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2019年国际血栓和止血学会（ISTH）：uniQure的B型血友病基因疗法展现出长达3年的临床益处和耐受性

2019-07-08 不详 MedSci原创

2019年国际血栓和止血学会uniQureB型血友病基因疗法临床益处耐受性
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在2019年第27届国际血栓和止血学会（ISTH）上，基因治疗公司uniQure NV公布其用于治疗严重B型血友病患者的AAV5基因疗法AMT-061的II期研究结果，对三名患者长达36周的随访后，FIX活性高达正常值的54％，平均值为正常值的45％。

在2019年第27届国际血栓和止血学会（ISTH）上，基因治疗公司uniQure NV公布其用于治疗严重B型血友病患者的AAV5基因疗法AMT-061的II期研究结果，对三名患者长达36周的随访后，FIX活性高达正常值的54％，平均值为正常值的45％。

并且在长达3.5年的随访中，用于治疗B型血友病患者的AMT-060在所有患者中的均具有临床益处和耐受性。

AMT-060是uniQure的第一代基因疗法，由携带野生型FIX基因的AAV5载体组成。

AMT-061由携带基因盒的AAV5病毒载体组成，该基因盒具有因子IX专利保护的Padua变体（FIX-Padua）。uniQure在美国和加拿大拥有多项已发布的专利。

"AMT-060的I / II期研究继续证明了uniQure基因治疗平台在血友病B中的长期安全性和耐受性，" 荷兰伊拉斯姆斯大学医学中心Frank WG Leebeek教授说，"我们现在已经证明了持久临床益处的证据，包括持续的FIX活性，改善的疾病表型以及在给药后长达3.5年的自发性出血的显着减少。这些数据对于uniQure的B型血友病计划是非常鼓舞人心的。"

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    2020-03-14 ligang4439

    #ISTH#

    0

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    2019-12-14 12498ca5m83暂无昵称

    #uniQure#

    0

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    2019-07-10 jeanqiuqiu

    #止血#

    0

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    2019-07-10 spoonycyy

    #B型血友病#

    0

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    2019-07-10 Smile2681

    #耐受性#

    0

  6. [GetPortalCommentsPageByObjectIdResponse(id=2085117, encodeId=1f7d208511e11, content=<a href='/topic/show?id=46ce100955d' target=_blank style='color:#2F92EE;'>#ISTH#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=36, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=10095, encryptionId=46ce100955d, topicName=ISTH)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=869698, createdName=ligang4439, createdTime=Sat Mar 14 23:44:00 CST 2020, time=2020-03-14, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1796590, encodeId=c5c81e965900d, content=<a href='/topic/show?id=ded018138d8' target=_blank style='color:#2F92EE;'>#uniQure#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=35, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=18138, encryptionId=ded018138d8, topicName=uniQure)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=32a12500167, createdName=12498ca5m83暂无昵称, createdTime=Sat Dec 14 23:44:00 CST 2019, time=2019-12-14, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1263072, encodeId=80f912630e244, content=<a href='/topic/show?id=ebb5631e076' target=_blank style='color:#2F92EE;'>#止血#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=34, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=63170, encryptionId=ebb5631e076, topicName=止血)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=6f7770, createdName=jeanqiuqiu, createdTime=Wed Jul 10 03:44:00 CST 2019, time=2019-07-10, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1322408, encodeId=0a76132240851, content=<a href='/topic/show?id=8a23382430' target=_blank style='color:#2F92EE;'>#B型血友病#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=36, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=3824, encryptionId=8a23382430, topicName=B型血友病)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=3516494, createdName=spoonycyy, createdTime=Wed Jul 10 03:44:00 CST 2019, time=2019-07-10, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1417698, encodeId=1868141e6989e, content=<a href='/topic/show?id=aada80351ae' target=_blank style='color:#2F92EE;'>#耐受性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=38, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=80351, encryptionId=aada80351ae, topicName=耐受性)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=43843300457, createdName=Smile2681, createdTime=Wed Jul 10 03:44:00 CST 2019, time=2019-07-10, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=369292, encodeId=d7f2369292cd, content=学习了，谢谢分享, beContent=null, objectType=article, channel=null, level=null, likeNumber=87, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=7c592194322, createdName=1209e435m98(暂无昵称), createdTime=Mon Jul 08 15:01:07 CST 2019, time=2019-07-08, status=1, ipAttribution=)]
    2019-07-08 1209e435m98(暂无昵称)

    学习了，谢谢分享

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