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Positive Update from Ongoing RestorAATion-2 Trial of WVE-006 in Alpha-1 Antitrypsin Deficiency

Wave Life Sciences Ltd. (WVE) | September 3, 2025

By Xander Turner

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Wave Life Sciences announced positive data from the ongoing RestorAATion-2 trial of WVE-006 in Alpha-1 Antitrypsin Deficiency. The trial showed durable production of serum AAT protein at levels associated with lower risk of AATD liver and lung diseases.

The data from 200 mg and 400 mg cohorts support monthly or less frequent subcutaneous dosing of WVE-006. The ongoing 400 mg multidose cohort is expected to deliver further increases in serum AAT.

Wave Life Sciences continues to demonstrate the well-tolerance and favorable safety profile of WVE-006 in patients with AATD.

Therapeutic Protein Production

Achieved durable production of serum AAT protein at levels associated with lower risk of AATD liver and lung diseases.

RNA Editing Success

Demonstrated the first-ever therapeutically restored physiological serum AAT production in a Pi*ZZ individual, crucial for AATD treatment.

Potential Dosage Regimen

Data supports monthly or less frequent subcutaneous dosing, with ongoing 400 mg multidose cohort expected to provide further benefits.

Safety Profile

WVE-006 continues to be well tolerated with a favorable safety profile in patients, indicating its potential as a transformative treatment option for AATD.

  • The positive data from the trial demonstrate significant clinical progress for individuals with AATD and highlight the potential of WVE-006 to provide a transformative treatment option.
  • The ability of WVE-006 to restore expression of AAT under physiological control and drive production of protective M-AAT protein is a major advancement in AATD treatment.

Wave Life Sciences has achieved promising results in the ongoing RestorAATion-2 trial, showing the effectiveness and potential of WVE-006 in treating Alpha-1 Antitrypsin Deficiency. The data support the continued development of WVE-006 as a transformative treatment option for individuals with AATD.