What Intellia Therapeutics does

Intellia Therapeutics is a clinical-stage biopharmaceutical company founded in 2014 and headquartered in Cambridge, Massachusetts, that develops CRISPR/Cas9-based gene editing therapies using both in vivo (lipid nanoparticle delivery to liver cells) and ex vivo (engineered cell therapy) approaches. Its lead candidates are lonvo-z (NTLA-2002) for hereditary angioedema, which targets the KLKB1 gene to durably reduce plasma kallikrein activity, and nex-z (NTLA-2001) for transthyretin amyloidosis, which targets the TTR gene; both are one-time treatments delivered via lipid nanoparticles. The technology platform is built on a Nobel Prize-winning foundation (co-founder Jennifer Doudna shared the 2020 Chemistry Nobel) and the company is the first to achieve a positive Phase 3 readout for an in vivo CRISPR gene editor, with a rolling BLA submission initiated in April 2026 and a planned H1 2027 US commercial launch for lonvo-z.

The company currently generates revenue almost entirely through collaboration and milestone payments — primarily from a 2016 partnership with Regeneron covering ATTR and hemophilia programs (under which Regeneron shares 25% of worldwide development costs and commercial profits for ATTR and holds a US co-promotion option) — rather than from product sales. FY2025 revenue was $67.7 million (+17% YoY) against a $412.7 million net loss, reflecting heavy investment in clinical development and pre-commercial buildout. The commercial organization is being constructed around a specialty pharmacy and hospital distribution model targeting rare disease specialists, led by SVP Commercial Maria Natale (former Takeda TAKHZYRO US launch lead), with approximately 32 commercial hires including 29 sales positions and 3 market access/HEOR roles. The company ended early 2026 with approximately $605 million in cash, extended by an April 2026 underwritten public offering of approximately $180 million at $10.75 per share, and carries a market capitalization of approximately $1.55-1.95 billion.

Additional pipeline programs include an allogeneic CD19 CAR-T cell therapy for B-cell-mediated autoimmune diseases partnered with Kyverna Therapeutics, the wholly owned AvenCell oncology CAR-T programs (AVC-201 and AVC-203), and a hemophilia B program co-developed with Regeneron. The company is publicly traded on NASDAQ under ticker NTLA and employs 251-500 people.

Name

Intellia Therapeutics

Legal name

Intellia Therapeutics, Inc.

Website

https://intelliatx.com

Company type

Public

Founded year

2014

Operating status

Operating

Headcount range

251–500 employees

Short description

Intellia Therapeutics is a clinical-stage biopharmaceutical company developing one-time CRISPR/Cas9 in vivo gene editing therapies delivered via lipid nanoparticles, with lonvo-z for hereditary angioedema in rolling BLA review and nex-z for ATTR amyloidosis in Phase 3, targeting a H1 2027 commercial launch.

Ownership category

akta.pro rank

Product category

Gene Therapy Pharmaceuticals

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Biological Product (except Diagnostic) Manufacturing (325414)

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Genome Editing Gene Therapies (CRISPR/TALEN/ZFN; in vivo/ex vivo) (HLAAACAD)

akta.pro secondary industries

Gene Therapy for Rare/Monogenic Diseases (HLAAACAE), Cell Therapy Enabling Technologies (gene editing, cell engineering, delivery methods) (HLAAABAL)

Keywords

• CRISPR gene editing
• In vivo therapeutics
• Lipid nanoparticle delivery
• Rare disease treatments
• Genome editing platform

Headquarters

HQ city

Cambridge

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Marketing or Sales, Operations, Infrastructure

Revenue model

1. Collaboration and Milestone Payments: Intellia's primary revenue model relies on collaboration payments and milestone income from pharmaceutical partners rather than commercial product revenue. The company receives development milestones and potential future royalties from Regeneron (ATTR program, hemophilia), AvenCell, and Kyverna partnerships.
2. Future Product Sales: Upon FDA approval and commercial launch, expected to generate revenue from product sales. Lonvo-z (if approved for HAE) and nex-z (if approved for ATTR amyloidosis) would be one-time treatments with potential premium pricing. FY2025 revenue was $67.7 million, up 17% year-over-year, driven by collaboration income from Regeneron.
3. Grant and Research Funding: The company has historically received grant funding and research support. FY2025 revenue of $67.7 million with 17% YoY growth, though still operating at a net loss of $412.7 million.

Pricing tiers

Go-to-market motion2 records

Distribution channels2 records

Marketing channels5 records

Core offering

Intellia Therapeutics is a clinical-stage biopharmaceutical company developing one-time CRISPR/Cas9-based gene editing therapies through in vivo (LNP delivery to liver hepatocytes) and ex vivo (engineered cell therapy) approaches. Its lead candidates are lonvo-z for hereditary angioedema (HAE) and nex-z for transthyretin (ATTR) amyloidosis, with partnered programs in hemophilia (Regeneron) and allogeneic CAR-T cell therapy (AvenCell, Kyverna).

Product overview

Intellia Therapeutics is a clinical-stage biopharmaceutical company developing CRISPR/Cas9-based gene editing therapies through both in vivo (direct delivery) and ex vivo (engineered cell therapy) approaches. The company's lead product candidates include lonvo-z (lonvoguran ziclumeran) for hereditary angioedema and nex-z (nexiguran ziclumeran) for ATTR amyloidosis, both delivered via lipid nanoparticles targeting liver cells. Additional pipeline programs include hemophilia treatments partnered with Regeneron and allogeneic CAR-T cell therapies for oncology through AvenCell.

