Thank you for reading my research on Seeking Alpha. If you want to learn even more about my method and how I discover these investment opportunities, please check out my subscription marketplace service, Compounding Healthcare , and sign up for a free trial.It has been over a year since my previous Crispr Therapeutics AG (NASDAQ: CRSP ) article , where I discussed the company's diversified gene-editing pipeline that was supported by a strong $2.1B cash position. At that time, CASGEVYHe is the leader of the ...

Biotech stock Crispr Therapeutics Inc's (NASDAQ:CRSP) June rally appears to be coming to an end. The shares are looking to snap a seven-day win streak, down 0.6% at $42.69 at last glance. Plus, after hitting its highest level since March, the stock is now flashing a historically bearish signal on the charts. Per Schaeffer's Senior Quantitative Analyst Rocky White, CRSP has moved up toward its 260-day moving average. More specifically, the security is within 0.75 of the trendline's 20-day average true range ...

Key Takeaways CRSP gained 14% in a month, driven by positive CTX310 in vivo gene therapy trial data. Casgevy, its approved ex vivo gene therapy, saw Q1 sales rise to $14.2M with 65 treatment centers activated. CRSP expects multiple pipeline updates in 2025, including early data for CTX320 and new in vivo programs.Shares of CRISPR Therapeutics (CRSP) have risen 14% over the past month, likely driven by encouraging results from the first candidates in its in vivo pipeline.CRSP’s Positive In Vivo Therapy Dat ...

The latest trading session saw CRISPR Therapeutics AG (CRSP) ending at $42.94, denoting a +2.02% adjustment from its last day's close. The stock outperformed the S&P 500, which registered a daily gain of 0.55%. At the same time, the Dow added 0.25%, and the tech-heavy Nasdaq gained 0.63%.Shares of the company have appreciated by 11.97% over the course of the past month, outperforming the Medical sector's gain of 3.49% and the S&P 500's gain of 6.29%.Investors will be eagerly watching for the performance of ...

CRISPR Therapeutics AG (NASDAQ:CRSP) Goldman Sachs 46th Annual Global Healthcare Conference June 9, 2025 3:20 PM ET Company Participants Samarth Kulkarni - CEO & Chairman Conference Call Participants Salveen Jaswal Richter - Goldman Sachs Group, Inc., Research Division Salveen Jaswal Richter Good afternoon. Thank you, everyone, for joining us. I'm Salveen Richter, biotechnology analyst at Goldman Sachs, and we're really pleased to have Sam Kulkarni, Dr. Sam Kulkarni, CEO and Chairman of CRISPR, here. Sam to ...

CRISPR Therapeutics (CRSP) FY Conference June 09, 2025 03:20 PM ET Speaker0 Good afternoon. Thank you everyone for joining us in Salveen Richter, Biotechnology Analyst at Goldman Sachs. We're really pleased to have Sam Kulkarni, Doctor. Sam Kulkarni, CEO and Chairman of CRISPR here. Sam, to start, could you give us a brief overview of the company at this point, your strategy, your pipeline and really kind of the key focus areas headed into the half of the year? Speaker1 Yes. Thank you for having us and it's ...

A month has gone by since the last earnings report for CRISPR Therapeutics AG (CRSP) . Shares have added about 11.1% in that time frame, outperforming the S&P 500.Will the recent positive trend continue leading up to its next earnings release, or is CRISPR Therapeutics due for a pullback? Before we dive into how investors and analysts have reacted as of late, let's take a quick look at its most recent earnings report in order to get a better handle on the important drivers.How Have Estimates Been Moving Sin ...

公司核心业务进展 - CRISPR Therapeutics是目前全球唯一一家获得CRISPR基因疗法批准的公司 该疗法是与Vertex Pharmaceuticals合作开发的[1] - 公司正在评估两种体内候选药物CTX310和CTX320 分别针对ANGPTL3和脂蛋白(a) 这两种物质与动脉粥样硬化性心脏病相关[2] - CTX310在I期试验中显示单剂量可使甘油三酯(TG)和低密度脂蛋白(LDL)水平出现剂量依赖性下降 峰值降幅分别达82%和81%[3] 研发管线规划 - CTX320的试验数据预计将在2025年第二季度公布[4] - 公司计划在年底前推进另外两个体内项目进入临床阶段[4] 行业竞争格局 - Intellia Therapeutics是主要竞争对手 拥有多个临床阶段体内基因编辑项目 包括针对遗传性血管性水肿(HAE)的NTLA-2002和转甲状腺素蛋白淀粉样变性(ATTR)的NTLA-2001[5] - Beam Therapeutics也在开发体内候选药物 包括针对α-1抗胰蛋白酶缺乏症(AATD)的BEAM-302和糖原累积病1a型(GSD1a)的BEAM-301[6] 财务与估值 - 公司股价表现落后于行业平均水平[7] - 按市净率(P/B)计算 公司当前交易价格为1.80倍 低于行业平均的3.10倍 也低于其五年均值2.41[10] - 过去60天内 2025年每股亏损预估从4.96美元扩大至5.54美元 2026年每股亏损预估从3.65美元增至4.11美元[12]

