SRSD-107

Sept. 22, 2025 -- CRISPR Therapeutics (NASDAQ: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, and Sirius Therapeutics, a clinical stage biotech company developing innovative small interfering RNA (siRNA) therapies for global markets, today announced that the first patient has been dosed in a Phase 2 clinical trial of SRSD107, a next-generation, long-acting Factor XI (FXI) siRNA for the prevention of venous thromboembolism (VTE) in patients undergoing total knee arthroplasty (TKA). SRSD107 is being co-developed by CRISPR Therapeutics and Sirius Therapeutics as part of a strategic collaboration to advance innovative treatments for cardiovascular and clotting-related diseases “We are pleased to announce that our Phase 2 clinical trial is now underway, and the first patient has been dosed,” said Naimish Patel, M.D., Chief Medical Officer of CRISPR Therapeutics. “Until now, existing anticoagulant options have been limited by bleeding risk, frequent dosing, and complex management challenges for patients with high thrombotic risk. SRSD107 offers the potential to reduce pathological thrombosis while minimizing bleeding risk, with sustained but reversible pharmacodynamic effects and the possibility of infrequent dosing. We look forward to exploring how this differentiated approach could meaningfully improve outcomes for patients in need.” “We are very excited to announce that the first patient has been dosed in our Phase 2 trial of SRSD107, marking a significant milestone for this program,” said Patrick Yue, M.D., Chief Medical Officer of Sirius Therapeutics. “This study will evaluate clinical efficacy as proof of concept for Factor XI inhibition using our siRNA approach, building on the positive results from our Phase 1 trials, and we look forward to the progress of this trial.” The ongoing Phase 2 clinical trial is a randomized, multicenter, global study evaluating the safety and efficacy of SRSD107 for the prevention of VTE in patients undergoing TKA. The trial will assess the anticoagulant effects and pharmacological profile of SRSD107 and help inform dose selection for future pivotal studies, with the goal of confirming its potential as a differentiated approach for reducing thrombotic risk in patients. SRSD107 is designed to selectively inhibit FXI, a key driver of pathological thrombosis, with minimal impact on normal hemostasis. In prior Phase 1 clinical trials conducted in Australia and China, single doses of SRSD107 were well tolerated and demonstrated strong, sustained pharmacodynamic effects, including reductions of over 93% in FXI levels, along with more than a twofold increase in activated partial thromboplastin time (aPTT) relative to baseline. These effects were sustained, with responses maintained for up to six months post-dosing. SRSD107 has the potential to be a best-in-class FXI inhibitor, achieving