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VX-634
- Development stage
- Phase 1
- Lead developer
- Vertex
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
VX-634 is an investigational small molecule developed by Vertex Pharmaceuticals for the treatment of alpha-1 antitrypsin deficiency (AATD). It acts as a corrector of the Z-AAT protein, which is misfolded due to mutations in the SERPINA1 gene. By promoting proper folding of this protein, VX-634 aims to restore functional alpha-1 antitrypsin levels and reduce toxic polymer accumulation in patients with AATD. The drug was evaluated in Phase 1 clinical trials for safety and pharmacokinetics but was discontinued after Vertex determined that its efficacy was not transformative enough for further development[1][2][3][4][6][8].
02
Targets
SERPINA1 (Alpha-1-antitrypsin)
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