Program Snapshot

The goal of the SCGE program is to accelerate the development of genome editing therapies into 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 share 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.

In The News

TARGETED Challenge Phase 1 Winners Announced by NIH

The National Institutes of Health (NIH) recently announced the winners for Phase 1 of the TARGETED (Targeted Genome Editor Delivery) Challenge. The challenge, which has three phases, supports the SCGE (Somatic Cell Genome Editing) program’s commitment to deliver genome editors to somatic cells throughout the body.

In Phase 1, there were 54 proposals from various research groups, universities, companies, and labs. The proposals outlined solutions to address one of two target areas: Programmable Delivery System for Gene Editing and Crossing the Blood-Brain Barrier. To see a full list of Phase 1 winners, click the button below.

Phase 2 of the Challenge is now open. Both Phase 1 Winners and new participants are invited to submit proposals. Phase 2 will have 10 winners that each win $250,000 USD and are eligible to compete in Phase 3. For more information on applying to Phase 2, check out this website.

Congratulations to the Phase 1 Winners, and best of luck to everyone entering Phase 2!

NIH Launches Phase 2 of the TARGETED Challenge

The National Institutes of Health (NIH) has launched the second phase of the Targeted Genome Editor Delivery (TARGETED) Challenge, a competition aimed at improving the state of in vivo delivery technologies for genome editors in two target areas. Participation in Phase 1 is not required to join Phase 2. More information is available on the Challenge website.

A Closer Look at the CRISPR/Cas9 Gene Therapy for Sickle Cell Disease

The National Center for Advancing Translational Sciences (NCATS) researchers Dr. P.J. Brooks and Dr. Jeanita Pritchett Clay wrote an article discussing the impact of this discovery on the African American community as well as the potential to apply the approach to other diseases.

New Funding Opportunity to Support Phase 2 of the SCGE Program

The purpose of this new funding opportunity is to support IND-enabling studies for the development of a novel in vivo genome editing therapeutic platform (genome editor plus delivery system) for two or more disease indications, using the same genome editor, route of administration, and delivery system.

NIH Launches Phase 2 of the SCGE Program with a Series of New Awards

The National Institutes of Health (NIH) has awarded over $140 million to help accelerate genome editing approaches from the lab to the clinic. The newly funded projects build upon the work performed though the Somatic Cell Genome Editing (SCGE) Program over the past five years and will focus on translating safe and effective genome editing therapeutics into the clinic.