HAELO: A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2002 in Participants With Hereditary Angioedema (HAE)

This Phase 3 study aims to evaluate the efficacy and safety of NTLA-2002 compared to placebo in participants with HAE.

Start Date15 Jan 2025

Sponsor / Collaborator

Intellia Therapeutics, Inc.

NCT05120830

/ Active, not recruitingPhase 1/2

Phase 1/2 Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2002 in Adults With Hereditary Angioedema (HAE)

This study will be conducted to evaluate the safety, tolerability, activity, pharmacokinetics, and pharmacodynamics of NTLA-2002 in adults with Hereditary Angioedema (HAE).

Start Date10 Dec 2021

Sponsor / Collaborator

Intellia Therapeutics, Inc.

100 Clinical Results associated with Lonvoguran

100 Translational Medicine associated with Lonvoguran

100 Patents (Medical) associated with Lonvoguran

Literatures (Medical) associated with Lonvoguran

15 Oct 2026·NEW ENGLAND JOURNAL OF MEDICINE

Lonvoguran Ziclumeran — In Vivo CRISPR Gene Editing in Hereditary Angioedema

Article

Author: Aygören-Pürsün, Emel ; Xu, Yuanxin ; Shah, Mrinal Y. ; Peter, Jonny ; Craig, Timothy J. ; Sutherland, Andrea ; Lebwohl, David ; Banerji, Aleena ; Lumry, William R. ; Riedl, Marc A. ; Butler, James S. ; Maag, David ; Leonard, John ; Abdelhady, Ahmed M. ; Cohn, Danny M. ; Farkas, Henriette ; Longhurst, Hilary J. ; Miller, Catherine R. ; Golden, Adele ; Jacobs, Joshua ; Gurugama, Padmalal ; Magerl, Markus

BACKGROUND:

Hereditary angioedema is a rare and potentially life-threatening genetic condition characterized by recurrent and debilitating swelling attacks. Lonvoguran ziclumeran (lonvo-z) - an investigational, in vivo gene-editing treatment based on clustered regularly interspaced short palindromic repeats (CRISPR) - is being developed as a single-dose treatment for hereditary angioedema.

METHODS:

In a phase 3, double-blind trial, we randomly assigned, in a 2:1 ratio, patients who were 16 years of age or older and had hereditary angioedema with C1 inhibitor deficiency to receive a single intravenous infusion of lonvo-z (at a dose of 50 mg) or placebo. The primary end point was the number of investigator-confirmed hereditary angioedema attacks per month (the monthly attack rate) from week 5 through week 28 after infusion.

RESULTS:

A total of 80 patients underwent randomization; 52 were assigned to receive lonvo-z and 28 to receive placebo. As of February 10, 2026, the median duration of follow-up was 7.5 months (range, 4.9 to 12.8). The least-squares mean monthly attack rate from weeks 5 through 28 was 0.26 (95% confidence interval [CI], 0.15 to 0.45) in the lonvo-z group and 2.10 (95% CI, 1.55 to 2.86) in the placebo group (relative difference, -87%; 95% CI, -93 to -78; P<0.001). Adverse events that occurred during or after infusion of lonvo-z or placebo were reported in 92% and 86% of the patients in the lonvo-z and placebo groups, respectively, from day 1 through week 28. Among the adverse events that occurred during or after infusion and more often in the lonvo-z group than in the placebo group, the most common (occurring in >10% of the patients in the lonvo-z group) included infusion-related reaction, headache, fatigue, back pain, and upper respiratory tract infection. No serious or grade 3 or higher adverse events were reported in the lonvo-z group.

CONCLUSIONS:

Among patients with hereditary angioedema, a single intravenous infusion of lonvo-z resulted in a significantly lower rate of hereditary angioedema attacks than placebo. (Funded by Intellia Therapeutics; HAELO ClinicalTrials.gov number, NCT06634420.).

