基因疗法Valoctocogene Roxaparvovec治疗严重血友病A:长期数据再次验证有效性和安全性

2020-06-01 Allan MedSci原创

BioMarin制药公司近日宣布,基因疗法Valoctocogene roxaparvovec治疗严重血友病A的开放标签I/II期研究取得了新结果。

BioMarin制药公司近日宣布,基因疗法Valoctocogene roxaparvovec治疗严重血友病A的开放标签I/II期研究取得了新结果,这些结果将于2020年6月14日至19日举行的世界血友病联盟(WFH)峰会上公布。

对6e13 vg / kg组的四年更新和对4e13 vg / kg组的三年更新表明,两个队列中的所有受试者自接受单剂量valoctocogene roxaparvovec以来均未接受VIII因子治疗。在这两个队列中,累积平均年度流血率(ABR)均小于1,且低于治疗前的基线水平。6e13 vg / kg队列第四年的平均ABR为1.3,而4e13 vg / kg队列第三年的平均ABR为0.5。在过去的一年中,6e13 vg / kg队列中85%(6/7)的患者和4e13 vg / kg队列中83%(5/6)的患者未发生自发性出血。

该研究的首席研究员John Pasi博士说:“这些额外的数据是基因疗法治疗严重血友病A的重要一步。对于这种破坏性疾病来说,这是一种好方法。每年的数据都增加了我们对安全性和有效性的了解”。

 

原始出处:

