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基因治疗IIIA型粘多糖贮积症的II/III期研究中，一名患者死亡

2020-10-16 MedSci原创 MedSci原创

IIIA型粘多糖贮积症腺相关病毒（AAV）基因疗法LYS-SAF302
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MPS IIIA的基因疗法LYS-SAF302由Lysogene开发，是由腺相关病毒（AAV）介导的基因疗法，旨在通过AAV递送功能性SGSH基因从而取代有缺陷的SGSH基因。

Lysogene周四披露，II/III期AAVance研究中有一名患者死亡，该患者正在接受基因疗法LYS-SAF302用于治疗IIIA型粘多糖贮积症（MPS IIIA）。Lysogene股价因这一消息下跌多达19％，目前还尚不清楚死因，但提到没有证据表明患者死亡与LYS-SAF302有关。

MPS IIIA的基因疗法LYS-SAF302由Lysogene开发，是由腺相关病毒（AAV）介导的基因疗法，旨在通过AAV递送功能性SGSH基因从而取代有缺陷的SGSH基因。

how gene therapy works

用于MPS IIIA的研究性基因疗法（LYS-SAF302）的三个要素是：

载体：AAVrh10

启动子：CAG

递送基因：完整的SGSH 基因

vector promoter transgene legend

Lysogene指出会“严格按照研究方案进行”研究剩余的18名患者。

图片来源：https://www.sarepta.com/science/gene-therapy-engine

原始出处：

https://www.firstwordpharma.com/node/1765418?tsid=4

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    2021-07-09 yige2004

    #粘多糖贮积症#

    0

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    2021-09-22 juliusluan78

    #III#

    0

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    2021-08-03 HinsMax

    #III期研究#

    0

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    2021-06-04 sunylz

    #患者死亡#

    0

  5. [GetPortalCommentsPageByObjectIdResponse(id=2055362, encodeId=e4292055362c3, 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=50, 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=Fri Jul 09 02:05:24 CST 2021, time=2021-07-09, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1916354, encodeId=8337191635413, content=<a href='/topic/show?id=c5a89550ff' target=_blank style='color:#2F92EE;'>#III#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=0, 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 Sep 22 09:05:24 CST 2021, time=2021-09-22, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2021375, encodeId=c37f20213e571, content=<a href='/topic/show?id=69d595e421' target=_blank style='color:#2F92EE;'>#III期研究#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=70, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9574, encryptionId=69d595e421, topicName=III期研究)], attachment=null, authenticateStatus=null, createdAvatar=http://thirdwx.qlogo.cn/mmopen/vi_32/qXbLjJB4FSzhQS3C5qQFMJkqicRIJFL6zHFwwhv0I95nzA6xicevQcEica0gk4A58UkbMMriadAgTY2b7N7Yd0ZsQg/132, createdBy=910c2500152, createdName=HinsMax, createdTime=Tue Aug 03 07:05:24 CST 2021, time=2021-08-03, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1983690, encodeId=e24e198369050, content=<a href='/topic/show?id=628553048e3' target=_blank style='color:#2F92EE;'>#患者死亡#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=66, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=53048, encryptionId=628553048e3, topicName=患者死亡)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=12de429, createdName=sunylz, createdTime=Fri Jun 04 10:05:24 CST 2021, time=2021-06-04, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1594818, encodeId=1e5b15948183f, content=<a href='/topic/show?id=b317956200' target=_blank style='color:#2F92EE;'>#III期#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=53, 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=Sat Oct 17 15:05:24 CST 2020, time=2020-10-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=892380, encodeId=a7b889238014, content=顶, beContent=null, objectType=article, channel=null, level=null, likeNumber=105, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20201011/7d12ce13e8cc40d0a226f0da164759e3/fcc097f99c8f4ce58672ebd6f717639c.jpg, createdBy=5f6f5424298, createdName=李小姐@, createdTime=Fri Oct 16 08:33:46 CST 2020, time=2020-10-16, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=892363, encodeId=9fbf89236304, content=顶, beContent=null, objectType=article, channel=null, level=null, likeNumber=95, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20201016/f2e3c0d9e191431c82d27c4d8fa3bcb7/d74d3f71c6b247379d19d88aa40f0acf.jpg, createdBy=dee45426404, createdName=ms2000001541278406, createdTime=Fri Oct 16 00:44:53 CST 2020, time=2020-10-16, status=1, ipAttribution=)]
    2020-10-17 智智灵药

