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细胞疗法MGTA-456治疗遗传性代谢疾病的全新II期数据

2020-02-20 Allan MedSci原创

细胞疗法MGTA-456遗传性代谢疾病IMD
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Magenta是一家临床阶段的生物技术公司，近日公布了其细胞疗法MGTA-456治疗遗传性代谢疾病的最新II期数据。MGTA-456是一种细胞疗法，旨在提供与患者良好匹配的高剂量造血干细胞（HSC），以确保患者的免疫和血液系统重建。

Magenta是一家临床阶段的生物技术公司，近日公布了其细胞疗法MGTA-456治疗遗传性代谢疾病的最新II期数据。MGTA-456是一种细胞疗法，旨在提供与患者良好匹配的高剂量造血干细胞（HSC），以确保患者的免疫和血液系统重建。

该II期临床试验正在探索MGTA-456治疗遗传性代谢异常（IMD）的有效性和安全性，IMD包括脑性肾上腺髓质营养不良（cALD）、IH型黏多糖贮积症（MPS I或Hurler综合征）、异色性白细胞营养不良（MLD）或球状细胞白细胞营养不良（GLD或Krabbe病）。这些是罕见的且快速进展的疾病，如果不治疗则会致命。

Magenta首席医学官John Davis博士表示：“现有治疗方法和正在研究的基因疗法均未能有效治疗遗传性代谢异常。但细胞疗法MGTA-456的临床试验证明，MGTA-456能够有效改善患者症状”。


原始出处：

https://www.firstwordpharma.com/node/1702382?tsid=4

本文系梅斯医学（MedSci）原创编译整理，转载需授权!

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#插入话题
  1. [GetPortalCommentsPageByObjectIdResponse(id=1860104, encodeId=6f0b18601043b, content=<a href='/topic/show?id=cc73951054b' target=_blank style='color:#2F92EE;'>#遗传性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=131, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=95105, encryptionId=cc73951054b, topicName=遗传性)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=a12645, createdName=智慧医人, createdTime=Sat Aug 29 06:51:00 CST 2020, time=2020-08-29, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1402007, encodeId=bbb4140200e92, content=<a href='/topic/show?id=193025463d8' target=_blank style='color:#2F92EE;'>#代谢疾病#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=136, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=25463, encryptionId=193025463d8, topicName=代谢疾病)], attachment=null, authenticateStatus=null, createdAvatar=http://thirdqq.qlogo.cn/qqapp/101296147/F9591315BE4091482318EFFCD39C9D08/100, createdBy=bf9f2374681, createdName=windight, createdTime=Sat Feb 22 14:51:00 CST 2020, time=2020-02-22, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=379461, encodeId=1d543e9461d9, content=学习了很有用不錯, beContent=null, objectType=article, channel=null, level=null, likeNumber=204, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/oLAjfB7s1ib3IzoRn621JdKCFUI4XvNELcibocrAVU6aUzEVDSaf3rYH08PUYUiagGqxAEhj8g9WqqNvD1CupIpCrmnR2OrqHZh/0, createdBy=7f181365728, createdName=飛歌, createdTime=Fri Feb 21 00:07:08 CST 2020, time=2020-02-21, status=1, ipAttribution=)]
    2020-08-29 智慧医人

    #遗传性#

    0

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    2020-02-22 windight

    #代谢疾病#

    0

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    2020-02-21 飛歌

    学习了很有用不錯

    0