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.
浙公网安备33011002015279
本网站未发布麻醉药品、精神药品、医疗用毒性药品、放射性药品、戒毒药品和医疗机构制剂的产品信息
收藏
登录后参与评论