Differentiator

Problem solved

Functional benefit

Products and services

• Lonvo-z (lonvoguran ziclumeran, NTLA-2002) A one-time in vivo CRISPR/Cas9-based gene editing therapy for hereditary angioedema (HAE) that targets the KLKB1 gene in hepatocytes to durably reduce plasma kallikrein activity and prevent excess bradykinin production, intended for HAE specialists and patients with recurrent life-threatening swelling attacks. Currently under rolling BLA submission to the FDA.
• Nex-z (nexiguran ziclumeran, NTLA-2001) A one-time in vivo CRISPR/Cas9-based gene editing therapy for transthyretin (ATTR) amyloidosis targeting the TTR gene in hepatocytes. In Phase 3 development for both ATTR amyloidosis with polyneuropathy (ATTRv-PN, MAGNITUDE-2) and ATTR amyloidosis with cardiomyopathy (ATTR-CM, MAGNITUDE). Partnered with Regeneron Pharmaceuticals.
• REGV131-LNP1265 (Hemophilia B) A CRISPR/Cas9-based treatment for hemophilia B using targeted transgene insertion capabilities, being co-developed with Regeneron Pharmaceuticals. Regeneron is advancing the hemophilia B (and hemophilia A research-stage) programs with Intellia eligible for development milestones and royalties.

Quantifiable outcome

• 87% reduction in mean monthly HAE attacks vs placebo (Phase 3 HAELO trial)
• +6 more outcomes

Named customers2 records

Segments3 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Three partnerships are on record, tiered core.

• Regeneron PharmaceuticalscoreStrategic or Co-development PartnerLicense and collaboration agreement to research, develop and potentially commercialize CRISPR/Cas9-based therapeutic products focused on genome editing in the liver. Regeneron shares 25% of worldwide development costs and commercial profits for the ATTR program and has an option to enter into a co-promotion agreement for U.S. commercialization of ATTR therapies.
• AvenCellcoreStrategic or Co-development PartnerAvenCell was launched by Intellia, Blackstone Life Sciences, Cellex Cell Professionals GmbH and GEMoaB (now AvenCell Europe GmbH) to develop next-generation universal CAR-T immunotherapies for difficult-to-treat cancers using Intellia's allogeneic cell engineering platform. AVC-201 and AVC-203 are wholly owned by AvenCell.
• Kyverna TherapeuticscoreStrategic or Co-development PartnerLicensing and collaboration agreement for the development of an allogeneic CD19 CAR-T cell therapy for the treatment of various B-cell-mediated autoimmune diseases. Intellia provides its differentiated allogeneic cell engineering platform.

Scale indicators9 records

Recent moves5 records

Expansion highlights6 records

Broad incumbents

• Alnylam Pharmaceuticals: Broader RNAi therapeutics incumbent with approved drugs targeting HAE (e.g., in this disease area family) and ATTR amyloidosis (AMVUTTRA). Represents the principal therapeutic alternative to Intellia's one-time gene editing approach, with an established commercial footprint in both lead indications.
• Moderna: Broader mRNA and LNP technology incumbent with extensive lipid nanoparticle delivery platform expertise. Comparable through LNP delivery technology overlap (the delivery vehicle for Intellia's in vivo CRISPR programs), though focused on vaccines and protein-encoding therapeutics rather than gene editing.
• Vertex Pharmaceuticals: Broad biotech incumbent with significant gene therapy exposure via the CASGEVY collaboration with CRISPR Therapeutics. Comparable as an established commercial-stage biotech operating in the curative medicine space Intellia targets, with deep rare-disease commercial expertise.
• Regeneron Pharmaceuticals: Strategic partner and broader biopharmaceutical incumbent with substantial gene therapy pipeline. Comparable through the active co-development arrangement covering Intellia's ATTR program (nex-z), with overlap in rare disease commercialization capability.

Emerging players

• Prime Medicine: Emerging prime editing company pursuing in vivo and ex vivo gene editing therapies. Comparable as a next-generation gene editing platform peer, though earlier-stage than Intellia with no Phase 3 programs.

Direct peers

• Editas Medicine: Direct peer developing CRISPR-based gene editing medicines, including Casgevy (now Verve/Vertex) and in vivo ophthalmology programs. Comparable as a clinical-stage CRISPR company using similar technology foundation, with overlapping competitive positioning in rare diseases.
• Sangamo Therapeutics: Clinical-stage gene editing and gene therapy peer using zinc finger nucleases (ZFN) and AAV delivery. Comparable as a platform-based genomic medicine developer with overlapping target diseases and similar business model, though using an earlier-generation editing modality.
• CRISPR Therapeutics: Direct CRISPR/Cas9 gene editing peer and pioneer; co-developed CASGEVY (exa-cel) with Vertex for sickle cell disease and beta-thalassemia — the first commercial CRISPR therapy. Comparable in core technology (CRISPR/Cas9) and platform-based approach, though historically focused on ex vivo applications.
• Beam Therapeutics: Direct peer in next-generation gene editing, developing base editing and prime editing therapies. Comparable as a clinical-stage gene editing platform targeting rare and serious diseases, with similar business model and therapeutic area overlap.
• Verve Therapeutics: In vivo gene editing peer using LNP delivery to edit genes in the liver for cardiovascular disease. Directly comparable in delivery modality (LNP-CRISPR) and target organ (liver), representing a clear competitor in the in vivo editing space.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks6 records

Key highlights6 records

Customer concentration

• YouTubeplayer.vimeo.com/video/769530132
• Xx.com/intelliatweets
• Facebookfacebook.com/133289660562078
• LinkedInlinkedin.com/company/intellia-therapeutics-inc-

Revenue estimate

Valuation estimate

Number of profiles

Profiles12 records

Funding overview

Funding rounds9 records

Investors9 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A1 record

Investments2 records

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Intellia Therapeutics