Program Snapshot

The goal of the SCGE program is to accelerate the development of genome editing therapies into clinic. For Phase 2, there are four program initiatives:

  1. Developing technologies and assays for safety and efficacy studies
  2. Optimizing genome editing-based therapeutic leads to support advancement towards clinical trials
  3. Supporting novel genome editing clinical trials for more than one disease
  4. Fostering collaboration and share new technologies and protocols with the public and research community

For ethical, legal, and safety reasons, the SCGE program does not support any research activities on genome editing in reproductive (germ) cells.

In The News

New Notice of Funding Opportunity from The NIH Somatic Cell Genome Editing Program

The NIH Common Fund issued a new Notice of Funding Opportunity (NOFO) “Technologies and Assays for Therapeutic Genome Editing INDs (U01, Clinical Trial Not Allowed)” to support the second phase (FY2023-2027) of the Somatic Cell Genome Editing (SCGE) program.   
The purpose of this NOFO to support the optimization and evaluation of IND-enabling technologies and assays to help accelerate the clinical development and evaluation of novel somatic cell genome editing therapeutics to treat a broad array of rare and common diseases.   

Applications are due on July 26, 2024, by 5:00 PM local time of applicant organization.  

TARGETED Challenge Phase 1 Winners Announced by NIH

The National Institutes of Health (NIH) recently announced the winners for Phase 1 of the TARGETED (Targeted Genome Editor Delivery) Challenge. In Phase 1, there were 54 proposals that outlined solutions to address one of two target areas: Programmable Delivery System for Gene Editing and Crossing the Blood-Brain Barrier. To see a full list of Phase 1 winners, click the button below.

NIH Launches Phase 2 of the TARGETED Challenge

The National Institutes of Health (NIH) has launched the second phase of the Targeted Genome Editor Delivery (TARGETED) Challenge, a competition aimed at improving the state of in vivo delivery technologies for genome editors in two target areas. Participation in Phase 1 is not required to join Phase 2. More information is available on the Challenge website.

A Closer Look at the CRISPR/Cas9 Gene Therapy for Sickle Cell Disease

The National Center for Advancing Translational Sciences (NCATS) researchers Dr. P.J. Brooks and Dr. Jeanita Pritchett Clay wrote an article discussing the impact of this discovery on the African American community as well as the potential to apply the approach to other diseases.

New Funding Opportunity to Support Phase 2 of the SCGE Program

The purpose of this new funding opportunity is to support 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.