Intellia Therapeutics (NASDAQ:NTLA) is developing one of the most promising new areas of therapeutic development: gene editing and repair using CRISPR/Cas9 technology. CRISPR/Cas9-based gene editing holds promise across a range of gene therapy applications, including blood disorders, cancer and other genetic based diseases. Intellia was founded in 2014 by Caribou Biosciences and Atlas Venture and it holds exclusive access to a broad intellectual property portfolio covering the application of CRISPR/Cas9 technology for human therapeutic use.
Intellia Therapeutics, Inc.
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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 sitespecific double-stranded break. By varying the guide RNA sequence one can target the Cas9 to other regions of the genome. 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.
In Jan 2015, Intellia signed 5 years R&D collaboration with Novartis to accelerate the ex vivo development of new CRISPR/Cas9- based therapies using chimeric antigen receptor T cells (CARTs) and hematopoetic stem cells (HSCs). Under the terms of the agreement, Novartis receives exclusive rights to develop all collaboration programs focused on engineered CARTs.
In May 2016, Intellia raised $108 Mn through IPO. Prior this, in Sep 2015, Intellia closed Series B round with $70 Mn led by OrbiMed HealthCare. Additional investors included Fidelity Management, Janus Capital Management, Foresite Capital, Sectoral Asset Management, EcoR1 Capital, Atlas Venture and Novartis.
In Nov 2014, Intellia closed Series A investment round with $ 15 Mn in financing led by Atlas Ventures and Novartis Institutes for Biomedical Research (NIBR).