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Can CRISPR Make The Jump To Therapeutics?

 
• By Alex Lash

Biomedical researchers are rapidly adopting the new gene-editing technology CRISPR/Cas9 for lab experiments, but can CRISPR also become the basis for new treatments of human disease? At least two venture-backed start-ups can't wait to find out.

It's late in the evening, and Emmanuelle Charpentier, PhD, is in a talkative mood. It's been about three years since the French microbiologist and her colleagues explained in a Nature paper how the innate immune system of some prokaryotes – bacteria and archaea – used two strands of RNA and an enzyme to chop up the DNA of invading viruses, and slowly, lights clicked on across the biomedical research world.

"I'm a microbiologist, I'm not a genome editing person," Charpentier says. "I wasn't expecting I would discover a totally new...

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