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: 

About the SCGE
Phase 1 Toolkit
Phase 2 Publications

In The News

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. Peter Marks, MD, PhD, is the Director for Center for Biologics Evaluation and Research (CBER) in the FDA. P.J. Brooks, PhD, is the Deputy Director in the Division of Rare Diseases Research Innovation in NIH NCATS.

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.

Brainwide silencing of prion protein by AAV-mediated delivery of an engineered compact epigenetic editor

SCGE researchers developed CHARM, a molecular-editing tool that’s small enough to be delivered to the brain to shut down the production of proteins that cause prion diseases. 

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.