EX-99.1 2 d154261dex991.htm EX-99.1 EX-99.1 Exhibit 99.1 Wave Life Sciences Announces Positive Update on RestorAATion-2 Trial: WVE-006 (GalNAc-RNA Editing) Achieves MZ-Like Phenotype Across Both Biweekly and Monthly Dosing 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 DN…
Open exhibit ↗Current Report · Items 7.01, 8.01, 9.01 · 8-K
Wave Life Sciences, Inc.
WVENASDAQEQUITYCurrent
Regulation FD Disclosure · Other Events
Item 7.01 Regulation FD Disclosure. On May 18, 2026, Wave Life Sciences Ltd. (the “Company” or “Wave”) issued a press release announcing a positive update 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).…
Company context
Wave Life Sciences (Nasdaq: WVE) is a biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health. Wave’s PRISM® platform combines multiple modalities, chemistry innovation, and deep insights in human genetics to deliver scientific breakthroughs that treat both rare and common disorders. Its toolkit of RNA-targeting modalities, including RNAi (SpiNA) and RNA editing (AIMers), provides Wave with unmatched capabilities for designing and sustainably delivering candidates that optimally address disease biology. Wave’s pipeline is focused on its obesity (WVE-007), alpha-1 antitrypsin deficiency (WVE-006) and PNPLA3 I148M liver disease (WVE-008) programs, and also includes clinical programs in Duchenne muscular dystrophy and Huntington’s disease, as well as several preclinical programs utilizing the company’s versatile RNA medicines platform. Driven by the calling to “Reimagine Possible,” Wave is leading the charge toward a world in which human potential is no longer hindered by the burden of disease. Wave is headquartered in Cambridge, MA. For more information on Wave’s science, pipeline and people, please visit www.wavelifesciences.com and follow Wave on X and LinkedIn.