deep reductions in FXI with the possibility of infrequent, semi-annual subcutaneous administration and offering reversibility not observed with other anti-FXI modalities. The addressable population includes patients with atrial fibrillation, VTE, cancer-associated thrombosis, chronic Coronary Artery Disease (CAD), chronic Peripheral Vascular Disease (PVD), end-stage renal disease requiring hemodialysis, and patients undergoing major orthopedic surgery, where bleeding risk limits existing therapies. Thrombosis, or blood clot formation, is the common underlying mechanism of most cases of myocardial infarction, ischemic stroke, and venous thromboembolism. Published data in The Lancet1 estimate that thromboembolic disorders are estimated to account for approximately one in four deaths worldwide. SRSD107 is a novel double-stranded small interfering ribonucleic acid (siRNA), that is designed to target the human coagulation factor XI (FXI) mRNA and inhibit FXI protein expression. By modulating the intrinsic coagulation pathway, SRSD107 has the potential to provide anticoagulant and antithrombotic effects. CRISPR Therapeutics and Sirius Therapeutics entered into a strategic collaboration in 2025 to develop and commercialize novel small interfering RNA (siRNA) therapies for thromboembolic disorders and other serious diseases. The lead program, SRSD107, is a long-acting siRNA targeting Factor XI (FXI) with the potential to offer best-in-class efficacy and safety. Under the agreement, the companies will co-develop SRSD107 and share costs and profits equally. CRISPR Therapeutics will lead commercialization in the U.S., while Sirius will lead in Greater China. The collaboration also provides CRISPR Therapeutics with the option to license up to two additional siRNA programs. This partnership expands CRISPR Therapeutics’ therapeutic portfolio into RNA-based medicines, complementing its ongoing efforts in gene editing and broadening its impact across serious and chronic diseases. For Sirius, the collaboration marks a major milestone in its mission to deliver innovative RNA-based therapies globally, leveraging deep expertise in siRNA design and delivery. Since its inception over a decade ago, CRISPR Therapeutics has evolved from a research-stage company advancing gene editing programs into a leader that celebrated the historic approval of the first-ever CRISPR-based therapy. The Company has a diverse portfolio of product candidates across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine, cardiovascular, autoimmune, and rare diseases. In 2018, CRISPR Therapeutics advanced the first-ever CRISPR/Cas9 gene-edited therapy into the clinic to investigate the treatment of sickle cell disease and transfusion-dependent beta thalassemia. Beginning in late 2023, CASGEVY® (exagamglogene autotemcel [exa-cel]) was approved in several countries to treat eligible patients with either of these conditions. The Nobel Prize-winning CRISPR technology has revolutionized biomedical research and represents a powerful, clinically validated approach with the potential to create a new class of potentially transformative medicines. To accelerate its efforts, CRISPR Therapeutics has formed strategic partnerships with leading companies, including Vertex Pharmaceuticals. