在15个镰状细胞和地中海贫血病人中,Moxafortide帮助了11个病人无法安全地进行基因治疗,克服了关键的治疗障碍。 Motixafortide helped 11 of 15 sickle cell and thalassemia patients fail-safe to gene therapy, overcoming a key treatment barrier.

艾尔米德有限公司报告了2025年一项实验的新数据,显示出以APHEXDA®销售的莫西克萨福蒂德在15名状细胞病或β- thalassemia患者中成功调动了11名患者的干细胞,而这些患者之前的plerixafor治疗失败,从而使得他们能够获得基因疗法. Ayrmid, Ltd. reported new real-world data from a 2025 study showing motixafortide, sold as APHEXDA®, successfully mobilized stem cells in 11 of 15 patients with sickle cell disease or beta-thalassemia who had failed prior plerixafor treatment, enabling access to gene therapies. 5名病人接受了基因治疗和移植,更多人正在制造中。 Five patients received gene therapy and engrafted, with more in manufacturing. 该药物单独在状细胞病和与G-CSF在β-血病中起作用,突出显示其有潜力克服卡斯格维和Lyfgenia等治疗治疗的主要障碍. The drug worked alone in sickle cell disease and with G-CSF in beta-thalassemia, highlighting its potential to overcome a major barrier to curative treatments like Casgevy and Lyfgenia.