Intellia

Corporate HQ: Cambridge, MA

Founded in 2014, Intellia Therapeutics is developing novel curative medicines and therapies, utilizing the promise of the CRISPR/Cas9 gene editing technology.

Intellia was founded by Caribou Biosciences and Atlas Venture, along with a consortium of leading scientists who have helped define the space. The company holds exclusive access to a broad intellectual property portfolio covering the application of CRISPR/Cas9 technology for human therapeutic use.

The CRISPR/Cas9 platform is a two component system: 1) the guide RNA and 2) the Cas9 nuclease. The guide RNA binds directly to the Cas9 protein and targets it to the DNA site of interest. Once bound, the Cas9 protein cuts the DNA generating a site-specific double-stranded break. Targeting the guide to sites of disease-causing mutations, the Cas9 creates a double-stranded break, harnessing the natural cellular repair process to either replace the mutation with the correct gene sequence, insert a replacement gene, or inactivate the gene. Specific guide RNA sequences can be created in hours, tested quickly in the laboratory, and manufactured and scaled with great efficiency for subsequent clinical testing.

For more detailed information, please visit: www.intelliatx.com