The RNA-editing race intensifies

Abstract

For small biotech companies hoping to strike a deal with larger drug developers, there’s no greater destination than the J.P. Morgan Healthcare Conference . In early January 2020, leaders from the start-up Shape Therapeutics made the annual pilgrimage to this mecca of biotechnology networking in San Francisco to make a pitch: What if you could edit someone’s genetic code without ever touching their DNA? The biotech industry is awash in companies using tools like CRISPR gene editing to fix or turn off problematic DNA. If gene editing works, it could provide a one-and-done cure. But some researchers are worried that if CRISPR slips up and cuts DNA at the wrong site , the damage could also be permanent. “Targeting DNA has a lot of all-or-nothing consequences,” says David Huss, head of research at Shape. At the conference, Huss explained to potential partners that Shape’s solution was to edit RNA instead