Fresh off CRISPR milestone, Intellia lays out 2022 vision – Boston Business Journal – Boston Business Journal

Intellia Therapeutics Inc. made history last summer when it became the first biotech company to successfully edit DNA inside the human body using CRISPR.
Now, Intellia (Nasdaq: NTLA) is planning to build on that momentum. The Cambridge company, which has already begun early-stage clinical trials of two in vivo gene-editing drugs, will launch a Phase 1/2 study of its ex vivo drug. Next year, Intellia plans to complete preclinical research for a third in vivo drug as well.
Intellia’s lead drug, NTLA-2001, was used last year to genetically alter liver cells in six patients with a genetic nerve disorder called transthyretin amyloidosis in a Phase 1 study, per interim trial results the company released at the time. CEO John Leonard told investors and attendees at the J.P. Morgan Healthcare conference this week that the company would present additional data from that study later this quarter, and said he has high hopes that the drug will usher in a wave of new possibilities for the company.
“These data support our belief that with our modular genome-editing technology, we may be able to target any genetic disease originating in the liver,” Leonard said. “We believe these groundbreaking data have opened the door to a new era of medicine.”
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Leonard spoke of turning Intellia into a “full-spectrum genome-editing company.” Over the next two years, he plans to expand the company’s range of disease targets as well as its technology, particularly what types of edits it can make. CRISPR has been likened to a microscopic pair of scissors that cuts a strand of DNA to knock out a gene; Leonard and his team are interested in a “knock-in” function as well.
Intellia is developing NTLA-2001 in collaboration with Regeneron Pharmaceuticals Inc. (Nasdaq: REGN). Over the last several months, the company has announced partnerships with California’s Kyverna Therapeutics for autoimmune diseases and the French company SparingVision for ocular diseases.
Chief Financial Officer Glenn Goddard said Intellia will continue to build on that partnership strategy, although he declined to offer further details.
“[The goal is] to have a platform that is unsurpassed in terms of what it’s able to accomplish,” Leonard said. “We want to be in a position where we can essentially introduce any edit that is therapeutically relevant.”
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