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Intellia Therapeutics

A U.S. clinical-stage genome editing company headquartered in Cambridge, Massachusetts. The company runs global clinical development through external trial sites, with in-house capabilities spanning research, development, regulatory and technical operations.

Founding and History

Intellia was founded in 2014 and is publicly traded on Nasdaq (NTLA). The company built its strategy around CRISPR/Cas9 genome editing, with a dual focus on in vivo gene editing (systemic delivery to edit genes inside the body) and ex vivo engineered cell therapies (editing cells outside the body before administration).

Therapy Areas and Focus

Intellia’s pipeline is centered on severe diseases where durable gene editing could offer long-term control with a single administration. Current focus areas include:

Genetic and rare diseases (including transthyretin amyloidosis and hereditary angioedema)
Liver-targeted indications (reflecting Intellia’s in vivo delivery approach)
Oncology and immunology (primarily through ex vivo cell therapy concepts and earlier programs)

Technology Platforms and Modalities

Intellia’s development engine combines:

In vivo CRISPR/Cas9 gene editing delivered systemically to target tissues (with the liver as a principal initial focus)
Ex vivo CRISPR-edited cell therapy approaches for selected oncology and immune indications
A modular approach to guide RNA design, Cas9 editing, and delivery optimization, with translational biomarker strategies intended to show proof of editing and proof of mechanism in early clinical studies

Key Personnel

John Leonard, MD, President and Chief Executive Officer
Glenn Goddard, Chief Financial Officer
David Lebwohl, MD, Chief Medical Officer
Laura Sepp-Lorenzino, PhD, Chief Scientific Officer
Eliana Clark, PhD, Chief Technology Officer
Derek Hicks, Chief Business Officer
James Basta, General Counsel and Corporate Secretary

Strategic Partnerships

Intellia has used collaborations to expand target coverage and accelerate development. A core collaboration with Regeneron supports discovery and development of selected in vivo gene-editing programs, including Intellia-led clinical development for its transthyretin amyloidosis program.

FAQ Section

Intellia develops CRISPR/Cas9-based genome editing therapies. The company’s core platform enables precise DNA edits either in vivo (editing within the body after systemic delivery) or ex vivo (editing cells outside the body and then administering them), with program design guided by measurable biomarkers of editing activity.

Intellia’s most advanced programs are in rare genetic diseases with strong mechanistic rationale for liver-targeted in vivo editing, including transthyretin amyloidosis and hereditary angioedema. The company also maintains earlier efforts in oncology and immunology through ex vivo approaches.

Key disclosed clinical-stage programs include:

Nexiguran ziclumeran (nex-z; previously NTLA-2001) for transthyretin amyloidosis (ATTR), including hereditary ATTR with polyneuropathy (ATTRv-PN) and ATTR cardiomyopathy (ATTR-CM), in Phase III development
Lonvoguran ziclumeran (lonvo-z; NTLA-2002) for hereditary angioedema (HAE), in Phase III (HAELO)
NTLA-3001, an in vivo targeted gene insertion program for alpha-1 antitrypsin deficiency (AATD), in Phase I/II

January 2026: the FDA lifted the clinical hold on the MAGNITUDE-2 Phase III trial of nex-z in ATTRv-PN, allowing enrollment and dosing to resume with updated monitoring measures.

Late 2025: the company reported completion of enrollment in the HAELO Phase III study of lonvo-z in hereditary angioedema, with topline data expected in the first half of 2026.

Across its lead programs, Intellia has emphasized durability of effect consistent with a one-time-editing approach, supported by biomarker readouts (such as target protein reduction for ATTR programs and clinical attack-rate reduction in HAE). The company’s more recent updates have also reflected heightened focus on safety monitoring and risk mitigation in late-stage ATTR development.

Near-term milestones include:

Ongoing execution and further regulatory dialogue for the ATTR Phase III program (with MAGNITUDE-2 resumed and the broader ATTR-CM program subject to ongoing FDA review)
HAELO Phase III topline results (H1 2026) and, if supportive, progression toward a U.S. filing pathway
Continued early clinical execution for NTLA-3001 in AATD

Intellia is led by executives with backgrounds spanning clinical drug development, genome editing science, technical operations, and biotech business development. The current leadership structure reflects a company operating multiple parallel clinical programs while managing the manufacturing and safety-monitoring requirements associated with systemic gene editing.

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