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
Dr. Luciano Marraffini
Assistant Professor in the Laboratory of Bacteriology,
The Rockefeller University
Blavatnik National Award
Recognized for major contributions to the basic molecular mechanisms and applied biology of CRISPRs
Dr. Marraffini has made seminal contributions to the field of CRISPR-Cas biology, including the discovery that these prokaryotic systems target DNA molecules. He is a 2012 Rita Allen Foundation Scholar and a 2011 Searle Scholar and is the recipient of a 2012 NIH Director's New Innovator Award and a 2015 Burroughs Wellcome Fund PATH Award.