ZYNTEGLO ™ is an FDA-approved gene therapy that has the potential to free people with beta-thalassemia from regular transfusions. In two phase 3 studies, about 9 out of 10 (89%; 32/36 patients) treated with ZYNTEGLO achieved transfusion independence and had a normal or near-normal median total hemoglobin of 11.5 g/dL.* Who is ZYNTEGLO for?

2022 年 8 月 17 日，美国食品和药物管理局FDA批准了蓝鸟生物公司 Zynteglo（betibeglogene autotemcel，beti-cel），这是第一个基于细胞的基因疗法，用于治疗需要定期输血的成人和儿童β-地中海贫血患者。

ZYNTEGLO is an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of adult and pediatric patients with β-thalassemia who require regular red blood cell (RBC ...

Betibeglogene autotemcel, sold under the brand name Zynteglo, is a gene therapy for the treatment for beta thalassemia. [ 1 ] [ 5 ] [ 2 ] It was developed by Bluebird Bio and was given breakthrough therapy designation by the US Food and Drug Administration in February 2015.

Tradename: ZYNTEGLO Manufacturer: bluebird bio Inc. Indication: For treatment of adult and pediatric patients with ß-thalassemia who require regular red blood cell (RBC) transfusions; Product ...

2022年8月19日 · Zynteglo 是一款基于 bluebird bio 的 LentiGlobin 平台的的慢病毒基因疗法，是一种单次给药的基因治疗产品， 首先从患者骨髓中提取造血干细胞，然后通过慢病毒将β-珠蛋白基因的修饰形式的功能性拷贝 （βA-T87Q-globin基因） 添加到患者自身造血干细胞中，恢复血红 ...

ZYNTEGLO is a one-time gene therapy to treat beta-thalassemia (also known as beta-thalassemia major or Cooley’s Anemia) in patients who require regular transfusions. Beta-thalassemia is caused by a change in the beta-globin gene, which causes the body to produce reduced or no beta-globin.

ZYNTEGLO™, also called beti-cel (betibeglogene autotemcel), is a one-time gene therapy for beta thalassemia.It was approved by the U.S. Food and Drug Administration (FDA) in 2022 for eligible children and adults with beta thalassemia major (also known as Cooley's anemia) who are dependent on regular transfusions of red blood cells.

2020年1月16日 · 近日，该公司宣布，在德国推出Zynteglo（LentiGlobin，含βA-T87Q珠蛋白编码基因的自体CD34+细胞），这是一种一次性的基因疗法，用于适合造血干细胞（HSC）移植但没有人类白细胞抗原（HLA）匹配的HSC供体、年龄在12岁以上、非β0/β0基因型输血依赖性β地中海贫血（TDT）患者的治疗。 在欧盟，Zynteglo于2019年5月获得有条件批准，成为全球首个治疗TDT的基因疗法。 在美国，Zynteglo尚未获得批准，蓝鸟生物已启动向FDA滚动提交生物制品 …

Zynteglo有望提供一种取代标准护理疗法（定期RBC输注+铁螯合）的治疗方案。 Zynteglo是一种一次性基因疗法，开发用于治疗β-地中海贫血和镰状细胞病 ...