RFA-RM-24-001: IND-enabling Studies for Platform Clinical Trials of Genome Editing in Multiple Diseases (U01 Clinical Trial Not Allowed)
Closed
This grant is closed. Who won this program's grants before is below, and similar open grants are at the end of the page. It is in our archive of National Institutes of Health notices from 2023.
National Institutes of Health offered grants of up to $2.0 million to nonprofits, colleges and universities, state and local governments, tribes, small businesses and businesses. Applications closed on February 22, 2024.
From the official notice on Grants.gov, last changed November 1, 2023. Checked November 7, 2026.
- Award ceiling$2.0 million
- Cost sharingNot required
- Funding typeCooperative Agreement
About this NIH notice
- Activity codeU01
- Clinical trialsNot allowed
- Funded byNCATS
- Median yearly award$3.3 million
- Typical project length4 years
- U01, Research Project Cooperative Agreement. Like an R01, but NIH staff are substantially involved in the work. Other open U01 notices.
Projects funded under RFA-RM-24-001 were run by National Center for Advancing Translational Sciences (NCATS, 2 projects). Contact the program officer at the institute that fits your project before you apply; the notice lists them.
Application due dates, page limits and budget limits are set in the notice. Read RFA-RM-24-001 in the NIH Guide.
Key dates
| Posted | November 1, 2023 |
|---|---|
| Deadline | February 22, 2024 |
| Archived by Grants.gov | March 29, 2024 |
Who can apply
This opportunity was open to nonprofits, colleges and universities, state and local governments, tribes, small businesses and businesses.
All 15 applicant types the notice lists
- 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 (see text field entitled "Additional Information on Eligibility" for clarification)
Other Eligible Applicants include the following: Alaska Native and Native Hawaiian Serving Institutions; Asian American Native American Pacific Islander Serving Institutions (AANAPISISs); Eligible Agencies of the Federal Government; Faith-based or Community-based Organizations; Hispanic-serving Institutions; Historically Black Colleges and Universities (HBCUs); Indian/Native American Tribal Governments (Other than Federally Recognized); Non-domestic (non-U.S.) Entities (Foreign Organizations); Regional Organizations; Tribally Controlled Colleges and Universities (TCCUs) ; U.S. Territory or Possession; Non-domestic (non-U.S.) Entities (Foreign Organizations) are not eligible to apply. Non-domestic (non-U.S.) components of U.S. Organizations are not eligible to apply. Foreign components, as defined in the NIH Grants Policy Statement, are allowed.
Is this a realistic fit?
- Projects NIH funded under this notice had a median yearly award of $3.3 million; it allows up to $2.0 million per award.
- Most of the program's money (72% over five years) went to colleges and universities.
- No cost sharing or match is required.
What the grant funds
The purpose of this NOFO is to provide support for applications for IND-enabling studies for the development of a novel in vivo genome editing therapeutic platform (genome editor plus delivery system) for two or more disease indications, using the same genome editor, route of administration, and delivery system. Through this initiative, NIH aims to explore the extent to which the use of a therapeutic genome editing platform can streamline the regulatory path for multiple disease indications and to disseminate this regulatory information (and supporting documentation) to the scientific community.
Funded under RFA-RM-24-001 so far
NIH funded 2 projects under RFA-RM-24-001 in fiscal 2024 to 2026, at 2 institutions. The median yearly award was $3.3 million.
| Project | Institution | Fiscal year | Award |
|---|---|---|---|
| AAV-mediated editing to treat human autosomal dominant hearing loss DFNA41 and DFNA2 | Massachusetts Eye and Ear Infirmary | 2026 | $4.6 million |
| Personalized prime editing as a platform for hepatic inborn errors of metabolism | Children's Hosp of Philadelphia | 2026 | $3.9 million |
Who has won Trans-NIH Research Support grants before
From fiscal 2021 to 2025, Trans-NIH Research Support (93.310) awarded $5.9 billion across 4,433 grant awards, an average of $1.3 million per award.
| Recipient | State | Awards | Amount |
|---|---|---|---|
| Duke University | North Carolina | 66 | $219 million |
| The Leland Stanford Junior University | California | 104 | $176 million |
| University of Pittsburgh - of the Commonwealth System of Higher Education | Pennsylvania | 82 | $154 million |
| The Johns Hopkins University | Maryland | 95 | $152 million |
| Yale Univ | Connecticut | 85 | $149 million |
Applying now
This notice no longer takes applications. Agencies often post the same opportunity again under a new number, so check the program's notices on Grants.gov and the similar open grants below.
The notice on Grants.gov keeps its full text and attachments, which is useful for planning a resubmission.
Questions people ask
Is RFA-RM-24-001 still open?
No. It closed on February 22, 2024.
Who can apply for IND-enabling Studies for Platform Clinical Trials of Genome Editing in Multiple Diseases (U01 Clinical Trial Not Allowed)?
National Institutes of Health accepted applications from nonprofits, colleges and universities, state and local governments, tribes, small businesses and businesses. The notice adds further conditions, so read its eligibility section.
What is the deadline for IND-enabling Studies for Platform Clinical Trials of Genome Editing in Multiple Diseases (U01 Clinical Trial Not Allowed)?
Closed February 22, 2024. Applications are no longer accepted; the program's newer notices are on this page when there are any.
How much funding is available?
Awards of up to $2.0 million each.
Who has been funded under RFA-RM-24-001?
NIH RePORTER lists 2 projects funded under RFA-RM-24-001 in fiscal 2024 to 2026, at 2 institutions. The most were at Massachusetts Eye and Ear Infirmary, Children's Hosp of Philadelphia.
Who has won Trans-NIH Research Support grants before?
The largest recipients over five fiscal years were Duke University, The Leland Stanford Junior University, University of Pittsburgh - of the Commonwealth System of Higher Education. The program made 4,433 awards worth $5.9 billion in that time.
Similar open grants
- Single Source for the Continuation of the Caring for OutPatiEnts after Acute Kidney Injury (COPE-AKI) Scientific and Data Research Center (U01 Clinical Trial Required)
- Single Source for the Continuation of the Epidemiology of Diabetes Interventions and Complications (EDIC) Study Research Center (Collaborative U01 Clinical Trial Not Allowed)
- Amyotrophic Lateral Sclerosis (ALS) Intermediate Patient Population Expanded Access (U01 Clinical Trial Required)
- Single Source for the Continuation of the Physiology of the Weight Reduced State (POWERS) Study (U01- Clinical Trials Required)
- Canine Cancer Immunotherapy Network (K9CIN; U01 Clinical Trial Not Allowed)
- Opportunities for Collaborative Research with the NIH Intramural Research Program (U01 Clinical Trial Optional)