Nexiguran ziclumeran

全球视野，深度视角 各位亲爱的股东，大家早上好中午好晚上好！ 朋友们，真的是突发。 2026年1月27日，基因编辑公司Intellia Therapeutics宣布其CRISPR基因编辑疗法III期临床试验MAGNITUDE-2计划恢复患者入组和给药！ 该临床试验在2025年10月29日暂停，当时报告了一起4级肝毒性不良反应。 美国食品药品监督管理局（FDA）已解除对MAGNITUDE-2 III期临床试验的新药临床试验申请（IND）的临床暂停。 公司已与FDA就MAGNITUDE-2的某些研究修改和缓解措施达成一致，包括加强对肝功能实验室检查的安全性监测。 该试验旨在评估nexiguran ziclumeran（nex-z）用于治疗遗传性转甲状腺素蛋白淀粉样变性伴多发性神经病（ATTRv-PN）患者。 NTLA-2001采用CRISPR-based体内基因编辑技术，敲除TTR基因的表达，payload（有效载荷）均采用LNP（Lipid nanoparticle，纳米脂质体）递送。 机制上，NTLA-2001通过CRISPR/Cas9在TTR基因上引入indel（插入缺失）突变，从而产生一个framshift突变（移码突变）进而阻止TTR蛋白的产生。 MAGNITUDE-2是一项随机、双盲、安慰剂对照试验，评估nex-z在ATTRv-PN患者中的疗效和安全性。 作为方案修正案的一部分，Intellia已将试验的目标入组人数从约50名患者增加至约60名ATTRv-PN患者。 该研究的主要终点是改良神经病变损伤评分的变化和血清TTR水平的变化，ATTRv-PN成人患者将以1:1的比例随机接受单剂55 mg的nex-z输注或安慰剂。 这里需要注意的是，恢复的是MAGNITUDE-2临床，MAGNITUDE仍然在暂停中。 也就是，评估nex-z用于治疗转甲状腺素蛋白淀粉样变性伴心肌病[ATTR-CM]患者的IND临床仍然暂停中。 Intellia与FDA的沟通仍在进行中，公司计划在就该项目的推进路径达成一致后提供最新进展。 在盘前交易中，Intellia股价一度涨超20%，但是开盘后有所回落。 不管咋样，这对患者来说可能是好消息。 Tags：#基因编辑#CRISPR #基因治疗 #Intellia #罕见病  END 各位股东看了么？赞了么？转发了么？别忘了点赞。谢谢！ 所有内容均不作为投资建议，信息均来自公开资料，星球更新更快。 扫码加入星球！ 循因缉药Elite PS:风里雨里，老编等你~ 近期原创: 重大突破！FDA 或允许MRD作为临床试验加速审批的终点指标 Illumina创始人创立的公司可能也许凉了 CFO缺钱了 多组学再次上榜！木头姐Big Ideas 2026发布！ 相关资料： 注1：https://www.globenewswire.com/news-release/2026/01/27/3226448/0/en/Intellia-Therapeutics-Announces-FDA-Lift-of-Clinical-Hold-on-MAGNITUDE-2-Phase-3-Clinical-Trial-in-ATTRv-PN.html

William Blair analysts called the hold lift “clearly positive,” demonstrating Intellia Therapeutics\' ability to work with the FDA on risk-mitigation measures to restart enrollment.\n The FDA is freeing Intellia Therapeutics\' CRISPR therapy from one of two clinical holds, allowing a phase 3 study to resume after a grade 4 liver event and subsequent patient death was reported in the sister study. The biotech’s stock is up 12% since yesterday, rising from $13.95 per share to $15.66 by 10:30 a.m. ET today. William Blair analysts called the hold lift “clearly positive,” demonstrating Intellia’s ability to work with the FDA on risk-mitigation measures to restart enrollment for the trial, which the biotech plans to resume as quickly as possible. Last fall, the FDA placed clinical holds on Intellia’s Magnitude and Magnitude-2 studies, which are evaluating the company’s experimental CRISPR therapy in patients with transthyretin amyloid cardiomyopathy (ATTR-CM) and hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN), respectively. Both diseases impact heart function.The hold followed Intellia’s voluntary pause in response to a grade 4 liver event—a case of Hy’s Law—in the ATTR-CM trial. Shortly after the regulatory hold was implemented, Intellia disclosed that the impacted patient had died.While the patient had several comorbidities, the reported cause of death was septic shock, indicating that that the liver enzyme elevation alone was unlikely to have been fatal. Intellia has said that the liver injury and enzyme elevations were likely due to its treatment, called nexiguran ziclumeran (nex-z), a CRISPR therapy designed to inactivate the TTR gene.Now, Intellia has implemented new risk-mitigation procedures designed to prevent dosing in potentially at-risk patients in the ATTRv-PN study, according to the William Blair analysts. Measures include enhanced safety monitoring of liver lab tests and boosting target enrollment from 50 patients to 60. Analysts now await updates on the Magnitude study in ATTR-CM, a condition that more commonly impacts the elderly, making the patient population automatically more at risk compared to the ATTRv-PN population. The study has already recruited more than 650 patients of an expected 1,200. Both factors are likely leading to higher scrutiny for Magnitude than Magnitude-2.Intellia’s nex-z therapy has secured both orphan drug and regenerative medicine advanced therapy tags from the FDA. The asset is one of the biotech’s most advanced clinical candidates, alongside lonvoguran ziclumeran, which is being tested out in hereditary angioedema (HAE).“We continue to believe in the efficacy of Intellia’s platform, and 2026 will be a major year for the promising HAE program, but safety events from nex-z have been and will continue to be a stock overhang until further regulatory clarity is gained on resumption of Magnitude and as investors gain comfort with new risk-mitigation strategies, in our view,” William Blair analysts wrote Jan. 27.