ReCor Medical, Inc.

地点：阿布扎比加拿大佛罗里达伦敦内华达州俄亥俄州 霍普·巴吉| 216.213.6192 香农·凯利| 克利夫兰诊所一项 1 期首次人体临床试验结果表明，使用 CRISPR-Cas9 进行一次性基因编辑疗法输注，可以安全地降低对现有药物有耐药性的脂质紊乱患者的低密度脂蛋白（“坏”）胆固醇和甘油三酯水平。 研究结果于今日在美国心脏协会 2025 年科学年会上以最新科学成果报告的形式公布，并同时发表在《新英格兰医学杂志》上。 这项包含15名患者的试验表明，治疗两周后，低密度脂蛋白胆固醇和甘油三酯水平均显著降低，并在至少60天内维持在较低水平，后续随访仍在进行中。短期随访期间，未发现与治疗相关的任何严重不良事件。 “这项疗法目前仍处于早期研发阶段，但如果未来的试验继续证实其安全性和有效性，那么它有望改变我们治疗血脂异常的方式，”克利夫兰诊所心脏病专家、该研究的第一作者卢克·拉芬医学博士表示。“与每日一次的药丸或每月一次的注射相比，这种疗法有望为高胆固醇患者提供一次安全且持久的输注。” 过高的低密度脂蛋白胆固醇（通常被称为“坏”胆固醇）会导致动脉粥样硬化——动脉壁上斑块积聚——从而增加心脏病发作或中风的风险。最新估计显示，截至2018年，26.4%的美国成年人低密度脂蛋白胆固醇水平升高。 高甘油三酯血症还会增加患心血管疾病的风险，包括心脏病发作和中风。美国约有25%的人患有高甘油三酯血症。 “对于患者而言，治疗依从性是一个重要问题。事实上，接受降胆固醇药物治疗的患者中，有一半会在一年内停止服药，”该研究的资深作者、克利夫兰诊所心脏、血管和胸外科研究所首席学术官史蒂文·尼森医学博士表示。“尽管基因编辑疗法的研究尚处于早期阶段，但目前试验中展现出的疗效令人鼓舞，并代表着药物研发的一个潜在新领域。” CRISPR-Cas9 是一种基因编辑技术，可用于修改或纠正人体 DNA 的特定区域，以治疗严重疾病。 这项试验测试了CTX310，一种实验性的CRISPR-Cas9基因编辑疗法，只需一次性输注即可完成。CTX310将CRISPR编辑机制导入肝脏，在那里它可以关闭一种名为ANGPTL3的基因。关闭该基因可以降低低密度脂蛋白胆固醇和甘油三酯水平，而这两种物质都与心脏病密切相关。先天性ANGPTL3基因功能缺失突变的人，终生患心脏病的风险较低，且未发现明显的不良后果。 该研究于2024年6月至2025年8月在澳大利亚、新西兰和英国的六个地点进行。研究参与者包括15名年龄在31岁至68岁之间的成年人。所有参与者均患有未控制的高甘油三酯和高低密度脂蛋白胆固醇水平。 在试验期间，受试者在接受皮质类固醇和抗组胺药预处理后，以0.1至0.8 mg/kg的剂量进行单次静脉输注CTX310。研究人员监测受试者治疗的安全性，以及该基因疗法对ANGPTL3、低密度脂蛋白胆固醇和甘油三酯水平的影响。在最高剂量下，CTX310平均可使低密度脂蛋白胆固醇和甘油三酯水平降低约50%。所有患者均接受了至少60天的安全性随访。 三名参与者出现了轻微反应，如背痛和恶心，用药物治疗后即可缓解；一名参与者在治疗前肝酶升高，治疗后肝酶暂时进一步升高，持续了几天，无需任何治疗即可恢复正常。 研究参与者将在试验结束后接受一年的监测，并按照美国食品药品监督管理局 (FDA) 对所有基因编辑疗法的建议，进行长达 15 年的长期安全随访。 拉芬博士补充道：“重要的是，CTX310没有出现任何严重的安全事件。然而，这是一项规模较小且持续时间相对较短的研究，我们需要在未来的试验中继续严格研究其安全性。” 未来的 2 期研究计划于 2026 年开始，重点关注更广泛的患者群体和更长期的结果。 该研究由瑞士楚格的 CRISPR Therapeutics AG 公司资助。 尼森博士曾担任多家制药公司的顾问，并负责监督艾伯维、安进、阿斯利康、CRSPR Therapeutics、Kardigan、礼来、诺华、新阿姆斯特丹制药和辉瑞等公司近期开展的临床试验。但他不接受来自商业机构的酬金、咨询费或其他任何形式的报酬。 拉芬博士目前或曾经担任Recor、美敦力、礼来、诺华、诺和诺德和Ripple Medical等公司的指导委员会或顾问委员会成员。他所在的机构曾获得阿斯利康、Arrowhead、Crispr Therapeutics、Kardigan、Mineralys和Retention等公司的研究经费。拉芬博士还从Belvoir Media Group和Elsevier获得版税。关于克利夫兰诊所 克利夫兰诊所是一家非营利性综合性学术医疗中心，集临床和住院治疗、科研和教育于一体。诊所位于俄亥俄州克利夫兰市，由四位杰出的医生于1921年创立，他们秉持合作、关爱和创新的原则，致力于提供卓越的患者护理。克利夫兰诊所开创了多项医学突破，包括冠状动脉搭桥手术和美国首例面部移植手术。克利夫兰诊所的精湛医术和卓越护理享誉美国乃至全球。克利夫兰诊所在全球拥有82,600名员工，其中包括5,786名受薪医生和研究人员，以及20,700名注册护士和高级执业人员，涵盖140个医学专科和亚专科。克利夫兰诊所拥有6,728张床位，包括位于克利夫兰市中心附近占地173英亩的主院区、23家医院和280家门诊机构，其分支机构遍布俄亥俄州东北部、佛罗里达州、内华达州拉斯维加斯等地。加拿大多伦多；阿联酋阿布扎比；以及英国伦敦。2024年，克利夫兰诊所医疗系统共接待门诊1570万人次，住院及观察33.3万人次，并完成32万例手术及其他医疗操作。患者来自美国各州以及112个国家。访问我们的网站clevelandclinic.org 。关注我们的新闻动态，请访问x.com/CleClinicNews。新闻及相关资源请访问 newsroom.clevelandclinic.org。 编者按： 克利夫兰诊所新闻服务中心可根据要求提供广播级采访和 B 卷素材。  新闻编辑室最新消息克利夫兰诊所在比奇伍德开设新诊所，专门提供神经科门诊服务 上传失败，网络异常。 重试克利夫兰诊所患者兼篮球明星将在第26届大克利夫兰体育奖颁奖典礼上荣获吉姆·多诺万勇气奖。克利夫兰诊所儿童医院成为美国首家采用磁辅助手术技术进行儿童胆囊切除术的医院 上传失败，网络异常。 重试克利夫兰诊所承诺捐赠200万美元支持地区食品银行 上传失败，网络异常。 重试VeloSano筹集了1450万美元，用于资助克利夫兰诊所的癌症研究。克利夫兰诊所研究人员公布了首个针对肥厚型心肌病的人体基因疗法的初步结果研究发现一种基因变异会增加非洲裔人群的心力衰竭风险Amazon One Medical 与克利夫兰诊所合作，在俄亥俄州东北部开设首家基层医疗诊所

The technology that many skeptics had previously given up on is now covered by the Centers for Medicare & Medicaid Services (CMS). CMS this week released the final national coverage determination (NCD), applicable to Medtronic’s Symplicity Spyral renal denervation (RDN) system, also known as the Symplicity blood pressure procedure, and Recor Medical’s Paradise ultrasound renal denervation (uRDN) system. The NCD provides Medicare patients with access to renal denervation procedures for the treatment of uncontrolled hypertension, or high blood pressure. Interestingly, in the proposal, patients were required to follow guideline-directed medical therapy for at least 3 months but in the final decision memo, this was reduced to six weeks. “This cuts the medical management criteria in half, which arguably is better than expected as it allows for a faster pathway to RDN than originally proposed,” wrote Ryan Zimmerman, a medtech analyst at BTIG. Zimmerman also noted that about 64% of Medicare claims had a hypertension diagnosis in FY21 and nearly half of all U.S. adults have high blood pressure (with 25% not under control). “We are very pleased with the final national coverage determination, which opens up patient access to renal denervation with the Symplicity Spyral RDN system, a safe and durable blood pressure-lowering procedure for patients who have not succeeded with medication or lifestyle changes alone,” said Jason Weidman, senior vice president and president of Medtronic’s coronary & renal denervation business. “This milestone enables physicians to offer patients more choices when managing hypertension, potentially leading to better outcomes for a population in urgent need of new options. We congratulate CMS and recognize all the work it took to get to this point.” The Symplicity Spyral RDN system is a minimally invasive procedure that delivers radiofrequency energy to nerves near the kidneys that can become overactive and contribute to high blood pressure. After sedation, the doctor inserts a single thin tube (known as a catheter) into the artery leading to the kidney. Once the tube is in place, the doctor administers energy to the system to calm the excessive activity of the nerves connected to the kidney. The tube is removed, leaving no implant behind. The Medtronic SPYRAL HTN global clinical program has studied RDN in more than 5,000 patients in the presence and absence of medication, and with high baseline cardiovascular risk, and is backed by experience in over 30,000 patients globally. The Symplicity Spyral RDN system has demonstrated sustained and durable drops in blood pressure out to three years in randomized control and real-world registry trials. In November 2023, FDA approved both Medtronic’s RDN system and Recor Medical’s Paradise ultrasound renal denervation system . “I would like to commend CMS for all the effort it took to issue this NCD. This is a transformative