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The U.S. Food and Drug Administration issued this draft guidance “for sponsors seeking approval for targeted individualized therapies by generating substantial evidence of effectiveness and safety when randomized controlled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. 

Meet the Expert: Andy Holt and Jacob Smith

In this video of the Meet the Expert Series, panelists discuss AAV manufacturing and considerations for IND-enabling studies. 

Next Steps Towards Personalized Gene Editing Platforms

SCGE researchers Rebecca Ahrens-Nicklas and Kiran Musunuru discuss the next steps towards creating personalized gene editing platforms, building on their work in which they created a bespoke corrective gene-editing therapy for an infant’s severe urea cycle disorder.

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: