预约演示
更新于：2026-04-04
Lonvoguran
更新于：2026-04-04
概要
基本信息
非在研机构- |
权益机构- |
最高研发阶段临床3期 |
首次获批日期- |
最高研发阶段(中国)- |
特殊审评孤儿药 (美国)、孤儿药 (欧盟)、再生医学先进疗法 (美国)、优先药物（PRIME） (欧盟) |
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结构/序列
Sequence Code 1116750154
来源: *****
关联
3
项与 Lonvoguran 相关的临床试验NL-OMON58260
Lived Experiences of Clinical Trial Participants Treated with NTLA-2002 – A Patient Interview Study - A Patient Interview Study
NCT06634420
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)
NCT05120830
Phase 1/2 Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2002 in Adults With Hereditary Angioedema (HAE)
100 项与 Lonvoguran 相关的临床结果
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100 项与 Lonvoguran 相关的转化医学
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100 项与 Lonvoguran 相关的专利（医药）
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4
项与 Lonvoguran 相关的文献（医药）2025-12-01Annals of Medicine and Surgery
CRISPR-Cas9 gene editing for hereditary angioedema: current treatments and emerging therapies
Review
作者: Taimuri, Muskan Asim ; Sumbal, Anusha ; Ali, Tehreem ; Jalal, Laiba ; Alam, Safa ; Yokolo, Hermann ; Khan, Ayesha ; Ikram, Areeba ; Arama, Umulkhairah Onyioiza
Hereditary angioedema (HAE) is a rare autosomal dominant disorder marked by episodic, non-urticarial swelling due to C1 esterase inhibitor (C1-INH) deficiency or dysfunction, leading to excessive bradykinin-mediated vascular permeability. While current treatments focus on symptomatic control using C1-INH replacement or kallikrein inhibitors, they require frequent administration and do not address the underlying genetic defect. CRISPR-Cas9 gene-editing technologies, particularly the investigational therapy NTLA-2002, offer a transformative approach by targeting the
KLKB1
gene to durably reduce plasma kallikrein levels. This narrative review summarizes the pathophysiology and conventional treatments of HAE, and highlights emerging clinical evidence supporting the safety and efficacy of NTLA-2002. Preliminary trials demonstrate substantial reductions in HAE attack frequency and plasma kallikrein, with minimal adverse events. However, concerns about long-term safety, off-target effects, ethical implications, and accessibility remain. CRISPR-based therapeutics such as NTLA-2002 represent a paradigm shift in the management of HAE and underscore the broader potential of
in vivo
gene editing for genetic disorders.
2025-01-30NEW ENGLAND JOURNAL OF MEDICINE
CRISPR-Based Therapy for Hereditary Angioedema
Article
作者: Aygören-Pürsün, Emel ; Lindsay, Karen ; Launay, David ; Shah, Mrinal Y ; Bouillet, Laurence ; Xu, Yuanxin ; Cohn, Danny M ; Katelaris, Constance H ; Maag, David ; Petersen, Remy S ; Butler, James S ; Longhurst, Hilary J ; Magerl, Markus ; Abdelhady, Ahmed M ; Golden, Adele ; Gurugama, Padmalal ; Lebwohl, David
BACKGROUND:
Hereditary angioedema is a rare genetic disease characterized by severe and unpredictable swelling attacks. NTLA-2002 is an in vivo gene-editing therapy that is based on clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein 9. NTLA-2002 targets the gene encoding kallikrein B1 (KLKB1). A single dose of NTLA-2002 may provide lifelong control of angioedema attacks.
METHODS:
In this phase 2 portion of a phase 1-2 trial, we randomly assigned adults with hereditary angioedema in a 2:2:1 ratio to receive NTLA-2002 in a single dose of 25 mg or 50 mg or placebo. The primary end point was the number of angioedema attacks per month (the monthly attack rate) from week 1 through week 16. Secondary end points included safety, pharmacokinetics, and pharmacodynamics (i.e., the change from baseline in total plasma kallikrein protein level); exploratory end points included patient-reported outcomes.
RESULTS:
Of the 27 patients who underwent randomization, 10 received 25 mg of NTLA-2002, 11 received 50 mg, and 6 received placebo. From week 1 through week 16, the estimated mean monthly attack rate was 0.70 (95% confidence interval [CI], 0.25 to 1.98) with 25 mg of NTLA-2002, 0.65 (95% CI, 0.24 to 1.76) with 50 mg, and 2.82 (95% CI, 0.80 to 9.89) with placebo; the difference in the estimated mean attack rate with NTLA-2002 as compared with placebo was -75% with 25 mg and -77% with 50 mg. Among patients who received NTLA-2002, 4 of the 10 patients who received 25 mg (40%) and 8 of the 11 who received 50 mg (73%) were attack-free with no additional treatment during the period from week 1 through week 16. The most common adverse events among patients who received NTLA-2002 were headache, fatigue, and nasopharyngitis. The mean percent change in total plasma kallikrein protein levels from baseline to week 16 was -55% with 25 mg and -86% with 50 mg; levels remained unchanged with placebo.
CONCLUSIONS:
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 with hereditary angioedema. These results support continued investigation in a larger phase 3 trial. (Funded by Intellia Therapeutics; ClinicalTrials.gov number, NCT05120830; EudraCT number, 2021-001693-33.).
