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CD-mPkd2
- Development stage
- Preclinical
- Lead developer
- University of Southern California
- Modality
- Gene Therapies, mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
CD-mPkd2 is a preclinical gene replacement therapy consisting of lipid nanoparticles (LNPs) functionalized with a collecting duct (CD)-targeting peptide and loaded with Pkd2 mRNA. Developed by researchers at the University of Southern California and Silver Spur Therapeutics, it is designed to target renal collecting duct epithelial cells, which are the primary site of cyst formation in autosomal dominant polycystic kidney disease (ADPKD). By delivering Pkd2 mRNA, CD-mPkd2 restores expression of the polycystin-2 (PC2) protein, thereby reversing established cystic disease, restoring renal architecture, and reducing the fibrotic and inflammatory microenvironment. It has also demonstrated cross-genetic applicability by attenuating disease progression in Pkd1-deficient models.
- Other names
- CD-m Pkd2CD-mPkd2 LNPCD-mPkd-2 LNPCD-mPkd 2 LNPCD-targeted LNP-mediated Pkd2 mRNAcollecting duct-targeted lipid nanoparticles delivering Pkd2 mRNA
02
Targets
PKD2 (Polycystin-2)
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