01 Oct 2026·Med

Genome editing for hereditary angioedema: A landmark trial and important unanswered questions

Article

Author: Margaglione, Maurizio ; Cancian, Mauro

Hereditary angioedema is a bradykinin-mediated genetic disorder increasingly controlled with effective long-term prophylaxis. In a phase 3 trial, a single dose of lonvoguran ziclumeran, an in vivo CRISPR-Cas9 therapy targeting KLKB1, significantly reduced attack rates. This approach represents a highly promising therapeutic strategy for HAE. However, it also raises important questions that require further investigation.

01 Sep 2026·Dermatologie (Heidelberg, Germany)

Article

Author: Dominas, Nina ; Fasshauer, Maria

Bradykinin-mediated angioedema, particularly hereditary angioedema due to C1 esterase inhibitor deficiency or dysfunction (HAE-C1INH), is caused by dysregulation of the kallikrein-kinin system with excessive bradykinin generation and subsequent increased vascular permeability. Modern HAE management is based on three major therapeutic strategies: on-demand treatment, short-term prophylaxis (STP), and long-term prophylaxis (LTP). On-demand treatment options include plasma-derived and recombinant C1 inhibitor (C1INH) concentrates, the bradykinin B2 receptor antagonist icatibant, and, more recently, the first orally available plasma kallikrein inhibitor, sebetralstat. Short-term prophylaxis prior to invasive medical procedures is performed as replacement therapy by intravenous administration of C1INH concentrates. The goal of long-term prophylaxis is complete disease control with reduction of attack frequency and/or severity and improvement of quality of life. LTP therapies include subcutaneous and intravenous C1INH preparations, the oral kallikrein inhibitor berotralstat, the anti-kallikrein monoclonal antibody lanadelumab, the factor XIIa inhibitor garadacimab, and the antisense oligonucleotide donidalorsen. Currently under development are the oral bradykinin B2 receptor antagonist deucrictibant, which is intended for both on-demand treatment and long-term prophylaxis in different formulations, long-acting antibodies, such as navenibart, and CRISPR/Cas9-based gene-editing therapies, such as NTLA-2002 with potential functional curative properties. In particular, orally available and long-acting therapies are expected to improve adherence, self-management, and quality of life in affected patients.

127

News (Medical) associated with Lonvoguran

29 Oct 2026

·www.biospace.com

Intellia Therapeutics Announces Upcoming Lonvoguran Ziclumeran (Lonvo-z) Investor Event and Seven Presentations at ACAAI 2026

