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

Frequently Asked Questions (FAQs)

What is the purpose of this NIH funding opportunity?

This opportunity supports IND-enabling, preclinical development work for somatic genome editing therapeutic leads. The goal is to move promising therapies closer to the point where an Investigational New Drug (IND) application could be submitted to the U.S. Food and Drug Administration (FDA), by funding the rigorous optimization, characterization, and evidence generation typically expected before first-in-human studies are proposed.

What is the official title and FOA number?

The opportunity is titled "IND-enabling Studies of Somatic Genome Editing Therapeutic Leads (U19, Clinical Trial Not allowed)" and the FOA number is RFA-RM-22-015.

Which NIH program is this associated with?

This FOA sits within the NIH Common Fund Somatic Cell Genome Editing (SCGE) Phase II Program, which aims to accelerate practical development of genome editing approaches that can be translated into therapeutic candidates.

What funding mechanism is used?

The mechanism is a U19 cooperative agreement. U19 indicates a multi-project structure, and the cooperative agreement structure indicates substantial NIH staff involvement during the project period (for example, coordinated planning, oversight, and progress tracking aligned with SCGE program goals).

What does "cooperative agreement" mean for how the project is run?

Because this is a cooperative agreement, NIH staff are expected to be substantially involved during the award. The funded program is expected to operate with coordinated planning and active progress monitoring rather than functioning as an entirely investigator-directed grant with minimal agency involvement.

Are clinical trials allowed under this award?

No. The FOA explicitly does not allow clinical trials. Funded work should remain preclinical and focused on development and IND-enabling studies rather than human testing.

What stage of research is this FOA aiming to support?

This FOA is intended for projects that have moved beyond early discovery and have credible proof-of-concept data already. It is positioned as a translational bridge between early proof-of-concept genome editing research and the formal regulatory pathway that precedes human trials.

What kind of starting evidence is expected for a therapeutic lead?

A central expectation is that 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.

Is this FOA meant for speculative or very early ideas?

No. The FOA is not seeking speculative concepts. It emphasizes leads that have already cleared an initial plausibility and early performance bar and now require systematic optimization and validation to become plausible therapeutic products.

What types of activities are considered "IND-enabling" in this FOA?

The FOA is centered on de-risking somatic 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, strengthening the efficacy case in relevant models, and developing data packages that would support regulatory interactions and an eventual IND submission.

What does "somatic genome editing" imply in the context of this opportunity?

The opportunity is focused on genome editing approaches intended for somatic (non-germline) cells, with an emphasis on translating these approaches toward therapeutic candidates through preclinical development and IND-enabling work.

What does the U19 "multi-project" structure require from applicants?

Applicants are expected to propose a coordinated set of projects under one integrated program. The U19 structure is described as functioning like an integrated development team rather than a collection of loosely related studies.

Why does the FOA emphasize team-based, milestone-oriented work?

The FOA is designed to support coordinated development efforts that can be planned, tracked, and assessed against defined progress expectations. This aligns with the translational goal of generating the kinds of optimized systems and preclinical evidence packages regulators expect prior to IND submission.

Does the FOA encourage platform or adaptable approaches?

Yes. The FOA highlights adaptability and notes that approaches should be potentially generalizable within a given cell or tissue context, so they can be adapted to address different pathogenic variants affecting the same target cell or tissue type.

What does "generalizable within a given cell or tissue context" mean here?

It reflects interest in strategies where core elements (such as delivery, editing modality, and tissue targeting) can potentially be reused and tailored across multiple variant targets, as long as the target cell or tissue type is the same.

Who is the awarding agency?

The awarding agency is the National Institutes of Health (NIH).

What is the activity category and CFDA listing provided?

The activity category is health, and the CFDA number provided is 93.310.

When was this FOA created and what was the listed closing date?

The FOA was created on April 11, 2022, and the original closing date listed was July 19, 2022.

Is the award size, award ceiling, or expected number of awards provided?

No. The supplied information does not include an award ceiling or the expected number of awards.

What kinds of organizations are eligible to apply?

Eligibility is broad across U.S.-based organizational types, including 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.

Are any additional applicant categories explicitly called out as eligible?

Yes. The FOA explicitly calls out 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.

Can a non-U.S. (foreign) institution apply as the primary applicant?

No. Non-domestic (non-U.S.) entities and non-domestic institutions are not eligible to apply as the primary applicant organization.

Can international collaborators or activities be included at all?

Yes. The FOA allows non-domestic components of U.S. organizations to participate, and foreign components (as defined by the NIH Grants Policy Statement) are allowed. In practice, a U.S. institution can lead the application and include certain justified international activities or collaborators, but a foreign institution cannot be the applicant of record.

How should applicants think about the overall intent of this opportunity?

The opportunity is best understood as supporting a translational bridge: it funds coordinated, multidisciplinary teams to do the development work needed to produce an IND-ready somatic genome editing therapeutic lead, while keeping the scope firmly preclinical and IND-enabling rather than clinical.