Intellia Therapeutics, Inc. (NTLA) is developing its lead in vivo genome-editing candidate, NTLA-2001, for the treatment of transthyretin (ATTR) amyloidosis. The company is collaborating with Regeneron Pharmaceuticals (REGN) for the development of NTLA-2001. NTLA-2001 is being studied for two indications: ATTR amyloidosis with polyneuropathy (ATTRv-PN) patients and ATTR amyloidosis with cardiomyopathy (ATTR-CM) patients. The phase III MAGNITUDE study evaluating NTLA-2001 to treat ATTR amyloidosis with cardi ...

CAMBRIDGE, Mass., June 14, 2024 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced the appointment of Brian Goff to its board of directors. “We are very pleased to welcome Brian to our board of directors. Brian’s extensive global commercialization experience, coupled with his track record of success leading rare disease product launches, will be invaluable to Intellia as ...

CAMBRIDGE, Mass., June 14, 2024 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced the appointment of Brian Goff to its board of directors. “We are very pleased to welcome Brian to our board of directors. Brian’s extensive global commercialization experience, coupled with his track record of success leading rare disease product launches, will be invaluable to Intellia as ...

Neither word has anything to do with money -- at least not yet. On June 2, Intellia Therapeutics' (NTLA -0.96%) CEO John Leonard remarked on the biotech's progress with one of its early-stage clinical programs called NTLA-2002, uttering two very important words that are sure to make at least a few savvy investors salivate. The stock briefly rose 8% above where it was before the announcement. But to the ears of other investors, the same two words imply a risk they'd rather not think about. Let's dive in and ...

CRISPR-Cas9 genome editing enzyme Artur Plawgo Intellia Therapeutics (NASDAQ:NTLA) recently reported strong data from the Phase I portion of an ongoing Phase I/II study for NTLA-2002, an in vivo CRISPR-based gene editing therapy for hereditary angioedema (HAE). I have previously written about CRISPR Therapeutics (CRSP) and rated the company a strong buy due to its progress in the commercialization of Casgevy, an ex vivo CRISPR-based gene editing therapy. While CRISPR Therapeutics remains a strong buy, certa ...

Intellia Therapeutics’ (NTLA) shares gained 10% on Jun 3 after the company reported encouraging long-term data from the phase I portion of its early to mid-stage study evaluating NTLA-2002 for treating hereditary angioedema (HAE). Shares of NTLA continued to gain another 1.1% in the after-market hours. NTLA-2002 is Intellia’s investigational single-dose CRISPR/Cas9 gene-editing therapy, which is being developed as a one-time cure for HAE. It is a rare genetic condition that is marked by severe inflammatory ...

From a technical perspective, Intellia Therapeutics, Inc. (NTLA) is looking like an interesting pick, as it just reached a key level of support. NTLA recently overtook the 50-day moving average, and this suggests a short- term bullish trend. The 50-day simple moving average is a widely used technical indicator that helps determine support or resistance levels for different types of securities. It's one of three major moving averages, but takes precedent because it's the first sign of an up or down trend. NT ...

财务表现 - Intellia Therapeutics, Inc. (NTLA)在2024年第一季度报告的每股亏损为1.06美元，比Zacks Consensus Estimate的每股亏损1.35美元窄[1]。 - 公司报告的亏损不包括一次性投资公平价值变动支出，包括这项支出在内，公司在2024年第一季度报告的每股亏损为1.12美元[2]。 - NTLA的总收入目前仅包括合作收入，2024年第一季度NTLA报告的收入为2,890万美元，而去年同期报告的收入为1,260万美元[3]。 - 公司的合作收入也超过了我们的模型估计的1,280万美元[4]。 - 研发支出总计1.118亿美元，比去年同期的数字增长了近15.1%[5]。 - 行政费用年同比增长约13.5%，达到3,110万美元，主要是由于股权补偿的增加[6]。 - 截至2024年3月31日，NTLA的现金、现金等价物和可市场出售证券价值为9.534亿美元，而截至2023年12月31日为10亿美元[7]。 合作与研发 - NTLA正在与Regeneron Pharmaceuticals (REGN)合作开发其研究中的体内基因编辑候选药NTLA-2001[8]。 - 公司计划在2024年下半年开始对NTLA-2002在HAE适应症中进行美国患者的关键第三阶段研究，取决于监管部门的反馈[9]。

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 001-37766 INTELLIA THERAPEUTICS, INC. (Exact Name of Registrant as Specified in its Charter) Delaware 36-4785571 (State or Other Jurisdiction ...

