The National Institutes of Health will launch an effort aimed at removing barriers that slow the adoption of genome editing for treating patients. This program, Somatic Cell Genome Editing, plans to award researchers approximately $190 million over six years beginning this year, pending availability of funds. These researchers will collaborate to improve the delivery mechanisms for targeting gene editing tools in patients, develop new and improved genome editors, develop assays for testing the safety and efficacy of the genome editing tools in animal and human cells, and assemble a genome editing toolkit containing the resulting knowledge, methods, and tools to be shared with the scientific community. The program is funded by NIH’s Common Fund.

"Genome editing technologies such as CRISPR/Cas9 are revolutionizing biomedical research," said NIH Director Francis S. Collins, M.D., Ph.D. "The focus of the Somatic Cell Genome Editing program is to dramatically accelerate the translation of these technologies to the clinic for treatment of as many genetic diseases as possible."

Many rare diseases, as well as some common disorders, are caused by changes in a person’s DNA, either through changes inherited from parents or those that occur during a person’s lifetime. Advances in genome editing made over the past decade now make it possible to precisely change the DNA code inside living cells. Despite widespread interest and investment in this field, many challenges remain preventing broad adoption of this technology in the clinic.

Somatic cells are any of the non-reproductive cells of the body, i.e. the cells that do not pass DNA down to the next generation. By focusing on somatic cells, any changes to the DNA introduced by the genome editing therapeutics will not be inherited.

Funding opportunity announcements for this program are expected to be issued within a month. This program is supported by the NIH Common Fund and is managed by a trans-agency Working Group representing multiple NIH Institutes and Centers, led by the National Center for Advancing Translational Sciences (NCATS).

About the NIH Common Fund: The NIH Common Fund encourages collaboration and supports a series of exceptionally high-impact, trans-NIH programs. Common Fund programs are managed by the Office of Strategic Coordination in the Division of Program Coordination, Planning, and Strategic Initiatives in the NIH Office of the Director in partnership with the NIH Institutes, Centers, and Offices.

 About the National Center for Advancing Translational Sciences (NCATS): NCATS conducts and supports research on the science and operation of translation – the process by which interventions to improve health are developed and implemented – to enable more treatments to get to more patients more quickly. 

About the National Institutes of Health (NIH): NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases.