Intellia Therapeutics

Intellia's ex vivo programs use CRISPR to create the therapy by using engineered human cells to treat cancer and autoimmune diseases.

[4] Intellia's proprietary non-viral gene knock out platform deploys lipid nanoparticles to deliver to the liver a two-part genome editing system: guide RNA specific to the disease-causing gene and messenger RNA that encodes the Cas9 enzyme, which carries out the precision editing.

The academic scientists involved in the founding included Rodolphe Barrangou, Rachel Haurwitz, Luciano Marraffini, Erik Sontheimer, and Derrick Rossi.

[15] In March 2017 Intellia and Regeneron, partners in co-developing a CRISPR-based treatment for transthyretin amyloidosis, presented data from a gene editing experiment in mice.

[16][17] By that time, University of California had lost a challenge to Broad's CRISPR patents, putting Intellia at a disadvantage relative to Editas.

Prior to Intellia, Goddard was the CFO at Generation Bio Company and senior vice president of finance at Agios Pharmaceuticals.

[21][22] In October 2020, Jennifer Doudna, one of Intellia's scientific co-founders, was awarded the 2020 Nobel Prize in Chemistry for the development of the CRISPR/Cas9 genome editing technology.

[23] In June 2021, Intellia made history by announcing interim Phase 1 data for NTLA-2001, demonstrating the ability to precisely edit target cells within the body to treat genetic disease with a single intravenous infusion of CRISPR.