Notice of Intent to Publish a Funding Opportunity Announcement for Platform Clinical Trials of Somatic Genome Editing for Multiple Diseases (UG3/UH3, Clinical Trial Required)
Notice Number:
NOT-RM-22-009

Key Dates

Release Date:
February 28, 2022
Estimated Publication Date of Funding Opportunity Announcement:
April 30, 2022
First Estimated Application Due Date:
July 19, 2022
Earliest Estimated Award Date:
April 01, 2023
Earliest Estimated Start Date:
April 01, 2023
Related Announcements

NOT-RM-22-006 - Notice of Intent to Publish a Funding Opportunity Announcement for Somatic Cell Genome Editing Translational Coordination and Dissemination Center (U24, Clinical Trial Not Allowed)
NOT-RM-22-007 - Notice of Intent to Publish a Funding Opportunity Announcement for Technologies and Assays for Therapeutic Genome Editing INDs (U01, Clinical Trial Not Allowed)
NOT-RM-22-008 - Notice of Intent to Publish a Funding Opportunity Announcement for IND-enabling Studies of Somatic Genome Editing Therapeutic Leads (U19, Clinical Trial Not allowed)

Issued by

Office of Strategic Coordination (Common Fund)

Purpose

The Somatic Cell Genome Editing (SCGE) program from the NIH Common Fund intends to issue Funding Opportunity Announcements (FOAs) for the second phase (FY2023-2027) of the program. In Phase 2 the SCGE program will accelerate the development of genome editing therapeutic agents by: facilitating Investigational New Drug (IND) application enabling studies; establishing pathways to regulatory approval; and disseminating successful strategies for initiating first in human clinical trials.

SCGE Phase 2 will include four FOAs: 1) an initiative for the development of technology/assays that support IND-submissions, 2) another for optimizing genome editing-based therapeutic leads for safety and efficacy, 3) a platform clinical trials initiative using genome editing therapeutics for more than one disease, 4) and a Translational Coordination and Dissemination Center (TCDC). Together these initiatives will comprise a consortium that will accelerate the optimization of promising clinical candidates toward IND filings and first in human clinical trials.

This Notice is being provided to allow potential applicants to the FOA for platform clinical trials initiative using genome editing therapeutics for more than one disease sufficient time to develop meaningful collaborations and responsive projects.

The FOA is expected to be published in Spring 2022 with expected application due dates in Summer 2022.

Details are provided below.

Research Initiative Details

Background

The NIH Somatic Cell Genome Editing (SCGE) program is funded through the NIH Common Fund, which supports cross-cutting programs that are expected to have exceptionally high impact. All Common Fund initiatives invite investigators to develop bold and innovative approaches to address problems that may seem intractable or to seize new opportunities that offer the potential for transformation of research processes.

The simplicity and broad applicability of targeted and programmable genome editing approaches, including but not limited to those based on CRISPR-Cas9, raise the possibility of a fundamentally new way to treat a variety of genetic diseases. However, many challenges need to be overcome before such techniques could be widely used in the clinic. To maximize the potential of genome editing technology, the SGCE program was developed to accelerate the translation of genome editing technology into clinical applications.

Based on input received from stakeholders from academia, industry, and regulatory agencies, as well as the substantial progress in the field of genome editing since the launch of the first five-year phase of the SCGE program, the second five-year phase of SCGE will focus on translating and accelerating safe and effective genome editing therapeutics into the clinic. Specifically, SCGE Phase 2 will support the following initiatives: 1) Technologies and Assays for Therapeutic Genome Editing INDs; 2) IND-enabling Studies of Somatic Genome Editing Therapeutic Leads; 3) Platform Clinical Trials of Somatic Genome Editing for Multiple Diseases and 4) Somatic Cell Genome Editing Translational Coordination and Dissemination Center.

The SCGE program will involve collaborative research by a consortium of grantees with differing expertise to develop, optimize and demonstrate genome editing technologies as treatments for human disease. Awardees from all four SCGE program components will form a consortium, governed by a steering committee of investigators and NIH staff that will develop consensus policies and procedures for Consortium-wide activities such as data and resource sharing. Collectively, these initiatives are intended to substantially expand the number of genetic diseases treated by in vivo genome editing, ultimately allowing these technologies to achieve their potential as a therapeutic platform to treat genetic disease.

Platform Clinical Trials of Genome Editors in Multiple Diseases

Phase 2 of the SCGE program will include a funding announcement for Platform Clinical Trials of Genome Editors in Multiple Diseases (UG3/UH3 Clinical Trial Required). The purpose of this FOA is to provide support for applications that propose a novel genome editing clinical trial that includes at least two different diseases, using the same genome editor, route of administration, and delivery system. It is expected that projects supported under this FOA will identify strategies to increase the efficiency of the process of obtaining regulatory approval for initiating and carrying out clinical genome editing trials of more than one disease, as compared to completely independent IND applications and clinical trials for each disease.

Funding Information

TBD

Estimated Total Funding
  • Pending availability of funds, estimated total funding is $10 mil in FY23, $20 mil per year in FY24-FY25, and $12 mil per year in FY26-FY27.
Expected Number of Awards
  • 2 awards
Estimated Award Ceiling
  • TBD
Primary Assistance Listing Number(s)
  • TBD
Anticipated Eligible Organizations
Public/State Controlled Institution of Higher Education
Private Institution of Higher Education
Nonprofit with 501(c)(3) IRS Status (Other than Institution of Higher Education)
Small Business
For-Profit Organization (Other than Small Business)
State Government
Indian/Native American Tribal Government (Federally Recognized)
County governments
Independent school districts
Public housing authorities/Indian housing authorities
Indian/Native American Tribally Designated Organization (Native American tribal organizations (other than Federally recognized tribal governments)
U.S. Territory or Possession
Indian/Native American Tribal Government (Other than Federally Recognized)
Regional Organization
Eligible Agencies of the Federal Government

Applications are not being solicited at this time. 

Inquiries

Please direct all inquiries to:

P.J. Brooks, Ph.D.

National Center for Advancing Translational Sciences (NCATS)

301-443-0513