Intellia Therapeutics
Corporate brand
UnclaimedClinical-stage biotechnology company developing CRISPR-based gene-editing medicines.
Last updated August 22, 2026
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Overview
Intellia Therapeutics is a clinical-stage biotechnology company developing potentially curative medicines based on CRISPR gene-editing technology. Founded in 2014 and headquartered in Cambridge, Massachusetts, the company works across two principal approaches. Its in vivo programs administer the editing system directly to patients, while its ex vivo programs edit human cells outside the body before those cells are used as a treatment. The company’s platform is intended to modify disease-causing genes with a single treatment or a limited number of interventions rather than manage symptoms continuously. Intellia’s principal in vivo technology uses lipid nanoparticles to deliver two components to target tissues: messenger RNA encoding the Cas9 nuclease and a guide RNA designed to recognize a particular genetic sequence. The company has emphasized liver-directed delivery, where the system can reach genes associated with hereditary diseases. Its best-known programs include NTLA-2001, an investigational treatment for transthyretin amyloidosis, and NTLA-2002, an investigational treatment for hereditary angioedema. The company has also described research programs involving alpha-1 antitrypsin deficiency, hemophilia, cancer and autoimmune disease, as well as work on additional editing modalities such as base editing and DNA writing. Intellia’s scientific origins are connected to the development of CRISPR-Cas9. Jennifer Doudna, one of the company’s scientific founders, helped develop the underlying technology with academic collaborators. Other scientists associated with the company’s formation included Rodolphe Barrangou, Rachel Haurwitz, Luciano Marraffini, Erik Sontheimer and Derrick Rossi. The company was initially backed by Atlas Venture and Novartis. Novartis supported its early work in part because of potential applications in engineered immune-cell therapies, leading to a collaboration and the creation of the eXtellia division for the CAR-T-related activities. The company became publicly traded in 2016. It subsequently established a major collaboration with Regeneron Pharmaceuticals, which obtained rights to apply Intellia’s CRISPR platform to multiple drug targets. Intellia’s development strategy has combined proprietary editing and delivery capabilities with partnerships intended to broaden the number of therapeutic targets it can pursue. It attracted substantial scientific attention in 2021 when it reported clinical data indicating that an intravenously administered CRISPR candidate could edit target cells inside the human body. Those data helped distinguish Intellia from approaches requiring cells to be removed, edited and returned to the patient. Intellia remains a development-stage company rather than a commercial medicine supplier. Its value proposition rests on precise genetic modification, non-viral delivery and the possibility of durable treatment effects. At the same time, its programs remain subject to clinical, regulatory, manufacturing and safety risks typical of advanced gene-editing development. Workforce reductions announced in 2024 and 2025 reflected a tighter prioritization of resources as the company advanced its lead programs.
