SCGE News

SCGE Investigational New Drug (IND) Application Toolkit for Gene Therapy

A primary goal of the SCGE Consortium is to submit IND applications for novel gene editing therapies. As part of this work, we have created a collection of useful resources, templates, and tools for filling out an IND application. Find these free, public resources at the link below. 

IND Application Toolkit

SCGE Spring 2026 Meeting Recap

The SCGE spring 2026 meeting was held in April in Philadelphia, PA. The event was full of scientific presentations, thoughtful discussions, and lots of collaboration. See a photo recap of the meeting at the link. 

FDA Launches Framework for Accelerating Development of Individualized Therapies for Ultra-Rare Diseases

The U.S. Food and Drug Administration issued this draft guidance “for sponsors seeking approval for targeted individualized therapies by generating substantial evidence of effectiveness and safety when randomized controlled trials are not feasible due to small patient populations.”

First SCGE Researcher Regulatory Documents Now Available

The first sets of regulatory documents (generated through regulatory interactions between consortium projects and the U.S. Food and Drug Administration (FDA)) are now available on the SCGE platform. Check out the documents at the button below.

Monitoring biological effects of somatic cell genome editing

Researchers from the SCGE Phase 1 Biological Effects Initiative published this review on monitoring biological effects of somatic cell genome editing.

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: