当地时间2026年6月2日，专注于新型基因编辑技术、处于临床阶段的创新生物医药科技企业正序生物(上海)宣布，中国首例碱基编辑临床治疗的镰刀型细胞贫血病(sickle cell disease，SCD)患者在接受CS-206注射液治疗后，已持续摆脱血管闭塞危象(VOCs，vaso-occlusive crises)超过15个月。患者于2025年2月14日接受CS-206回输，末次输血时间为回输后第11天。自回输至今，患者未再出现VOCs并持续脱离贫血状态，已达到主要疗效终点。随访数据展现了正序生物CS-206注射液良好的安全性和有效性。

患者是一位来自尼日利亚的21岁女性，治疗前有反复发作的严重VOCs。在2025年2月接受正序生物CS-206注射液治疗后，患者快速高效地实现了造血重建：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个月内胎儿血红蛋白(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3个月起，HbF与HbS比例持续稳定在6:4。未发现产品相关的不良事件。

CS-206注射液是正序生物自主开发的一款针对镰刀型细胞贫血病(简称“镰贫”)的碱基编辑药物，利用由上海科技大学自主研发的自主原创的高精准变形式碱基编辑器tBE(transformer Base Editor)(Wang et al., Nat Cell Biol, 2021)，对采集的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，从而快速提升患者胎儿血红蛋白水平。该疗法可有效抑制红细胞镰变，显著减少血管闭塞危象和溶血。

与基于CRISPR技术的基因编辑疗法相比，CS-206注射液具有不需要切断DNA双链即可对单个碱基进行精准校正的底层技术优势，不会引发患者基因组DNA大片段缺失、染色体突变、脱靶突变等风险，更安全高效;同时还能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。

镰刀型细胞贫血病是由于β-珠蛋白基因突变使红细胞呈镰刀状的遗传性疾病，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。全球约有3.5%的人口携带相关突变基因，每年约有30万婴儿出生时患有此病，在非洲、地中海沿岸、中东和南亚等地区发病率较高。它与β-地中海贫血同属于血红蛋白病。血红蛋白病是全世界最普遍的单基因遗传病之一，全世界约有7%的人口携带异常血红蛋白基因，每年约有40万名婴儿出生时患有此病。

基于tBE技术、采用相同治疗流程的正序生物CS-101注射液(针对β-地中海贫血)已经在临床试验中成功治愈十余位来自中国(Lai et al., Nature, 2026)、老挝、马来西亚、巴基斯坦等国内外β-地贫患者，截至2026年5月底，所有接受CS-101治疗的患者均已持续摆脱输血依赖超过15个月，最长的已超过30个月。

目前，正序生物正在全球范围内持续招募CS-206注射液针对镰贫的IIT试验患者，并加速推进基因编辑药物CS-101及CS-206的临床与上市进程，致力于为全球血红蛋白病患者提供疗效更优、安全性更高、性价比更佳的治疗方案。

(特别感谢广西医科大学第一附属医院、上海科技大学及上海临床研究中心)

English Version

High-Precision Base-Editing Therapy Demonstrates Durable VOC-Free Efficacy and Favorable Safety in Sickle Cell Disease

CorrectSequence Therapeutics Reports Positive 15-Month Follow-Up Data for CS-206

Shanghai, June 2, 2026 — CorrectSequence Therapeutics Co., Ltd. (Correctseq), a clinical-stage biotechnology company pioneering transformer Base Editing (tBE) technology for severe diseases, announced that the first sickle cell disease (SCD) patient being treated in China with its high-precision base-editing therapy CS-206 has remained free of vaso-occlusive crises (VOCs) for more than 15 months following engraftment. Starting from 60 Days after the last red-cell transfusion, the patient has remained free from VOCs and anemia for 13 consecutive months, achieving the primary efficacy endpoint. The follow-up data demonstrated favorable safety and efficacy of CS-206.

The patient, a 21-year-old woman from Nigeria, had experienced recurrent severe VOCs prior to treatment with CS-206. After receiving CS-206 treatment in February 2025, the patient achieved rapid and efficient hematopoietic reconstitution, with neutrophil engraftment observed on Day 13 and platelet counts exceeding 50×10^9/L on Day 21 post-treatment. Within one month after treatment, fetal hemoglobin (HbF) levels increased significantly and continuously, while sickle hemoglobin (HbS) levels declined substantially and persistently. Beginning at Month 3 after treatment, the HbF-to-HbS ratio stabilized at approximately 6:4 and has remained stable thereafter. No product-related adverse events have been observed to date.

