Data reinforce WVE-006's potential to address both lung and liver manifestations of AATD with a durable, convenient, and safe therapy capable of recapitulating the protective MZ-like phenotype with monthly dosing 

On track for feedback from FDA on potential accelerated approval pathway mid-2026

WVE-006 generated wild-type M-AAT comprising 64% of total AAT and reduced harmful Z-AAT by 71%, with 11.9 µM total AAT in the 200 mg biweekly cohort; effects were consistent with 400 mg monthly dosing regimen, with 13.6 µM total AAT; editing was sustained at least three months following last dose

Restored dynamic AAT production with two new observations of elevated AAT during acute phase responses following mild upper respiratory infections, building on prior observation of 20.6 µM of total AAT during an acute phase response two weeks post-single 200 mg dose

RNA editing offers distinct safety advantages (reversible, no bystander edits or off-target edits) over DNA editing; WVE-006 continues to be generally safe and well tolerated with no liver toxicities

Wave to host investor webcast at 5:30 p.m. ET today

CAMBRIDGE, Mass., May 18, 2026 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (NASDAQ:WVE), a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health, today announced updated data from the ongoing RestorAATion-2 trial of WVE-006, its investigational, GalNAc-conjugated, subcutaneously delivered RNA editing oligonucleotide (AIMer) for alpha-1 antitrypsin deficiency (AATD). These data further affirm WVE-006's potential as a novel therapeutic addressing both lung and liver AATD by generating healthy, wild-type M-AAT, reducing harmful Z-AAT, and restoring the ability to dynamically produce functional AAT protein when needed.

Individuals with Pi*ZZ AATD cannot produce healthy, wild-type M-AAT, have unhealthy, mutant Z-AAT aggregates in liver, and cannot dynamically increase AAT, a protein responsible for protecting lungs against ongoing damage. Heterozygous Pi*MZ individuals have low risk of lung or liver disease and circulating M-AAT levels ranging from 57% to 71% of total (mean=64%), based on analysis of natural history study samples measured with the same assay used in RestorAATion-2.

The only approved treatment for AATD is weekly intravenous plasma-derived augmentation therapy, which only addresses lung manifestations and may leave individuals living with AATD at risk if AAT protein levels fall too low during an acute phase response. There are no approved therapies for AATD liver disease.