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Boston, Massachusetts and San Francisco, California. CRISPR THERAPEUTICS® standard character mark and design logo are trademarks and registered trademarks of CRISPR Therapeutics AG. CASGEVY® and the CASGEVY logo are registered trademarks of Vertex Pharmaceuticals Incorporated. All other trademarks and registered trademarks are the property of their respective owners. Sirius is a global, clinical-stage biotech company developing innovative siRNA therapies focusing on the treatment of chronic diseases. The Company’s pipeline is centered around three key franchises with mega blockbuster potential: coagulation disorders, cardiometabolic diseases, and obesity. Sirius’ most advanced investigational programs include SRSD107, a FXI inhibitor targeting the anticoagulation market, SRSD216, an inhibitor of Lp(a) synthesis intended to address atherosclerotic cardiovascular disease, and SRSD384, an INHBE inhibitor for managing obesity. Founded in 2021 by a world-class leadership team and investors, Sirius has established an innovation center in the United States and a translational medicine center in China. Sirius has raised nearly US$150 million in funding to date from OrbiMed, Creacion Ventures, Hankang Capital, Delos Capital, and BioTrack Capital. 1 Lozano R, Naghavi M, Foreman K, et al. Global and regional mortality from 235 causes of death for 20 age groups in 1990 and 2010: a systematic analysis for the Global Burden of Disease Study 2010. Lancet. 2012;380, 2095-1128. The content above comes from the network. if any infringement, please contact us to modify.

Sirius NEWS 靖因药业 2025 年 9 月 22 日，美国圣地亚哥、中国上海、瑞士楚格及美国波士顿——靖因药业是一家临床阶段的生物技术公司，致力于为全球市场开发创新的小干扰RNA (siRNA) 疗法；CRISPR Therapeutics（纳斯达克股票代码：CRSP）是一家专注于研发用于治疗严重疾病的变革性基因药物的生物制药公司。今日双方宣布，SRSD107用于预防全膝关节置换术后患者的静脉血栓栓塞症 (VTE)的II期临床试验已完成首例患者给药。SRSD107是一款新一代、长效、特异性肝靶向的凝血因子XI (FXI) siRNA疗法。 SRSD107由靖因药业和CRISPR Therapeutics 共同开发，是双方战略合作的一部分，旨在推进心血管和凝血相关疾病的创新疗法。 靖因药业首席医疗官Patrick Yue医学博士表示：“我们非常高兴地宣布，SRSD107的II期临床试验已完成首例患者给药，标志着该项目取得了重要的里程碑。本研究将基于I期临床试验的积极成果，作为概念验证，评估通过siRNA疗法抑制凝血XI因子的临床疗效。我们期待着这项试验的进展。” CRISPR Therapeutics首席医疗官 Naimish Patel医学博士表示，“我们很高兴宣布SRSD107的II期临床试验现已正式启动，并完成首位患者给药。现有的抗凝药物选择仍面临诸多挑战和限制，包括出血风险高、需频繁给药，以及在高血栓风险患者中的管理复杂性。SRSD107有望在降低出血风险的同时，有效减少病理性血栓形成，并具有持续，但是可逆的药效学作用和低频给药的潜力。我们期待这一差异化疗法能为有需求的患者带来实质性的临床获益。” 正在进行的II期临床试验是一项全球多中心、随机对照研究，旨在评估SRSD107在接受全膝关节置换术患者中预防静脉血栓栓塞 (VTE) 的安全性和有效性。该试验将评估 SRSD107的抗凝作用和药理学特性，并为未来关键性研究的剂量选择提供依据，以验证其作为降低患者血栓风险的差异化治疗策略的潜力。 