milestone for patients across the U.S. who continue to battle uncontrolled hypertension,” said Lara Barghout, CEO of Recor Medical. “By formally recognizing the proven clinical value of renal denervation and extending Medicare coverage, CMS is paving the way for broader, more equitable access to this breakthrough therapy. This decision not only reinforces the robust body of evidence supporting the Paradise uRDN system as a safe and effective adjunctive treatment but also delivers renewed hope to patients striving for better hypertension control and improved cardiovascular health.” Recor has been focused on developing and testing the Paradise uRDN system as an adjunctive treatment option for uncontrolled hypertension since 2009. The RADIANCE global program studied the Paradise uRDN system in three independently powered, sham-controlled, randomized clinical trials in over 500 patients with uncontrolled hypertension: RADIANCE II and RADIANCE-HTN SOLO, which studied patients with mild-moderate hypertension in an “off-meds” setting, and RADIANCE-HTN TRIO, which studied patients with resistant hypertension on standardized triple antihypertensive therapy. Each trial met its prescribed primary efficacy endpoint with a favorable safety profile consistently observed following ultrasound RDN treatment. Recor has also initiated the U.S. Global Paradise System post-approval study, which intends to collect real-world clinical evidence with long-term follow-up in up to 1,000 patients with uncontrolled hypertension. The Paradise uRDN system is a novel ultrasound-based RDN technology designed to lower blood pressure by denervating the sympathetic nerves surrounding the renal arteries, reducing the overactivity that can lead to hypertension. The Paradise uRDN system delivers two to three doses of 360-degree ultrasound energy — lasting seven seconds each — through the main renal arteries to the surrounding nerves. The Paradise catheter features the exclusive HydroCooling system, which circulates sterile water through the balloon catheter during the procedure to help protect the renal artery wall.

A Big Win for Renal Denervation CMS released the final national coverage determination on renal denervation, applicable to Medtronic’s Symplicity Spyral system and Recor Medical’s Paradise system. Getting Ahead of the 'Curve' Curve Biosciences raised $40M to advance its AI-powered blood tests for chronic disease monitoring. Curve spun out of Stanford University about three years ago. Taking TCT by 'Storm' Penumbra took TCT by storm last Sunday with the results of its landmark STORM-PE randomized controlled trial. The trial found that the use of mechanical thrombectomy with anticoagulation achieved superior reduction in right heart strain compared to anticoagulation therapy alone in patients with acute intermediate to high-risk pulmonary embolism.