Wave achieves first-ever therapeutic RNA editing in humans in RestorAATion-2 trial of WVE-006

Wave Life Sciences Ltd.

Single 200 mg dose in first two Pi*ZZ patients produced mean total AAT ~11 µM at day 15, with edited M-AAT >60% of total.
Wild-type M-AAT reached mean 6.9 µM at day 15, consistent with MZ genotype with low AATD risk.
WVE-006 well-tolerated; all adverse events mild/moderate, no SAEs; multidose data expected 2025.
Wave eligible for up to $525M milestones + royalties from GSK; Research Day Oct 30 to show pipeline.
AATD affects ~200,000 in US/EU; current therapy limited to weekly IV augmentation (>$1.4B sales); no approved liver disease therapy.

This headline and bullets were generated automatically by deepseek-v4-flash:cloud@v2 from the public filing. Read the source on SEC.gov before relying on any specific claim. Not investment advice. See methodology for how this pipeline works.