BioCentury
ARTICLE | Distillery Therapeutics

Renal

February 28, 2017 11:51 PM UTC

Studies in patient samples and mice suggest inhibiting miR-17 could help treat autosomal dominant polycystic kidney disease (ADPKD). In patient kidney samples, levels of miR-17 were higher than in samples from healthy volunteers. In three mouse models of ADPKD, kidney-specific knockout of miR-17 decreased proliferation of cyst epithelial cells and increased kidney function and survival compared with normal miR-17 expression and in a fourth mouse model, kidney-specific knockout of miR-17 decreased renal fibrosis. In one of the models, an anti-miR-17 oligonucleotide decreased proliferation of cyst epithelial cells and increased kidney function compared with vehicle. In patient-derived primary cyst epithelial cells, the anti-miR-17 oligonucleotide decreased proliferation and cyst growth compared with control oligos. Next steps by Regulus Therapeutics Inc. include testing the safety and efficacy of additional undisclosed anti-miR-17 compounds in mouse models of PKD.

Regulus Therapeutics Inc. has the anti-miR-17 compound RGLS4326 in preclinical testing to treat PKD...