CS-101

SHANGHAI, Aug. 26, 2025 /PRNewswire/ -- CorrectSequence Therapeutics Co., Ltd. (Correctseq), a clinical-stage biotechnology company pioneering transformer Base Editing (tBE) technology for the treatment of severe diseases, today announced the successful treatment of the first sickle cell disease (SCD) patient using its high-precision base-editing therapy CS-101. Continue Reading Picture: The patient, a 21-year-old woman from Nigeria, had previously suffered from recurrent VOCs. For six months after receiving CS-101, she remains crisis-free and in good health. The breakthrough was achieved in an Investigator-Initiated Trial (IIT) conducted in collaboration with the First Affiliated Hospital of Guangxi Medical University. The patient demonstrated a significant and sustained increase in fetal hemoglobin (HbF) levels, accompanied by a marked reduction in sickle hemoglobin (HbS). Six months post-treatment, the HbF-to-HbS ratio stabilized at 6.5:3.5, with total hemoglobin consistently exceeding 120 g/L. Over the six-month follow-up period, the patient experienced no vaso-occlusive crises (VOCs) and successfully resumed normal daily activities — marking the first reported clinical treatment of sickle cell disease in China using base editing technology. Picture: The patient, a 21-year-old woman from Nigeria, had previously suffered from recurrent VOCs. For six months after receiving CS-101, she remains crisis-free and in good health. Sickle Cell Disease and the Need for Curative Therapies Hemoglobinopathies — including SCD and β-thalassemia (β-thal) — are the world's most common group of monogenic diseases, with approximately 7% of the global population carrying a pathogenic variant. Each year, around 400,000 newborns are affected. SCD, caused by mutations in the β-globin gene, leads to abnormal, sickle-shaped red blood cells, resulting in chronic anemia, recurrent pain crises, increased infection risk, and progressive organ damage. Globally, about 3.5% of the population carries the sickle mutation, with roughly 300,000 affected infants born annually, especially in Africa, the Mediterranean, the Middle East, and South Asia. Existing treatments such as symptomatic drugs and blood transfusions can alleviate symptoms but do not offer a cure. Hematopoietic stem cell transplantation (HSCT) can be curative, but the requirement for a matched donor limits accessibility. Gene-editing approaches, particularly base editing, can activate fetal hemoglobin in a patient's own blood cells — eliminating the need for a donor and offering a shorter, potentially safer treatment path. CS-101: Precision Base Editing for Hemoglobinopathies CS-101 is a gene-editing therapy based on the high-precision transformer Base Editor (tBE) platform (Wang et al., Nat Cell Biol, 2021), developed by Correctseq's scientific co-founders. The therapy targets β-hemoglobinopathies by precisely modifying a regulatory element in the promoter of the γ-globin genes (HBG1/2), mimicking naturally occurring single-nucleotide variants found in individuals with hereditary persistence of fetal hemoglobin. This reactivates γ-globin expression to produce functional HbF, which prevents sickling and reduces hemolysis. In the IIT, the patient began with a baseline hemoglobin level of 67.3 g/L. Following CS-101 treatment in February 2025, she achieved rapid hematopoietic recovery, with neutrophil engraftment in 13 days and platelet counts surpassing 50×10⁹/L in 21 days. Her HbF levels rose from 4.4% to 34.6% within one month, and since the third month post-treatment, HbF levels have remained above 60% while HbS has stayed