https://www.firstwordpharma.com/node/1728658?tsid=4

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 (5)
#插入话题
  1. [GetPortalCommentsPageByObjectIdResponse(id=1862791, encodeId=d07f1862e9130, content=<a href='/topic/show?id=0b69542242' target=_blank style='color:#2F92EE;'>#CTO#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=46, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=5422, encryptionId=0b69542242, topicName=CTO)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c66088, createdName=gao_jian4217, createdTime=Sat Feb 20 06:18:17 CST 2021, time=2021-02-20, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1993319, encodeId=5bea1993319d4, content=<a href='/topic/show?id=b2f1280466' target=_blank style='color:#2F92EE;'>#ARVO#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=2804, encryptionId=b2f1280466, topicName=ARVO)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=636e2500187, createdName=12498da4m32(暂无昵称), createdTime=Mon Apr 19 21:18:17 CST 2021, time=2021-04-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2049302, encodeId=d22f2049302dc, content=<a href='/topic/show?id=4363e929ad' target=_blank style='color:#2F92EE;'>#Gene#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=7929, encryptionId=4363e929ad, topicName=Gene)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=63a4277, createdName=cy0324, createdTime=Mon May 03 19:18:17 CST 2021, time=2021-05-03, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1858349, encodeId=1c2618583491b, content=<a href='/topic/show?id=21311323521' target=_blank style='color:#2F92EE;'>#OCT#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=34, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=13235, encryptionId=21311323521, topicName=OCT)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=19c567, createdName=habb, createdTime=Mon Mar 01 20:18:17 CST 2021, time=2021-03-01, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1338216, encodeId=1e211338216da, content=<a href='/topic/show?id=cd2a262068' target=_blank style='color:#2F92EE;'>#APA#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=34, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=2620, encryptionId=cd2a262068, topicName=APA)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=ce072500140, createdName=12498b3em08(暂无昵称), createdTime=Wed Jun 03 00:18:17 CST 2020, time=2020-06-03, status=1, ipAttribution=)]
    2021-02-20 gao_jian4217
  2. [GetPortalCommentsPageByObjectIdResponse(id=1862791, encodeId=d07f1862e9130, content=<a href='/topic/show?id=0b69542242' target=_blank style='color:#2F92EE;'>#CTO#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=46, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=5422, encryptionId=0b69542242, topicName=CTO)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c66088, createdName=gao_jian4217, createdTime=Sat Feb 20 06:18:17 CST 2021, time=2021-02-20, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1993319, encodeId=5bea1993319d4, content=<a href='/topic/show?id=b2f1280466' target=_blank style='color:#2F92EE;'>#ARVO#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=2804, encryptionId=b2f1280466, topicName=ARVO)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=636e2500187, createdName=12498da4m32(暂无昵称), createdTime=Mon Apr 19 21:18:17 CST 2021, time=2021-04-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2049302, encodeId=d22f2049302dc, content=<a href='/topic/show?id=4363e929ad' target=_blank style='color:#2F92EE;'>#Gene#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=7929, encryptionId=4363e929ad, topicName=Gene)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=63a4277, createdName=cy0324, createdTime=Mon May 03 19:18:17 CST 2021, time=2021-05-03, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1858349, encodeId=1c2618583491b, content=<a href='/topic/show?id=21311323521' target=_blank style='color:#2F92EE;'>#OCT#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=34, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=13235, encryptionId=21311323521, topicName=OCT)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=19c567, createdName=habb, createdTime=Mon Mar 01 20:18:17 CST 2021, time=2021-03-01, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1338216, encodeId=1e211338216da, content=<a href='/topic/show?id=cd2a262068' target=_blank style='color:#2F92EE;'>#APA#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=34, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=2620, encryptionId=cd2a262068, topicName=APA)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=ce072500140, createdName=12498b3em08(暂无昵称), createdTime=Wed Jun 03 00:18:17 CST 2020, time=2020-06-03, status=1, ipAttribution=)]
  3. [GetPortalCommentsPageByObjectIdResponse(id=1862791, encodeId=d07f1862e9130, content=<a href='/topic/show?id=0b69542242' target=_blank style='color:#2F92EE;'>#CTO#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=46, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=5422, encryptionId=0b69542242, topicName=CTO)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c66088, createdName=gao_jian4217, createdTime=Sat Feb 20 06:18:17 CST 2021, time=2021-02-20, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1993319, encodeId=5bea1993319d4, content=<a href='/topic/show?id=b2f1280466' target=_blank style='color:#2F92EE;'>#ARVO#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=2804, encryptionId=b2f1280466, topicName=ARVO)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=636e2500187, createdName=12498da4m32(暂无昵称), createdTime=Mon Apr 19 21:18:17 CST 2021, time=2021-04-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2049302, encodeId=d22f2049302dc, content=<a href='/topic/show?id=4363e929ad' target=_blank style='color:#2F92EE;'>#Gene#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=7929, encryptionId=4363e929ad, topicName=Gene)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=63a4277, createdName=cy0324, createdTime=Mon May 03 19:18:17 CST 