RARE Daily

Intellia Raises $600 Million to Advance Gene Editing Therapeutic Pipeline

June 30, 2021

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Fresh on the heels of demonstrating proof of concept for its CRISPR/Cas9 technology for in vivo gene editing in patients with ATTR amyloidosis, Intellia Therapeutics raised $600 million in a follow-on offering as its shares hit an all time high.

Intellia priced the underwritten public offering of 4.1 million shares of its common stock at $145.00 per share. The company also granted the underwriters a 30-day option to purchase up to an additional 620,689 shares of its common stock, before deducting underwriting discounts and commissions and offering expenses.

Intellia and partner Regeneron Pharmaceuticals had just reported landmark data that supported the efficacy of in vivo CRISPR genome editing in humans. In a phase 1 dose escalation study in patients with transthyretin (ATTR) amyloidosis, a rare, progressive, and deadly disease in which mutations in the TTR gene cause the liver to produce misshapen transthyretin (TTR) protein that misfold and build up as amyloid in the body, a single intravenous infusion with Intellia’s NTLA-2001 led to dose-dependent reductions in serum TTR, with mean reductions of 52 percent among the three patients in the low dose group, and 87 percent among the three patients in the high dose group, including one patient with a 96 percent reduction.

NTLA-2001 is the first experimental CRISPR therapy candidate to be administered systemically to edit genes inside the human body. Intellia uses lipid nanoparticles to deliver a two-part genome editing system to the liver—a guide RNA specific to the disease-causing gene and a messenger RNA that encodes the Cas9 enzyme, which carries out the precision editing.

CRISPR has already been used effectively to treat sickle cell disease and beta-thalessemia, but in those cases, the gene editing took place outside the body, with the edited gene then being inserted back into the body.

Intellia is currently enrolling patients into part 3 of the dose escalation study, after which it plans to move into pivotal studies for both polyneuropathy and cardiomyopathy manifestations of ATTR amyloidosis.

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