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MZ-301
- Development stage
- Unknown
- Lead developer
- Maze Therapeutics
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
MZ-301 is an orally bioavailable small molecule inhibitor of Apolipoprotein L1 (APOL1) currently in clinical development by Maze Therapeutics. The drug is designed to treat APOL1-mediated chronic kidney disease by potently blocking the pore-forming function of the APOL1 protein. High-risk genetic variants of APOL1 are known to cause podocyte cytotoxicity and lead to progressive renal decline; MZ-301 aims to mitigate this damage as a disease-modifying therapy. Preclinical data demonstrated that MZ-301 inhibits APOL1-dependent cytotoxicity in human podocytes and leads to a dose-dependent resolution of albuminuria in animal models of kidney disease.
02
Targets
APOL1 (Apolipoprotein L1)
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