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Rumi Scientific PKD program
- Development stage
- Preclinical
- Lead developer
- Rumi Scientific
- Modality
- Small Molecules
01
Overview
Rumi Scientific's PKD program is a discovery-stage initiative focused on developing small molecule therapeutics for Polycystic Kidney Disease (PKD), including Autosomal Dominant Polycystic Kidney Disease (ADPKD). The program utilizes the company's proprietary Nephroid platform—a human stem cell-derived kidney organoid system—to identify compounds that modulate biological pathways associated with PKD1 and PKD2 mutations. These small molecules are designed to reduce the formation and expansion of renal cysts, aiming to slow disease progression and preserve kidney function.
- Other names
- PKD1/PKD2 small molecule program
02
Targets
ENPP1PKD2 (Polycystin-2)
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