Gene Therapy Side Effects: Verve Therapeutics Halts Enrollment Of Lead Gene Therapy Trial In Patients With Bad Cholesterol
Gene Therapy Side Effects: Verve Therapeutics Halts Enrollment Of Lead Gene Therapy Trial In Patients With Bad Cholesterol
Verve Therapeutics Inc (NASDAQ:VERV) announced an update from the Heart-1 Phase 1b trial of VERVE-101.
Verve Therapeutics Inc（纳斯达克股票代码：VERV）宣布了 VERVE-101 Heart-1 1b 期试验的最新情况。
VERVE-101 is being evaluated in patients with a genetic condition that leads to high cholesterol levels, which raises the risk of heart disease. The treatment is supposed to permanently turn off a protein called PCSK9, which helps reduce blood cholesterol levels.
正在对患有导致高胆固醇水平的遗传病患者进行 VERVE-101 评估，这会增加患心脏病的风险。该治疗本应永久关闭一种叫做 PCSK9 的蛋白质，它有助于降低血液中的胆固醇水平。
The company said that one of the six patients treated with a 0.45-mg/kg dose of VERVE-101 experienced grade 3 increases in a particular liver enzyme and a grade 3 case of thrombocytopenia (low blood platelets).
该公司表示，在接受0.45mg/kg剂量的 VERVE-101 治疗的六名患者中，有一例出现了特定肝酶的3级升高和3级血小板减少症（低血小板）的病例。
The participant did not experience any bleeding or other symptoms related to the laboratory abnormalities, which resolved fully within a few days.
参与者没有出现任何与实验室异常相关的出血或其他症状，这些症状在几天内完全消退。
In consultation with the study's independent data and safety monitoring board (DSMB), Verve has decided to pause enrollment in the Heart-1 clinical trial.
经与该研究的独立数据和安全监测委员会（DSMB）协商，Verve决定暂停Heart-1临床试验的注册。
Verve is investigating the laboratory abnormalities and expects to work with regulatory authorities to define a path forward for VERVE-101 based on those results.
Verve 正在调查实验室异常情况，并预计将与监管机构合作，根据这些结果确定 VERVE-101 的前进方向。
Verve is now prioritizing the development of VERVE-102 and the initiation of the Heart-2 clinical trial.
Verve 现在优先开发 VERVE-102 和启动 Heart-2 临床试验。
VERVE-102 uses the same base editor and guide RNA for PCSK9 but a different lipid nanoparticle (LNP) delivery system than VERVE-101.
VERVE-102 对 PCSK9 使用相同的碱基编辑器和指南 RNA，但脂质纳米颗粒 (LNP) 输送系统与 VERVE-101 不同。
Verve has received regulatory clearances for the Heart-2 clinical trial in the U.K. and Canada. It plans to initiate the Heart-2 clinical trial in patients with HeFH or premature coronary artery disease in the second quarter of 2024.
Verve已获得英国和加拿大的Heart-2临床试验的监管许可。它计划在2024年第二季度启动针对HefH或早产冠心病患者的Heart-2临床试验。
VERVE-101 is a novel, investigational gene editing medicine designed to be a single-course treatment that permanently turns off the PCSK9 gene in the liver to reduce disease-driving low-density lipoprotein cholesterol.
VERVE-101 是一种新型的研究性基因编辑药物，旨在作为一种单疗程疗法，永久关闭肝脏中的 PCSK9 基因，从而降低导致疾病的低密度脂蛋白胆固醇。
Verve and Beam Therapeutics Inc (NASDAQ:BEAM) entered into a collaboration and license agreement in April 2019, under which Verve received exclusive access to Beam's base editing, gene editing, and delivery technologies for human therapeutic applications against certain cardiovascular targets.
Verve and Beam 治疗公司（纳斯达克股票代码：BEAM） 已输入 于2019年4月签订合作和许可协议，根据该协议，Verve获得了Beam针对某些心血管靶标的人体治疗应用的基础编辑、基因编辑和交付技术的独家使用权。
In October 2023, Verve announced the expansion of its relationship with Eli Lilly And Co (NYSE:LLY), which has acquired from Beam Therapeutics certain product rights to Verve's cardiovascular in vivo gene editing programs targeting PCSK9 and ANGPTL3, as well as a third undisclosed cardiovascular disease target.
2023年10月，Verve宣布扩大与礼来公司（纽约证券交易所代码：LLY）的关系，后者已从Beam Therapeutics手中收购了Verve针对 PCSK9 和 ANGPTL3 的心血管活体基因编辑计划的某些产品版权，以及第三个未公开的心血管疾病靶标。
Price Action: VERV stock is down 36.0% at $8.18, and BEAM shares are down 7.37% at $30.29 on the last check Tuesday.
价格走势：在周二的最后一次检查中，VERV股价下跌36.0％，至8.18美元，BEAM股价下跌7.37％，至30.29美元。
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