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Wave Life Sciences plunges 21% after RNA-editing therapy trial data underwhelms
ByAinvest
Wednesday, Sep 3, 2025 8:26 am ET1min read
Wave Life Sciences dropped 21% premarket after announcing Phase 1b/2a trial data for its RNA-editing therapy WVE-006, developed with GSK, for alpha-1-antitrypsin deficiency. The therapy aims to correct the genetic mutation causing the rare genetic disorder. The company's stock price fell following the announcement.
Wave Life Sciences (NASDAQ: WVE) experienced a significant drop in its stock price, falling by 21% premarket following the announcement of positive data from the ongoing Phase 1b/2a RestorAATion-2 trial of WVE-006 for Alpha-1 Antitrypsin Deficiency (AATD). The trial demonstrated durable production of serum AAT protein at therapeutic levels following 200 mg doses, marking a significant milestone in the development of RNA editing technology for AATD.The trial achieved notable clinical milestones, including the first-ever demonstration of therapeutically restored physiological serum AAT production in a Pi*ZZ individual during an acute phase response. This breakthrough indicates that WVE-006 can dynamically generate wild-type M-AAT protein when needed, potentially reducing the risk of lung and liver diseases associated with AATD.
The data also supports monthly or less frequent subcutaneous dosing, with 400 mg monthly multidose cohort data expected in Q1 2026. The favorable safety profile, with only mild to moderate adverse events reported, further strengthens the clinical potential of WVE-006.
Wave Life Sciences, in partnership with GSK, continues to advance its pipeline of RNA editing candidates, with plans to share new preclinical data at Research Day in the fall of 2025 and to initiate clinical development of additional RNA editing programs in 2026.
The company is eligible for up to $525 million in milestones plus tiered royalties on net sales for WVE-006, further highlighting the potential commercial value of this breakthrough therapy.
Wave Life Sciences will host an investor conference call today at 8:30 a.m. ET to review the RestorAATion-2 data.
References:
[1] https://www.stocktitan.net/news/WVE/wave-life-sciences-announces-positive-update-from-ongoing-restor-aa-aji2b2yvr2vz.html
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