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).

Meet the Expert: Nick Buss, Ilan Irony, Allen Callaway

In this video of the Meet the Expert Series, panelists discuss non-clinical study design and regulatory insights for a seamless transition.

SCGE Fall 2025 Meeting

The SCGE Consortium met for an in-person meeting in Arlington, VA. The event was full of great presentations and insightful discussions. Check out a photo recap of the event below.

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: Deanna Portero and Stefano Benvenuti

In this video of the Meet the Expert Series, Deanna Portero and Stefano Benvenuti discuss alternative business models for non-commercially viable diseases.

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: