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Intellia Therapeutics

Intellia Therapeutics

Intellia Therapeutics is a biotechnology company founded in 2014, based in Cambridge, MA that develops biopharmaceuticals using CRISPR-Cas9 gene editing technology.

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.

Therapeutic Products

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.

Funding
Series A

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.

Series B

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.

Timeline

April 12, 2021
Intellia Therapeutics Inc. announce the appointment of Georgia Keresty, Ph.D., M.P.H., to the company's Board of Directors.
June 1, 2020
Regeneron Pharmaceuticals and Intellia Therapeutics announce that they will be expanding an existing collaboration.

Funding rounds

Patents

Products

People

Name
Role
LinkedIn

Andrew D. Schiermeier, Ph.D.

Executive Vice President, Development and Corporate Strategy

Glenn Goddard

Executive Vice President, Chief Financial Officer

Intellia Therapeutics

Founder

Jennifer King, Ph.D.

Senior Vice President, Business Development

Jennifer Mound Smoter

Senior Vice President, External Affairs & Communications

Further reading

Title
Author
Link
Type
Date

Intellia Therapeutics Presents New Data in In Vivo and Ex Vivo Programs at the 26th Annual Congress of the European Society of Gene and Cell Therapy

Intellia Therapeutics, Inc.

Web

Documentaries, videos and podcasts

Title
Date
Link

CRISPR: Editing our genetic instructions | Rachel Haurwitz | TEDxSanFrancisco

October 26, 2016

Intellia Therapeutics

October 24, 2017

Companies

Company
CEO
Location
Products/Services

News

Title
Author
Date
Publisher
Description
Kyle LaHucik
October 12, 2021
FierceBiotech
Intellia Therapeutics was the first to prove gene editing can work in a human and now the famed CRISPR/Cas9 biotech is beefing up its ocular disease pipeline with a 10% equity stake in SparingVision.
Heather McKenzie
June 28, 2021
BioSpace
Interim results from the phase 1 study presented at the Peripheral Nerve Society Annual Meeting (PNS) demonstrated that the investigational therapy, NTLA-2001, greatly reduced the disease-causing protein after a single infusion.
Annalee Armstrong
June 25, 2021
FierceBiotech
Interim results are in for Intellia and partner Regeneron's in vivo CRISPR/Cas9 genome editing candidate, NTLA-2001, in patients with transthyretin (ATTR) amyloidosis: and the numbers look good. This is the first time gene editing has been proven to work in humans, which "opens up a whole new area of therapies for patients that wasn't there."
BioSpace
June 24, 2021
BioSpace
Intellia Therapeutics, Inc. today announced the appointment of James Basta, J.D., as Executive Vice President, General Counsel and Corporate Secretary.
Nick Paul
June 22, 2021
FierceBiotech
Blackstone Life Sciences, Cellex Cell Professionals and Intellia Therapeutics have teamed to create a CAR-T therapy startup. The biotech begins life with $250 million from Blackstone, universal CAR-T platforms from Cellex and CRISPR/Cas9 genome editing technology from Intellia.
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References

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