Drugs / NTLA-2002
Regulatory milestones approvals, filings & regulatory actions · 2 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed | US (FDA) | — | angioedema, hereditary, 4 | 2026-04-27 | “initiated a rolling submission of a biologics license application (BLA) to the U.S. Food and Drug Administration (FDA) seeking approval of lonvo-z” intelliatx.com ↗ |
| Filed | US (FDA) | — | angioedema, hereditary, 4 | 2023-02-23 | “submitted an IND application for NTLA-2002 to the U.S. Food and Drug Administration (FDA)” intelliatx.com ↗ |
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/21 trial · 1 met primary | ||||||
| Phase 1/2 | NCT05120830 | Dec 2021 → Apr 2024 overdue | hereditary angioedema | Intellia Therapeutics | Active not recruiting | Met primary |
| Comparator or background therapy1 trial | ||||||
| Phase 3 | NCT06634420 Comparator | Jan 2025 → Feb 2026 overdue | hereditary angioedema with C1Inh deficiency | Intellia Therapeutics | Active not recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 21
| Date | Issuer | Release |
|---|---|---|
| 2026-09-08 | Intellia Therapeutics | Regulatory Intellia Therapeutics Announces FDA Acceptance of Biologics License Application with Priority Review for Lonvoguran Ziclumeran (Lonvo-z) for Hereditary Angioedema (HAE) intelliatx.com ↗ |
| 2026-06-13 | Intellia Therapeutics | Results Intellia Therapeutics Reports Additional Positive Phase 3 Results for Lonvoguran Ziclumeran (lonvo-z) in Patients with Hereditary Angioedema intelliatx.com ↗
Today, Intellia reported data for the trial’s other key secondary endpoints: |
| 2026-04-27 | Intellia Therapeutics | Results Intellia Therapeutics Reports Positive Phase 3 Results in Hereditary Angioedema, Marking a Global First for In Vivo Gene Editing intelliatx.com ↗
Phase 3 HAELO trial of lonvoguran ziclumeran (lonvo-z) met primary and all key secondary endpoints; favorable safety and tolerability data observed |
| 2026-04-27 | Intellia Therapeutics | Regulatory Intellia Therapeutics Initiates Rolling Submission of Biologics License Application to FDA for Lonvoguran Ziclumeran (lonvo-z) as a One-Time Treatment for Hereditary Angioedema intelliatx.com ↗ |
| 2026-03-03 | Intellia Therapeutics | Results Intellia Therapeutics Presents Longer-Term Clinical Data for Lonvoguran Ziclumeran (lonvo-z); Hereditary Angioedema (HAE) Patient-Focused Research at AAAAI 2026 intelliatx.com ↗
In this pooled Phase 1/2 analysis (n=32), a one-time 50 mg dose of lonvo-z led to deep, stable and durable reductions in plasma kallikrein across all patients with up to three years of follow-up. |
| 2025-11-08 | Intellia Therapeutics | Results Intellia Therapeutics Presents Positive Pooled Phase 1/2 Data of Lonvoguran Ziclumeran (lonvo-z) in Patients with Hereditary Angioedema intelliatx.com ↗
Intellia Therapeutics, Inc. (NASDAQ: NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today presented positive clinical data from a pooled analysis of all patients who received a 50 milligram (mg) dose of lonvo-z in the company’s ongoing Phase 1/2 clinical trial in patients with hereditary angioedema (HAE). |
| 2025-06-15 | Intellia Therapeutics | Results Intellia Therapeutics Announces Positive Three-Year Data from Phase 1 Trial of Lonvoguran Ziclumeran (lonvo-z) in Patients with Hereditary Angioedema (HAE) at the European Academy of Allergy and Clinical Immunology Congress intelliatx.com ↗
With up to three years of follow-up, a single dose of lonvo-z led to a 98% mean reduction in monthly HAE attack rate in all 10 patients |
| 2024-10-24 | Intellia Therapeutics | Results Intellia Presents Positive Results from the Phase 2 Study of NTLA-2002, an Investigational In Vivo CRISPR Gene Editing Treatment for Hereditary Angioedema (HAE) intelliatx.com ↗
Results were published online today in The New England Journal of Medicine and will be presented on Saturday, October 26 at the 2024 American College of Allergy, Asthma & Immunology (ACAAI) Scientific Meeting in Boston, Massachusetts. |
| 2024-06-02 | Intellia Therapeutics | Results Intellia Therapeutics Announces Positive Long-Term Data from Ongoing Phase 1 Study of NTLA-2002, an Investigational In Vivo CRISPR Gene Editing Treatment for Hereditary Angioedema (HAE) intelliatx.com ↗
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced long-term data from the Phase 1 portion of the ongoing Phase 1/2 study of NTLA-2002. |
| 2024-01-31 | Intellia Therapeutics | Results Intellia Therapeutics Announces Publication of Positive Interim Phase 1 Data for NTLA-2002 in Patients with Hereditary Angioedema in the New England Journal of Medicine intelliatx.com ↗
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced that interim results from the Phase 1 portion of the Phase 1/2 study of NTLA-2002 were published online in the New England Journal of Medicine (NEJM). |
| 2023-11-14 | Intellia Therapeutics | Regulatory Intellia Therapeutics Receives European Union Orphan Drug Designation for NTLA-2002, an Investigational In Vivo CRISPR Genome Editing Treatment for Hereditary Angioedema intelliatx.com ↗
today announced that the European Commission (EC) has granted orphan drug designation to NTLA-2002 for the treatment of hereditary angioedema (HAE). |
