Program Snapshot

The NIH Common Fund’s Somatic Cell Genome Editing (SCGE) program aims to reduce the burden of diseases caused by genetic changes. Genome editing technologies present an exciting prospect for treatments and possibly even cure for these diseases. During its first 5-year phase (FY18-FY23), SCGE developed quality tools to perform and assess effective genome editing tools in non-reproductive (“somatic”) cells of the body. In its second phase (FY23-27), SCGE will accelerate the translation of genome editing therapies into the clinic.

SCGE Phase 1

SGCE worked to improve the efficacy and specificity of genome editing approaches. The SCGE program developed new methods and improved systems that delivered genome editing machinery into various tissues with greater specificity, including tissues that present an unmet need and that are harder to reach such as the brain, ear, heart, and lung. SCGE made significant discoveries of new or optimized editors that edit target genomes with improved efficacy and novel functionality, including a prime editor that could correct up to 89% of known genetic variants associated with human diseases. The SCGE program developed new methods to assess unintended biological effects, such as high-throughput technologies that quickly identify high specific target sites and follow genome-wide activity of editors over time. Through the development of better animal models, the SCGE program has tested and validated a number of genome editing tools and delivery systems. The program has developed a genome editing toolkit that broadly disseminates program tools and learnings, opening this space to a wider range of the biomedical research community.

Learn more about the goals of SCGE Phase 1

An audio described version of this video is also available.

SCGE Phase 1 Consortium

Click the image below to view the interactive Somatic Cell Genome Editing Consortium map.

SCGE Phase 2

SCGE will accelerate the translation of genome editing therapies into the clinic by developing targeted delivery technologies; advancing clinical development and evaluation of novel genome editing therapeutics; establishing new regulatory pathways to lay the groundwork for clinical trials that assess the safety and efficacy of promising genome editing therapies to treat multiple diseases; and disseminating successful strategies for starting clinical trials through a publicly accessible platform.

SCGE phase 2 will consist of four initiatives:

• Developing technologies and assays for safety and efficacy studies
• Optimizing genome editing-based therapeutic leads to support advancement towards clinical trials
• Supporting novel genome editing clinical trials for more than one disease
• Fostering collaboration and share new technologies and protocols with the public and research community

Existing gene editing technologies have great potential but are not able to deliver gene editing tools to many target tissues and cell types in sufficient quantities, which hinders clinical application. To realize the promise of genome editing for treating many genetic diseases, SCGE launched the multi-phase TARGETED (Targeted Genome Editor Delivery) Challenge to improve the current state of in vivo delivery technologies for genome editors in two Target Areas: 1. Programmable Delivery System for Gene Editing, and 2. Crossing the Blood-Brain Barrier. The TARGETED Challenge will help improve these technologies and speed their translation from the lab into the clinic.

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