WVE-006
Alpha-1 Antitrypsin Deficiency
Phase 1Active - Accelerating regulatory engagement
Key Facts
Indication
Alpha-1 Antitrypsin Deficiency
Phase
Phase 1
Status
Active - Accelerating regulatory engagement
Company
About WaVe Life Sciences
Wave Life Sciences is a clinical-stage biotech focused on developing stereopure oligonucleotide therapeutics via its proprietary PRISM® platform. The company has built a diversified pipeline targeting both rare genetic disorders and common diseases, with key programs in obesity (WVE-007), alpha-1 antitrypsin deficiency (WVE-006), Duchenne muscular dystrophy, and Huntington's disease. Its strategy combines validated human genetics, a multi-modal RNA-targeting approach, and stereochemistry control to aim for best-in-class efficacy and tolerability. Recent positive interim Phase 1 data in obesity and strategic moves to accelerate its RNA editing portfolio underscore its momentum.
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| Drug | Company | Phase |
|---|---|---|
| VX-407 | Vertex Pharmaceuticals | Phase 2 |