Gene-editing techniques such as CRISPR-Cas9 are already transforming research, but have a ways to go before they will prove their worth as human therapeutics. The National Institutes of Health (NIH) wants to speed things up—and is putting $190 million into a program to help make that happen.
The six-year project promises to tackle a lot of the issues that are seen as barriers to the adoption of genome editing for treating patients. That includes working on ways to deliver gene-editing drugs into target tissues, new and improved editing techniques and assays to test their safety that could become a "toolkit" for scientists working in this area.
The NIH’s involvement comes at a time when human trials of CRISPR are still at the planning stages in the U.S., where much of the research on the technique was carried out, but are already underway in China where regulatory obstacles to testing new therapies are not as high. According to a Wall Street Journal article, 86 patients have already received CRISPR treatment in China.
The rivalry between the two nations has been dubbed "Sputnik 2.0," in a nod back to the 20th century space race between the U.S. and Soviet Union
Last week, researchers at the University of Pennsylvania said they will shortly start a trial of CRISPR-modified T cells in cancer patients, while CRISPR Therapeutics and Vertex said in December they are hoping to start the first European trial of a gene-editing drug in beta-thalassemia patients before the end of the year.
All the studies envisaged to date involve ex vivo CRISPR therapy in which gene editing is done outside the body on cells that are then reintroduced into the patient. The ultimate ambition, however, is to be able to deliver gene-editing drugs systemically that will zero in and correct genetic defects in situ.
The NIH program will focus on genome editing in somatic cells that don’t pass genetic material into the next generation. By focusing on somatic cells, any changes to the DNA introduced by the genome editing therapeutics will not be inherited, says the Institutes.
NIH Director Francis Collins, M.D., Ph.D., said the investment—which will be made from the NIH Common Fund used for high-impact projects—is intended to “dramatically accelerate the translation of these technologies to the clinic for treatment of as many genetic diseases as possible.”
There are still battles ongoing about the intellectual property associated with the emerging tech, as well as its safety, with a recent paper suggesting that the immune responses to CRISPR-Cas9 could hinder its use to treat disease and cause serious side effects.
“Advances in genome editing made over the past decade now make it possible to precisely change the DNA code inside living cells,” said the NIH. “Despite widespread interest and investment in this field, many challenges remain preventing broad adoption of this technology in the clinic.”