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Sanfilippo综合征A型的基因疗法：基因疗法ABO-102的I/II期临床试验取得阶段性成果

2019-07-26 Allan MedSci原创

ABO-102Sanfilippo综合征A型AAV9
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Abeona Therapeutics是一家基因和细胞治疗领域的领导者，今天宣布其正在进行的I/II期临床试验已经取得阳性数据，该试验评估了腺相关病毒9（AAV9）基因疗法ABO-102治疗Sanfilippo综合征A型（MPS IIIA）的有效性和安全性。结果显示，患儿在使用高剂量ABO-102治疗后的12-18个月仍保持神经认知发育。

Abeona Therapeutics是一家基因和细胞治疗领域的领导者，今天宣布其正在进行的I/II期临床试验已经取得阳性数据，该试验评估了腺相关病毒9（AAV9）基因疗法ABO-102治疗Sanfilippo综合征A型（MPS IIIA）的有效性和安全性。结果显示，患儿在使用高剂量ABO-102治疗后的12-18个月仍保持神经认知发育。

Abeona Therapeutics首席执行官，医学博士João Siffert说：“我们对这些最新结果感到鼓舞，这项试验证明了单次静脉注射ABO-102治疗MPS IIIA患儿的有效性。要知道，目前尚无经过批准的药物来治疗MPS IIIA”。


原始出处：

http://www.firstwordpharma.com/node/1655101#axzz5ug3cIKrI

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    2020-03-24 839640778

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    2019-10-30 一叶知秋

    #Sanfilippo综合征A型#

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    2020-04-05 医生2402

    #I期临床#

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28 00:48:00 CST 2019, time=2019-07-28, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1598030, encodeId=552b15980302b, content=<a href='/topic/show?id=6c6f1e62c5' target=_blank style='color:#2F92EE;'>#ABO#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=89, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=1762, encryptionId=6c6f1e62c5, topicName=ABO)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=b6c918580135, createdName=闆锋旦, createdTime=Sun Jul 28 00:48:00 CST 2019, time=2019-07-28, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=370289, encodeId=4d753e0289d9, content=向科研人员致敬！, beContent=null, objectType=article, channel=null, level=null, likeNumber=119, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=e5171668917, createdName=坚强007, createdTime=Fri Jul 26 10:42:56 CST 2019, time=2019-07-26, status=1, ipAttribution=)]
    2019-11-17 kksonne

    #II期临床试验#

    0

  5. 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28 00:48:00 CST 2019, time=2019-07-28, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1598030, encodeId=552b15980302b, content=<a href='/topic/show?id=6c6f1e62c5' target=_blank style='color:#2F92EE;'>#ABO#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=89, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=1762, encryptionId=6c6f1e62c5, topicName=ABO)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=b6c918580135, createdName=闆锋旦, createdTime=Sun Jul 28 00:48:00 CST 2019, time=2019-07-28, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=370289, encodeId=4d753e0289d9, content=向科研人员致敬！, beContent=null, objectType=article, channel=null, level=null, likeNumber=119, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=e5171668917, createdName=坚强007, createdTime=Fri Jul 26 10:42:56 CST 2019, time=2019-07-26, status=1, ipAttribution=)]
    2019-09-22 12498717m66(暂无昵称)

    #ABO-102#

    0

  6. 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28 00:48:00 CST 2019, time=2019-07-28, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1598030, encodeId=552b15980302b, content=<a href='/topic/show?id=6c6f1e62c5' target=_blank style='color:#2F92EE;'>#ABO#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=89, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=1762, encryptionId=6c6f1e62c5, topicName=ABO)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=b6c918580135, createdName=闆锋旦, createdTime=Sun Jul 28 00:48:00 CST 2019, time=2019-07-28, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=370289, encodeId=4d753e0289d9, content=向科研人员致敬！, beContent=null, objectType=article, channel=null, level=null, likeNumber=119, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=e5171668917, createdName=坚强007, createdTime=Fri Jul 26 10:42:56 CST 2019, time=2019-07-26, status=1, ipAttribution=)]
    2019-07-28 zhouqu_8

