OBiO Technology Congratulates on The First Clinical Gene Editing Therapy to Treat An Overseas Patient in China by CorrectSequence Therapeutics
OBiO Technology Congratulates on The First Clinical Gene Editing Therapy to Treat An Overseas Patient in China by CorrectSequence Therapeutics
SHANGHAI, Aug. 5, 2024 /PRNewswire/ -- Recently, CorrectSequence Therapeutics Co., Ltd. (Correctseq) announced a significant milestone in their base editing therapy CS-101 for transfusion-dependent β-thalassemia. Utilizing their pioneering transformer Base Editor (tBE), Correctseq has successfully cured the first overseas patient with transfusion-dependent β-thalassemia in a clinical trial in collaboration with the First Affiliated Hospital of Guangxi Medical University. The patient has achieved a sustained transfusion-free status for over two months, with the hemoglobin level stabilized at above 120 g/L. As a result, the patient has been able to resume a normal life. This milestone marks China's first documented report of an overseas patient being clinically cured through gene editing therapy. OBiO Technology (Shanghai) Corp., Ltd. (OBiO Technology) extends its heartfelt congratulations and best wishes to the patient who has been cured, and warmly congratulates the partner Correctseq on this significant progress.
上海，2024 年 8 月 5 日 /PRNewswire/ — 最近，CorrectSequence Therapeutics Co., Ltd.（CorrectSeq）宣布其治疗输血依赖性 β-地中海贫血的基础编辑疗法 CS-101 取得了重要的里程碑。Correctseq利用其开创性的变压器基础编辑器（tBE），在与广西医科大学第一附属医院合作的临床试验中成功治愈了首位输血依赖型β-地中海贫血的海外患者。患者已持续保持两个多月的无输血状态，血红蛋白水平稳定在120 g/L以上。因此，患者得以恢复正常生活。这一里程碑标志着中国首次记录在案的海外患者通过基因编辑疗法获得临床治愈的报告。奥生物科技（上海）有限公司（OBio Technology）向治愈的患者表示衷心的祝贺和良好的祝愿，并热烈祝贺合作伙伴Correctseq取得的这一重大进展。
The Phase I clinical trial for the treatment of β-thalassemia with CS-101 is proceeding steadily. In tandem, a clinical trial targeting sickle cell disease (SCD) with CS-101 are in active preparation. A global recruitment program for SCD patients is recently launched.
使用 CS-101 治疗 β-地中海贫血的 I 期临床试验正在稳步进行中。同时，一项针对 CS-101 的镰状细胞病 (SCD) 的临床试验正在积极准备中。最近启动了一项针对SCD患者的全球招募计划。
OBiO Technology is a leading CDMO company specializing in cell and gene therapy in China, equipped with 15 GMP vector production lines and 20 GMP cell therapy production lines. As a reliable partner with Correctseq, OBiO Technology has provided solid support for CS-101, including process development, analytical development, production and IND filling services. OBiO Technology celebrates the significant achievements made in treating patients with CS-101, affirming the successful collaboration between the two companies. Driven by innovation and united in purpose, we believe our close partnership will bring hope and a brighter future to patients worldwide!
OBio Technology是一家领先的CDMO公司，专门从事中国细胞和基因治疗，拥有15条GMP载体生产线和20条GMP细胞疗法生产线。作为 Correctseq 的可靠合作伙伴，oBio Technology 为 CS-101 提供了坚实的支持，包括工艺开发、分析开发、生产和 IND 填充服务。OBio Technology 庆祝在治疗 CS-101 患者方面取得的重大成就，肯定了两家公司之间的成功合作。在创新的推动下，目标一致，我们相信我们的紧密伙伴关系将为全球患者带来希望和更光明的未来！
About CorrectSequence Therapeutics
关于 CorrectSequence 疗法
CorrectSequence TherapeuticsTM (CorrectseqTM), is a clinical-stage biotech company employing its proprietary transformer Base Editor (tBE) to pioneer next-generation gene editing therapies. Our leading pipeline candidate, CS-101, utilizing innovative base editor targeting HBG, curing β-hemoglobinopathies, has obtained IND approval from the China NMPA. Clinical data demonstrate its superior performance. Proof-of-concept (POC) data in mice for in vivo pipelines using tBE-editing therapies via lipid nanoparticle (LNP) delivery are available, including targets for metabolic dysfunction and associated diseases. Ex vivo multiplex editing of T cells on multiple targets simultaneously preserved T cell growth and function in vivo compared to non-edited cells, establishing tBE as the ideal gene editing tool for the next-generation cell therapy development. We are developing multiple pipeline programs targeting genetic diseases, metabolic disorders, and cardiovascular diseases.
CorrectSequence TherapeuticsTM（CorrectSeqTM）是一家处于临床阶段的生物技术公司，使用其专有的变压器基础编辑器（tBe）来开创下一代基因编辑疗法。我们的领先候选药物 CS-101 已获得中国国家药品监督管理局的临床试验批准，它使用针对HBG的创新碱基编辑器来治疗β-血红蛋白病。临床数据表明其卓越的性能。可获得通过脂质纳米颗粒 (LNP) 输送的 TBE 编辑疗法的体内管道小鼠的概念验证 (POC) 数据，包括代谢功能障碍和相关疾病的靶标。与未经编辑的细胞相比，对多个靶标上的 t 细胞进行体外多重编辑可同时保持体内 t 细胞的生长和功能，从而确立了 tBE 作为下一代细胞疗法开发的理想基因编辑工具。我们正在开发针对遗传疾病、代谢障碍和心血管疾病的多个管道项目。
Please refer to our homepage for more information about the tBE and its therapeutics applications at
请参阅我们的主页，了解有关 tBE 及其治疗应用的更多信息，网址为
About OBiO Technology
关于 OBio 科技
Established in 2013, OBiO Technology is a pioneering Contract Research Organization (CRO) and Contract Development and Manufacturing Organization (CDMO) in gene and cell therapy, offering comprehensive solutions. Our unwavering dedication ensures high-quality CRO and CDMO services for consumers worldwide across preclinical, IND, clinical, and commercial stages, including plasmids, mRNA, AAV, LVV, Ad viral vectors, cell therapy (CAR-T, NK, Treg, iPSCs), exosomes, and cutting-edge technologies like inducible viral vector packaging, ultralow endotoxin processes, and AAVneO screening for tissue-specific AAV variants. Guided by our mission "Enable Gene Therapy for Better Lives", we are dedicated to delivering top-tier services globally. From bench to clinic, we are committed to advancing your product and benefiting populations worldwide.
oBio Technology成立于2013年，是基因和细胞疗法领域的开创性合同研究组织（CRO）和合同开发与制造组织（CDMO），提供全面的解决方案。我们坚定不移的奉献精神确保在临床前、IND、临床和商业阶段为全球消费者提供高质量的 CRO 和 CDMO 服务，包括质粒、mRNA、AAV、LVV、AD 病毒载体、细胞疗法（car-T、Nk、Treg、iPSC）、外泌体以及诱导性病毒载体包装、超低内毒素工艺和 Aavneo 组织筛查等尖端技术特定的 AAV 变体。在 “让基因疗法改善生活” 使命的指导下，我们致力于在全球范围内提供一流的服务。从实验室到诊所，我们致力于改进您的产品并惠及全球人群。
SOURCE OBiO Technology (Shanghai) Corp., Ltd.
来源 OBio 科技（上海）有限公司