Study demonstrates clinical proof of mechanism with statistically significant increases in polycystin levels following RGLS4326 treatment in the first cohort of patients with ADPKD
Trends indicate further increases in polycystin levels may be achievable with longer term and, potentially, less frequent, dosing
Preclinical proof of mechanism demonstrated in relevant mouse model that harbor Pkd1 mutation equivalent to human ADPKD
miR-17 validated as a target for ADPKD treatment
PR Newswire
SAN DIEGO, June 25, 2021