Wave Life Sciences Reveals RNA Editing Breakthrough, Reports First-Ever Clinical Proof-Of-Mechanism In Humans
Wave Life Sciences Reveals RNA Editing Breakthrough, Reports First-Ever Clinical Proof-Of-Mechanism In Humans
Wave Life Sciences Ltd. (NASDAQ:WVE) stock is trading higher on Wednesday after the company revealed proof-of-mechanism data from the ongoing Phase 1b/2a RestorAATion-2 study of WVE-006 in alpha-1 antitrypsin deficiency (AATD), an inherited genetic disorder that causes low levels of a protein (AAT) that protects the lungs.
Wave Life Sciences Ltd.（纳斯达克：WVE）股价周三上涨，因公司公布了WVE-006在α-1抗胰蛋白酶缺乏症（AATD）1b/2a RestorAATion-2研究中的机制证据数据。AATD是一种遗传性疾病，导致蛋白质（AAT）水平低，该疾病可保护肺部。
The condition can cause lung and liver damage.
该疾病可能导致肺部和肝脏损伤。
WVE-006 is designed to address AATD-related lung disease, liver disease, or both.
WVE-006旨在治疗AATD相关的肺部疾病、肝脏疾病或两者兼有。
Also Read: Wave Life Sciences Stock Surges On Promising Interim Data From Mid-Stage Study Of Muscular Disorder Drug.
此外阅读：Wave Life Sciences股价因中期肌肉疾病药物研究中有希望的临时数据而大涨。
The company adds that today's proof-of-mechanism data are the first-ever clinical demonstration of RNA editing in humans.
该公司补充称，今天的机制验证数据是人类中首次临床展示RNA编辑的证据。
These data are from the first single-dose cohort (200 mg) in RestorAATion-2 and include the first two patients with "ZZ" AATD (Pi*ZZ AATD) to reach day 57.
这些数据来自RestorAATion-2的第一个单剂量队列（200毫克），包括首批两名达到第57天的“ZZ”AATD（Pi*ZZ AATD）患者。
Individuals with Pi*ZZ AATD do not naturally produce wild-type alpha-1 antitrypsin (M-AAT) protein; therefore, the presence of M-AAT protein is confirmation of successful editing of mutant Z-AAT mRNA.
Pi*ZZ AATD的患者不会自然产生野生型α-1抗胰蛋白酶（m-AAT）蛋白，因此，m-AAt蛋白的存在确认了成功编辑突变Z-AAt mRNA。
Additionally, restoring 50% M-AAT would be consistent with the heterozygous "MZ" genotype with low risk of AATD lung and liver disease.
此外，恢复50%的m-AAt将与低风险的AATD肺部和肝脏疾病的杂合子“MZ”基因型一致。
Circulating wild-type M-AAT protein in plasma reached a mean of 6.9 micromolar at day 15, representing more than 60% of total AAT.
血浆中循环的野生型m-AAt蛋白在第15天达到6.9微摩尔，占总AAt的60%以上。
Increases in neutrophil elastase inhibition from baseline were consistent with the production of functional M-AAT.
与基线相比，中性粒细胞弹性蛋白酶抑制增加与功能m-AAt的产生一致。
WVE-006 has been well-tolerated and has a favorable safety profile to date. All adverse events in RestorAATion-2 and the ongoing RestorAATion-1 trial of healthy volunteers are mild to moderate, with no serious adverse events reported.
WVE-006迄今耐受性良好，安全性良好。RestorAATion-2和正在进行中的健康志愿者RestorAATion-1试验中所有不良事件均为轻度至中度，未报告严重不良事件。
The RestorAATion-2 trial is ongoing, and Wave expects to share multidose data in 2025.
RestorAATion-2试验正在进行中，Wave预计于2025年分享多剂量数据。
GSK plc (NYSE:GSK) has the exclusive global license for WVE-006.
GSk plc（纽交所：GSK）拥有WVE-006的全球独家许可。
Development and commercialization responsibilities will transfer to GSK after Wave completes the RestorAATion-2 study.
开发和商业化责任将在Wave完成RestorAATion-2研究后转移给GSk。
Wave is eligible for up to $525 million in milestones and sales-based royalties.
Wave有资格获得高达$52500万的里程碑和基于销售的特许使用费。
Price Action: WVE stock is up 74.4% at $14.93 at last check Wednesday.
股票价格走势：最后查看星期三，WVE股价上涨74.4%，至14.93美元。
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Image created using artificial intelligence via Midjourney.
图像由Midjourney通过人工智能创建。
