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治疗粘多糖贮积症的基因疗法LYS-SAF302 的II/III期临床试验AAVance正式开始

2019-02-18 不详 网络

LYS-SAF302粘多糖贮积症AAVance
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Lysogene是一家专注于针对中枢神经系统（CNS）疾病的基因治疗的先驱生物制药公司，Sarepta 是罕见疾病精准基因医学的领导者，两家公司近日宣布第一名患者已经接受了AAVance临床试验，AAVance是LYS-SAF302的全球II/III期临床试验，LYS-SAF302是一种治疗II型粘多糖贮积症（MPS IIIA）的基因疗法。

Lysogene是一家专注于针对中枢神经系统（CNS）疾病的基因治疗的先驱生物制药公司，Sarepta 是罕见疾病精准基因医学的领导者，两家公司近日宣布第一名患者已经接受了AAVance临床试验，AAVance是LYS-SAF302的全球II/III期临床试验，LYS-SAF302是一种治疗II型粘多糖贮积症（MPS IIIA）的基因疗法。

AAVance是一项单臂试验，旨在评估一次性递送携带N-磺基葡糖胺磺基水解酶（SGSH）基因的重组腺相关病毒载体rh.10的有效性。MPS IIIA由SGSH基因突变引起，该基因参与产生长链糖分子分解和处理所必需的酶。LAF-SAF302旨在提供SGSH基因的功能性拷贝，并允许大脑分泌缺失的酶。该试验的目的是显示MPS IIIA患者的神经发育状态的改善或稳定。该试验将在美国和欧洲的8个地点招募20名患者。

Lysogene的创始人兼首席执行官Karen Aiach说：“在AAVance试验中入组第一名患者是解决这种无法治疗的神经退行性疾病的重要一步。我们的目标是通过在大脑中提供永久性功能性酶来稳定或改善MPS IIIA患者的临床状态”。


原始出处：

http://www.firstwordpharma.com/node/1625380#axzz5fc4uA1RS

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    2019-11-05 yige2004

    #粘多糖贮积症#

    0

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    2019-11-23 医生2402

    #I期临床#

    0

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    2019-12-23 kksonne

    #II期临床试验#

    0

  4. 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    2019-04-17 juliusluan78

    #III#

    0

  5. 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    2019-08-15 839640778

    #I期临床试验#

    0

  6. [GetPortalCommentsPageByObjectIdResponse(id=2055361, encodeId=934620553615d, content=<a href='/topic/show?id=9445e6231f0' target=_blank style='color:#2F92EE;'>#粘多糖贮积症#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=35, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=76231, encryptionId=9445e6231f0, topicName=粘多糖贮积症)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=66962500501, createdName=yige2004, createdTime=Tue Nov 05 16:58:00 CST 2019, time=2019-11-05, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1652860, encodeId=2fb4165286074, 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=38, 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=Sat Nov 23 05:58:00 CST 2019, time=2019-11-23, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1931447, encodeId=5b95193144e6a, 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=37, 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=Mon Dec 23 02:58:00 CST 2019, time=2019-12-23, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1916069, encodeId=58fe191606956, content=<a href='/topic/show?id=c5a89550ff' target=_blank style='color:#2F92EE;'>#III#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=49, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9550, encryptionId=c5a89550ff, topicName=III)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=e68e415, createdName=juliusluan78, createdTime=Wed Apr 17 11:58:00 CST 2019, time=2019-04-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2088723, encodeId=d75a2088e2357, content=<a href='/topic/show?id=53961018913' target=_blank style='color:#2F92EE;'>#I期临床试验#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=29, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=10189, encryptionId=53961018913, topicName=I期临床试验)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=45b2276, createdName=839640778, createdTime=Thu Aug 15 22:58:00 CST 2019, time=2019-08-15, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1271861, encodeId=dc8512e1861c0, content=<a href='/topic/show?id=9fef169483' target=_blank style='color:#2F92EE;'>#AAV#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=30, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=1694, encryptionId=9fef169483, topicName=AAV)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=8ba7103, createdName=xuqianhua, createdTime=Tue Feb 19 23:58:00 CST 2019, time=2019-02-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1524673, encodeId=2b5215246e353, 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=29, 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=Tue Feb 19 23:58:00 CST 2019, time=2019-02-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1588015, encodeId=f63e1588015d2, content=<a href='/topic/show?id=056095e171' target=_blank style='color:#2F92EE;'>#III期临床试验#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=29, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9571, encryptionId=056095e171, topicName=III期临床试验)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=d24217580122, createdName=guihongzh, createdTime=Tue Feb 19 23:58:00 CST 2019, time=2019-02-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1594559, encodeId=87c71594559d7, content=<a href='/topic/show?id=b317956200' target=_blank style='color:#2F92EE;'>#III期#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=27, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9562, encryptionId=b317956200, topicName=III期)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=a12645, createdName=智智灵药, createdTime=Tue Feb 19 23:58:00 CST 2019, time=2019-02-19, status=1, ipAttribution=)]
    2019-02-19 xuqianhua