History
Intellia Therapeutics was established in November 2014 to develop biopharmaceuticals based on CRISPR gene editing. Its formation brought together venture investors, pharmaceutical-industry executives and academic scientists associated with the emergence of CRISPR-Cas9 as a programmable genome-editing system. Atlas Venture backed the company, while Novartis also provided early support because of its interest in applying CRISPR to engineered immune-cell therapies. Nessan Bermingham, an Atlas Venture partner, became the founding chief executive, and John Leonard, an experienced drug-development executive, served as founding chief scientific officer. Scientific founders and advisers associated with the company included Jennifer Doudna, Rodolphe Barrangou, Rachel Haurwitz, Luciano Marraffini, Erik Sontheimer and Derrick Rossi. The company’s intellectual-property position developed alongside a broader dispute over ownership and priority in CRISPR patents. Intellia licensed relevant intellectual property from Caribou Biosciences, which had licensed University of California-related inventions associated with Doudna and her collaborators. The patent landscape became strategically important because Intellia was competing with other CRISPR companies for access to foundational technology and therapeutic applications. In November 2014, Intellia raised a reported $15 million Series A round, followed by a reported $70 million Series B financing in September 2015. In January 2015, it entered a collaboration with Novartis covering CRISPR applications in CAR-T and research involving hematopoietic stem cells, including possible applications in beta thalassemia and sickle-cell disease. Intellia created the eXtellia Therapeutics division to administer the CAR-T collaboration. The company later shifted increasing attention toward in vivo editing, particularly the challenge of delivering CRISPR components safely and efficiently to organs such as the liver. In December 2016, Intellia moved into an approximately 80,000-square-foot laboratory and office facility in Cambridge. Around the same period, it obtained a license to a lipid-nanoparticle drug-delivery system. The system allowed CRISPR components to be packaged for administration and protected from rapid degradation in the bloodstream. The identity of the original licensor was not initially disclosed publicly, although the company later disclosed that the delivery technology was licensed from Novartis in connection with its public offering. Intellia and Regeneron began presenting preclinical work on transthyretin amyloidosis in 2017. In April of that year, the companies announced a broader strategic agreement under which Regeneron received exclusive rights to apply Intellia’s platform to as many as ten drug targets, with up to five potentially outside the liver. The arrangement included an upfront payment, potential milestone payments and royalties. Intellia indicated that part of the proceeds would support bioinformatics capabilities for evaluating therapeutic targets. In December 2017, John Leonard became chief executive officer. The company continued to expand its management team, naming Glenn Goddard executive vice president and chief financial officer in October 2019. In October 2020, Jennifer Doudna shared the Nobel Prize in Chemistry with Emmanuelle Charpentier for the development of CRISPR-Cas9 genome-editing technology. Although the award recognized academic scientific work rather than a company product, it increased public visibility for the technology and for companies founded around its therapeutic potential. Intellia’s most important clinical milestone came in June 2021, when it announced interim Phase 1 data for NTLA-2001. The results provided early evidence that a CRISPR medicine delivered by intravenous infusion could edit a disease-related gene directly inside the human body. The findings were subsequently published in the New England Journal of Medicine in August 2021. This was a significant distinction from ex vivo approaches, in which cells are edited outside the body and then administered to a patient. In September and November 2022, Intellia presented early clinical data for NTLA-2002, its hereditary-angioedema program. The reports included initial evidence of potential clinical benefit in addition to laboratory biomarker changes. The company has continued to describe NTLA-2001 and NTLA-2002 as core in vivo programs while maintaining research in cancer, autoimmune disease, alpha-1 antitrypsin deficiency and hemophilia. It has also investigated newer forms of genome engineering, including base editing and DNA writing. Intellia entered a period of workforce and portfolio prioritization in 2024 and 2025. It announced an approximately 15% employee reduction in January 2024 and a further net workforce reduction of approximately 27% in the following year. These actions occurred as the company concentrated resources on its clinical-stage programs and managed the financial and operational demands of developing gene-editing medicines. Intellia remains an active, publicly traded, clinical-stage biotechnology company without a commercially marketed therapy identified in the supplied reference material.
- 2025
Further workforce reduction
[1]The company announced a net workforce reduction of approximately 27%.
- 2024
Workforce reduction
[1]Intellia announced a reduction of approximately 15% of its employees.
- 2022
Early NTLA-2002 clinical data
[1]The company presented initial clinical findings for its hereditary-angioedema candidate.
- 2021
First reported human in vivo CRISPR clinical data
[1]NTLA-2001 data provided early clinical evidence for direct, intravenous CRISPR editing inside the human body.
- 2017
Regeneron strategic agreement
[1]Regeneron obtained rights to use Intellia’s CRISPR platform across multiple drug targets under a collaboration including upfront, milestone and royalty economics.
- 2016
Initial public offering
[1]Intellia completed its initial public offering and became a publicly traded biotechnology company.
- 2015
Novartis collaboration established
[1]Intellia and Novartis agreed to collaborate on CRISPR applications in CAR-T and hematopoietic stem-cell diseases.
