institution can lead the application and include certain international activities or collaborators when justified, but a foreign institution cannot be the applicant of record.

Taken together, the opportunity is best understood as a translational bridge between early proof-of-concept genome editing research and the formal regulatory pathway that precedes human trials. The NIH is using this U19 to assemble coordinated, multidisciplinary teams that can do the hard development work needed to produce an IND-ready therapeutic lead in somatic genome editing, while keeping the funded scope firmly on preclinical, IND-enabling studies rather than clinical testing.

• The National Institutes of Health in the health sector is offering a public funding opportunity titled "IND-enabling Studies of Somatic Genome Editing Therapeutic Leads (U19, 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 2022-04-11.
• Applicants must submit their applications by 2022-07-19. (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.

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