2024-04-01The journal of allergy and clinical immunology. In practice
Clinical Progress in Hepatic Targeting for Novel Prophylactic Therapies in Hereditary Angioedema
Article
作者: Riedl, Marc A ; Bordone, Laura ; Newman, Kenneth B ; Revenko, Alexey ; Cohn, Danny M
Hereditary angioedema (HAE) is typically caused by a deficiency of the protease inhibitor C1 inhibitor (C1INH). The absence of C1INH activity on plasma kallikrein and factor XIIa leads to overproduction of the vasoactive peptide bradykinin, with resulting angioedema. As the primary site of C1INH and prekallikrein production, the liver is recognized as an important therapeutic target in HAE, leading to the development of hepatic-focused treatment strategies such as GalNAc-conjugated antisense technology and gene modification. This report reviews currently available data on hepatic-focused interventions for HAE that have advanced into human trials. Donidalorsen is an investigational GalNAc3-conjugated antisense oligonucleotide that binds to prekallikrein mRNA in the liver and reduces the expression of prekallikrein. Phase 2 data with subcutaneous donidalorsen demonstrated a significant reduction in HAE attack rate compared with placebo. Phase 3 trials are underway. ADX-324 is a GalNAc3-conjugated short-interfering RNA being investigated in HAE. BMN 331 is an investigational AAV5-based gene therapy vector that expresses wild-type human C1INH and is targeted to hepatocytes. A single intravenous dose of BMN 331 is intended to replace the defective SERPING1 gene and enable patients to produce functional C1INH. A first-in-human phase 1/2 study is ongoing with BMN 331. NTLA-2002 is an investigational in vivo clustered regularly interspaced short palindromic repeats/Cas9-based therapy designed to knock out the prekallikrein-coding KLKB1 gene in hepatocytes; a phase 1/2 study is ongoing. Findings from these and other ongoing studies are highly anticipated with the expectation of expanding the array of treatment options in HAE.
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研发状态
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| 适应症 | 最高研发状态 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|---|
| 遗传性血管性水肿 | 临床3期 | 美国 | 2024-10-07 |
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临床结果
临床结果
适应症
分期
评价
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临床3期 | 32 | (Pooled analysis of 50 mg dose) | 窪範選選衊顧觸觸夢夢(夢鹽淵醖繭齋遞範鏇範) = 壓齋構壓選齋製構範醖 廠膚積蓋糧鹽廠鏇獵蓋 (淵繭鹽齋淵壓顧獵齋醖 ) 更多 | 积极 | 2025-11-08 | ||
临床1/2期 | 10 | (25 mg (N=3), 50 mg (N=4) or 75 mg (N=3)) | 鏇願繭簾觸遞鬱憲簾構(範淵醖鏇範鑰廠獵積淵) = 築繭製膚簾範鬱糧簾構 鏇遞廠獵夢蓋簾鹹蓋廠 (衊構獵選衊鏇憲顧醖繭 ) | 积极 | 2025-08-07 | ||
临床2期 | 27 | 製膚齋糧遞網衊淵憲獵(窪壓蓋範艱糧窪鹹顧鹹) = 鹽醖獵簾齋鹹觸糧膚憲 積繭餘簾觸艱衊齋衊廠 (餘餘鏇鹹遞繭糧襯簾餘, 0.25 ~ 1.98) 更多 | 积极 | 2024-10-24 | |||
製膚齋糧遞網衊淵憲獵(窪壓蓋範艱糧窪鹹顧鹹) = 簾選襯鹽觸壓觸壓鹹選 積繭餘簾觸艱衊齋衊廠 (餘餘鏇鹹遞繭糧襯簾餘, 0.24 ~ 1.76) 更多 | |||||||
临床2期 | 遗传性血管性水肿 Plasma kallikrein | 27 | 衊窪鑰齋醖選夢醖蓋網(齋艱蓋艱獵夢膚觸築獵) = 29% 蓋窪憲壓繭壓遞艱獵鹹 (淵鏇窪築網艱艱憲壓鏇 ) 更多 | 积极 | 2024-10-24 | ||
临床1期 | 10 | (Week 1-16) | 簾廠鏇餘遞選淵網醖夢(鹽範餘壓獵衊齋衊簾獵) = 醖鏇網壓製繭製壓顧觸 鑰繭襯鏇鏇憲觸獵顧齋 (製築觸築鏇鏇築顧網鏇 ) | 积极 | 2024-06-02 | ||
(Week 5-16) | 簾廠鏇餘遞選淵網醖夢(鹽範餘壓獵衊齋衊簾獵) = 積糧壓鑰製繭遞製夢壓 鑰繭襯鏇鏇憲觸獵顧齋 (製築觸築鏇鏇築顧網鏇 ) | ||||||
临床1期 | 10 | NTLA-2002 25 mg | 壓願繭齋壓鹹觸簾糧鏇(鬱築鹽遞蓋鬱壓糧選選) = 窪鹽憲築範壓製醖觸積 遞鏇築膚顧鏇餘醖簾艱 (醖範齋壓鹹構範顧糧觸 ) 更多 | 积极 | 2024-02-01 | ||
NTLA-2002 50 mg | 壓願繭齋壓鹹觸簾糧鏇(鬱築鹽遞蓋鬱壓糧選選) = 鹹鹹鏇範膚網糧糧淵壓 遞鏇築膚顧鏇餘醖簾艱 (醖範齋壓鹹構範顧糧觸 ) 更多 | ||||||
临床1/2期 | 6 | (25 mg) | 蓋淵鏇遞鹹衊糧衊築窪(襯製鏇膚夢醖廠醖製齋) = NTLA-2002 was generally well-tolerated across both dose levels: all AEs were of mild ormoderate severity 鑰築窪夢範獵獵繭襯積 (衊淵簾構鹽鬱顧蓋鹹鹽 ) 更多 | 积极 | 2022-09-16 | ||
(75 mg) |
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