公司介绍 - Vertex Pharmaceuticals在2025年6月20日美国糖尿病协会(ADA)第85届科学会议上公布zimislecel最新进展 [1][2] - 公司高管团队包括细胞与基因疗法研究高级副总裁Felicia Pagliuca（剑桥大学生物学博士）等核心成员参与会议 [1][4] 会议内容 - 会议通过线上线下结合方式举行 特别提及《新英格兰医学杂志》(NEJM)相关文章发布与夏至日的时间节点 [2] - 讨论重点聚焦于Vertex的1型糖尿病(T1D)研发项目进展 但未披露具体临床数据或疗效结果 [3] 参会机构 - 参会分析师来自摩根大通、Leerink Partners、杰富瑞、Evercore ISI等知名投行研究部门 [1]

纪要涉及的公司和行业 - **公司**：Vertex Pharmaceuticals（VRTX）[1] - **行业**：制药行业，专注于1型糖尿病治疗领域 [7] 纪要提到的核心观点和论据 1型糖尿病现状 - **患者数量与需求**：北美和欧洲约有400万人被诊断患有1型糖尿病，其中约6万名患者有严重低血糖事件，未满足的医疗需求高 [11] - **传统治疗不足**：自1921年胰岛素发现以来，治疗方式基本未变，无法满足患者及其家庭需求，会导致急性和长期并发症，降低生活质量 [9] - **技术进步仍有局限**：尽管有泵和连续血糖监测仪等技术进步，但近10%的患者仍有严重低血糖事件，40%的患者无法达到血糖控制目标 [12][49] Zamyla Cell疗法介绍 - **疗法原理**：从干细胞制造的胰岛细胞疗法，可分化为葡萄糖反应性、胰岛素产生的干细胞衍生胰岛，通过输注到肝门静脉，用无类固醇免疫抑制方案保护细胞 [13] - **临床试验进展**：关键试验（1、2、3期研究）进展迅速，预计今年夏天完成入组和给药，2026年提交监管申请 [15] - **监管指定**：获得美国再生医学先进疗法指定（RMAT）、快速通道指定，欧洲PRIME指定，英国创新许可和准入途径下的创新护照 [15] 临床试验数据 - **疗效数据**：12名接受全剂量治疗且随访至少12个月的患者，恢复了有临床意义和持续的内源性胰岛素分泌，改善了葡萄糖耐量，消除了严重低血糖事件，达到血糖控制目标，10人停用胰岛素 [21][23][27] - **安全数据**：多数不良事件为轻度或中度，与研究药物有因果关系的主要归因于免疫抑制治疗，与Zamyla Cell相关的主要是短暂的肝转氨酶升高，无严重不良事件 [25][26] 疗法优势与潜力 - **解决供体胰岛问题**：与已故供体胰岛移植相比，Zamyla Cell细胞质量高、供应充足，有望改变严重1型糖尿病患者的治疗模式 [31][39] - **患者接受度高**：患者认为免疫抑制治疗风险可接受，能减轻疾病负担，改善生活质量 [33][34][39] - **创新研发前景**：Vertex正在进行新型免疫疗法、基因编辑、下一代技术等研究，有望扩大治疗人群 [59][60] 其他重要但是可能被忽略的内容 - **未来研究方向**：Vertex正在研究替代免疫抑制药物和方案、基因编辑低免疫方法等技术 [14] - **制造能力准备**：Vertex在波士顿有制造团队，与Lonza和TreeFrog合作，扩大制造能力，以满足产品需求 [60][61] - **潜在适用人群扩展**：除严重低血糖患者外，肾移植患者、全胰腺切除患者等也可能从Zamyla Cell疗法中受益 [68][69]

福泰制药（VRTX）针对1型糖尿病的干细胞疗法Zimislecel（VX-880）展示正面数据。 ...

In the latest close session, Vertex Pharmaceuticals (VRTX) was down 1.68% at $440.87. The stock fell short of the S&P 500, which registered a loss of 0.22% for the day. Meanwhile, the Dow gained 0.08%, and the Nasdaq, a tech-heavy index, lost 0.51%. Heading into today, shares of the drugmaker had gained 3.31% over the past month, outpacing the Medical sector's loss of 0% and the S&P 500's gain of 0.45%.The investment community will be paying close attention to the earnings performance of Vertex Pharmaceutic ...

Popular themes on Wall Street today include artificial intelligence (AI), the market for weight management medicines, and the potential impact of tariffs on broader equities and the economy. In 10 years, there will probably be a different set of trending topics on The Street, but those changes won't stop the market from delivering competitive returns over the next decade.Those who want to cash in on that can purchase exchange-traded funds (ETFs) that track the performance of some major index, or invest in i ...

Vortex Energy Plans Geophysical Survey at Robinsons River Salt Project to Assess Hydrogen Storage PotentialVANCOUVER, British Columbia, June 12, 2025 (GLOBE NEWSWIRE) -- Vortex Energy Corp. (CSE: VRTX) (OTC: VTECF) (FSE: AA3) (“Vortex” or the “Company”) is pleased to announce plans for a detailed geophysical survey at its flagship Robinsons River Salt Project (the “Project”) in Newfoundland. This marks the start of summer exploration activities that are focused on evaluating the project’s suitability for la ...

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) Goldman Sachs 46th Annual Global Healthcare Conference Call June 10, 2025 2:40 PM ET Company Participants David M. Altshuler - Executive VP & Chief Scientific Officer Reshma Kewalramani - CEO, President & Director Conference Call Participants Salveen Jaswal Richter - Goldman Sachs Group, Inc., Research Division Salveen Jaswal Richter Good afternoon, everyone. Thank you so much for joining us. I'm Salveen Richter, biotechnology at Goldman Sachs and really ple ...

Vertex Pharmaceuticals (VRTX) FY Conference June 10, 2025 02:40 PM ET Speaker0 Launch, we are now seeing the building of momentum as we build up that franchise. And then most recently, the launch of Gernavix in acute pain. We're keenly focused on those three launches. As we also think about the next wave of innovation that's either in or nearing Phase III development. There are three actually four programs that are already in Phase III development, and the one, POV in membranous is coming very soon. And the ...

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) Bernstein 41st Annual Strategic Decisions Conference May 30, 2025 8:00 AM ET Company Participants Reshma Kewalramani - President & CEO Conference Call Participants Will Pickering - Bernstein Will Pickering All right. Well, I think we can get started. Good morning, everyone. My name is Will Pickering. I cover U.S. Biotech at Bernstein. I am privileged to be joined today by Vertex's CEO, Reshma Kewalramani. And Reshma, welcome. Reshma Kewalramani Thank you so ...

Vertex Pharmaceuticals (VRTX) FY 2025 Conference May 30, 2025 08:00 AM ET Speaker0 Alright. Well, I think we can get started. Good morning, everyone. My name is Will Pickering. I cover US Biotech at Bernstein. I am privileged to be joined today by Vertex CEO Reshma K. Walramani. And Reshma, welcome. Speaker1 Thank you so much, Will. Good morning. Speaker0 Well, why don't we, you know, start with giving you know, maybe Reshma, if you wanna make a few kind of framing comments for the discussion, and then we c ...