In 1996, 32,655 Americans died from AIDS. A year later, that number was 16,685. The difference? Protease inhibitors — drugs that prevented HIV from building more copies of itself inside human cells. Acting with record speed, the Food and Drug Administration approved three such drugs in 1996 — the products of a high-intensity race between scientists at Merck, Roche, and Abbott Laboratories.
“I feel sort of the same energy now, all these years later,” said John Leonard, a former National Institutes of Health virologist who led Abbott’s work on its first-generation AIDS drug, speaking at a STAT virtual event Thursday. Now the president and CEO of Intellia Therapeutics, Leonard joined STAT senior medical writer Matthew Herper to discuss the pace of clinical advances in genome editing. “It’s the nature of the adventure,” said Leonard. “Thinking about 1979, 1980, inconceivable stuff back then is mundane today.”
Intellia is toward the front of a pack of CRISPR companies all aiming to turn genome editing into a standard of clinical care — first for genetic disorders, and eventually for more common diseases. A little over a year ago, Intellia began testing whether it would be possible to precisely alter the DNA of particular cells within the human body to treat genetic diseases with a one-time intravenous infusion of CRISPR components. And last June, in a landmark moment for the field, the company shared interim trial data from the first six patients showing that not only could they safely deliver CRISPR to the right cells, but that it could make the right changes too.
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