Intellia Therapeutics Announces Big Gene Editing Breakthrough in Early-Phase Trials

For the first time, CRISPR technology has been used to successfully treat disease in vivo, or inside the human body. That big medical news was announced Saturday by the biotech startup Intellia Therapeutics and its partner Regeneron, which said their gene-editing techniques reduced the amount of harmful liver protein associated with a genetic nerve disorder.

What is CRISPR? It stands for “clustered regularly interspaced short palindromic repeats,” and it’s one of those things humans found in nature and then copied.

• Bacteria use CRISPR to repel viruses, but humans have harnessed it to ctrl+c, ctrl+v DNA sequences, potentially leading to a revolution in treating disease.
• The two scientists who made that breakthrough in 2012, Jennifer Doudna and Emmanuelle Charpentier, won the Nobel Prize in Chemistry last year (Doudna is also a cofounder of Intellia).

Quote du jour: “There’s a feeling like we’re walking through a door here into all kinds of new possibilities. And there’s not many moments in medicine where you get to experience that,” Intellia CEO John Leonard said.

Looking ahead...expect Intellia shares, which have gained 233% since its 2016 IPO, to pop today.