公司战略与目标 - 公司专注于开发针对严重疾病的变革性基因药物 [3] - 目前已有改进疗法CASGEVY上市 由合作伙伴Vertex主导商业化 [4] - 正在推进第二至第五个产品的研发 未来6-12个月的临床试验结果将决定公司战略方向 [4] 研发管线与技术平台 - 拥有多项临床前项目 基于可扩展的基因编辑技术平台 [5] - 技术平台可应用于多种疾病领域 但需根据资源分配选择性推进 [5] - 长期目标是通过平台技术实现管线指数级扩张 建立可持续的生物技术业务模式 [5]

纪要涉及的行业和公司 - **行业**：生物科技行业 - **公司**：CRISPR Therapeutics（CRSP）、Vertex、Sirius Therapeutics、Novartis、Anthos 纪要提到的核心观点和论据 公司目标与战略 - **核心目标**：为严重疾病创造变革性的基因药物，成为可持续发展的生物科技公司 [3] - **战略方向**：基于CRISPR基因编辑技术，构建四个关键业务领域，推进多个临床试验和临床前项目，通过临床数据确定未来战略方向 [4][5] - **发展阶段**：2024年为基础建设年，2025年是转折点，有望成为行业领先的生物科技公司，拥有强大资产负债表 [7][8] 业务领域与产品管线 1. **血红蛋白病领域（Heme Franchise）** - **核心产品**：KASJEVY，由合作伙伴Vertex主导商业化，全球可治疗患者达6万，市场潜力巨大 [8][10] - **市场拓展**：2025年计划通过CMMI试点模式、拓展美国以外市场、增加制造投资等方式扩大市场份额 [11][12] - **创新方向**：通过靶向调理和体内治疗方法，进一步扩大治疗人群，包括非洲等服务不足地区 [13] 2. **CAR - T平台（CAR T Platform）** - **核心产品**：CTX - 112，用于癌症和自身免疫性疾病治疗，一期临床试验数据显示，总体缓解率67%，完全缓解率50%，高剂量组效果更佳 [14][15] - **竞争优势**：具有较低成本、高可扩展性、深度B细胞耗竭和长期缓解潜力，有望成为同类最佳疗法 [16] 3. **体内平台（In Vivo Platform）** - **CTX - 310**：靶向ANGPTL3，一期试验显示可显著降低甘油三酯和LDL，安全性良好，有望成为同类最佳疗法，可治疗多种适应症 [17][19][21] - **CTX - 320**：靶向脂蛋白A（LPA），在NHP研究中显示可降低95%血浆LPA，持续两年，美国约有6000万患者有治疗需求 [24][25][26] - **其他项目**：CTX - 320后续有两个项目推进，分别针对血管紧张素原（治疗难治性高血压）和罕见病，糖尿病项目CTX - 211也在推进中 [27][28] 4. **1型糖尿病领域（Type One Diabetes Franchise）**：致力于提供可产生胰岛素的胰岛细胞，减少胰岛素注射需求，预计2025年提供更新信息 [28][29] 业务发展与合作 - **与Sirius Therapeutics合作**：引入siRNA技术，针对Factor XI靶点，用于全膝关节置换等领域，有望在抗凝领域创造数十亿美元机会 [8][31][35] - **未来业务发展策略**：以成为行业领先生物科技公司为目标，谨慎评估收购和合作机会，保持业务发展活动的积极性 [39][40] 财务与盈利 - **财务状况**：拥有18.6亿美元的强大资产负债表，KASJEVY已开始产生收入 [8][31][44] - **盈利目标**：谨慎使用资金，确保投资回报，预计未来实现可持续盈利 [49][50] 其他重要但可能被忽略的内容 - **KASJEVY治疗效果**：接受治疗的镰状细胞病患者能够攀登乞力马扎罗山，地中海贫血患者可能实现输血独立 [9][10][43] - **CTX - 112治疗效果**：在复发难治性肿瘤患者中，CTX - 112显示出良好的治疗反应 [15] - **体内平台技术优势**：体内平台基于脂质纳米颗粒和mRNA，具有高可扩展性和低成本的特点 [27] - **LPA检测情况**：LPA检测在美国尚未纳入指南，但在医学会议上受到广泛关注，预计美国有超过6000万患者受高LPA影响 [24][25][26] - **地域市场机会**：海湾沿岸国家市场对投资界来说是一个惊喜，Vertex预计该地区可治疗患者超过2.3万 [47][48]