内分泌科

订阅

MedSci主页 / 所有栏目 / 内分泌科

基因疗法PR001治疗神经性戈谢病：IND已生效

2019-12-27 Allan MedSci原创

神经性戈谢病基因疗法PR001FDA
关注

戈谢病（GD）即葡糖脑苷脂病，是一种家族性糖脂代谢疾病，为染色体隐性遗传。Prevail Therapeutics是一家生物技术公司，致力于为神经退行性疾病患者开发基于腺病毒（AAV）的基因疗法，该公司近日宣布，美国FDA已通知Prevail，PR001用于治疗神经性戈谢病（nGD）的IND已生效，并且Prevail可能会着手进行临床试验。

戈谢病（GD）即葡糖脑苷脂病，是一种家族性糖脂代谢疾病，为染色体隐性遗传。Prevail Therapeutics是一家生物技术公司，致力于为神经退行性疾病患者开发基于腺病毒（AAV）的基因疗法，该公司近日宣布，美国FDA已通知Prevail，PR001用于治疗神经性戈谢病（nGD）的IND已生效，并且Prevail可能会着手进行临床试验。

Prevail提交了非临床数据，其中未观察到PR001相关的安全事件或不良反应事件，从而支持了以较高剂量进行I/II期临床试验的可行性。


原始出处：

http://www.firstwordpharma.com/node/1690053#axzz69CnI0iLA

本文系梅斯医学（MedSci）原创编译整理，转载需授权!

版权声明：
本网站所有内容来源注明为“梅斯医学”或“MedSci原创”的文字、图片和音视频资料，版权均属于梅斯医学所有。非经授权，任何媒体、网站或个人不得转载，授权转载时须注明来源为“梅斯医学”。其它来源的文章系转载文章，或“梅斯号”自媒体发布的文章，仅系出于传递更多信息之目的，本站仅负责审核内容合规，其内容不代表本站立场，本站不负责内容的准确性和版权。如果存在侵权、或不希望被转载的媒体或个人可与我们联系，我们将立即进行删除处理。
在此留言
评论区 (3)
#插入话题
  1. [GetPortalCommentsPageByObjectIdResponse(id=904261, encodeId=5c32904261b6, content=<a href='/topic/show?id=2d545380647' target=_blank style='color:#2F92EE;'>#戈谢病#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=109, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=53806, encryptionId=2d545380647, topicName=戈谢病)], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20211210/ede9c51184aa4f54ae6c1164cfb8eeac/967dbf3eff2943c7bf3ccfec3a776ab0.JPG, createdBy=a33789389, createdName=lifefamily, createdTime=Wed Dec 02 11:57:27 CST 2020, time=2020-12-02, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2035449, encodeId=c776203544905, content=<a href='/topic/show?id=d382e42956d' target=_blank style='color:#2F92EE;'>#神经性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=37, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=74295, encryptionId=d382e42956d, topicName=神经性)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=927d310, createdName=hywen7326, createdTime=Wed Aug 05 20:26:00 CST 2020, time=2020-08-05, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1469147, encodeId=0ead146914eb9, content=<a href='/topic/show?id=211f98250b' target=_blank style='color:#2F92EE;'>#IND#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=26, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9825, encryptionId=211f98250b, topicName=IND)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=16377007930, createdName=xiaogang319, createdTime=Sun Dec 29 07:26:00 CST 2019, time=2019-12-29, status=1, ipAttribution=)]
    2020-12-02 lifefamily

    #戈谢病#

    0

  2. [GetPortalCommentsPageByObjectIdResponse(id=904261, encodeId=5c32904261b6, content=<a href='/topic/show?id=2d545380647' target=_blank style='color:#2F92EE;'>#戈谢病#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=109, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=53806, encryptionId=2d545380647, topicName=戈谢病)], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20211210/ede9c51184aa4f54ae6c1164cfb8eeac/967dbf3eff2943c7bf3ccfec3a776ab0.JPG, createdBy=a33789389, createdName=lifefamily, createdTime=Wed Dec 02 11:57:27 CST 2020, time=2020-12-02, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2035449, encodeId=c776203544905, content=<a href='/topic/show?id=d382e42956d' target=_blank style='color:#2F92EE;'>#神经性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=37, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=74295, encryptionId=d382e42956d, topicName=神经性)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=927d310, createdName=hywen7326, createdTime=Wed Aug 05 20:26:00 CST 2020, time=2020-08-05, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1469147, encodeId=0ead146914eb9, content=<a href='/topic/show?id=211f98250b' target=_blank style='color:#2F92EE;'>#IND#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=26, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9825, encryptionId=211f98250b, topicName=IND)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=16377007930, createdName=xiaogang319, createdTime=Sun Dec 29 07:26:00 CST 2019, time=2019-12-29, status=1, ipAttribution=)]
    2020-08-05 hywen7326

    #神经性#

    0

  3. [GetPortalCommentsPageByObjectIdResponse(id=904261, encodeId=5c32904261b6, content=<a href='/topic/show?id=2d545380647' target=_blank style='color:#2F92EE;'>#戈谢病#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=109, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=53806, encryptionId=2d545380647, topicName=戈谢病)], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20211210/ede9c51184aa4f54ae6c1164cfb8eeac/967dbf3eff2943c7bf3ccfec3a776ab0.JPG, createdBy=a33789389, createdName=lifefamily, createdTime=Wed Dec 02 11:57:27 CST 2020, time=2020-12-02, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2035449, encodeId=c776203544905, content=<a href='/topic/show?id=d382e42956d' target=_blank style='color:#2F92EE;'>#神经性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=37, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=74295, encryptionId=d382e42956d, topicName=神经性)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=927d310, createdName=hywen7326, createdTime=Wed Aug 05 20:26:00 CST 2020, time=2020-08-05, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1469147, encodeId=0ead146914eb9, content=<a href='/topic/show?id=211f98250b' target=_blank style='color:#2F92EE;'>#IND#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=26, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9825, encryptionId=211f98250b, topicName=IND)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=16377007930, createdName=xiaogang319, createdTime=Sun Dec 29 07:26:00 CST 2019, time=2019-12-29, status=1, ipAttribution=)]
    2019-12-29 xiaogang319

    #IND#

    0