Vertex obtains FDA clearance for ADPKD IND with VX-407, targeting specific PKD1 gene variants.
Vertex Pharmaceuticals announces FDA clearance for Investigational New Drug (IND) application of VX-407, a small molecule corrector targeting autosomal dominant polycystic kidney disease (ADPKD) in patients with specific PKD1 gene variants. ADPKD is a life-shortening genetic kidney disease affecting 250,000 people in the US and Europe; currently, no treatments address the disease's underlying biology.
March 21, 2024
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