2021, time=2021-05-03, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1858349, encodeId=1c2618583491b, content=<a href='/topic/show?id=21311323521' target=_blank style='color:#2F92EE;'>#OCT#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=34, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=13235, encryptionId=21311323521, topicName=OCT)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=19c567, createdName=habb, createdTime=Mon Mar 01 20:18:17 CST 2021, time=2021-03-01, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1338216, encodeId=1e211338216da, content=<a href='/topic/show?id=cd2a262068' target=_blank style='color:#2F92EE;'>#APA#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=34, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=2620, encryptionId=cd2a262068, topicName=APA)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=ce072500140, createdName=12498b3em08(暂无昵称), createdTime=Wed Jun 03 00:18:17 CST 2020, time=2020-06-03, status=1, ipAttribution=)]
    2021-05-03 cy0324
  4. [GetPortalCommentsPageByObjectIdResponse(id=1862791, encodeId=d07f1862e9130, content=<a href='/topic/show?id=0b69542242' target=_blank style='color:#2F92EE;'>#CTO#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=46, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=5422, encryptionId=0b69542242, topicName=CTO)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c66088, createdName=gao_jian4217, createdTime=Sat Feb 20 06:18:17 CST 2021, time=2021-02-20, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1993319, encodeId=5bea1993319d4, content=<a href='/topic/show?id=b2f1280466' target=_blank style='color:#2F92EE;'>#ARVO#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=2804, encryptionId=b2f1280466, topicName=ARVO)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=636e2500187, createdName=12498da4m32(暂无昵称), createdTime=Mon Apr 19 21:18:17 CST 2021, time=2021-04-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2049302, encodeId=d22f2049302dc, content=<a href='/topic/show?id=4363e929ad' target=_blank style='color:#2F92EE;'>#Gene#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=7929, encryptionId=4363e929ad, topicName=Gene)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=63a4277, createdName=cy0324, createdTime=Mon May 03 19:18:17 CST 2021, time=2021-05-03, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1858349, encodeId=1c2618583491b, content=<a href='/topic/show?id=21311323521' target=_blank style='color:#2F92EE;'>#OCT#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=34, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=13235, encryptionId=21311323521, topicName=OCT)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=19c567, createdName=habb, createdTime=Mon Mar 01 20:18:17 CST 2021, time=2021-03-01, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1338216, encodeId=1e211338216da, content=<a href='/topic/show?id=cd2a262068' target=_blank style='color:#2F92EE;'>#APA#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=34, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=2620, encryptionId=cd2a262068, topicName=APA)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=ce072500140, createdName=12498b3em08(暂无昵称), createdTime=Wed Jun 03 00:18:17 CST 2020, time=2020-06-03, status=1, ipAttribution=)]
    2021-03-01 habb
  5. [GetPortalCommentsPageByObjectIdResponse(id=1862791, encodeId=d07f1862e9130, content=<a href='/topic/show?id=0b69542242' target=_blank style='color:#2F92EE;'>#CTO#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=46, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=5422, encryptionId=0b69542242, topicName=CTO)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c66088, createdName=gao_jian4217, createdTime=Sat Feb 20 06:18:17 CST 2021, time=2021-02-20, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1993319, encodeId=5bea1993319d4, content=<a href='/topic/show?id=b2f1280466' target=_blank style='color:#2F92EE;'>#ARVO#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=2804, encryptionId=b2f1280466, topicName=ARVO)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=636e2500187, createdName=12498da4m32(暂无昵称), createdTime=Mon Apr 19 21:18:17 CST 2021, time=2021-04-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2049302, encodeId=d22f2049302dc, content=<a href='/topic/show?id=4363e929ad' target=_blank style='color:#2F92EE;'>#Gene#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=7929, encryptionId=4363e929ad, topicName=Gene)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=63a4277, createdName=cy0324, createdTime=Mon May 03 19:18:17 CST 2021, time=2021-05-03, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1858349, encodeId=1c2618583491b, content=<a href='/topic/show?id=21311323521' target=_blank style='color:#2F92EE;'>#OCT#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=34, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=13235, encryptionId=21311323521, topicName=OCT)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=19c567, createdName=habb, createdTime=Mon Mar 01 20:18:17 CST 2021, time=2021-03-01, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1338216, encodeId=1e211338216da, content=<a href='/topic/show?id=cd2a262068' target=_blank style='color:#2F92EE;'>#APA#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=34, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=2620, encryptionId=cd2a262068, topicName=APA)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=ce072500140, createdName=12498b3em08(暂无昵称), createdTime=Wed Jun 03 00:18:17 CST 2020, time=2020-06-03, status=1, ipAttribution=)]

相关资讯

NEJM:使用重组因子VIII治疗的重症血友病A患者有较高的抑制剂发生率

中和抗–VIII因子抗体产生(抑制剂)治疗重度血友病A患者可能取决于替代治疗的集中使用。原始出处:Flora Peyvandi,Pier M. Mannucci,Isabella Garagiola,et al.A Randomized Trial of Factor VIII and Neutralizing Antibodies in Hemophilia A,NEJM,2016.5.26

拓展阅读

NEJM:使用重组因子VIII治疗的重症血友病A患者有较高的抑制剂发生率

中和抗–VIII因子抗体产生(抑制剂)治疗重度血友病A患者可能取决于替代治疗的集中使用。原始出处:Flora Peyvandi,Pier M. Mannucci,Isabella Garagiola,et al.A Randomized Trial of Factor VIII and Neutralizing Antibodies in Hemophilia A,NEJM,2016.5.26