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.（納斯達克：WVE）股價週三上漲，因公司公佈了WVE-006在α-1抗胰蛋白酶缺乏症（AATD）1b/2a RestorAATion-2研究中的機制證據數據。AATD是一種遺傳性疾病，導致蛋白質（AAT）水平低，該疾病可保護肺部。
The condition can cause lung and liver damage.
該疾病可能導致肺部和肝臟損傷。
WVE-006 is designed to address AATD-related lung disease, liver disease, or both.
WVE-006旨在治療AATD相關的肺部疾病、肝臟疾病或兩者兼有。
Also Read: Wave Life Sciences Stock Surges On Promising Interim Data From Mid-Stage Study Of Muscular Disorder Drug.
此外閱讀：Wave Life Sciences股價因中期肌肉疾病藥物研究中有希望的臨時數據而大漲。
The company adds that today's proof-of-mechanism data are the first-ever clinical demonstration of RNA editing in humans.
該公司補充稱，今天的機制驗證數據是人類中首次臨床展示RNA編輯的證據。
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.
這些數據來自RestorAATion-2的第一個單劑量隊列（200毫克），包括首批兩名達到第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的患者不會自然產生野生型α-1抗胰蛋白酶（m-AAT）蛋白，因此，m-AAt蛋白的存在確認了成功編輯突變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，恢復50%的m-AAt將與低風險的AATD肺部和肝臟疾病的雜合子「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.
血漿中循環的野生型m-AAt蛋白在第15天達到6.9微摩爾，佔總AAt的60%以上。
Increases in neutrophil elastase inhibition from baseline were consistent with the production of functional M-AAT.
與基線相比，中性粒細胞彈性蛋白酶抑制增加與功能m-AAt的產生一致。
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和正在進行中的健康志願者RestorAATion-1試驗中所有不良事件均爲輕度至中度，未報告嚴重不良事件。
The RestorAATion-2 trial is ongoing, and Wave expects to share multidose data in 2025.
RestorAATion-2試驗正在進行中，Wave預計於2025年分享多劑量數據。
GSK plc (NYSE:GSK) has the exclusive global license for WVE-006.
GSk plc（紐交所：GSK）擁有WVE-006的全球獨家許可。
Development and commercialization responsibilities will transfer to GSK after Wave completes the RestorAATion-2 study.
開發和商業化責任將在Wave完成RestorAATion-2研究後轉移給GSk。
Wave is eligible for up to $525 million in milestones and sales-based royalties.
Wave有資格獲得高達$52500萬的里程碑和基於銷售的特許使用費。
Price Action: WVE stock is up 74.4% at $14.93 at last check Wednesday.
股票價格走勢：最後查看星期三，WVE股價上漲74.4%，至14.93美元。
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Image created using artificial intelligence via Midjourney.
圖像由Midjourney通過人工智能創建。