Oral presentations to include four-year follow-up data from patients who received a one-time 50 milligram (mg) dose of lonvo-z; insights on new hereditary angioedema (HAE) treatment goals Poster presentations to include patient perspective about life after lonvo-z treatment Intellia to host investor webcast on Thursday, November 12 at 10 a.m. ET CAMBRIDGE, Mass., Oct. 29, 2026 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (Nasdaq: NTLA), a leading biopharmaceutical company focused on revolutionizing medicine leveraging CRISPR gene editing and other core technologies, today announced two oral presentations and five poster presentations at the American College of Allergy, Asthma & Immunology (ACAAI) 2026 Annual Scientific Meeting, taking place November 12-16 in Phoenix, Arizona. Intellia also announced that it will host an investor webcast on Thursday, November 12, at 8 a.m. MT / 10 a.m. ET to share highlights from the presentations, provide perspectives from HAE key opinion leaders and discuss the company’s preparations for a planned commercial launch of lonvo-z in HAE. Oral Presentation Details: Title: Four-Year Durability/Safety of Lonvoguran Ziclumeran (Lonvo-z) 50 mg in Patients with Hereditary Angioedema Session: Distinguished Industry and Late-Breaking Oral Abstracts – Session 2 Date and Time: Saturday, November 14, 2026, from 4:43 – 4:53 p.m. MT Presenter: Danny Cohn, M.D., Ph.D., Associate Professor, Amsterdam University Medical Center, Department of Vascular Medicine, University of Amsterdam; Phase 1/2 and HAELO principal investigator Title: Attack-Free and Long-Term Prophylaxis-Free Status Following Treatment with Lonvoguran Ziclumeran in Hereditary Angioedema Session: Distinguished Industry and Late-Breaking Oral Abstracts – Session 2 Date and Time: Saturday, November 14, 2026, from 4:53 – 5:03 p.m. MT Presenter: Emel Aygören-Pürsün, M.D., Department for Children and Adolescents, Angioedema Centre, University Hospital Frankfurt, Goethe University; Phase 1/2 and HAELO principal investigator Poster Presentation Details: Title: Lonvoguran Ziclumeran Demonstrated Consistent Benefit in All Hereditary Angioedema Patient Subgroups in Ph3 HAELO Study Poster Session: Monitor 17 Date and Time: Friday, November 13, 2026, from 3:05 – 3:20 p.m. MT Presenter: Marc Riedl, M.D., Professor of Medicine, Clinical Director of the U.S. Hereditary Angioedema Association (HAEA) Angioedema Center, University of California San Diego; HAELO principal investigator Title: Lived Experiences of Patients Treated with Lonvoguran Ziclumeran, a One-Time CRISPR Treatment for Hereditary Angioedema Poster Session: Monitor 17 Date and Time: Friday, November 13, 2026, from 3:20 – 3:35 p.m. MT Presenter: Padmalal Gurugama, MBBS, M.S., M.D., MRCP, FRCPath, Consultant, Clinical Immunology and Allergy, Cambridge University Hospitals NHS Foundation Trust; Phase 1/2 and HAELO principal investigator Title: Long-Term Prophylaxis for Hereditary Angioedema: Treatment Use, Patterns, and Gaps in a U.S. Claims Database Poster Session: Monitor 18 Date and Time: Friday, November 13, 2026, from 4:05 – 4:20 p.m. MT Presenter: Raffi Tachdjian, M.D., M.P.H., Associate Clinical Professor of Medicine and Pediatrics, Division of Allergy and Clinical Immunology, David Geffen School of Medicine, University of Los Angeles; HAELO principal investigator Title: A One-Time Treatment of Lonvoguran Ziclumeran Improves Quality of Life in Patients with Hereditary Angioedema Poster Session: Monitor 17 Date and Time: Friday, November 13, 2026, from 4:35 – 4:50 p.m. MT Presenter: William Lumry, M.D., Clinical Professor of Internal Medicine, Allergy and Immunology Division, University of Texas Southwestern Medical School, Medical Director, AARA Research Center; HAELO principal investigator Title: Integrated Analysis of Lonvoguran Ziclumeran Phase 1/2/3 Studies in Hereditary Angioedema: Efficacy, Safety, and Tolerability Poster Session: Monitor 16 Date and Time: Friday, November 13, 2026, from 5:35 – 5:50 p.m. MT Presenter: Aleena Banerji, M.D., Professor at Harvard Medical School, Director of Clinical Care, Center for Drug and Vaccine Allergy at Massachusetts General Hospital; HAELO principal investigator The presentations will be made available on the Scientific Publications & Presentations page of at the start of each respective session. Intellia Therapeutics Investor Webcast Intellia will host a live webcast on Thursday, November 12, 2026, at 8 a.m. MT / 10 a.m. ET to review data from the ACAAI presentations, share perspectives from key opinion leaders and discuss the company’s preparations for a planned commercial launch of lonvo-z in HAE. To join the webcast, visit the Events page of the Investors & Media section of the company’s website at . A replay of the webcast will be available on Intellia’s website for approximately 90 days following the call. About Lonvo-z Based on Nobel Prize-winning CRISPR/Cas9 technology, lonvo-z has the potential to become the first one-time treatment for hereditary angioedema (HAE). Lonvo-z is an in vivo CRISPR gene editing candidate that is intended to permanently lower kallikrein by inactivating the kallikrein B1 ( KLKB1 ) gene with a single dose that is administered in an outpatient setting. Lonvo-z has received five notable regulatory designations: Orphan Drug and Regenerative Medicine Advanced Therapy (RMAT) Designations by the U.S. Food and Drug Administration (FDA), the Innovation Passport by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA), Priority Medicines (PRIME) Designation by the European Medicines Agency, as well as Orphan Drug Designation (ODD) by the European Commission. In September 2026, the FDA accepted the Biologics License Application (BLA) for lonvo-z and granted Priority Review with a PDUFA target action date of March 10, 2027. About Hereditary Angioedema Hereditary angioedema (HAE) is a rare, genetic disease characterized by severe, recurring and unpredictable inflammatory attacks in various organs and tissues of the body, which can be painful, debilitating and life-threatening. It is estimated that one in 50,000 people are affected by HAE. There are preventative and on-demand treatment options to help manage the condition, including long- and short-term prophylaxis used to prevent swelling attacks. Current treatment options often include lifelong therapies, which may require chronic intravenous (IV) or subcutaneous (SC) administration as often as twice per week or daily oral administration to ensure constant pathway suppression for disease control. Despite chronic administration, breakthrough attacks still occur. Kallikrein inhibition is a clinically validated strategy for the preventive treatment of HAE attacks. About Intellia Therapeutics Intellia Therapeutics, Inc. (Nasdaq: NTLA) is a leading biopharmaceutical company focused on revolutionizing medicine leveraging CRISPR gene editing and other core technologies. The company’s mission is to transform the lives of people with severe diseases by developing and commercializing potentially curative treatments. With deep scientific, technical and clinical development experience, Intellia aims to reset the standard for medicine by durably treating the root causes of disease. Learn more at intelliatx.com and follow us @intelliatx . Forward-Looking Statements This press release contains “forward-looking statements” of Intellia Therapeutics, Inc. (“Intellia” or the “Company”) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellia’s beliefs and expectations concerning: the success and advancement of its program for lonvoguran ziclumeran (“lonvo-z”) for the treatment of hereditary angioedema (“HAE”), including its expectations regarding review and approval of its biologics license application (“BLA”) for lonvo-z, such as the timing of such review and approval based on the Prescription Drug User Fee Act ("PDUFA") target action date of March 10, 2027 for the BLA; its belief that lonvo-z could permanently lower kallikrein by inactivating the kallikrein B1 (KLKB1) gene with a single dose that is administered in an outpatient setting; and its expectations regarding its preparations for and the potential success of the commercial launch of lonvo-z, if approved. Any forward-looking statements in this press release are based on management’s current expectations and beliefs of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: uncertainties related to the conduct of clinical studies and other development and commercialization requirements for its product candidates, including lonvo-z, including risks related to the review and approval of the BLA for lonvo-z and the ability to develop and successfully commercialize lonvo-z or any of Intellia’s product candidates; risks related to Intellia’s ability to protect and maintain its intellectual property position; risks related to Intellia’s relationship with third parties, including its contract manufacturers, collaborators, licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; risks related to the results of preclinical studies or clinical studies not being predictive of future results in connection with future studies; the risk that clinical study results will not be positive; and risks related to the potential delay of planned clinical trials due to regulatory feedback or other developments. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellia’s actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in Intellia’s most recent annual report on Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in Intellia’s other filings with the Securities and Exchange Commission, including its recent quarterly report on Form 10-Q. All information in this press release is as of the date of the release, and Intellia undertakes no duty to update this information unless required by law. Investor Contact: Jason Fredette Vice President, Investor Relations and Corporate Communications Intellia Therapeutics, Inc. jason.fredette@intelliatx.com Media Contact: Mike Tattory Vice President LifeSci Communications mtattory@lifescicomms.com

