Program Snapshot

The goal of the SCGE program is to accelerate the development of genome editing therapies into the 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 sharing 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.

Learn more about individual and collaborative research projects, as well as the SCGE program and deliverables, below: 

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.  

Meet The Expert: Peter Marks and P.J. Brooks

In the first webinar of the SCGE Meet the Expert series, Drs. Peter Marks and P.J. Brooks discuss the current landscape of the gene therapy regulatory and approval process, and the vision for where this can go in the future. Meet the Expert is a webinar series presented by the Somatic Cell Genome Editing (SCGE) Consortium. This series aims to connect researchers within the consortium and the greater scientific community with experts in the gene therapy space.

NOSI: Administrative Supplement

The National Institutes of Health (NIH) released a Notice of Special Interest (NOSI): Administrative supplements to active Common Fund awards to support replication studies. Find more details at the link below.

Bespoke Gene Therapy Consortium (BGTC) Regulatory Playbook 

The Bespoke Gene Therapy Consortium (BGTC) recognized the need for a guiding framework for the development and regulatory submission of adeno-associated virus (AAV) gene therapies for rare diseases. The BGTC drew upon their consortium subject matter experts and the collective experience of the scientific community to create a playbook that aims to bring safe, effective, and transformative gene therapies to patients in need.

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.