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“基因魔剪”CRISPR商业化加速,细胞治疗成**站

2018-06-29 佚名 基因谷

高盛最近的一项针对生物技术领域的研究报告《基因组革命》显示,中国的研究人员已经在测试CRISPR编辑的人类细胞方面领先一步,而全球的商业巨头也纷纷将战略市场瞄准了中国。

高盛最近的一项针对生物技术领域的研究报告《基因组革命》显示,中国的研究人员已经在测试CRISPR编辑的人类细胞方面领先一步,而全球的商业巨头也纷纷将战略市场瞄准了中国。

神奇的“基因魔剪”

CRISPR之所以备受推崇,是因为这种基因编辑技术相比较传统手段更高效更精准:2012年,来自美国和奥地利的科学家共同改进了CRISPR-Cas9系统,并在其发表的研究论文中预示:CRISPR可作为一种高效而特异的RNA介导的基因编辑工具。2013年,华人科学家张锋等人利用CRISPR进行哺乳动物细胞的基因编辑,开启了CRISPR作为可编程的基因编辑工具的新纪元。

仅在过去一年里,几十位世界各地的研究人员就发布科学论文详细阐述自己的研究结果,他们利用CRISPR剪断和取代不需要的DNA来开发治疗癌症、HIV、失明、慢性疼痛、肌肉萎缩症和亨廷顿症等疾病的疗法。

“CRISPR技术的商业化在细胞治疗上可能是最容易的,会涉及到把免疫细胞拿到体外通过CRISPR技术进行基因编辑。在疾病的应用上,以相对而言比较简单的艾滋病治疗为例:可以把人体T细胞或造血干细胞其中的一个基因CCR5敲掉, 这将导致艾滋病毒无法侵染这类改造过的T细胞,从而患者身体的免疫系统能够重建。”默克生命科学科研解决方案战略负责人刘颖在接受第一财经采访时表示。

近几年开启转型之路的默克公司在基因编辑上押下重注,目前他们已经在全球7个国家拥有CRISPR剪切后进行基因插入的专利。

“目前的CRISPR技术和基因编辑技术,仍处于早期研发阶段,所以可能我们所有的研究者,还需要5-10年的时间,才能逐渐地适应CRISPR在生命科学领域的应用,包括让他们使用这种技术针对一些具体的疾病做一些初步的诊断。”默克生命科学中国董事总经理卫政熹如此表示,“即使如此,我们的业务依然呈现了爆发式的增长。到目前为止,我们已在全球范围拥有了74名合作伙伴,包括哈佛医学院、哥伦比亚大学、斯坦福大学和约翰·霍普金斯大学等世界一流院校,同济大学是我们的第75名合作伙伴,将获得默克的基因组编辑工具的使用权。”

这家企业在6月20日宣布了和中国的数项合作,与基因编辑CRISPR技术相关的合作 -与同济大学建立CRISPR核心合作伙伴关系成为了重要一环。

“现在在中国地区的政府管理方面,我们看到了诸多积极的变化,包括审批速度的加快,对于一些以前管得较严的地方,现在都有逐渐的放开,这都是积极的发展态势。这为中国未来生物制药、生物科技的前沿发展奠定了坚实的基础,这也是我们非常看好中国市场的原因。”卫政熹表示。

中国:九项临床试验正在进行

近几年,全球资本市场对于CRISPR这项“诺奖级别”的生命科学技术相当推崇:在刚刚过去的一年,三家CRISPR基因编辑技术上市公司(Editas Medicine、CRISPR Therapeutics、Intellia Therapeutics)的累计市值翻了三番,现在已经接近60亿美元。

高盛分析师Salveen Richter表示,当一群欧美生物先锋还在排队进行其首次人体试验时,中国研究人员就已经在测试CRISPR编辑的人类细胞方面拔得了头筹。

这份报告指出,截至2018年2月底,在已登记的测试CRISPR编辑细胞治疗多种癌症、HIV感染临床研究中,中国占了九项,而美国则仅有一项。这9项研究都由中国的顶级公立医院发起/赞助,据报道,共有超过80名患者正在接受这些基因药物的治疗。

“对于CRISPR的应用领域之一细胞治疗而言,现在我们国家的政策环境非常好,我们现在都是备案制,只要你这个机构允许做,就可以把项目拿出来,然后到国家去备案,备案好了以后就可以做临床研究了。”同济大学生命科学与技术学院院长高绍荣在接受第一财经采访时如此表示。

据他透露,目前我国基于CRISPR的探索仍处于研发阶段,“但过不了很久,我们就可以进入量产的阶段。”他表示,“中国的发展非常迅速,要达到欧美国家相同的水平可能不需要花费那么多时间。而一旦这个技术应用到了生物制药领域,会是非常有用的,这可能是一个诺贝尔奖级别的领域。”

除了政策的支持外,大量生命科学“海归派”的回流也让中国的这一领域充满活力。“由创新驱动的中国经济正由高速增长阶段向高质量发展阶段转变,与此同时中国的尖端技术也展现出了其强大的竞争力,如干细胞研究就是一个例子。另外中国是世界上最早的一个允许基因治疗的国家、线病毒P51治疗的一个国家,所以说结合现在的良好环境,该行业在中国,未来大有可为。”卫政熹说。

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    2018-07-01 yuandd
  4. [GetPortalCommentsPageByObjectIdResponse(id=1771783, encodeId=d6cf1e717832f, content=<a href='/topic/show?id=62754005931' target=_blank style='color:#2F92EE;'>#商业#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=57, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=40059, encryptionId=62754005931, topicName=商业)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=fd6238344589, createdName=zhangxingru, createdTime=Sun Feb 10 16:29:00 CST 2019, time=2019-02-10, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1683447, encodeId=e4d9168344ea9, content=<a href='/topic/show?id=a576102854fe' target=_blank style='color:#2F92EE;'>#魔剪#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=63, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=102854, encryptionId=a576102854fe, topicName=魔剪)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c09b27973273, createdName=neurosurgeon, createdTime=Mon Nov 26 08:29:00 CST 2018, time=2018-11-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1276080, encodeId=72e412e60806a, content=<a href='/topic/show?id=755d5211a0' target=_blank style='color:#2F92EE;'>#CRISPR#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=54, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=5211, encryptionId=755d5211a0, topicName=CRISPR)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=583c133, createdName=yuandd, createdTime=Sun Jul 01 07:29:00 CST 2018, time=2018-07-01, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1506757, encodeId=31261506e575e, content=<a href='/topic/show?id=afce40064d1' target=_blank style='color:#2F92EE;'>#商业化#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=74, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=40064, encryptionId=afce40064d1, topicName=商业化)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=8b549798360, createdName=yeaweam, createdTime=Sun Jul 01 07:29:00 CST 2018, time=2018-07-01, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=327988, encodeId=f56932e98870, content=学习了.获益匪浅.感谢分享, beContent=null, objectType=article, channel=null, level=null, likeNumber=129, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://cdnapi.center.medsci.cn/medsci/head/2018/02/27/c53a212859edcad4f60a54feffff50bb.jpg, createdBy=67cf1525321, createdName=13718711b3m, createdTime=Fri Jun 29 16:49:32 CST 2018, time=2018-06-29, status=1, ipAttribution=)]
    2018-07-01 yeaweam
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    2018-06-29 13718711b3m

    学习了.获益匪浅.感谢分享

    0

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