Orphan DrugPriority ReviewBreakthrough Therapy

13 Oct 2026

·www.biospace.com

Intellia Therapeutics Appoints Jing L. Marantz, M.D., Ph.D., as Chief Medical Officer

CAMBRIDGE, Mass., Oct. 13, 2026 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (Nasdaq: NTLA), a leading biopharmaceutical company focused on revolutionizing medicine leveraging CRISPR gene editing and other core technologies, today announced the appointment of Jing L. Marantz, M.D., Ph.D., as Executive Vice President and Chief Medical Officer, effective October 26, 2026. Dr. Marantz is an accomplished drug development leader with more than 25 years of experience in the biopharmaceutical industry that includes the successful advancement of several now-approved rare disease therapies. In this role, Dr. Marantz will lead the company’s translational, development, regulatory and medical affairs functions. “Jing is an exceptional leader with a track record of success in the rare disease space across the full lifecycle from development through regulatory approval and commercialization,” said John Leonard, M.D., Intellia President and Chief Executive Officer. “We are delighted to welcome her as our Chief Medical Officer during this exciting and momentous time at Intellia. Her unique combination of development and business experience will be critical for our next phase as we seek to commercialize our first potential in vivo gene editing product, advance our Phase 3 clinical trials in transthyretin amyloidosis and further expand our pipeline.” “I am thrilled to join Intellia, where we have the opportunity to profoundly improve patient lives by delivering durable efficacy while minimizing treatment burden leveraging Nobel-prize-winning science,” said Dr. Marantz. “I look forward to working with the company’s talented team to advance our clinical programs with the goal of bringing new therapeutic options to patients with serious diseases.” Dr. Marantz’s leadership experience spans research and development, medical affairs, business development and portfolio strategy across multiple specialties and rare diseases. Most recently, she served as Chief Medical Officer for Scholar Rock, where she led the development organization in advancing multiple clinical programs across neuromuscular, oncology and obesity, culminating in the U.S. approval of ISEMBYLD, the first muscle-targeted therapy for Spinal Muscular Atrophy. During her tenure at Scholar Rock, Dr. Marantz led simultaneous regulatory submissions to the U.S. Food and Drug Administration and European Medicines Agency followed by successful engagement with Japan’s Pharmaceuticals and Medical Devices Agency, achieving alignment for a Japanese New Drug Application submission without clinical trial data in Japanese patients. Prior to her role at Scholar Rock, Dr. Marantz held senior leadership positions at Krystal Biotech, Acceleron, Alnylam, Alexion, Biogen, ARIAD and Millennium Pharmaceuticals, playing key roles in the development and commercialization of over a dozen products across hematology/oncology, neurology and rare diseases. She is a member of the Board of Directors for Arcturus Therapeutics, Inc. Dr. Marantz received her medical training from Tongji Medical College, a Ph.D. in Biochemistry and Molecular Biology from the Medical University of South Carolina, and an MBA from the University of California at Berkeley. About Intellia Therapeutics Intellia Therapeutics, Inc. (Nasdaq: NTLA) is a leading biopharmaceutical company focused on revolutionizing medicine leveraging CRISPR gene editing and other core technologies. The company’s mission is to transform the lives of people with severe diseases by developing and commercializing potentially curative treatments. With deep scientific, technical and clinical development experience, Intellia aims to reset the standard for medicine by durably treating the root causes of disease. Learn more at intelliatx.com and follow us @intelliatx . Forward-Looking Statements This press release contains “forward-looking statements” of Intellia Therapeutics, Inc. (“Intellia” or the “Company”) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellia’s beliefs and expectations concerning: its ability, and the impact of its executive leadership on its ability to commercialize lonvoguran ziclumeran (“lonvo-z”), its first potential in vivo gene editing product, advance its Phase 3 clinical trials of nexiguran ziclumeran (“nex-z”) in transthyretin amyloidosis and further expand its pipeline. Any forward-looking statements in this press release are based on management’s current expectations and beliefs of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: uncertainties related to the conduct of clinical studies and other development and commercialization requirements for its product candidates, including lonvo-z and nex-z, including risks related to the ability to develop and successfully commercialize lonvo-z, nex-z or any of Intellia’s product candidates; risks related to Intellia’s ability to protect and maintain its intellectual property position; risks related to Intellia’s relationship with third parties, including its contract manufacturers, collaborators, licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; risks related to the results of preclinical studies or clinical studies not being predictive of future results in connection with future studies; the risk that clinical study results will not be positive; and risks related to the potential delay of planned clinical trials or regulatory filings due to regulatory feedback or other developments. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellia’s actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in Intellia’s most recent annual report on Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in Intellia’s other filings with the Securities and Exchange Commission, including its quarterly report on Form 10-Q. All information in this press release is as of the date of the release, and Intellia undertakes no duty to update this information unless required by law. Investor Contact: Jason Fredette Vice President, Investor Relations and Corporate Communications Intellia Therapeutics, Inc. jason.fredette@intelliatx.com Media Contact: Mike Tattory Vice President LifeSci Communications mtattory@lifescicomms.com This press release was published by a CLEAR® Verified individual.

