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（納斯達克股票代碼：VERV）宣佈了 VERVE-101 Heart-1 1b 期試驗的最新情況。
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.
正在對患有導致高膽固醇水平的遺傳病患者進行 VERVE-101 評估，這會增加患心臟病的風險。該治療本應永久關閉一種叫做 PCSK9 的蛋白質，它有助於降低血液中的膽固醇水平。
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).
該公司表示，在接受0.45mg/kg劑量的 VERVE-101 治療的六名患者中，有一例出現了特定肝酶的3級升高和3級血小板減少症（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.
參與者沒有出現任何與實驗室異常相關的出血或其他症狀，這些症狀在幾天內完全消退。
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.
經與該研究的獨立數據和安全監測委員會（DSMB）協商，Verve決定暫停Heart-1臨床試驗的註冊。
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 正在調查實驗室異常情況，並預計將與監管機構合作，根據這些結果確定 VERVE-101 的前進方向。
Verve is now prioritizing the development of VERVE-102 and the initiation of the Heart-2 clinical trial.
Verve 現在優先開發 VERVE-102 和啓動 Heart-2 臨床試驗。
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 對 PCSK9 使用相同的鹼基編輯器和指南 RNA，但脂質納米顆粒 (LNP) 輸送系統與 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已獲得英國和加拿大的Heart-2臨床試驗的監管許可。它計劃在2024年第二季度啓動針對HefH或早產冠心病患者的Heart-2臨床試驗。
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 是一種新型的研究性基因編輯藥物，旨在作爲一種單療程療法，永久關閉肝臟中的 PCSK9 基因，從而降低導致疾病的低密度脂蛋白膽固醇。
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 治療公司（納斯達克股票代碼：BEAM） 已輸入 於2019年4月簽訂合作和許可協議，根據該協議，Verve獲得了Beam針對某些心血管靶標的人體治療應用的基礎編輯、基因編輯和交付技術的獨家使用權。
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宣佈擴大與禮來公司（紐約證券交易所代碼：LLY）的關係，後者已從Beam Therapeutics手中收購了Verve針對 PCSK9 和 ANGPTL3 的心血管活體基因編輯計劃的某些產品版權，以及第三個未公開的心血管疾病靶標。
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.
價格走勢：在週二的最後一次檢查中，VERV股價下跌36.0％，至8.18美元，BEAM股價下跌7.37％，至30.29美元。
Photo via Wikimedia Commons
照片來自維基共享資源