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婴儿型庞贝氏病AAV基因治疗药物获IND受理

2022-03-18 锦篮基因 锦篮基因

锦篮基因
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庞贝氏病是一种由于GAA基因突变导致的遗传疾病，患者体内酸性葡萄糖苷酶活性降低或几乎失活而造成糖原降解障碍，贮积在肌肉肝脏中并导致一系列严重症状直至威胁生命。根据发病年龄、病情进展和疾病严重程度等情况

庞贝氏病是一种由于GAA基因突变导致的遗传疾病，患者体内酸性葡萄糖苷酶活性降低或几乎失活而造成糖原降解障碍，贮积在肌肉肝脏中并导致一系列严重症状直至威胁生命。根据发病年龄、病情进展和疾病严重程度等情况临床上可分为婴儿型庞贝氏病（IOPD）和晚发型庞贝氏病（LOPD）。IOPD发病凶险，严重危及患儿生命。当前临床可及的治疗用药物尚不能满足庞贝病患者的长期医疗需求，尤其是不能改善IOPD的临床困境。

GC301腺相关病毒注射液是一款基因治疗药物，有效成分是重组腺相关病毒载体搭载GAA基因表达序列，通过单次静脉输注，一次性抵达全身各系统脏器，达到长期改善GAA酶活力水平、持续清除累积糖原和恢复肌肉力量的目的。国际同类产品均为针对LOPD设计开发。

不仅是庞贝氏病，现如今随着更多的罕见病的遗传病因被明确，罕见病患者有机会获得准确的诊断结果，罕见病患者也已经成为被社会广泛认识、关注和理解的群体。不放弃每一位罕见病患者是国家和社会的决心，促进中国罕见病基因药物从基础走向临床和市场是锦篮团队多年来长久的坚持，我们相信在社会各界的共同协同努力下，罕见病治疗难题将会被加速破解。截止至今，锦篮基因已推动3项罕见病基因治疗候选药物进入临床试验审评阶段，其临床适应症分别为脊髓性肌萎缩症、基因缺陷型高甘油三酯血症以及本次获得受理的庞贝氏病，我们也期待在不久的未来它们能真正走向临床并为患者带来现实获益及生活改善。

图片

▲ 图片来源：CDE官网截图

关于锦篮基因

北京锦篮基因科技有限公司成立于2018年，是一家专注于基因治疗药物创新研发的生物科技平台公司。公司专注于AAV载体递送技术介导的遗传性神经肌肉疾病、遗传代谢疾病、溶酶体疾病以及眼科疾病等领域基因治疗产品研发，以推动中国罕见病基因药物从基础走向临床和市场为使命，致力于让“无药可治”变成“一劳永逸”治愈，造福患者及家庭；通过罕见病基因药物研发和临床应用，更深入认识生命健康，过渡到慢性病和其它重大疾病的治疗和康复。

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    2022-03-26 周虎

    #治疗药物#

    0

  2. [GetPortalCommentsPageByObjectIdResponse(id=1921868, encodeId=10e819218685a, content=<a href='/topic/show?id=4f0a6469837' target=_blank style='color:#2F92EE;'>#治疗药物#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=39, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=64698, encryptionId=4f0a6469837, topicName=治疗药物)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c9d3459, createdName=周虎, createdTime=Sat Mar 26 05:14:32 CST 2022, time=2022-03-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1204722, encodeId=88da1204e22f3, content=学习学习了, beContent=null, objectType=article, channel=null, level=null, likeNumber=52, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20211111/81c0b64745c644319545ec26d7ef0b34/9ddb2864d5df4dd9bf4305e380bffc96.jpg, createdBy=c7835705079, createdName=屋顶瞄爱赏月, createdTime=Mon Mar 21 20:40:47 CST 2022, time=2022-03-21, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1271886, encodeId=392212e1886ea, content=<a href='/topic/show?id=9fef169483' target=_blank style='color:#2F92EE;'>#AAV#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=36, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=1694, encryptionId=9fef169483, topicName=AAV)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=8ba7103, createdName=xuqianhua, createdTime=Sun Mar 20 07:14:32 CST 2022, time=2022-03-20, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1469191, encodeId=60e514691917c, content=<a href='/topic/show?id=211f98250b' target=_blank style='color:#2F92EE;'>#IND#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=50, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9825, encryptionId=211f98250b, topicName=IND)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=16377007930, createdName=xiaogang319, createdTime=Sun Mar 20 07:14:32 CST 2022, time=2022-03-20, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1203905, encodeId=4abd120390544, content=好, beContent=null, objectType=article, channel=null, level=null, likeNumber=69, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=fa8c2337585, createdName=12283265m64暂无昵称, createdTime=Fri Mar 18 22:08:39 CST 2022, time=2022-03-18, status=1, ipAttribution=)]
    2022-03-21 屋顶瞄爱赏月

