Gene Editing, Research Institutes

Reckoning the potential of CRISPR/Cas9 tech, NIH launches $190M genome editing research initiative

Francis Collins, the director of the National Institutes of Health CQ/Roll Call Getty

As the first human trial involving CRISPR comes into view in the United States, the NIH has stepped up with a $190 million research initiative to help float all the boats riding on the wave of new genome editing technologies.

Named Somatic Cell Genome Editing, the program will award funds over six years to researchers collaborating to expand the toolkit for the whole scientific community.  improving the delivery mechanisms, developing better editors, designing assays to evaluate those editing tools, and assembling a package of knowledge and methods. Ultimately, the hope is to remove barriers to broad adoption of the tech in the clinic.

“Genome editing technologies such as CRISPR/Cas9 are revolutionizing biomedical research,” said director Francis Collins in a 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.”

As its name suggests, the program focuses on somatic cells — non-reproductive cells that do not pass DNA down to the next generation. In other words, any changes to the DNA introduced by these kinds of therapeutics would not be inherited. That avoids the stickiest ethical concerns tied to the use of US taxpayer funds.

This new investment by the NIH comes at a pivotal moment for gene editing.

The pioneers have had their patent fight, and star-studded new players such as Exonics and Tmunity are starting to come on the scene. With China racing ahead in the clinic — 86 patients have had their genes edited, per the Wall Street Journal — developing the infrastructure needed to advance next-gen tech benefits all players.

Mary Ellen Perry

In fact, the specific directions of the program are the product of a brainstorming workshop with industry experts, patient advocates and academic researchers. That’s according to Mary Ellen Perry, who first presented the concept to a group of NIH insiders in September. What comes out of this program, she said, could impact everything from common disorders to rare diseases.

“It’s almost difficult to think of a condition that this might not be applicable to,” Perry said at the meeting.

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