Executive ChangePhase 3Gene Therapy

08 Sep 2026

·www.fiercebiotech.com

Pharvaris' latest phase 3 win builds case for HAE drug with FDA verdict pending

Pharvaris’ deucrictibant has tasted success in a second phase 3 study, strengthening the case for the therapy to treat hereditary angioedema (HAE) patients as the Swiss biotech awaits an FDA approval decision. The biotech has been evaluating a 40-mg once-daily extended-release formulation of its oral bradykinin B2 receptor antagonist against placebo in the late-stage CHAPTER-3 study. The trial enrolled 85 patients with three types of HAE: HAE type 1, HAE type 2 or HAE with normal C1 inhibitor. The study hit its primary endpoint by demonstrating an 83% reduction in the rate of monthly attacks compared with placebo after 24 weeks, Pharvaris reported in a Sept. 8 release . This reduction was boosted to 87% when looking specifically at the 80 patients with HAE type 1 or 2, the Swiss biotech noted. Deucrictibant also aced all of the secondary endpoints (PDF), according to Pharvaris, including the number of attacks treated with on-demand medication, the number of severe attacks and the proportion of time without angioedema symptoms. The Zug, Switzerland-based company filed an immediate-release formulation of deucrictibant for FDA approval in July to treat HAE attacks, with the agency setting an April 23, 2027, decision date. If approved, deucrictibant will give HAE patients an alternative to KalVista’s Ekterly, an existing oral HAE attack treatment. That approval submission followed a separate phase 3 win for immediate-release deucrictibant in December 2025, when the drug demonstrated its potential as an on-demand treatment for episodes of swelling and related symptoms that affect people with HAE. The company also plans to submit an NDA for deucrictibant as a prophylactic for bradykinin-mediated angioedema attacks in the first half of 2027. Pharvaris CEO Berndt Modig said this morning’s readout represented a “landmark moment for the company and the community as a whole.” “Deucrictibant could be the first and only oral therapy to offer injectable-like efficacy and a well-tolerated profile in on-demand treatment and prophylaxis with our two unique formulations,” Modig explained. “Pharvaris now plans to redefine disease management by uniting on-demand treatment and long-term prophylaxis within a single therapeutic franchise.” Pharvaris’ stock was trading up 14% at $41.44 after markets opened, compared with a $35.25 closing price on Friday. HAE is a rare genetic condition that causes recurrent, unpredictable and potentially life-threatening swelling of the face, limbs, gastrointestinal tract and airways. Intellia has its own HAE option in the works courtesy of the in vivo gene-editing therapy lonvoguran ziclumeran, which also saw phase 3 success earlier this year.

Phase 3Clinical ResultNDAGene TherapyDrug Approval

100 Deals associated with Lonvoguran

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Indication	Highest Phase	Country/Location	Organization	Date
Hereditary Angioedema	NDA/BLA	United States	Intellia Therapeutics, Inc.	27 Apr 2026
Hereditary Angioedema Types I and II	Phase 2	Australia	Intellia Therapeutics, Inc.	10 Dec 2021
Hereditary Angioedema Types I and II	Phase 2	France	Intellia Therapeutics, Inc.	10 Dec 2021
Hereditary Angioedema Types I and II	Phase 2	Germany	Intellia Therapeutics, Inc.	10 Dec 2021
Hereditary Angioedema Types I and II	Phase 2	Netherlands	Intellia Therapeutics, Inc.	10 Dec 2021
Hereditary Angioedema Types I and II	Phase 2	New Zealand	Intellia Therapeutics, Inc.	10 Dec 2021
Hereditary Angioedema Types I and II	Phase 2	United Kingdom	Intellia Therapeutics, Inc.	10 Dec 2021

