First SCGE Researcher Regulatory Documents Now Available

One goal of phase 2 of the SCGE program is to disseminate documents generated through regulatory interactions between consortium projects and the U.S. Food and Drug Administration (FDA) to help rare disease communities and researchers. These first sets of regulatory documents are now available on the SCGE platform. They are from the SCGE projects “Postnatal and Prenatal Therapeutic Base Editing for Metabolic Diseases” and “Personalized prime editing as a platform for hepatic inborn errors of metabolism“. 

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.

SCGE Researchers Create a Personalized Gene Editing Therapy to Treat a Rare Genetic Disease

A team of SCGE researchers created a patient-specific gene editing therapy to treat an inborn error of metabolism in an infant. This groundbreaking research was led by Drs. Rebecca Ahrens-Nicklas (CHOP) and Kiran Musunuru (Penn).

Base Editing in Rare Diseases

In a scientific first, SCGE researchers supported used base editing to change single DNA bases and disrupt the repetitive sequence mutation and applied this technique in laboratory models of Huntington’s disease and Friedreich’s ataxia.

Meet the Expert: James Valentine and Richard Lewis

In this video of the Meet the Expert Series, panelists use their legal and U.S. Food and Drug Administration (FDA) expertise to provide practical guidance to rare disease researchers. 

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: