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
Wave Life Sciences reported positive Phase 1b/2a data for WVE-006 in alpha-1 antitrypsin deficiency (AATD), showing robust reduction of toxic Z-AAT by 71% and restoration of wild-type M-AAT to 64% of total AAT in the biweekly dosing cohort. The monthly dosing cohort (400 mg) showed similar efficacy with 68% Z-AAT reduction and 59% M-AAT restoration, supporting a convenient subcutaneous dosing regimen. Editing effects were sustained for at least three months after the last dose. The data demonstrates restored dynamic AAT production during acute phase responses, a key unmet need in AATD.
Key figures
- Drug Name
- WVE-006
- Phase Of Trial
- Phase 1b/2a
- Total aat monthly
- 13.6 µM
- Total aat biweekly
- 11.9 µM
- Acute phase aat spike
- 20.6 µM
- Z aat reduction monthly
- 67.7%
- M aat percentage monthly
- 58.7%
- Z aat reduction biweekly
- 70.5%
- M aat percentage biweekly
- 64.4%
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