SRSD107旨在选择性抑制凝血因子XI (FXI), 这一因子是病理性血栓形成的关键驱动因素，同时将对正常止血过程影响极小。在此前于澳大利亚和中国开展的I期临床试验中，SRSD107单次给药表现出良好的耐受性，并表现出强效且持久的药效学作用，包括FXI水平降低超过95%，以及活化部分凝血活酶时间 (aPTT) 相对于基线延长超过两倍。这些药效持续显著，给药后反应可持续达六个月。SRSD107有望成为同类最佳的 FXI抑制剂，不仅能实现大幅降低FXI水平，还具备每半年一次的低频次皮下注射的潜力，并提供其他FXI抑制剂所不具备的可逆性。其潜在适应人群包括房颤患者、静脉血栓栓塞症患者、癌症相关血栓患者、慢性冠状动脉疾病 (CAD)和慢性外周血管疾病 (PVD)患者、需接受血液透析的终末期肾病患者，以及因出血风险限制现有治疗方案的重大骨科手术患者。 关于血栓栓塞性疾病 血栓形成，即血液凝块的生成，是心肌梗死、缺血性脑卒中及静脉血栓栓塞症等多数疾病的共同发病机制。《柳叶刀》期刊发布，血栓栓塞性疾病估计占全球死亡人数的四分之一。 关于SRSD107 SRSD107是一款新型双链小干扰核糖核酸（siRNA）疗法，。该药物特异性靶向人类凝血因子XI（FXI）的mRNA，抑制FXI蛋白表达。通过调节内源性凝血途径，SRSD107有望实现抗凝和抗血栓形成的双重功效。 关于CRISPR Therapeutics与靖因药业的合作 CRISPR Therapeutics与靖因药业于2025年达成战略合作，共同开发和商业化用于治疗血栓栓塞性疾病及其他严重疾病的新型小干扰RNA（siRNA）疗法。双方的首个合作项SRSD107是一款靶向凝血因子XI（FXI）的长效siRNA药物，有望在疗效和安全性方面实现同类最优。根据协议，双方将共同开发SRSD107，并按比例分担成本与分享利润。CRISPR Therapeutics将负责该产品在美国地区的商业化，靖因将负责大中华区的商业化。合作协议还包括CRISPR Therapeutics对最多两个siRNA项目的授权选择权。此次合作不仅拓展了CRISPR Therapeutics在RNA疗法领域的，并与其基因编辑研发形成互补，也进一步扩大了其在严重及慢性疾病领域的影响力。对于靖因药业而言，此次合作是其全球推广创新siRNA疗法的重要里程碑，充分发挥其在siRNA设计与递送方面的深厚技术优势。 关于靖因药业 靖因药业是一家全球化临床阶段的生物技术公司，致力于开发治疗慢性疾病的创新siRNA疗法。公司研发管线聚焦三大具有重磅药物潜力的治疗领域：凝血功能障碍、心血管代谢疾病和肥胖症。其中进展最快的研究性项目包括针对抗凝血市场的FXI抑制剂SRSD107、治疗动脉粥样硬化性心血管疾病的脂蛋白(a)抑制剂SRSD216，以及用于肥胖管理的INHBE抑制剂SRSD384。 靖因药业由世界级管理团队和投资者于2021年创立，在美国设立创新研发中心，并在中国建立转化医学中心。公司迄今已获得近1.5亿美元融资，投资方包括奥博资本、泓元资本、汉康资本、康禧资本以及博远资本。 关于CRISPR Therapeutics 十余年来，CRISPR Therapeutics已从推进基因编辑项目的研究阶段公司发展成为史上首款CRISPR疗法获批的行业领导者。公司拥有覆盖血红蛋白病、肿瘤学、再生医学、心血管、自身免疫病及罕见病等广泛疾病领域的多样化候选产品管线。2018年，CRISPR Therapeutics率先将全球首款CRISPR/Cas9基因编辑疗法推进临床，用于镰状细胞病和输血依赖性β地中海贫血的治疗。自2023年底起，CASGEVY®（exagamglogene autotemcel [exa-cel]）已在多个国家获批用于治疗上述两种适应症的合格患者。这项荣获诺贝尔奖的CRISPR技术彻底改变了生物医学研究，代表了一种经过临床验证的强大方法，有望开创具有变革潜力的新型药物类别。为加速拓展研发进程，CRISPR Therapeutics已与福泰制药等行业领军企业建立战略合作。CRISPR Therapeutics AG总部位于瑞士楚格，其全资美国子公司CRISPR Therapeutics, Inc.及研发运营中心位于马萨诸塞州波士顿和加利福尼亚州旧金山。了解更多信息请访问www.crisprtx.com。 CRISPR THERAPEUTICS® 标准字符标志和设计标识是CRISPR Therapeutics AG的商标及注册商标。CASGEVY® 及CASGEVY标识是Vertex Pharmaceuticals Incorporated的注册商标。所有其他商标及注册商标均归各自所有者所有。 靖因药业前瞻性声明 本新闻稿可能包含基于靖因药业当前信念、假设和预期的"前瞻性声明"，这些声明并非历史事实，通常可通过"将会"、"可能"、"预计"、"相信"、"计划"、"打算"、"预计"等类似含义的词语识别。尽管我们认为这些预测合理，但未来事件本身具有不确定性，实际结果或表现可能与预期存在重大差异。敬请注意，任何前瞻性声明均受显著已知及未知风险与不确定性的影响。本文件所有前瞻性声明均需参照本节警示声明的限定。新闻稿所提供信息截至发布之日，基于我们当时认为合理的假设，除适用法律要求外，我们无义务更新任何前瞻性声明。 CRISPR Therapeutics前瞻性声明 本新闻稿中涉及非历史事实的陈述均属《1995年私人证券诉讼改革法》所界定的"前瞻性声明"。由于此类陈述存在风险和不确定性，实际结果可能与表达或暗示的内容存在重大差异。包括但不限于Patel博士及Yue博士在本新闻稿中的陈述，以及涉及以下方面的所有声明：(i) CRISPR Therapeutics临床前研究、临床试验及管线产品与项目，包括对数据、安全性和有效性的普遍预期；(ii) 本新闻稿所载数据及利用正在进行或计划中临床试验数据设计及启动后续试验的能力；(iii) SRSD107临床项目的进展状态及开发时间表；(iv) CRISPR Therapeutics的战略与目标；(v) 合作双方未来活动及与靖因药业合作预期收益；(vi) 基因编辑与递送技术及疗法的治疗价值、开发与商业化潜力（包括CRISPR/Cas9）。导致前瞻性声明不确定的风险因素包括但不限于CRISPR Therapeutics最新10-K表格年度报告及后续向美国证券交易委员会提交文件中"风险因素"章节讨论的内容。现有及潜在投资者请注意不应过度依赖这些仅代表发布之日观点的前瞻性声明。CRISPR Therapeutics否认任何更新或修订新闻稿中前瞻性声明的义务（法律要求除外）。本新闻稿讨论的研究性疗法不构成对疗效或安全性的结论，无法保证任何研究性疗法能成功完成临床开发或获得监管批准。