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A Phase 1b/2a Open-label Single Ascending Doses (SAD) and Multiple Ascending Doses (MAD) Research Study to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics in Participants with AATD Pi*ZZ on WVE-006 (RestorAATion-2)

Trial Profile

A Phase 1b/2a Open-label Single Ascending Doses (SAD) and Multiple Ascending Doses (MAD) Research Study to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics in Participants with AATD Pi*ZZ on WVE-006 (RestorAATion-2)

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 06 Mar 2025

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At a glance

  • Drugs WVE-006 (Primary)
  • Indications Alpha 1-antitrypsin deficiency
  • Focus Adverse reactions
  • Acronyms RestorAATion; RestorAATion 2
  • Sponsors WaVe life Sciences
  • Most Recent Events

    • 04 Mar 2025 According to a WaVe life Sciences media release, In the first quarter of 2025, company initiated multi-dosing in the first cohort of RestorAATion-2, where patients are receiving 200 mg subcutaneous doses every two weeks, and initiated the second single dose cohort of RestorAATion-2 at 400 mg
    • 16 Oct 2024 According to a WaVe life Sciences media release, company expects to share multidose data in 2025.
    • 16 Oct 2024 According to a WaVe life Sciences media release, company Announces First-Ever Therapeutic RNA Editing in Humans Achieved in this trial

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