Intellia Therapeutics

Intellia Therapeutics is a biotechnology and synthetic biology company using genetic engineering to create medical therapies that is headquartered in Cambridge, Massachusetts and was founded in 2014 by Andy May, Derrick Rossi, Erik Sontheimer, Jennifer A. Doudna, Luciano Marraffini, Nessan Bermingham, Rachel E. Haurwitz, and Rodolphe Barrangou.

Intellia Therapeutics has in vivo programs focused on developing treatments for liver diseases, including transthyretin amyloidosis, alpha-1 antitrypsin deficiency, hepatitis B virus, and inborn errors of metabolism. The company also has proprietary programs for developing engineered cell therapies to treat oncological and autoimmune diseases. In partnerships, they are also working on chimeric antigen receptor T cells and hematopoietic cells. Intellia has collaborations with Novartis Institutes for BioMedical Research, Regeneron Pharmaceuticals and Caribou Biosciences.

In 2018, Intellia Therapeutics began an arbitration proceeding against Caribou Biosciences, alleging Caribou had broken terms of a key license between the two companies by using and seeking to license two patent families invented or controlled by Caribous to third parties. Caribou licensed its technology for human therapeutic use to Intellia, which according to Caribou CEO Rachel Haurwiz includes the foundational CRISPR-Cas9 IP invented by Jennifer Doudna and colleagues, but does not include other types of CRISPR and non-CRISPR systems.

In a collaboration between Intellia and Regeneron Pharmaceuticals, researchers achieved higher than clinically required levels (supratherapeutic levels) of expression for the model mouse gene Factor 9 (F9), that codes for a blood clotting protein, by CRISPR-mediated targeted insertion into the liver where blood clotting proteins are produced. Intellia’s modular lipid nanoparticle (LNP) delivery system was used to deliver CRISPR-Cas9 with a modular adeno-associated viral (AAV) insertion template. The F9 gene codes for Factor IX (FIX), a blood-clotting protein often missing or defective in hemophilia B patients. Intellia’s proprietary bi-directional template was used and resulted in levels of FIX 40-300 times higher than those able to prevent bleeding episodes in hemophilia B patients.

For alpha-1 antitrypsin deficiency (AATD), which affects the liver and lungs, researchers used their system to insert a donor template DNA for the SERPINA1 gene into mice which resulted in clinically relevant blood protein levels in mice. Non-human primate studies conducted by Intellia with their transthyretin amyloidosis (ATTR) program demonstrated gene editing in liver, where approximately 35-40 percent rate of liver editing resulted in therapeutically meaningful reduced expression of TTR. The disease ATTR is endemic in certain populations in Portugal and the research was conducted in collaborations with researchers at Regeneron and the University of Porto in Portugal.

On November 18, 2014 Intellia Therapeutics completed their series A funding round with $15 million in capital from Novartis (lead investor), Atlas Ventures (lead investor), and Caribou Biosciences.

On September 15, 2015 Intellia Therapeutics completed their series B funding round with $70 million in funding from Orbimed (lead investor), Sectoral Asset Management, Janus Capital Group, Foresite Capital, Fidelity Management and Research Company, EcoR1 Capital, and Atlas Ventures.