作者: 【OTL-200白血病基因疗法获欧洲药品管理局加速评审资格】Orchard Therapeutics,一家领先的商业阶段生物制药公司,致力于通过创新的基因疗法改变严重和危及生命的罕见疾病患者的生命。今天宣布,欧洲药品管理局人类使用药物产品委员会已经批准了 OTL-200的加速评估, OTL-200是一种基因疗法,与意大利米兰的圣拉斐尔- Telet...
【OTL-200白血病基因疗法获欧洲药品管理局加速评审资格】Orchard Therapeutics ,一家领先的商业阶段生物制药公司,致力于通过创新的基因疗法改变严重和危及生命的罕见疾病患者的生命。今天宣布,欧洲药品管理局人类使用药物产品委员会已经批准了 OTL-200的加速评估, OTL-200是一种基因疗法,与意大利米兰的圣拉斐尔- Telethon ...
Orchard Therapeutics is a fully integrated commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies. Orchard’s portfolio ofex vivo, auto...
“Receipt of RMAT designation for OTL-200 underscores both the severe nature of MLD and the transformative potential of the therapy for young patients suffering from this devastating, fatal neurodegenerative condition,” said Bobby Gaspar, M.D., Ph.D., chief executive of...
Compassionate use of OTL-200 for patients with metachromatic leukodystrophydoi:10.1016/j.ymgme.2022.107254Paul J. OrchardAshish GuptaJennifer BraunLaura A. AdangNishitha R. PillaiTroy LundJulie B. EisengartElsevier Inc.Molecular Genetics and Metabolism...