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PKD1 gene therapy construct
- Development stage
- Preclinical
- Lead developer
- Sonotherapeutics
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
PKD1 gene therapy construct is a nonviral genetic medicine being developed by SonoThera for the treatment of autosomal dominant polycystic kidney disease (ADPKD). The therapy consists of an optimized DNA construct expressing a full-size, codon-optimized PKD1 protein under the control of a proximal tubular epithelial cell-specific promoter. It is designed to be delivered to renal cells using SonoThera's proprietary RIPPLE™ ultrasound-mediated gene delivery (UMGD) platform, which utilizes acoustic energy profiles and microbubbles to temporarily permeabilize cell membranes. This nonviral approach aims to restore functional polycystin-1 expression in cystic kidney cells while avoiding the payload size constraints and immunogenicity associated with viral vectors.
- Other names
- SonoThera PKD1 gene therapy constructnonviral PKD1 gene therapy construct
02
Targets
ENPP1
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