European Commission Approves First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY (Exagamglogene Autotemcel), for the Treatment of Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT)
European Commission Approves First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY (Exagamglogene Autotemcel), for the Treatment of Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT)
CRISPR Therapeutics (NASDAQ:CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, announced today that the European Commission has granted conditional marketing authorization to CASGEVY (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene edited therapy. CASGEVY is approved for the treatment of patients who are 12 years of age and older with severe sickle cell disease (SCD) characterized by recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT), for whom hematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen matched related HSC donor is not available.
专注于为严重疾病开发变革性基因药物的生物制药公司CRISPR Therapeutics（纳斯达克股票代码：CRSP）今天宣布，欧盟委员会已向CRISPR/Cas9基因编辑疗法CASGEVY（exagamglogene autotemcel [exa-cel]）提供了有条件的上市许可。CASGEVY 获准用于治疗 12 岁及以上的严重镰状细胞病 (SCD) 患者，其特征是复发性血管闭塞危象 (VOC) 或输血依赖性 β 地中海贫血 (TDT)，适合造血干细胞 (HSC) 移植，且没有与之相关 HSC 供体相匹配的人类白细胞抗原。
译文内容由第三方软件翻译。