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VX-407

Development stage: Phase 2
Lead developer: Vertex
Modality: Small Molecules
Administration: Oral

Overview

VX-407 is a first-in-class small molecule corrector under development by Vertex Pharmaceuticals for the treatment of autosomal dominant polycystic kidney disease (ADPKD), specifically targeting patients with a subset of *PKD1* gene variants. The drug is designed to correct defective PC1 protein function in these patients, addressing the underlying genetic cause of ADPKD rather than just managing symptoms. VX-407 is administered orally and represents a precision medicine approach for this indication. As of early 2025, it is in Phase I clinical trials, with plans for further proof-of-concept studies.

Targets

ENPP1

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