Capricor公司细胞疗法Deramiocel治疗杜氏肌营养不良(DMD)的3期试验HOPE-3数据发表于《柳叶刀》,106例患者中,上肢功能衰减速度减缓54%,心脏功能也获改善。这是首个在晚期DMD人群中做出阳性结果的3期随机双盲试验,但尚未获批,8月22日将迎来FDA审批结果。
McDonald CM et al. Deramiocel heart-derived cellular therapy in advanced Duchenne muscular dystrophy (HOPE-3): a phase 3, randomised, double-blind, placebo-controlled trial. The Lancet, 2026. (2026-07-29 在线发表)
Capricor Therapeutics. The Lancet Publishes HOPE-3 Data for Capricor Therapeutics' Deramiocel in Duchenne Muscular Dystrophy [新闻稿]. GlobeNewswire, 2026-07-29. 来源:https://www.globenewswire.com/news-release/2026/07/29/3335380/0/en/the-lancet-publishes-hope-3-data-for-capricor-therapeutics-deramiocel-in-duchenne-muscular-dystrophy.html
生物通. 柳叶刀发表杜氏肌营养不良症的突破性临床试验结果, 2026-07-31. 来源:https://m.ebiotrade.com/newsf/2026-7/2026730172822379.htm
A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy (HOPE-3). ClinicalTrials.gov NCT05126758. 来源:https://clinicaltrials.ucsd.edu/trial/NCT05126758
FDA Cellular, Tissue, and Gene Therapies Advisory Committee, July 29, 2026 Meeting Briefing Document.