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FDA授予基因疗法SPIRO-2101“孤儿药称号”,用于治疗囊性纤维化

2020-09-26 Allan MedSci原创

囊性纤维化(CF)是一种常见的遗传疾病,此病症最常影响肺脏,但也常发生于胰脏、肝脏、肾脏以及肠。长期影响包含肺部感染所导致的呼吸困难以及积痰。

囊性纤维化(CF)是一种常见的遗传疾病,此病症最常影响肺脏,但也常发生于胰脏、肝脏、肾脏以及肠。长期影响包含肺部感染所导致的呼吸困难以及积痰。

Spirovant Sciences是一家专注于基因疗法的生物制药公司,Spirovant今天宣布,美国食品药品监督管理局(FDA)已授予其主要候选药物SPIRO-2101“孤儿药称号”,用于治疗囊性纤维化(CF)。

SPIRO-2101是一种吸入式腺相关病毒(AAV)基因疗法,旨在治疗具有囊性纤维化跨膜电导调节剂(CFTR)基因突变的CF患者。SPIRO-2101包含经过改良的AAV衣壳,该衣壳经过改造后对人类呼吸道上皮细胞具有高度的特异性

Spirovant Sciences首席执行官Joan Lau博士说:“囊性纤维化是一种令人难以置信的破坏性疾病。SPIRO-2101孤儿药称号的获得强调了为这些患者提供治疗选择的紧迫性,特别是那些具有非常罕见突变亚型的患者”。

 

原始出处:

https://www.firstwordpharma.com/node/1760314?tsid=4

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    2021-08-31 wolongzxh
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    2020-09-27 14818eb4m67暂无昵称

    学习

    0

  4. [GetPortalCommentsPageByObjectIdResponse(id=1675937, encodeId=a91416e5937fe, content=<a href='/topic/show?id=7395403e580' target=_blank style='color:#2F92EE;'>#囊性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=73, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=40375, encryptionId=7395403e580, topicName=囊性)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=926927300775, createdName=wolongzxh, createdTime=Tue Aug 31 10:54:07 CST 2021, time=2021-08-31, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1365251, encodeId=aa9a136525148, content=<a href='/topic/show?id=dfa045434e1' target=_blank style='color:#2F92EE;'>#孤儿药称号#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=88, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=45434, encryptionId=dfa045434e1, topicName=孤儿药称号)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=cd0d217, createdName=huiwelcome, createdTime=Mon Sep 28 03:54:07 CST 2020, time=2020-09-28, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=888638, encodeId=2dcf888638fe, content=学习, beContent=null, objectType=article, channel=null, level=null, likeNumber=115, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=64245268311, createdName=14818eb4m67暂无昵称, createdTime=Sun Sep 27 07:59:19 CST 2020, time=2020-09-27, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=888518, encodeId=97e888851859, content=<a href='/topic/show?id=a22d4038010' target=_blank style='color:#2F92EE;'>#囊性纤维化#</a>的<a href='/topic/show?id=567041e8055' target=_blank style='color:#2F92EE;'>#基因治疗#</a>提上日程,未来有明确基因的原因导致的疾病,基因治疗可能是终极解决方案, beContent=null, objectType=article, channel=null, level=null, likeNumber=251, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=40380, encryptionId=a22d4038010, topicName=囊性纤维化), TopicDto(id=41780, encryptionId=567041e8055, topicName=基因治疗)], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20220519/c2ab253484ee4527a2d4e9589a4821ac/45de9bf494a54becb2ea4369c9d11e85.jpg, createdBy=7a3710, createdName=lovetcm, createdTime=Sat Sep 26 13:35:47 CST 2020, time=2020-09-26, status=1, ipAttribution=)]
    2020-09-26 lovetcm

    #囊性纤维化##基因治疗#提上日程,未来有明确基因的原因导致的疾病,基因治疗可能是终极解决方案

    0

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