Reported Late Sunday, Bluebird Bio's Long-Term Data Presented Supports Beti-cel As A Potentially Curative Gene Therapy For Β-Thalassemia Patients
Reported Late Sunday, Bluebird Bio's Long-Term Data Presented Supports Beti-cel As A Potentially Curative Gene Therapy For Β-Thalassemia Patients
Updated data from studies of beti-cel in 63 patients with transfusion-dependent beta-thalassemia showed sustained transfusion independence and improvements in iron management through up to 9 years of follow-up (n=1)
对63名输血依赖性β-地中海贫血患者的beti-cel研究的最新数据显示，在长达9年的随访中，输血独立性持续，铁管理有所改善（n=1）
All four parent studies are complete, and all patients have transitioned to a long-term follow-up study (LTF-303)
所有四项家长研究均已完成，所有患者均已过渡到长期随访研究（LTF-303）
beti-cel is U.S. Food and Drug Administration (FDA) approved and marketed in the U.S. as ZYNTEGLO
beti-cel 已获得美国食品药品监督管理局 (FDA) 的批准，并以 ZYNTEGLO 的名义在美国上市
Updated data from bluebird bio inc.'s (NASDAQ:BLUE) gene therapy program in transfusion-dependent (TDT) beta-thalassemia were presented today at the 65th American Society of Hematology (ASH) Annual Meeting & Exposition. Updated follow-up data showed sustained treatment effect, reduced iron management burden and improved quality of life measures in patients with beta-thalassemia who require regular red blood cell transfusions following treatment with betibeglogene autotemcel (beti-cel) (FDA approved and marketed in the U.S. as ZYNTEGLO).
来自蓝鸟生物公司的更新数据。”s（纳斯达克股票代码：BLUE）输血依赖性（TDT）β-地中海贫血的基因疗法项目今天在美国血液学会（ASH）第65届年会和博览会上发布。最新的随访数据显示，在使用betibeglogene autotemcel（beti-cel）（美国食品药品管理局批准并在美国以ZYNTEGLO的名义上市）治疗后需要定期输血红细胞的β-地中海贫血患者，其治疗效果持续，铁管理负担减轻，生活质量得到改善。
"Long-term results presented at ASH 2023 showed durable transfusion independence and a continued positive safety profile in patients with beta-thalassemia treated with our beti-cel gene therapy through up to nine years of follow-up," said Richard Colvin, M.D., Ph.D., chief medical officer, bluebird bio. "These data represent the longest follow-up with a gene therapy for patients with beta-thalassemia requiring regular transfusions and continue to show that beti-cel is a potentially curative therapy across ages and genotypes, through the achievement of durable transfusion independence and normal or near-normal hemoglobin levels."
蓝鸟生物首席医学官理查德·科尔文医学博士、博士说：“在ASH 2023上公布的长期结果显示，通过长达九年的随访，使用我们的beti-cel基因疗法治疗的β-地中海贫血患者具有持久的输血独立性，并且安全性持续良好。”“这些数据代表了需要定期输血的β-地中海贫血患者的基因疗法随访时间最长，并继续表明，通过实现持久的输血独立性和正常或接近正常的血红蛋白水平，beti-cel是一种跨年龄和基因型的潜在治疗方法。”
译文内容由第三方软件翻译。