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葛兰素史克将罕见病干细胞基因治疗技术转交给Orchard公司

2018-04-13 MedSci MedSci原创

干细胞基因治疗罕见病
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Orchard Therapeutics公司是英国的一家生物技术公司，一直致力于罕见病的造血干细胞基因疗法。该治疗手段是通过提取患者自体干细胞，体外进行基因修复后再回输到患者体内进行治疗，选用患者自身的干细胞可大幅度缩减因干细胞配对而延误的时间。

Orchard Therapeutics公司是英国的一家生物技术公司，一直致力于罕见病的造血干细胞基因疗法。该治疗手段是通过提取患者自体干细胞，体外进行基因修复后再回输到患者体内进行治疗，选用患者自身的干细胞可大幅度缩减因干细胞配对而延误的时间。

此次交易中，Orchard公司成功抢购全球制药巨头葛兰素史克公司（GSK）开发的罕见病基因疗法，交易后GSK将成为Orchard的投资者，坐拥19.9%的股权以及公司董事会席位。

Orchard收购的项目包括Strimvelis，这是2016年欧盟批准的第一个针对腺苷脱氨酶严重联合免疫缺陷症（ADA-SCID）儿童的离体自体基因疗法，以及两项正在进行的异染性脑白质营养不良的晚期临床项目和威斯科特奥尔德里奇综合征，和一种用于β地中海贫血的临床计划。在完成1型粘多糖病（MPS1或Hurler综合征），慢性肉芽肿性疾病和球状细胞脑白质营养不良的临床证据收集后，Orchard还将获得独家授权GSK合作伙伴Telethon / Ospedale San Raffaele的三项额外临床前计划。这些计划都将补充Orchard公司的临床和临床前基因疗法。

Orchard公司首席执行官Mark Rothera表示："收购这GSK的组合产品会进一步推动了Orchard成为一家全球性，完全整合的，引领罕见疾病基因治疗的公司。"

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  1. [GetPortalCommentsPageByObjectIdResponse(id=1315764, encodeId=6dd91315e6450, content=<a href='/topic/show?id=6f0188218ee' target=_blank style='color:#2F92EE;'>#葛兰素史克#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=25, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=88218, encryptionId=6f0188218ee, topicName=葛兰素史克)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=d58a435, createdName=sjq027, createdTime=Sun Apr 15 13:23:00 CST 2018, time=2018-04-15, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1467307, encodeId=7296146e30750, content=<a href='/topic/show?id=ef32e9539e8' target=_blank style='color:#2F92EE;'>#罕见#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=29, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=79539, encryptionId=ef32e9539e8, topicName=罕见)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=29f36868190, createdName=syscxl, createdTime=Sun Apr 15 13:23:00 CST 2018, time=2018-04-15, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=305533, encodeId=365830553362, content=厉害了我的哥, beContent=null, objectType=article, channel=null, level=null, likeNumber=1, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/oLAjfB7s1ib3IzoRn621JdKCFUI4XvNELcibocrAVU6aUzEVDSaf3rYH08PUYUiagGqxAEhj8g9WqqNvD1CupIpCrmnR2OrqHZh/0, createdBy=7f181365728, createdName=飛歌, createdTime=Sat Apr 14 00:12:40 CST 2018, time=2018-04-14, status=1, ipAttribution=)]
    2018-04-15 sjq027

    #葛兰素史克#

    0

  2. [GetPortalCommentsPageByObjectIdResponse(id=1315764, encodeId=6dd91315e6450, content=<a href='/topic/show?id=6f0188218ee' target=_blank style='color:#2F92EE;'>#葛兰素史克#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=25, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=88218, encryptionId=6f0188218ee, topicName=葛兰素史克)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=d58a435, createdName=sjq027, createdTime=Sun Apr 15 13:23:00 CST 2018, time=2018-04-15, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1467307, encodeId=7296146e30750, content=<a href='/topic/show?id=ef32e9539e8' target=_blank style='color:#2F92EE;'>#罕见#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=29, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=79539, encryptionId=ef32e9539e8, topicName=罕见)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=29f36868190, createdName=syscxl, createdTime=Sun Apr 15 13:23:00 CST 2018, time=2018-04-15, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=305533, encodeId=365830553362, content=厉害了我的哥, beContent=null, objectType=article, channel=null, level=null, likeNumber=1, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/oLAjfB7s1ib3IzoRn621JdKCFUI4XvNELcibocrAVU6aUzEVDSaf3rYH08PUYUiagGqxAEhj8g9WqqNvD1CupIpCrmnR2OrqHZh/0, createdBy=7f181365728, createdName=飛歌, createdTime=Sat Apr 14 00:12:40 CST 2018, time=2018-04-14, status=1, ipAttribution=)]
    2018-04-15 syscxl

    #罕见#

    0

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    2018-04-14 飛歌

    厉害了我的哥

    0