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT06634420 (HAELO, Pubmed \| N Engl J Med)	Phase 3	Hereditary Angioedema	80	Lonvoguran ziclumeran (lonvo-z) 50 mg	kfelqlplnh(apahbhikyu) = qyrlwhhlok whmffarjoh (gumyrxfdks, 0.15 - 0.45) View more	Positive	15 Oct 2026
				Placebo	kfelqlplnh(apahbhikyu) = ygfvcntcbd whmffarjoh (gumyrxfdks, 1.55 - 2.86) View more
NCT05120830 (AAAAI2026)	Phase 1/2	Hereditary Angioedema	21	Lonvo-z 50mg	xwognjkavf(icnqqhnkur) = mnkgayzkhj vfhvrpeyzy (vekyemvxci ) View more	Positive	27 Feb 2026
NCT05120830 \| NCT06262399 (Company_Website \| Corporate Publications) Manual	Phase 3	Hereditary Angioedema	32	Lonvoguran Ziclumeran (Pooled analysis of 50 mg dose)	yeiykgqmjw(nnjjnwjbkk) = hyblkzpgxn mpvrlfjgyi (itcbkpcofv ) View more	Positive	08 Nov 2025
NCT05120830 (Company_Website) Manual	Phase 1/2	Hereditary Angioedema	10	lonvo-z (25 mg (N=3), 50 mg (N=4) or 75 mg (N=3))	imnyjkskim(pyzkhqsqqg) = vbhmunovuv wgmltadbnz (zfwpckewba )	Positive	07 Aug 2025
NCT05120830 (AAAAI2025)	Phase 1/2	Hereditary Angioedema	27	NTLA-2002 50mg	ymkcqfsjwt(iyinlrkzmv) = ppprnunpua zyyutcvcxg (jtwqupnlus, 17.15)	Positive	28 Feb 2025
				Placebo	ymkcqfsjwt(iyinlrkzmv) = ezlocnxlaj zyyutcvcxg (jtwqupnlus, 10.39)
NCT05120830 (Pubmed) Manual	Phase 2	Hereditary Angioedema	27	NTLA-2002NTLA-2002 25 mg	bjwusfysoz(cxtgdleqmc) = jnycergred ssfcdrdgrn (jcdxlnkxls, 0.25 - 1.98) View more	Positive	24 Oct 2024
				NTLA-2002NTLA-2002 50 mg	bjwusfysoz(cxtgdleqmc) = dhffztrmdd ssfcdrdgrn (jcdxlnkxls, 0.24 - 1.76) View more
NCT05120830 (NTLA-2002, ACAAI2024)	Phase 2	Hereditary Angioedema Plasma kallikrein	27	NTLA-2002 25mg	fqolxguxmd(ibpcnhqwkh) = 29% gsnftqyink (frypgxxggh ) View more	Positive	24 Oct 2024
				NTLA-2002 50mg
ISTH2024	Phase 1/2	Hereditary Angioedema	10	NTLA-2002 25mg	gxdgxiohve(wcjgimqril) = The most frequent adverse events (AEs) were infusion-related reactions and fatigue. No treatment-emergent serious AEs or ≥Grade 3 AEs were observed. No patients experienced thromboembolic or bleeding events. lxfesucngj (aopdmnqpqa ) View more	Positive	25 Jun 2024
				NTLA-2002 50mg
NCT05120830 (Company_Website) Manual	Phase 1	Hereditary Angioedema	10	NTLA-2002 (Week 1-16)	guheeyfoiw(pkjgndauux) = jyqoooaalx xsjldqjotr (pqxxdvaxbo )	Positive	02 Jun 2024
				NTLA-2002 (Week 5-16)	guheeyfoiw(pkjgndauux) = wnygztexyb xsjldqjotr (pqxxdvaxbo )
NCT05120830 (Literature) Manual	Phase 1	Hereditary Angioedema	10	NTLA-2002 25 mg	ynjndrxkee(mdqzcvuwgm) = xhgjwynbby ugqqwhoqqq (zlkdkxinzp ) View more	Positive	01 Feb 2024
				NTLA-2002 50 mg	ynjndrxkee(mdqzcvuwgm) = ehtviszedi ugqqwhoqqq (zlkdkxinzp ) View more

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