风湿免疫科

订阅

MedSci主页 / 所有栏目 / 风湿免疫科

FDA授予OTL-103再生医学高级治疗（RMAT）认定用于治疗Wiskott-Aldrich综合征

2019-08-04 Allan MedSci原创

Wiskott-Aldrich综合征OTL-103再生医学高级治疗（RMAT）
关注

Orchard是一家领先的生物制药公司，致力于通过创新的基因疗法改善罕见疾病患者的生活。Orchard近日宣布，其OTL-103已经获得FDA的再生医学高级治疗（RMAT）认定。OTL-103是基于离体自体造血干细胞（HSC）的基因疗法，用于治疗Wiskott-Aldrich综合征（WAS）。

Orchard是一家领先的生物制药公司，致力于通过创新的基因疗法改善罕见疾病患者的生活。Orchard近日宣布，其OTL-103已经获得FDA的再生医学高级治疗（RMAT）认定。OTL-103是基于离体自体造血干细胞（HSC）的基因疗法，用于治疗Wiskott-Aldrich综合征（WAS）。

Wiskott-Aldrich综合征（WAS）是由X染色体上WAS基因突变所导致的，兼有原发性免疫缺陷病和遗传性血液病表现的特殊类型疾病。虽然典型WAS的临床表现特征比较明显，经相关基因（WAS）及其产物蛋白（WASP）检测可以确诊，但由于发病率较低，病例严重程度差异显著。


原始出处：

http://www.firstwordpharma.com/node/1656499#axzz5vaProhET

本文系梅斯医学（MedSci）原创编译整理，转载需授权!

评论区 (4)
#插入话题
  1. [GetPortalCommentsPageByObjectIdResponse(id=1668902, encodeId=03e416689027b, content=<a href='/topic/show?id=227518e107b' target=_blank style='color:#2F92EE;'>#Wiskott-Aldrich综合征#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=87, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=18710, encryptionId=227518e107b, topicName=Wiskott-Aldrich综合征)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=ffcc26471069, createdName=guojianrong, createdTime=Mon Sep 09 07:28:00 CST 2019, time=2019-09-09, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1316369, encodeId=1ee713163692b, content=<a href='/topic/show?id=f3789349c3' target=_blank style='color:#2F92EE;'>#ICH#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=79, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9349, encryptionId=f3789349c3, topicName=ICH)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c192448, createdName=diushouji, createdTime=Tue Aug 06 00:28:00 CST 2019, time=2019-08-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1431595, encodeId=06991431595de, 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=64, 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=Tue Aug 06 00:28:00 CST 2019, time=2019-08-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1469410, encodeId=becc146941004, content=<a href='/topic/show?id=9d6522e613' target=_blank style='color:#2F92EE;'>#ALD#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=75, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=2276, encryptionId=9d6522e613, topicName=ALD)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=64f67025397, createdName=zhangjingnwpu, createdTime=Tue Aug 06 00:28:00 CST 2019, time=2019-08-06, status=1, ipAttribution=)]
    2019-09-09 guojianrong

    #Wiskott-Aldrich综合征#

    0

  2. [GetPortalCommentsPageByObjectIdResponse(id=1668902, encodeId=03e416689027b, content=<a href='/topic/show?id=227518e107b' target=_blank style='color:#2F92EE;'>#Wiskott-Aldrich综合征#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=87, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=18710, encryptionId=227518e107b, topicName=Wiskott-Aldrich综合征)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=ffcc26471069, createdName=guojianrong, createdTime=Mon Sep 09 07:28:00 CST 2019, time=2019-09-09, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1316369, encodeId=1ee713163692b, content=<a href='/topic/show?id=f3789349c3' target=_blank style='color:#2F92EE;'>#ICH#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=79, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9349, encryptionId=f3789349c3, topicName=ICH)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c192448, createdName=diushouji, createdTime=Tue Aug 06 00:28:00 CST 2019, time=2019-08-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1431595, encodeId=06991431595de, 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=64, 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=Tue Aug 06 00:28:00 CST 2019, time=2019-08-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1469410, encodeId=becc146941004, content=<a href='/topic/show?id=9d6522e613' target=_blank style='color:#2F92EE;'>#ALD#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=75, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=2276, encryptionId=9d6522e613, topicName=ALD)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=64f67025397, createdName=zhangjingnwpu, createdTime=Tue Aug 06 00:28:00 CST 2019, time=2019-08-06, status=1, ipAttribution=)]
    2019-08-06 diushouji