below 40%. No VOCs or treatment-related adverse events have been reported. A Safer, More Effective Approach Compared with conventional CRISPR/Cas9-based therapies, CS-101 delivers efficient hematopoietic recovery and higher HbF-to-HbS ratio, without the risks of large DNA deletions, chromosomal rearrangements, or off-target mutations. To date, CS-101 has been used to treat nearly 20 patients with β-thalassemia or SCD in clinical trials. The first β-thalassemia patient treated has remained transfusion-free for over 22 months. This positions CS-101 as a potential best-in-class gene-editing therapy — and the world's first base editing treatment — for β-hemoglobinopathies. Global Development and Next Steps The Phase I trial of CS-101 for β-thalassemia has been completed, with all patients achieving transfusion independence. Pivotal Phase II/III trials are set to begin soon, and global recruitment for both SCD and β-thalassemia trials is now underway. Correctseq is committed to advancing CS-101 — a first-in-class, China-originated gene-editing therapy — to deliver safe, effective, and accessible treatments for patients worldwide living with severe hemoglobin disorders. About CorrectSequence Therapeutics CorrectSequence Therapeutics (Correctseq), incubated at ShanghaiTech University, is dedicated to leveraging innovative gene-editing technologies to transform the lives of people with severe diseases. The company has developed multiple state-of-the-art base-editing systems that offer exceptional precision, minimize off-target effects, and enhance in vivo editing efficiency. Correctseq's mission is to discover, develop, manufacture, and commercialize curative genetic medicines across a broad range of conditions. Its robust pipeline spans genetic disorders, metabolic diseases, and cardiovascular conditions, with several programs already advancing toward clinical development. For more information, visit . Acknowledgments: The First Affiliated Hospital of Guangxi Medical University, ShanghaiTech University, Shanghai Clinical Research and Trial Center. Media Contact: Business Cooperate: [email protected] Clinical Trial Recruitment: [email protected] Photo - WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

8月26日，正序生物宣布，与广西医科大学第一附属医院合作开展的针对镰刀型细胞贫血病（SCD）的碱基编辑药物CS-101注射液的研究者发起的临床研究（IIT）成功“治愈”首位患者，患者在治疗后胎儿血红蛋白（HbF）水平显著且持续上升，镰状血红蛋白（HbS）水平显著且持续降低，治疗后6个月起HbF与HbS比例稳定在6.5:3.5，总血红蛋白浓度稳定至120 g/L以上，治疗后6个月中没有出现血管闭塞危象（VOCs，vaso-occlusive crises），已回归到正常生活中。 血红蛋白病是一种单基因遗传病，常见的主要有β-地中海贫血（β-地贫）和镰刀型细胞贫血病。每年大约有40万名婴儿出生时患有血红蛋白病。其中，镰刀型细胞贫血病临床表现为慢性溶血性贫血、再发性疼痛危象、易感染、慢性局部缺血导致器官组织损害等，严重时可危及生命。 目前，治疗镰刀型细胞贫血病的常规疗法为药物治疗和输血治疗，这两种对症治疗的方式只能缓解疾病带来的痛苦，不能实现彻底治愈。另外还有一种治愈性治疗策略为造血干细胞移植，传统的异体造血干细胞移植需要等待匹配的供体，在实际情况中常常难以实现。 正序生物CS-101注射液是一款针对β-血红蛋白病的碱基编辑药物。CS-101注射液通过采集患者自体造血干细胞，利用tBE对患者自体造血干细胞中的HBG1/2启动子区域进行精准碱基编辑，模拟健康人群中天然存在的有益碱基突变，重新激活γ-珠蛋白的表达，再将编辑后的造血干细胞回输至患者体内，使得患者胎儿血红蛋白浓度快速升高，有效抑制红细胞镰变，显著减少血管闭塞危象和溶血。 CS-101注射液治疗的首位镰贫患者是一名21岁女性，在接受CS-101治疗前总血红蛋白浓度为67.3 g/L，有反复发作的血管闭塞危象。患者在2025年2月接受tBE编辑自体干细胞移植治疗后，便快速高效地实现了造血重建，治疗后第13天获得中性粒细胞植入，第21天血小板浓度达到50x10^9/L以上。治疗后1个月胎儿血红蛋白比例从治疗前的基线水平4.4%迅速上升至34.6%，治疗后3个月胎儿血红蛋白比例持续大于60%，镰状血红蛋白比例持续小于40%。治疗后6个月中，没有出现VOCs，且未发现产品相关的不良事件。 CS-101可以使患者迅速地完成造血重建，更快速并大幅提升胎儿血红蛋白水平从而降低镰刀状血红蛋白的占比，更高效且稳定地阻止红细胞发生镰变；同时不会引发患者DNA大片段缺失、染色体突变、脱靶突变等风险，显示出了更好的有效性和安全性。CS-101注射液已经在临床试验中成功“治愈”了近20例β-地贫患者和镰刀型细胞贫血病患者，首位β-地贫患者在接受CS-101治疗后已经摆脱输血依赖超过22个月。 目前，正序生物CS-101注射液针对β-地贫的1期临床试验已经完成，所有患者在治疗后均成功摆脱输血依赖，关键性临床试验（2/3期临床试验）即将开始。针对镰贫的IIT试验和针对β-地贫的关键性临床试验的全球招募正在同步进行。 参考资料： [1] 中国首例碱基编辑临床治疗镰刀型细胞贫血病获得成功.From https://mp.weixin.qq.com/s/xWrXRHSL2ZML68Nr58adYA 免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。