镰状细胞病
输血依赖型β-地中海贫血
RM001是一款在研的自体CD34+造血干细胞和祖细胞(HSPC)疗法,用于治疗输血依赖型β-地中海贫血。该疗法采用CRISPR/Cas9在体外编辑细胞的HBG1/2启动子,以提高γ-珠蛋白表达和胎儿血红蛋白(HbF)的产生。
镰状细胞病
RM001是一款在研的自体CD34+造血干细胞和祖细胞(HSPC)疗法,用于治疗镰状细胞病(SCD)。该疗法采用CRISPR/Cas9在体外编辑细胞的HBG1/2启动子,以提高γ-珠蛋白表达和胎儿血红蛋白(HbF)的产生。
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EHA 2026(European Hematology Association)年会报道:DURABLE CLINICAL BENEFITS WITH RM001 (AUTOLOGOUS HBG1/2 PROMOTER-MODIFIED CD34+ HEMATOPOIETIC STEM AND PROGENITOR CELLS) FOR TRANSFUSION-DEPENDENT B-THALASSEMIA Link ↗ -
ASH 2025(American Society of Hematology)年会报道:All 19 subjects with transfusion-dependent β-thalassemia achieved transfusion independence after treatment with RM001 (autologous HBG1/2 promoter-modified CD34+ hematopoietic stem and progenitor cells) Link ↗ -
EHA 2025 年会报道:Updated Safety and Efficacy Results of RM001, Autologous HBG1/2 Promoter-Modified CD34+ Hematopoietic Stem and Progenitor Cells, in Treating Transfusion-Dependent β-Thalassemia Link ↗ -
ASH 2024 年会报道:Updated Safety and Efficacy Results of RM001, Autologous HBG1/2 Promoter-Modified CD34+ Hematopoietic Stem and Progenitor Cells, in Treating Transfusion-Dependent β-Thalassemia Link ↗ -
EHA 2024 年会报道:Safety and Efficacy of RM001, Autologous HBG1/2 Promoter-Modified CD34+ Hematopoietic Stem and Progenitor Cells, in Transfusion-Dependent β-Thalassemia Link ↗ - 瑞风生物公布地贫基因编辑药物RM001临床一期进展,助力开启中国基因编辑药物时代 Link ↗
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ASH 2023 年会报道:Safety and Efficacy of RM001 (Autologous HBG1/2 Promoter-modified CD34+ Hematopoietic Stem and Progenitor Cells) in Patients with Transfusion-Dependent β-Thalassemia Link ↗ -
EHA 2023 年会报道:Safety and Efficacy of RM001 in Patients with Transfusion-Dependent β-Thalassemia: Early Results from the Ongoing Study of Autologous HBG1/2 Promoter-Modified CD34+ Hematopoietic Stem and Progenitor Cells Link ↗ -
ASH 2022 年会报道:Preliminary Result of the Safety and Efficacy of Autologous HBG1/2 Promoter-Modified CD34+ Hematopoietic Stem and Progenitor Cells (RM001) in Transfusion-Dependent β-Thalassemia Link ↗ - 瑞风生物地贫基因编辑药物RM001临床研究进展于2022第64届美国血液学年会(ASH)公布 Link ↗
- 瑞风生物β-地中海贫血基因编辑药物IND获国家药监局批准 Link ↗
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EHA 2022 年会报道:Initial Safety and Efficacy Study of RM001, Autologous HBG1/2 Promoter-Modified CD34+ Hematopoietic Stem and Progenitor Cells, in Transfusion-Dependent β-Thalassemia Link ↗ - 瑞风生物携全球首个新靶点基因编辑地贫临床成果亮相2022欧洲血液学年会(EHA) Link ↗
- 首个突破!我国基因编辑技术成功治疗成人重型地贫 Link ↗
- 全球首个新靶点治疗重型β地贫,瑞风生物取得新突破 Link ↗