CS-206 is a proprietary base-editing therapy independently developed by Correctseq for the treatment of SCD. The therapy utilizes the highly precise transformer Base Editor (tBE), an original gene-editing technology developed by Correctseq’s scientific co-founders (Wang et al., Nature Cell Biology, 2021), to precisely edit the HBG1/2 promoter region in autologous hematopoietic stem cells collected from patients. The editing mimics naturally occurring beneficial mutations found in healthy individuals, thereby reactivating γ-globin expression and rapidly increasing fetal hemoglobin levels.

This therapy effectively suppresses red blood cell sickling and significantly reduces vaso-occlusive crises and hemolysis. Compared with CRISPR-based gene-editing therapies for SCD, CS-206 offers the technological advantage of precise single-base correction without introducing DNA double-strand breaks, thereby avoiding risks such as large genomic deletions, chromosomal abnormalities, and off-target mutations. It is a safer and more efficient therapeutic approach. In addition, the therapy enables rapid hematopoietic recovery and substantial elevation of fetal hemoglobin levels, reducing the proportion of sickle hemoglobin and more effectively and durably preventing red blood cell sickling.

SCD is a hereditary hemoglobin disorder caused by mutations in the β-globin gene. Patients may experience chronic anemia, recurrent pain crises, infections, and progressive organ damage, with severe cases becoming life-threatening. Approximately 3.5% of global population carries the sickle cell mutation gene, and around 300,000 babies are born with the disease each year, with particularly high prevalence in Africa, the Mediterranean, the Middle East, and South Asia. SCD and β-thalassemia are both classified as hemoglobinopathies, among the world’s most common monogenic inherited diseases. Approximately 7% of global population carries abnormal hemoglobin genes, with an estimated 400,000 affected births annually worldwide.

Based on the same tBE technology platform and therapeutic pathway, CS-101 developed by Correctseq for β-thalassemia has successfully cured more than ten β-thalassemia patients from China (Lai et al., Nature, 2026), Laos, Malaysia, Pakistan in clinical trials. As of the end of May 2026, all patients treated with CS-101 have remained transfusion-independent for more than 15 months, with the longest duration exceeding 30 months.

Global recruitment for the investigator-initiated trial (IIT) of CS-206 for SCD is currently ongoing. Correctseq is accelerating the clinical development and commercialization of both CS-101 and CS-206, with the goal of providing hemoglobinopathy patients worldwide with safer, more effective, and more affordable treatment options.

About CorrectSequence Therapeutics

CorrectSequence Therapeutics (Correctseq), is a clinical-stage biotech company employing its proprietary transformer Base Editor (tBE) to pioneer next-generation gene editing therapies. The company has developed multiple state-of-the-art base-editing systems that offer exceptional precision, minimize off-target effects, and enhance ex vivo and in vivo editing efficiency. Its robust pipeline spans genetic disorders, metabolic diseases, and cardiovascular conditions, with several programs already advancing toward clinical development.

For more information, visit www.correctsequence.com.

Acknowledgments: The First Affiliated Hospital of Guangxi Medical University, ShanghaiTech University, Shanghai Clinical Research and Trial Center.

关于正序生物

正序生物(CorrectSequence TherapeuticsTM)

是一家专注于新型基因编辑技术、处于临床阶段的生物医药科技公司，致力于利用自主原创的碱基编辑体系，开发突破性精准疗法，造福全球患者和家庭。

公司基于以变形式碱基编辑器tBE为代表的自主知识产权碱基编辑系统搭建了融合多治疗领域的新药发现平台，可长期开发和筛选针对多种遗传性疾病或罕见病的有效治疗靶点。所创建的多种精准疗法，在动物体内实现了疾病治疗靶点上的高效的编辑效率和未检出脱靶的安全性。目前，公司针对遗传疾病、代谢疾病、心血管疾病、肿瘤等布局了多条管线。首条管线CS-101正在IND临床试验阶段，成功治愈多位海内外β-地中海贫血患者;CS-206处于IIT研究阶段，成功治愈首例镰刀型细胞贫血病患者;体内管线CS-121已进入IIT研究阶段，成功治疗多位高血脂患者。

正序生物孵化自上海科技大学，拥有世界一流的创新技术平台和管线研发能力。在中国科学院上海高等研究院拥有先进的研发和CMC开发中心，在北京华贸中心设立有临床注册和运营中心。目前，公司汇聚了数十位来自全球基因治疗技术开发、药物研发、工艺开发与生产、临床开发和质量与合规等领域的优秀生物医药专家，管理团队和科研团队拥有平均十年以上工业界经验，硕士及以上学位比例超过65%，核心技术人员毕业于国内外顶尖名校。