财务数据和关键指标变化 - 公司现金、现金等价物和有价证券约为9.534亿美元,较2023年12月31日的10亿美元减少 [49] - 第一季度合作收入为2890万美元,较2023年同期的1260万美元增加1630万美元,主要由于与AvenCell合作的2100万美元非现金收入确认 [50] - 第一季度研发费用为1.118亿美元,较2023年同期的9710万美元增加1470万美元,主要由于核心项目的推进 [51] - 第一季度管理费用为3110万美元,较2023年同期的2740万美元增加370万美元,主要由于股份支付费用增加 [52] 各条业务线数据和关键指标变化 - 公司正在推进两个领先的体内CRISPR疗法的关键III期临床试验,预计今年年底将有5项临床试验在进行,其中3项处于III期 [14] - NTLA-2001(ATTR)的III期"Magnitude"试验在美国和全球范围内已开始给患者给药,目前已有30多名患者给药,另有40多名正在筛选中 [21][22] - 公司还计划于今年年底启动NTLA-2001(ATTR多发性神经病)的III期试验,这将是一项小规模的约50名患者的安慰剂对照试验,主要针对无法获得沉默剂的地区 [23][24][25][26] - NTLA-2002(HAE)的长期安全性和持久性数据将于6月2日在欧洲过敏和临床免疫学年会上公布,包括有多少患者完全无发作的数据 [28][29] - 公司计划于今年下半年启动NTLA-2002的III期关键试验 [32] - 公司计划今年开始首次人体试验NTLA-3001(α1抗胰蛋白酶缺乏症),这是公司自主开发的基因插入项目,有望单次给药即可恢复正常水平 [34][35][36] 各个市场数据和关键指标变化 - ATTR心肌病患者尽管使用了TTR稳定剂,但仍继续出现心力衰竭加重、住院、中风和心脏病发作等情况,这种疾病最终仍是致命的 [20] - 在HAE市场,目前约70%的患者使用慢性预防性治疗,这一比例还在不断增加,许多患者仍在寻求更好的疗效和更方便的治疗方式 [31] 公司战略和发展方向及行业竞争 - 公司正在推进基因编辑技术在肝脏以外的5种新组织类型的应用,包括骨髓、大脑、眼睛、肺和肌肉 [42][43][44] - 公司正在与外部创新者建立合作关系,以推进这些研发工作,已经产生了至少12个潜在的药物候选物 [43] - 公司正在通过开发多样化的编辑和递送工具来推进模块化平台,用于体内和体外应用 [13] - 公司强调在每个开发阶段都注重安全性和治疗活性,致力于将尖端科学工具转化为实际的医疗治疗 [13] - 公司认为其CRISPR疗法的单次给药、无需长期激素治疗和住院等优势,可以克服其他基因疗法面临的关键障碍,有望在大型和快速增长的商业市场上取得成功 [8][9] 管理层对经营环境和未来前景的评论 - 公司CEO表示,公司正在进入下一个增长阶段,正在推动CRISPR技术的边界,从基因敲除到基因插入,从肝脏靶向到更广泛的组织 [10][11] - 公司预计今年将有5项临床试验在进行,其中3项处于III期,并计划于2026年提交NTLA-2002的生物制品许可申请,这将是首个获批的体内CRISPR疗法 [14] - 公司有信心NTLA-2001和NTLA-2002可以颠覆ATTR和HAE的治疗格局,提供潜在的最佳临床方案 [8][9] - 公司认为其专有的LNP制剂、新型基因编辑工具和差异化的同种异体细胞疗法方法,将有助于实现安全性和治疗活性的目标 [13] 问答环节重要的提问和回答 问题1 **Yanan Zhu提问** 询问ATTR心肌病III期试验的患者入组进度,包括美国和非美国站点的数量,以及每个站点每月的患者入组数 [57] **John Leonard回答** 公司不会提供具体的患者入组数据和站点数量,但表示目前入组进度超出预期,体现了医生和患者对该疗法的热情 [58][59][60] 问题2 **Maury Raycroft提问** 询问ATTR多发性神经病III期试验设计为何只有50例患者,与既往同类试验相比样本量较小的原因 [64] **David Lebwohl回答** FDA认为TTR降低程度与疗效改善相关,公司与FDA就试验设计达成一致,主要针对无法获得沉默剂的地区患者 [67][68][69][70][71] 问题3 **Luca Issi提问** 询问ATTR心肌病III期试验是否会根据竞争对手HELIOS-B试验结果调整设计,如是否考虑按整体人群和单药组分别设主要终点 [76] **John Leonard和David Lebwohl回答** 公司设计的Magnitude试验已经非常谨慎保守,会密切关注HELIOS-B结果,必要时可能会对试验进行调整,但目前公司有信心NTLA-2001可以成为该领域的最佳选择 [77][78][79]