Genome therapy is a part of precision medicine that is slowly going mainstream as policy clarifies reimbursement. The National Institutes of Health, for instance, is working on removing barriers that slow the adoption of genome editing for treating patients.
NIH announced this week the launch of Somatic Cell Genome Editing, a program aimed at picking up the pace in which the technology moves to the clinic for treatment.
Starting this year, NIH will award an estimated $190 million to researchers over six years, pending the availability of funds. The program is funded by NIH's Common Fund.
The researchers would be expected to 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.
"Genome editing technologies such as CRISPR/Cas9 are revolutionizing biomedical research," NIH Director Francis Collins, MD, said by statement. "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, the NIH said.
Advances in genome editing made over the past decade now make it possible to precisely change the DNA code inside living cells.
Somatic cells are any of the non-reproductive cells of the body, those 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 would not be inherited.
Despite widespread interest and investment in the field, many challenges remain in preventing broad adoption of this technology in the clinic.
One challenge is reimbursement. In November, the American Medical Association said it wanted clarity from policymakers for how insurance covers genomic tests and other precision medicine treatment.
Funding opportunity announcements for the program are expected to be issued within a month.