| 2023-10-13 | Intellia Therapeutics | Regulatory Intellia Therapeutics Receives Priority Medicines (PRIME) Designation From the European Medicines Agency for NTLA-2002, an Investigational In Vivo CRISPR Genome Editing Treatment for Hereditary Angioedema intelliatx.com ↗
CAMBRIDGE, Mass., Oct. 13, 2023 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing potentially curative therapeutics leveraging CRISPR-based technologies, today announced that the European Medicines Agency (EMA) has granted Priority Medicine (PRIME) designation to NTLA-2002 for the treatment of hereditary angioedema (HAE). |
| 2023-06-11 | Intellia Therapeutics | Results Intellia Therapeutics Announces New Positive Clinical Data from Phase 1 Study of NTLA-2002, an Investigational In Vivo CRISPR Genome Editing Treatment for Hereditary Angioedema (HAE) intelliatx.com ↗ |
| 2023-03-21 | Intellia Therapeutics | Regulatory Intellia Therapeutics Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to NTLA-2002 for the Treatment of Hereditary Angioedema intelliatx.com ↗
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing potentially curative therapies leveraging CRISPR-based technologies, today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to NTLA-2002 for the treatment of hereditary angioedema (HAE). |
| 2023-03-02 | Intellia Therapeutics | Regulatory Intellia Therapeutics Announces FDA Clearance of Investigational New Drug (IND) Application for NTLA-2002, an In Vivo CRISPR-Based Investigational Therapy for the Treatment of Hereditary Angioedema (HAE) intelliatx.com ↗ |
| 2023-01-11 | Intellia Therapeutics | Regulatory Intellia Therapeutics Awarded Innovation Passport in the United Kingdom for NTLA-2002, an Investigational Genome Editing Treatment for Hereditary Angioedema intelliatx.com ↗ |
| 2022-11-12 | Intellia Therapeutics | Results Intellia Therapeutics Presents New Interim Data from First-in-Human Study of NTLA-2002 for the Treatment of Hereditary Angioedema (HAE) at the American College of Allergy, Asthma & Immunology 2022 Annual Scientific Meeting intelliatx.com ↗
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing potentially curative therapeutics leveraging CRISPR-based technologies, today announced updated data from an ongoing Phase 1/2 clinical study of NTLA-2002 for the treatment of hereditary angioedema (HAE). |
| 2022-09-16 | Intellia Therapeutics | Results Intellia Therapeutics Announces Positive Interim Clinical Data for its Second Systemically Delivered Investigational CRISPR Candidate, NTLA-2002 for the Treatment of Hereditary Angioedema (HAE) intelliatx.com ↗ |
| 2022-09-01 | Intellia Therapeutics | Regulatory Intellia Therapeutics Receives U.S. FDA Orphan Drug Designation for NTLA-2002, an Investigational CRISPR Therapy for the Treatment of Hereditary Angioedema intelliatx.com ↗
today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for Intellia’s in vivo CRISPR/Cas9 genome editing candidate, NTLA-2002, for the treatment of hereditary angioedema (HAE). |
| 2021-10-06 | Intellia Therapeutics | Regulatory Intellia Therapeutics Receives Authorization to Initiate Phase 1/2 Clinical Trial of NTLA-2002 for the Treatment of Hereditary Angioedema intelliatx.com ↗
today announced the authorization of its Clinical Trial Application (CTA) by the New Zealand Medicines and Medical Devices Safety Authority (MEDSAFE) to initiate a Phase 1/2 study evaluating NTLA-2002 for the treatment of adults with hereditary angioedema (HAE). |
| 2020-05-12 | Intellia Therapeutics | Results Intellia Therapeutics Reports Progress on CRISPR/Cas9 AML Cancer Therapy Using Proprietary Cell Engineering Process at the 23rd Annual Meeting of the American Society of Gene and Cell Therapy intelliatx.com ↗
Intellia is also providing an update on NTLA-2002, its newest development candidate for the treatment of hereditary angioedema (HAE). |
All press releases naming this drug 34 releases
Evidence & citations 7 cited values
Every value below carries the sentence it was read from. 4 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | NTLA-2002 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT06634420 ↗ |
| Known as | lonvo-z | “Lonvoguran ziclumeran (lonvo-z) - an investigational, in vivo gene-editing treatment based on clustered regularly interspaced short palindromic repeats (CRISPR) - is being...” PMID 42294842 ↗ Jun 2026 |
| Known as | lonvoguran ziclumeran | “Lonvoguran ziclumeran (lonvo-z) - an investigational, in vivo gene-editing treatment based on clustered regularly interspaced short palindromic repeats (CRISPR) - is being...” PMID 42294842 ↗ Jun 2026 |
| Action | Inhibit | “NTLA-2002 administered in a single dose of 25 mg or 50 mg reduced angioedema attacks and led to robust and sustained reduction in total plasma kallikrein levels in patients...” PMID 39445704 ↗ Oct 2024 |
| Modality | CRISPR / gene editing | “CRISPR/Cas9 gene editing system delivered by LNP for IV administration” NCT05120830 ↗ |
| Route | Intravenous | “CRISPR/Cas9 gene editing system delivered by LNP for IV administration” NCT05120830 ↗ |
| Target | KLKB1 | “NTLA-2002 targets the gene encoding kallikrein B1 (KLKB1).” PMID 39445704 ↗ Oct 2024 |