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FDA暂停Verve碱基编辑疗法IND申请,导致股价下跌30.49%!

FDA Verve 碱基编辑疗法
细胞基因治疗前沿
2022/11/09
2682


Verve Therapeutics是一家临床阶段的生物技术公司,致力于通过碱基编辑技术治疗心血管疾病。Verve公司在周一发布公告,表示其开发的碱基编辑疗法VERVE-101被FDA暂停了IND申请。该疗法用于治疗杂合子家族性高胆固醇血症 (HeFH),它有望通过一次性治疗,永久降低患者心血管疾病的发作风险。


Verve表示在今年10月公司向FDA提交了VERVE-101的IND申请,并在2022年11月4日收到了FDA的暂停通知。该公司预计将在30天内收到FDA的正式信函。Verve计划在与FDA接触前提供更新,并打算与FDA密切合作,尽快解决IND申请暂停的问题。

投资者们对于Verve关于FDA暂停IND申请公告的回应则是股票抛售,导致周一上午公告发布后,Verve的股价在周一的交易中下跌30.49%,从周五的收盘价31.29美元跌至周一收盘价21.75美元。



关于VERVE-101

VERVE-101药物基本信息(部分)
截图来源:药融云全球药物研发数据库

VERVE-101是Verve Therapeutics与在碱基编辑疗法研发领先的公司Beam Therapeutics合作开发的,通过在体内对肝脏中PCSK9基因进行单碱基编辑,降低PCSK9表达和低密度脂蛋白胆固醇(LDL-C)水平的基因编辑疗法。该疗法可用于治疗杂合子家族性高胆固醇血症 (HeFH),HeFH是动脉粥样硬化性心血管疾病(ASCVD)的一种亚型。据药融云数据库显示,目前该药针对家族性高胆固醇血症动脉粥样硬化的临床试验已进入临床1期试验阶段。

VERVE-101适应症研发现状
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截图来源:药融云全球药物研发数据库

HeFH患者通常携带LDL受体(LDLR)基因突变,导致LDLR表达下降,引起血液中LDL-C水平升高。随着时间的推移,LDL-C在心脏动脉中蓄积,导致血流减少或阻塞,最终导致心脏病发作。PCSK9 基因表达的 PCSK9 蛋白能够与与肝细胞表面的 LDL 受体(LDL-R)结合,使LDL-R 降解,从而升高血浆中LDL-C水平。降低 PCSK9基因的表达或抑制 PCSK9 蛋白与 LDL-R 的结合,就能降低血浆中低密度脂蛋白胆固醇水平,从而预防心血管疾病的发生。


VERVE-101碱基编辑疗法旨在通过脂质纳米颗粒(LNP)递送药物,该药物精确的通过碱基编辑永久关闭肝脏中PCSK9基因的表达,从而降低低密度脂蛋白胆固醇(LDL-C),实现对包括 HeFH在内的心血管疾病的预防和治疗。


信息参考:
[1] Verve Therapeutics官网
[2] 药融云数据库


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*声明:本文由入驻摩熵医药的相关人员撰写或转载,观点仅代表作者本人,不代表摩熵医药的立场。
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