    #AAV#

    0

  7. [GetPortalCommentsPageByObjectIdResponse(id=2055361, encodeId=934620553615d, content=<a href='/topic/show?id=9445e6231f0' target=_blank style='color:#2F92EE;'>#粘多糖贮积症#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=35, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=76231, encryptionId=9445e6231f0, topicName=粘多糖贮积症)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=66962500501, createdName=yige2004, createdTime=Tue Nov 05 16:58:00 CST 2019, time=2019-11-05, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1652860, encodeId=2fb4165286074, 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=38, 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=Sat Nov 23 05:58:00 CST 2019, time=2019-11-23, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1931447, encodeId=5b95193144e6a, 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=37, 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=Mon Dec 23 02:58:00 CST 2019, time=2019-12-23, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1916069, encodeId=58fe191606956, content=<a href='/topic/show?id=c5a89550ff' target=_blank style='color:#2F92EE;'>#III#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=49, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9550, encryptionId=c5a89550ff, topicName=III)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=e68e415, createdName=juliusluan78, createdTime=Wed Apr 17 11:58:00 CST 2019, time=2019-04-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2088723, encodeId=d75a2088e2357, content=<a href='/topic/show?id=53961018913' target=_blank style='color:#2F92EE;'>#I期临床试验#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=29, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=10189, encryptionId=53961018913, topicName=I期临床试验)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=45b2276, createdName=839640778, createdTime=Thu Aug 15 22:58:00 CST 2019, time=2019-08-15, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1271861, encodeId=dc8512e1861c0, content=<a href='/topic/show?id=9fef169483' target=_blank style='color:#2F92EE;'>#AAV#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=30, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=1694, encryptionId=9fef169483, topicName=AAV)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=8ba7103, createdName=xuqianhua, createdTime=Tue Feb 19 23:58:00 CST 2019, time=2019-02-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1524673, encodeId=2b5215246e353, 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=29, 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=Tue Feb 19 23:58:00 CST 2019, time=2019-02-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1588015, encodeId=f63e1588015d2, content=<a href='/topic/show?id=056095e171' target=_blank style='color:#2F92EE;'>#III期临床试验#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=29, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9571, encryptionId=056095e171, topicName=III期临床试验)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=d24217580122, createdName=guihongzh, createdTime=Tue Feb 19 23:58:00 CST 2019, time=2019-02-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1594559, encodeId=87c71594559d7, content=<a href='/topic/show?id=b317956200' target=_blank style='color:#2F92EE;'>#III期#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=27, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9562, encryptionId=b317956200, topicName=III期)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=a12645, createdName=智智灵药, createdTime=Tue Feb 19 23:58:00 CST 2019, time=2019-02-19, status=1, ipAttribution=)]
    2019-02-19 guihongzh

    #II期临床#

    0

  8. 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    2019-02-19 guihongzh

    #III期临床试验#

    0

  9. 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    2019-02-19 智智灵药

    #III期#

    0