文章核心观点 - 公司公布lonvo - z治疗遗传性血管性水肿（HAE）的三年随访数据，显示其疗效显著、安全性良好，全球3期HAELO试验提前完成筛查 [1][2][6] 公司介绍 - Intellia Therapeutics是临床阶段基因编辑公司，专注用基于CRISPR的疗法革新医学 [1][9] lonvo - z介绍 - 基于CRISPR/Cas9技术，通过使激肽释放酶B1（KLKB1）基因失活预防HAE发作，有望成为HAE的一次性治疗方法，已获五项重要监管认定 [8] 研究情况 1/2期研究 - 研究评估lonvo - z对I型或II型HAE成人患者的安全性和有效性，1期确定剂量水平，两部分入组均已完成 [5][7] - 1期研究中，分别给予25mg（N = 3）、50mg（N = 4）、75mg（N = 3）的lonvo - z单次静脉输注，截至2月12日数据截止时，患者中位近2年无发作且无需治疗，三年随访显示单次给药使每月HAE发作率平均降低98%，血浆激肽释放酶蛋白持续显著降低 [2] 3期HAELO试验 - 评估50mg lonvo - z的安全性和有效性，已提前完成筛查，超半数患者来自美国，公司未来将更新入组情况 [4][6] 安全性 - 各剂量水平下lonvo - z耐受性良好，安全性与2024年EAACI公布的数据一致，最常见不良事件为输液相关反应（IRRs），多为1级且患者均完成全剂量输注，随访3年未观察到治疗引起的严重不良事件，给药28天后未观察到治疗相关不良事件 [3] 临床开发计划 - 预计2025年下半年公布正在进行的1/2期研究2期部分的新的长期数据，2026年上半年公布3期HAELO研究结果，2026年提交生物制品许可申请（BLA）以支持2027年在美国上市 [2][4][10]

分组1 - 会议记录了2025年6月11日上午9点举行的Intellia Therapeutics年度股东大会 [1] - 公司董事会主席Frank Verwil主持会议并介绍了董事会和高管团队成员 [2][3][4] - 公司独立注册会计师事务所Deloitte Touche LLP的Jim Holt出席会议，可回答相关问题 [5] 分组2 - 会议记录日期为2025年4月14日，当天公司已向所有在册股东邮寄代理材料互联网可用性通知 [6] - 会议法定人数方面，已收到代表82,824,215股的代理投票，占已发行普通股总数103,582,731股的约80% [7] 分组3 - 会议提出六项提案，包括选举三名III类董事、批准任命审计机构、高管薪酬相关投票、批准股权奖励计划等 [8][10] - 投票时间为2025年6月11日上午9:05至9:06，所有有权投票的股东可在线投票 [12][13] 分组4 - 投票结果显示，所有提案均获通过，其中关于高管薪酬非约束性咨询投票频率的提案，多数投票支持一年一次 [14][15] - 会议期间未收到与会议讨论事项相关的问题，最终会议于2025年6月11日休会 [16][17] 问答环节所有提问和回答 问题: 无 - 无相关问题提出 [16]

Shares of Intellia Therapeutics, Inc. (NTLA) have gained 6.1% over the past four weeks to close the last trading session at $7.69, but there could still be a solid upside left in the stock if short-term price targets of Wall Street analysts are any indication. Going by the price targets, the mean estimate of $38.32 indicates a potential upside of 398.3%.The average comprises 25 short-term price targets ranging from a low of $7 to a high of $106, with a standard deviation of $24.94. While the lowest estimate ...

Shares of Intellia Therapeutics (NTLA) were down 22.9% yesterday after it announced an update from the ongoing phase III study, evaluating the investigational in vivo genome-editing candidate, nexiguran ziclumeran (nex-z), for treating ATTR amyloidosis with cardiomyopathy (ATTR-CM).Per the company, one of the patients in the phase III MAGNITUDE study, which evaluated nex-z for treating ATTR-CM, experienced grade 4 liver transaminase elevations, indicating a notable increase in liver enzymes. The patient was ...

Intellia Therapeutics Inc NTLA said on Wednesday that a single, recent, asymptomatic patient has had grade 4 (severe) liver transaminase elevations (liver enzymes) based on laboratory tests in its Phase 3 MAGNITUDE study.The trial is evaluating nexiguran ziclumeran (nex-z, also known as NTLA-2001) in transthyretin amyloidosis with cardiomyopathy.These elevations appear to be resolving without hospitalization or medical intervention and have fallen to grade 3 ALT and grade 2 AST elevations.The company has en ...

Persistent volatility has plagued the S&P 500 for much of 2025, prompting widespread caution among many investors amid the uncertainties of trade battles, tariffs, domestic policy, and more. Investors might remember that many of the biggest rallies take place during these volatile periods, and there may be potential to win big gains for those with at least a moderate tolerance for risk. Healthcare firms are already known for being volatile, with smaller companies in particular experiencing significant spike ...

NEW YORK, May 22, 2025 (GLOBE NEWSWIRE) -- Bragar Eagel & Squire, P.C., a nationally recognized shareholder rights law firm, is investigating potential claims against Intellia Therapeutics, Inc. (NASDAQ:NTLA) on behalf of long-term stockholders following a class action complaint that was filed against Intellia on February 11, 2025 with a Class Period from July 30, 2024 to January 8, 2025. Our investigation concerns whether the board of directors of Intellia have breached their fiduciary duties to the compan ...

An updated edition of the March 28, 2025, article.The rapid evolution of genomics, the study of genes and their functions, in recent times has paved the way for a revolutionary era in genetic medicine. Given the constant need for innovative medical treatments, genomics holds the key to the future of the medical world.In simpler terms, genomics includes the study of a complete set of genes, their work process and way of interacting with each other and the environment. The primary focus is to evaluate all the ...

Deep, durable and consistent reductions in TTR were sustained at two years, following a one-time dose of nex-zClinically meaningful improvements in ATTRv-PN related outcomes observed at 24 months compared to baseline, including in patients who were previously progressing on patisiranContinue to observe generally favorable safety and tolerability data in the full Phase 1 cohort with no new drug-related adverse events within the follow-up periodEnrollment continues to progress well in MAGNITUDE-2, which is de ...

Shares of Intellia Therapeutics, Inc. (NTLA) have gained 14.9% over the past four weeks to close the last trading session at $7.88, but there could still be a solid upside left in the stock if short-term price targets of Wall Street analysts are any indication. Going by the price targets, the mean estimate of $40.08 indicates a potential upside of 408.6%.The mean estimate comprises 25 short-term price targets with a standard deviation of $26.66. While the lowest estimate of $9 indicates a 14.2% increase fro ...