Updated data from over 60 patients showed consistent, deep and durable serum TTR reduction achieved with a single dose of NTLA-2001, including in 29 patients who have now reached 12 months or more of follow-upNTLA-2001 was generally well-tolerated across both polyneuropathy and cardiomyopathy arms at all dose levels tested55 mg dose of NTLA-2001 selected for further evaluation in the global pivotal Phase 3 trial expected to begin by year-end CAMBRIDGE, Mass., Nov.

来自60多名患者的最新数据显示，单剂量NTLA-2001可实现一致，深入和持久的血清TTR降低，包括29名现已达到12个月或更长时间随访的患者NTLA-2001在所有剂量水平测试的多发性神经病和心肌病组中均具有良好的耐受性，在全球关键阶段选择了55 mg剂量的NTLA-2001进行进一步评估3试验预计将于年底开始，马萨诸塞州剑桥市，11月。

02, 2023 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing potentially curative therapies leveraging CRISPR-based technologies, today presented additional interim results from its ongoing Phase 1 study of NTLA-2001, an investigational, in vivo CRISPR/Cas9 genome editing therapy in development as a single-dose treatment for transthyretin (ATTR) amyloidosis.

022023（GLOBE NEWSWIRE）-Intellia Therapeutics，Inc。（纳斯达克股票代码：NTLA），一家领先的临床阶段基因组编辑公司，专注于利用基于CRISPR的技术开发潜在的治愈性疗法，今天提出了其正在进行的额外中期结果NTLA-2001的1期研究，一项调查，体内CRISPR/Cas9基因组编辑疗法正在开发中，作为运甲状腺素蛋白（ATTR）淀粉样变性的单剂量治疗。

Results were presented in an oral presentation at the 4th International ATTR Amyloidosis Meeting, held Nov. 2–3 in Madrid, Spain. “With 65 patients reported from the Phase 1 study, this update represents the largest clinical dataset for an in vivo CRISPR-based investigational therapy. These positive interim results add to the growing body of data that demonstrates deep and durable reductions of serum TTR after a single dose of NTLA-2001.

结果在11月2日至3日在西班牙马德里举行的第四届国际ATTR淀粉样变性会议上的口头报告中提出。“在第一阶段研究报告的65例患者中，本次更新代表了基于体内CRISPR的研究治疗的最大临床数据集。这些积极的中期结果增加了越来越多的数据，证明了血清TTR的深度和持久降低单剂量的NTLA-2001。

The consistent and profound levels of reduction in all patients bolster our confidence that NTLA-2001 could potentially reset the standard of care for ATTR amyloidosis — both for treating the disease and how response is evaluated,” said Intellia President and Chief Executive Officer John Leonard, M.D.

Intellia总裁兼首席执行官John Leonard说，所有患者的持续而深刻的减少水平增强了我们的信心，即NTLA-2001可能会重置ATTR淀粉样变性的护理标准-既用于治疗疾病又如何评估反应。

“We have also observed early signals of clinical activity in the initial cohorts and look forward to presenting the first clinical data beyond.

“我们还在最初的队列中观察到了临床活动的早期信号，并期待提供除此之外的第一批临床数据。