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MD44
- Development stage
- Preclinical
- Lead developer
- University of California, San Francisco
- Modality
- Small Molecules
01
Overview
MD44 is a first-generation small-molecule polycystin-1 (PC1) trafficking corrector under development for the treatment of autosomal dominant polycystic kidney disease (ADPKD). Discovered through a focused chemical screen at the University of California San Francisco (UCSF), MD44 restores the ciliary localization and protein maturation of folding- and trafficking-defective PKD1 variants, including PC1^R2215W, R2220W, and T3135M. This rescue is associated with enhanced glycosylation maturation and increased surface delivery of PC1, promoting productive folding and endoplasmic reticulum export to modify cyst-promoting signaling.
02
Targets
ENPP1
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