- 2014
Company founded
[1]Intellia Therapeutics was founded in November to develop biopharmaceuticals using CRISPR gene-editing technology.
Products and positioning
A CRISPR gene-editing specialist focused on one-time or potentially durable treatments for genetic, immune and oncological diseases.
In vivo gene-editing therapeutic candidate
NTLA-2001
An investigational CRISPR-based therapy for transthyretin amyloidosis. It uses intravenous delivery to direct genome editing toward the liver, where the target gene is expressed. Early clinical work was notable because it offered evidence that a CRISPR candidate could edit disease-related cells directly inside patients rather than requiring cell removal and laboratory manipulation.
In vivo gene-editing therapeutic candidate
NTLA-2002
An investigational CRISPR-based candidate for hereditary angioedema. The program is designed to edit a disease-relevant target in the liver and is intended to provide durable reduction of the biological driver of attacks. Initial clinical results were reported in 2022, and the supplied reference material describes the program as being in Phase 3 studies.
In vivo gene-editing therapeutic candidate
Lonvo-z
An investigational CRISPR therapy candidate for hereditary angioedema. The supplied reference material identifies Lonvo-z as an ongoing Phase 3 in vivo program; detailed product, regulatory and commercial information is not provided.
Engineered-cell therapeutic platform
Ex vivo CRISPR programs
A group of programs in which human cells are modified outside the body using CRISPR and then prepared as therapeutic products. Intellia has associated this approach with cancer and autoimmune disease research, including collaborations in engineered immune-cell therapies.
Brand decisions
2025
Further workforce and portfolio prioritizationStrategyBackground
Intellia continued to manage resources while advancing its clinical pipeline.
Action taken
The company announced a net workforce reduction of approximately 27%.
Aftermath
The action further narrowed the company’s operating base around its priority programs.
2024
Operating-cost and workforce prioritizationStrategyBackground
As a clinical-stage biotechnology company, Intellia needed to concentrate resources on its most advanced development programs.
Action taken
The company reduced its workforce by approximately 15%.
Aftermath
The reduction reflected portfolio and resource prioritization; detailed financial effects are not provided in the supplied material.
2021
Clinical advancement of NTLA-2001Product launchBackground
Intellia’s lead in vivo program was designed to test whether CRISPR components could be delivered systemically and edit a disease-related gene in patients.
Action taken
The company reported interim Phase 1 data after a single intravenous infusion of NTLA-2001.
Aftermath
The findings became an important proof point for systemic in vivo gene editing and were later published in the New England Journal of Medicine.
2017
Regeneron multi-target CRISPR collaborationM&ABackground
Intellia sought to expand the number of therapeutic targets accessible through its CRISPR platform and to support target-selection capabilities.
Action taken
Intellia granted Regeneron exclusive rights to use the platform on up to ten targets, with up to five potentially outside the liver, while agreeing to co-develop other targets.
Aftermath
The agreement brought an upfront payment, potential milestones and royalties and made Regeneron a major development partner.
Upfront payment. $75 million (2017 agreement announcement)
2015
Creation of eXtellia for the Novartis collaborationStrategyBackground
Novartis was interested in using CRISPR for CAR-T and hematopoietic stem-cell applications, while Intellia was building a broader therapeutic platform.
Action taken
Intellia created the eXtellia Therapeutics division to manage the CAR-T-related collaboration with Novartis.
Aftermath
The arrangement gave Intellia an early pharmaceutical partner and a dedicated structure for ex vivo and immune-cell applications.
Recent events
Reported
Intellia announces pricing of public stock offering[2]
Intellia Therapeutics announced pricing for an underwritten public offering of 4,794,521 common shares at $36.50 per share and granted the underwriters a 30-day option to buy additional shares.
Reported
Intellia reports fourth-quarter and full-year 2019 results[3]
Intellia Therapeutics reported operational highlights and financial results for the fourth quarter and year ended December 31, 2019.
Sources3 sources
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