Beam公司披露首个基于基因编辑的镰状细胞患者的临床数据：一例患者死亡

Beam therapeutics对第一批接受其前精神基因编辑治疗镰状细胞疾病的患者进行了初步观察。

Beam Therapeutics gave an initial look at the first handful of patients who received its experimental gene-edited treatment for sickle cell disease.

该生物技术公司表示，截至7月2日，已经有6名患有严重镰状细胞疾病的患者使用了该公司的产品，一种通过碱基编辑来激活具有功能的血红蛋白。

As of Ju­ly 2, six peo­ple with se­vere sick­le cell dis­ease have been dosed with its ther­a­py, which us­es base edit­ing to turn on a func­tion­ing fe­tal form of the blood pro­tein he­mo­glo­bin, the biotech said Tues­day.

在1/2期临床试验中，一名患者在接受BEAM-101治疗4个月后死于呼吸衰竭，可能与接受基因编辑治疗所需的预处理有关。

One pa­tient in the Phase 1/2 tri­al died from res­pi­ra­to­ry fail­ure four months af­ter get­ting BEAM-101, which was deemed like­ly re­lat­ed to the busul­fan con­di­tion­ing re­quired to re­ceive the gene-edit­ed ther­a­py.