    #综合征#

    0

  7. 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28 00:48:00 CST 2019, time=2019-07-28, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1598030, encodeId=552b15980302b, content=<a href='/topic/show?id=6c6f1e62c5' target=_blank style='color:#2F92EE;'>#ABO#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=89, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=1762, encryptionId=6c6f1e62c5, topicName=ABO)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=b6c918580135, createdName=闆锋旦, createdTime=Sun Jul 28 00:48:00 CST 2019, time=2019-07-28, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=370289, encodeId=4d753e0289d9, content=向科研人员致敬！, beContent=null, objectType=article, channel=null, level=null, likeNumber=119, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=e5171668917, createdName=坚强007, createdTime=Fri Jul 26 10:42:56 CST 2019, time=2019-07-26, status=1, ipAttribution=)]
    2019-07-28 guihongzh

    #II期临床#

    0

  8. 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createdAvatar=, createdBy=30bd35, createdName=一叶知秋, createdTime=Wed Oct 30 18:48:00 CST 2019, time=2019-10-30, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1652919, encodeId=384016529196a, content=<a href='/topic/show?id=5b011018e07' target=_blank style='color:#2F92EE;'>#I期临床#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=72, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=10187, encryptionId=5b011018e07, topicName=I期临床)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=fc1f24335670, createdName=医生2402, createdTime=Sun Apr 05 07:48:00 CST 2020, time=2020-04-05, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1931474, encodeId=39e519314e47b, content=<a href='/topic/show?id=a3d89601f2' target=_blank style='color:#2F92EE;'>#II期临床试验#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=72, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9601, encryptionId=a3d89601f2, topicName=II期临床试验)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=39a0295, createdName=kksonne, createdTime=Sun Nov 17 19:48:00 CST 2019, time=2019-11-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2077005, encodeId=bb7020e700564, content=<a href='/topic/show?id=d2471e6407' target=_blank style='color:#2F92EE;'>#ABO-102#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=75, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=1764, encryptionId=d2471e6407, topicName=ABO-102)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=951d2500061, createdName=12498717m66(暂无昵称), createdTime=Sun Sep 22 22:48:00 CST 2019, time=2019-09-22, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1431585, encodeId=64681431585aa, content=<a href='/topic/show?id=f9b9e9124a8' target=_blank style='color:#2F92EE;'>#综合征#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=73, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=79124, encryptionId=f9b9e9124a8, topicName=综合征)], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/aLGWoFXAyMbIu3qymFOyheQLjPSX3OUs5GmkyBlcCOwTPIeq3why9NGibxxUqYo6hcx8qZLHZFgNPnBK1yzWeOFpyg2OnWOt0/0, createdBy=fa4716, createdName=zhouqu_8, createdTime=Sun Jul 28 00:48:00 CST 2019, time=2019-07-28, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1524722, encodeId=58001524e224f, content=<a href='/topic/show?id=4ba395994d' target=_blank style='color:#2F92EE;'>#II期临床#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=104, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9599, encryptionId=4ba395994d, topicName=II期临床)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=139811614978, createdName=guihongzh, createdTime=Sun Jul 28 00:48:00 CST 2019, time=2019-07-28, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1598030, encodeId=552b15980302b, content=<a href='/topic/show?id=6c6f1e62c5' target=_blank style='color:#2F92EE;'>#ABO#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=89, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=1762, encryptionId=6c6f1e62c5, topicName=ABO)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=b6c918580135, createdName=闆锋旦, createdTime=Sun Jul 28 00:48:00 CST 2019, time=2019-07-28, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=370289, encodeId=4d753e0289d9, content=向科研人员致敬！, beContent=null, objectType=article, channel=null, level=null, likeNumber=119, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=e5171668917, createdName=坚强007, createdTime=Fri Jul 26 10:42:56 CST 2019, time=2019-07-26, status=1, ipAttribution=)]
    2019-07-28 闆锋旦

    #ABO#

    0

  9. 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28 00:48:00 CST 2019, time=2019-07-28, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1598030, encodeId=552b15980302b, content=<a href='/topic/show?id=6c6f1e62c5' target=_blank style='color:#2F92EE;'>#ABO#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=89, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=1762, encryptionId=6c6f1e62c5, topicName=ABO)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=b6c918580135, createdName=闆锋旦, createdTime=Sun Jul 28 00:48:00 CST 2019, time=2019-07-28, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=370289, encodeId=4d753e0289d9, content=向科研人员致敬！, beContent=null, objectType=article, channel=null, level=null, likeNumber=119, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=e5171668917, createdName=坚强007, createdTime=Fri Jul 26 10:42:56 CST 2019, time=2019-07-26, status=1, ipAttribution=)]
    2019-07-26 坚强007

    向科研人员致敬！

    0

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