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    Intellia Therapeutics

    Also known as Intellia, NTLA

    Intellia Therapeutics is a biotechnology company developing CRISPR therapies given directly into the body.[1] The FDA accepted its application for lonvo-z, a one-time treatment for hereditary angioedema, with a target action date of 10 March 2027, and the company says it would be the first approved in vivo CRISPR therapy.[2] Its nex-z Phase 3 trials were put on hold in 2025 after a severe liver event and resumed in 2026.[3][4]

    Editor reviewedStrict sourcingUpdated Gene editing and gene therapyHealth and medicineLife sciences
    Key facts

    Intellia Therapeutics develops in vivo gene-editing therapies based on crispr-cas9.[1] An early study of its NTLA-2001 was published in 2021. That lipid nanoparticle carried Cas9 mRNA and a guide RNA against the TTR gene, and it lowered blood TTR protein by a mean 87% at the 0.3 mg/kg dose.[5] This page summarizes company disclosures and published data. It is not medical or investment advice.

    Lonvo-z for hereditary angioedema

    Lonvo-z (lonvoguran ziclumeran) is a one-time, outpatient treatment. It is designed to permanently lower kallikrein by inactivating the KLKB1 gene.[1]

    • Phase 3 results. In the HAELO trial, one infusion cut attacks by 87% versus placebo over weeks 5 to 28. The mean monthly attack rate was 0.26 versus 2.10. 62% of treated patients were attack-free and therapy-free, versus 11% on placebo.[6] The results were published in NEJM in June 2026.[7]
    • Regulatory path. Intellia began a rolling application in April 2026.[8] On 8 September 2026 it said the FDA had accepted the application with Priority Review and set a target action date of 10 March 2027.[2]
    • Published evidence. The NEJM report describes 80 patients randomized 2:1 to a single 50 mg infusion or placebo, with a median 7.5 months of follow-up at the 10 February 2026 cutoff and no serious or Grade 3 or higher adverse events in the lonvo-z group.[9]
    • Designations. Lonvo-z holds FDA Orphan Drug and RMAT designations, the UK Innovation Passport, EMA PRIME and European Commission orphan status, and the FDA has said it is not currently planning an advisory committee meeting.[10]
    • Launch plans. The company plans a US launch in the first half of 2027 if lonvo-z is approved.[11] Hereditary angioedema is estimated to affect one in 50,000 people, and existing options often mean lifelong therapy.[12]

    Intellia says lonvo-z would be the world’s first in vivo CRISPR-based therapy if approved.[2]

    Nex-z and the 2025 clinical hold

    Nex-z (nexiguran ziclumeran) targets the TTR gene. It is being developed with Regeneron for transthyretin amyloidosis with cardiomyopathy and hereditary ATTR with polyneuropathy.[13]

    On 29 October 2025 the FDA placed clinical holds on both Phase 3 trials, MAGNITUDE and MAGNITUDE-2. A patient dosed in MAGNITUDE had Grade 4 liver transaminase elevations and increased bilirubin.[3] According to Intellia’s August 2026 quarterly report, the patient died on 5 November 2025. The principal investigator reported the cause as septic shock secondary to a perforated duodenal ulcer, and the clinical course also included acute liver injury treated with corticosteroids.[14] The FDA lifted the MAGNITUDE-2 hold in January 2026 and the MAGNITUDE hold in March 2026, after the company agreed to changes including enhanced liver monitoring.[4] As of August 2026, Intellia expected to finish MAGNITUDE-2 enrollment in the second half of 2026.[15]

    What to watch

    Questions readers ask

    What is lonvo-z?

    An investigational one-time in vivo CRISPR/Cas9 treatment for hereditary angioedema that is designed to permanently lower kallikrein by inactivating the KLKB1 gene, given in an outpatient setting.[1]

    When will the FDA decide on lonvo-z?

    The FDA's target action date is 10 March 2027, under Priority Review. Intellia plans a US launch in the first half of 2027 if it is approved.[2][11]

    Why were Intellia's nex-z trials put on hold?

    In October 2025 the FDA placed holds on the MAGNITUDE and MAGNITUDE-2 Phase 3 trials after a patient had Grade 4 liver enzyme elevations and raised bilirubin. That patient died on 5 November 2025; the investigator attributed the death to septic shock from a perforated duodenal ulcer, in a course that also included acute liver injury. The MAGNITUDE-2 hold was lifted in January 2026 and the MAGNITUDE hold in March 2026, with enhanced liver monitoring.[3][14][4]

    What did Intellia's genomic analysis of nex-z patients find?

    In August 2026 the company said an analysis of more than 600 patient samples found the highest observed liver transaminase elevations in patients carrying one specific HLA allele, and that it would give genotyping results to investigators and to patients enrolling or screening in the Phase 3 trials.[18]

    Sources

    Each numbered claim is a statement we checked against the sources listed with it. Status shows how well established it is.