    #III期#

    0

  6. [GetPortalCommentsPageByObjectIdResponse(id=2055362, encodeId=e4292055362c3, 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=50, 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=Fri Jul 09 02:05:24 CST 2021, time=2021-07-09, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1916354, encodeId=8337191635413, content=<a href='/topic/show?id=c5a89550ff' target=_blank style='color:#2F92EE;'>#III#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=0, 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 Sep 22 09:05:24 CST 2021, time=2021-09-22, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2021375, encodeId=c37f20213e571, content=<a href='/topic/show?id=69d595e421' target=_blank style='color:#2F92EE;'>#III期研究#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=70, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9574, encryptionId=69d595e421, topicName=III期研究)], attachment=null, authenticateStatus=null, createdAvatar=http://thirdwx.qlogo.cn/mmopen/vi_32/qXbLjJB4FSzhQS3C5qQFMJkqicRIJFL6zHFwwhv0I95nzA6xicevQcEica0gk4A58UkbMMriadAgTY2b7N7Yd0ZsQg/132, createdBy=910c2500152, createdName=HinsMax, createdTime=Tue Aug 03 07:05:24 CST 2021, time=2021-08-03, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1983690, encodeId=e24e198369050, content=<a href='/topic/show?id=628553048e3' target=_blank style='color:#2F92EE;'>#患者死亡#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=66, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=53048, encryptionId=628553048e3, topicName=患者死亡)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=12de429, createdName=sunylz, createdTime=Fri Jun 04 10:05:24 CST 2021, time=2021-06-04, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1594818, encodeId=1e5b15948183f, content=<a href='/topic/show?id=b317956200' target=_blank style='color:#2F92EE;'>#III期#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=53, 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=Sat Oct 17 15:05:24 CST 2020, time=2020-10-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=892380, encodeId=a7b889238014, content=顶, beContent=null, objectType=article, channel=null, level=null, likeNumber=105, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20201011/7d12ce13e8cc40d0a226f0da164759e3/fcc097f99c8f4ce58672ebd6f717639c.jpg, createdBy=5f6f5424298, createdName=李小姐@, createdTime=Fri Oct 16 08:33:46 CST 2020, time=2020-10-16, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=892363, encodeId=9fbf89236304, content=顶, beContent=null, objectType=article, channel=null, level=null, likeNumber=95, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20201016/f2e3c0d9e191431c82d27c4d8fa3bcb7/d74d3f71c6b247379d19d88aa40f0acf.jpg, createdBy=dee45426404, createdName=ms2000001541278406, createdTime=Fri Oct 16 00:44:53 CST 2020, time=2020-10-16, status=1, ipAttribution=)]
    2020-10-16 李小姐@

    顶

    0

  7. [GetPortalCommentsPageByObjectIdResponse(id=2055362, encodeId=e4292055362c3, 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=50, 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=Fri Jul 09 02:05:24 CST 2021, time=2021-07-09, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1916354, encodeId=8337191635413, content=<a href='/topic/show?id=c5a89550ff' target=_blank style='color:#2F92EE;'>#III#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=0, 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 Sep 22 09:05:24 CST 2021, time=2021-09-22, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2021375, encodeId=c37f20213e571, content=<a href='/topic/show?id=69d595e421' target=_blank style='color:#2F92EE;'>#III期研究#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=70, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9574, encryptionId=69d595e421, topicName=III期研究)], attachment=null, authenticateStatus=null, createdAvatar=http://thirdwx.qlogo.cn/mmopen/vi_32/qXbLjJB4FSzhQS3C5qQFMJkqicRIJFL6zHFwwhv0I95nzA6xicevQcEica0gk4A58UkbMMriadAgTY2b7N7Yd0ZsQg/132, createdBy=910c2500152, createdName=HinsMax, createdTime=Tue Aug 03 07:05:24 CST 2021, time=2021-08-03, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1983690, encodeId=e24e198369050, content=<a href='/topic/show?id=628553048e3' target=_blank style='color:#2F92EE;'>#患者死亡#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=66, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=53048, encryptionId=628553048e3, topicName=患者死亡)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=12de429, createdName=sunylz, createdTime=Fri Jun 04 10:05:24 CST 2021, time=2021-06-04, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1594818, encodeId=1e5b15948183f, content=<a href='/topic/show?id=b317956200' target=_blank style='color:#2F92EE;'>#III期#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=53, 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=Sat Oct 17 15:05:24 CST 2020, time=2020-10-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=892380, encodeId=a7b889238014, content=顶, beContent=null, objectType=article, channel=null, level=null, likeNumber=105, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20201011/7d12ce13e8cc40d0a226f0da164759e3/fcc097f99c8f4ce58672ebd6f717639c.jpg, createdBy=5f6f5424298, createdName=李小姐@, createdTime=Fri Oct 16 08:33:46 CST 2020, time=2020-10-16, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=892363, encodeId=9fbf89236304, content=顶, beContent=null, objectType=article, channel=null, level=null, likeNumber=95, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20201016/f2e3c0d9e191431c82d27c4d8fa3bcb7/d74d3f71c6b247379d19d88aa40f0acf.jpg, createdBy=dee45426404, createdName=ms2000001541278406, createdTime=Fri Oct 16 00:44:53 CST 2020, time=2020-10-16, status=1, ipAttribution=)]
    2020-10-16 ms2000001541278406

    顶

    0

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