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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