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VX-864
- Development stage
- Discontinued
- Lead developer
- Vertex
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
VX-864 is an investigational small molecule developed by Vertex Pharmaceuticals for the treatment of alpha-1 antitrypsin deficiency (AATD), a genetic disorder characterized by misfolding and polymerization of the Z variant of alpha-1 antitrypsin (AAT) protein. VX-864 acts as a corrector molecule, designed to promote proper folding of the mutant Z-AAT protein, thereby increasing plasma levels of functional AAT. Clinical studies demonstrated that VX-864 could significantly increase circulating functional AAT levels in patients with the PiZZ genotype; however, the magnitude of effect was considered insufficient for substantial clinical benefit. The development program was discontinued due to limited efficacy and non-serious rash events observed in some patients[3][6][8].
- Other names
- vx 864vx864vx-864
02
Targets
SERPINA1 Z (Alpha-1-antitrypsin Z variant protein)
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