    1. [1]

      Lonvo-z (lonvoguran ziclumeran) is Intellia's one-time, in vivo CRISPR/Cas9 treatment for hereditary angioedema, designed to permanently lower kallikrein by inactivating the KLKB1 gene, and given in an outpatient setting. confirmedas of 2026-10-10

    2. [2]

      On 8 September 2026 Intellia said the FDA had accepted its lonvo-z application with Priority Review and a target action date of 10 March 2027; the company says lonvo-z would be the world's first in vivo CRISPR-based therapy if approved. confirmedas of 2026-10-10

    3. [3]

      On 29 October 2025 the FDA placed clinical holds on Intellia's Phase 3 MAGNITUDE and MAGNITUDE-2 trials of nex-z after a patient dosed in MAGNITUDE had Grade 4 liver transaminase elevations and increased bilirubin. confirmedas of 2026-10-10

    4. [4]

      The FDA lifted the clinical hold on Intellia's MAGNITUDE-2 trial in January 2026 and the hold on MAGNITUDE in March 2026, after the company agreed to study modifications including enhanced monitoring of liver laboratory tests; enrollment then advanced in both Phase 3 trials. confirmedas of 2026-08-06

    5. [5]

      In a 2021 NEJM study, NTLA-2001, a lipid nanoparticle carrying Cas9 mRNA and a guide RNA targeting the TTR gene, given by infusion, lowered blood TTR protein by a mean 87% at the 0.3 mg/kg dose, with mainly mild adverse events. confirmedas of 2026-10-10

    6. [6]

      In the Phase 3 HAELO trial, a single infusion of lonvo-z reduced hereditary angioedema attacks by 87% versus placebo over weeks 5 to 28 (mean monthly attack rate 0.26 versus 2.10), and 62% of treated patients were attack-free and therapy-free versus 11% on placebo. confirmedas of 2026-10-10

    7. [7]

      The HAELO Phase 3 results were published in the New England Journal of Medicine in June 2026. confirmedas of 2026-10-10

    8. [8]

      Intellia began a rolling Biologics License Application to the FDA for lonvo-z in April 2026. confirmedas of 2026-10-10

    9. [9]

      The HAELO Phase 3 trial randomized 80 patients 2:1 to a single 50 mg infusion of lonvo-z or placebo; median follow-up was 7.5 months as of 10 February 2026, and no serious or Grade 3 or higher adverse events were reported in the lonvo-z group. confirmedas of 2026-06-13

    10. [10]

      Lonvo-z holds five regulatory designations - FDA Orphan Drug and Regenerative Medicine Advanced Therapy, the UK MHRA Innovation Passport, EMA PRIME and European Commission Orphan Drug - and the FDA told Intellia in September 2026 that it was not currently planning an advisory committee meeting on the application. confirmedas of 2026-09-08

    11. [11]

      Intellia plans a US launch of lonvo-z in the first half of 2027 if it is approved. reportedas of 2026-10-10· forecast

    12. [12]

      Intellia states that hereditary angioedema is estimated to affect one in 50,000 people and that current treatment options often include lifelong therapies. confirmedas of 2026-09-08

    13. [13]

      Nex-z (nexiguran ziclumeran) is Intellia's CRISPR/Cas9 therapy targeting the TTR gene for transthyretin (ATTR) amyloidosis with cardiomyopathy and hereditary ATTR with polyneuropathy, developed with Regeneron. confirmedas of 2026-10-10

    14. [14]

      Intellia's August 2026 quarterly report states that the MAGNITUDE participant who had Grade 4 liver transaminase elevations and increased bilirubin after a nex-z dose died on 5 November 2025, and that the principal investigator reported the cause as septic shock secondary to a perforated duodenal ulcer, with a clinical course that also included acute liver injury treated with corticosteroids and an autopsy report supporting the clinical diagnoses. confirmedas of 2026-08-06

    15. [15]

      As of August 2026 Intellia expected to complete enrollment in MAGNITUDE-2 in the second half of 2026. reportedas of 2026-08-06· forecast

    16. [16]

      In November 2025 CRISPR Therapeutics reported Phase 1 data, published in NEJM, for CTX310, an LNP-delivered CRISPR therapy that edits the ANGPTL3 gene in liver cells; at the highest dose mean ANGPTL3 fell 73%, triglycerides 55% and LDL cholesterol 49%, with no treatment-related serious adverse events reported. confirmedas of 2026-10-10

    17. [17]

      Intellia reported $628.4 million in cash, cash equivalents and marketable securities as of 30 June 2026, which it expects to fund operations at least into 2028, and in September 2026 it announced a non-dilutive debt facility with OrbiMed of up to $400 million. confirmedas of 2026-09-04

    18. [18]

      In August 2026 Intellia said that a genomic analysis of more than 600 patient samples from nex-z trials, run with Regeneron and outside experts, found the highest observed liver transaminase elevations in patients carrying one specific HLA allele, and that it would give HLA genotyping results to investigators and to patients enrolled or entering screening in the ongoing Phase 3 trials. confirmedas of 2026-08-06

    Revision history (2)
    1. Page created.
    2. Refresh: added the published HAELO trial details, lonvo-z's regulatory designations, the HLA finding in nex-z, the reported cause of the 2025 trial death, the March 2026 MAGNITUDE hold lift and the 2026 financing.

    Created Oct 10, 2026. Last reviewed by an editor on Oct 10, 2026. Next scheduled review: Jan 10, 2027.

    Cite this page

    "Intellia Therapeutics." ContentLora, updated Oct 10, 2026. https://contentlora.com/wiki/intellia-therapeutics

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    CRISPR-Cas9 UsesLipid nanoparticle … UsesCRISPR Therapeutics Competitor
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