    学习学习了

    0

  3. [GetPortalCommentsPageByObjectIdResponse(id=1921868, encodeId=10e819218685a, content=<a href='/topic/show?id=4f0a6469837' target=_blank style='color:#2F92EE;'>#治疗药物#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=39, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=64698, encryptionId=4f0a6469837, topicName=治疗药物)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c9d3459, createdName=周虎, createdTime=Sat Mar 26 05:14:32 CST 2022, time=2022-03-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1204722, encodeId=88da1204e22f3, content=学习学习了, beContent=null, objectType=article, channel=null, level=null, likeNumber=52, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20211111/81c0b64745c644319545ec26d7ef0b34/9ddb2864d5df4dd9bf4305e380bffc96.jpg, createdBy=c7835705079, createdName=屋顶瞄爱赏月, createdTime=Mon Mar 21 20:40:47 CST 2022, time=2022-03-21, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1271886, encodeId=392212e1886ea, content=<a href='/topic/show?id=9fef169483' target=_blank style='color:#2F92EE;'>#AAV#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=36, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=1694, encryptionId=9fef169483, topicName=AAV)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=8ba7103, createdName=xuqianhua, createdTime=Sun Mar 20 07:14:32 CST 2022, time=2022-03-20, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1469191, encodeId=60e514691917c, content=<a href='/topic/show?id=211f98250b' target=_blank style='color:#2F92EE;'>#IND#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=50, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9825, encryptionId=211f98250b, topicName=IND)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=16377007930, createdName=xiaogang319, createdTime=Sun Mar 20 07:14:32 CST 2022, time=2022-03-20, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1203905, encodeId=4abd120390544, content=好, beContent=null, objectType=article, channel=null, level=null, likeNumber=69, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=fa8c2337585, createdName=12283265m64暂无昵称, createdTime=Fri Mar 18 22:08:39 CST 2022, time=2022-03-18, status=1, ipAttribution=)]
    2022-03-20 xuqianhua

    #AAV#

    0

  4. [GetPortalCommentsPageByObjectIdResponse(id=1921868, encodeId=10e819218685a, content=<a href='/topic/show?id=4f0a6469837' target=_blank style='color:#2F92EE;'>#治疗药物#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=39, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=64698, encryptionId=4f0a6469837, topicName=治疗药物)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c9d3459, createdName=周虎, createdTime=Sat Mar 26 05:14:32 CST 2022, time=2022-03-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1204722, encodeId=88da1204e22f3, content=学习学习了, beContent=null, objectType=article, channel=null, level=null, likeNumber=52, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20211111/81c0b64745c644319545ec26d7ef0b34/9ddb2864d5df4dd9bf4305e380bffc96.jpg, createdBy=c7835705079, createdName=屋顶瞄爱赏月, createdTime=Mon Mar 21 20:40:47 CST 2022, time=2022-03-21, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1271886, encodeId=392212e1886ea, content=<a href='/topic/show?id=9fef169483' target=_blank style='color:#2F92EE;'>#AAV#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=36, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=1694, encryptionId=9fef169483, topicName=AAV)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=8ba7103, createdName=xuqianhua, createdTime=Sun Mar 20 07:14:32 CST 2022, time=2022-03-20, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1469191, encodeId=60e514691917c, content=<a href='/topic/show?id=211f98250b' target=_blank style='color:#2F92EE;'>#IND#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=50, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9825, encryptionId=211f98250b, topicName=IND)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=16377007930, createdName=xiaogang319, createdTime=Sun Mar 20 07:14:32 CST 2022, time=2022-03-20, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1203905, encodeId=4abd120390544, content=好, beContent=null, objectType=article, channel=null, level=null, likeNumber=69, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=fa8c2337585, createdName=12283265m64暂无昵称, createdTime=Fri Mar 18 22:08:39 CST 2022, time=2022-03-18, status=1, ipAttribution=)]
    2022-03-20 xiaogang319

    #IND#

    0

  5. [GetPortalCommentsPageByObjectIdResponse(id=1921868, encodeId=10e819218685a, content=<a href='/topic/show?id=4f0a6469837' target=_blank style='color:#2F92EE;'>#治疗药物#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=39, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=64698, encryptionId=4f0a6469837, topicName=治疗药物)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c9d3459, createdName=周虎, createdTime=Sat Mar 26 05:14:32 CST 2022, time=2022-03-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1204722, encodeId=88da1204e22f3, content=学习学习了, beContent=null, objectType=article, channel=null, level=null, likeNumber=52, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20211111/81c0b64745c644319545ec26d7ef0b34/9ddb2864d5df4dd9bf4305e380bffc96.jpg, createdBy=c7835705079, createdName=屋顶瞄爱赏月, createdTime=Mon Mar 21 20:40:47 CST 2022, time=2022-03-21, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1271886, encodeId=392212e1886ea, content=<a href='/topic/show?id=9fef169483' target=_blank style='color:#2F92EE;'>#AAV#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=36, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=1694, encryptionId=9fef169483, topicName=AAV)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=8ba7103, createdName=xuqianhua, createdTime=Sun Mar 20 07:14:32 CST 2022, time=2022-03-20, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1469191, encodeId=60e514691917c, content=<a href='/topic/show?id=211f98250b' target=_blank style='color:#2F92EE;'>#IND#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=50, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9825, encryptionId=211f98250b, topicName=IND)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=16377007930, createdName=xiaogang319, createdTime=Sun Mar 20 07:14:32 CST 2022, time=2022-03-20, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1203905, encodeId=4abd120390544, content=好, beContent=null, objectType=article, channel=null, level=null, likeNumber=69, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=fa8c2337585, createdName=12283265m64暂无昵称, createdTime=Fri Mar 18 22:08:39 CST 2022, time=2022-03-18, status=1, ipAttribution=)]
    2022-03-18 12283265m64暂无昵称

    好

    0