    #ICH#

    0

  3. [GetPortalCommentsPageByObjectIdResponse(id=1668902, encodeId=03e416689027b, content=<a href='/topic/show?id=227518e107b' target=_blank style='color:#2F92EE;'>#Wiskott-Aldrich综合征#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=87, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=18710, encryptionId=227518e107b, topicName=Wiskott-Aldrich综合征)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=ffcc26471069, createdName=guojianrong, createdTime=Mon Sep 09 07:28:00 CST 2019, time=2019-09-09, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1316369, encodeId=1ee713163692b, content=<a href='/topic/show?id=f3789349c3' target=_blank style='color:#2F92EE;'>#ICH#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=79, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9349, encryptionId=f3789349c3, topicName=ICH)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c192448, createdName=diushouji, createdTime=Tue Aug 06 00:28:00 CST 2019, time=2019-08-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1431595, encodeId=06991431595de, 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=64, 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=Tue Aug 06 00:28:00 CST 2019, time=2019-08-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1469410, encodeId=becc146941004, content=<a href='/topic/show?id=9d6522e613' target=_blank style='color:#2F92EE;'>#ALD#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=75, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=2276, encryptionId=9d6522e613, topicName=ALD)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=64f67025397, createdName=zhangjingnwpu, createdTime=Tue Aug 06 00:28:00 CST 2019, time=2019-08-06, status=1, ipAttribution=)]
    2019-08-06 zhouqu_8

    #综合征#

    0

  4. [GetPortalCommentsPageByObjectIdResponse(id=1668902, encodeId=03e416689027b, content=<a href='/topic/show?id=227518e107b' target=_blank style='color:#2F92EE;'>#Wiskott-Aldrich综合征#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=87, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=18710, encryptionId=227518e107b, topicName=Wiskott-Aldrich综合征)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=ffcc26471069, createdName=guojianrong, createdTime=Mon Sep 09 07:28:00 CST 2019, time=2019-09-09, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1316369, encodeId=1ee713163692b, content=<a href='/topic/show?id=f3789349c3' target=_blank style='color:#2F92EE;'>#ICH#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=79, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9349, encryptionId=f3789349c3, topicName=ICH)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c192448, createdName=diushouji, createdTime=Tue Aug 06 00:28:00 CST 2019, time=2019-08-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1431595, encodeId=06991431595de, 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=64, 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=Tue Aug 06 00:28:00 CST 2019, time=2019-08-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1469410, encodeId=becc146941004, content=<a href='/topic/show?id=9d6522e613' target=_blank style='color:#2F92EE;'>#ALD#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=75, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=2276, encryptionId=9d6522e613, topicName=ALD)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=64f67025397, createdName=zhangjingnwpu, createdTime=Tue Aug 06 00:28:00 CST 2019, time=2019-08-06, status=1, ipAttribution=)]
    2019-08-06 zhangjingnwpu

    #ALD#

    0

相关资讯

Blood：基因疗法对Wiskott-Aldrich综合征重度患者的疗效。

Wiskott-Aldrich综合征（又称湿疹血小板减少伴免疫缺陷综合征）是一种隐性伴性遗传性疾病，只发生于男性儿童，以血小板减少伴小血小板、湿疹、免疫缺陷、易患自身免疫性疾病和恶性肿瘤为临床特点。

Lancet haemat： 慢病毒造血干细胞基因编辑疗法用于Wiskott-Aldrich综合征患儿的临床疗效和安全性评估

Wiskott-Aldrich综合征是一种罕见的危及生命的X染色体连锁的原发性免疫缺陷，其特征是微血小板减少、感染、湿疹、自身免疫和恶性疾病。慢病毒载体介导的造血干/祖细胞(HSPC)基因疗法是一种潜在的治疗方法，或可替代异基因HSPC移植。