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EST-201
- Development stage
- Preclinical
- Lead developer
- Estuary Biotherapeutics
- Modality
- Gene Therapies
01
Overview
EST-201 is an investigational, locally delivered adeno-associated virus (AAV) gene therapy designed to treat autosomal dominant polycystic kidney disease (ADPKD) in patients with a PKD2 mutation. Developed by Estuary Biotherapeutics based on research from the Yale School of Medicine, the therapy delivers a functional, full-length copy of the PKD2 gene (which encodes the polycystin-2 protein, a calcium-permeable cation channel) directly to kidney epithelial cells. By restoring polycystin-2 expression, EST-201 aims to address the genetic root cause of ADPKD, potentially halting or reversing cyst formation and progressive kidney damage rather than merely slowing decline.
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