In ADPKD, mutations in the PKD1 or PKD2 gene cause changes in polycystin 1 or 2 (PC1 and PC2) expression, which in turn interferes with several intracellular signaling pathways that are e.g., involved in cell proliferation, fluid secretion, and ciliary function. The...
Preclinical studies aiming to assess kidney function traditionally include readouts such as serum creatinine and/or blood urea nitrogen (BUN). However, the use of readouts that are more clinically relevant such as glomerular filtration rate (GFR) is highly encouraged,...
Salsalate is a pro-drug dimer of salicylate belonging to the family of NSAIDs, activating AMPK through direct interactions with the drug-binding domain of the AMPK beta-1 isoform. As such, Salsalate arises as a key candidate for drug repurposing in ADPKD. To...
In this newsletter we present in-house validation data of Salsalate on disease progression in the ADPKD mouse model. One of key therapeutic strategies undertaken to provide new and efficacious treatments for ADPKD is targeting key signaling pathways that drive cystic...
A significant challenge in the ADPKD research field is demonstrating efficacy of novel compounds in preclinical research models using clinically relevant endpoints. To date, total kidney volume (TKV) is the only FDA and EMA-approved imaging biomarker that is used as a...
The lead optimisation phase is a crucial process during which you investigate and select the most promising preclinical candidate(s) to progress to efficacy testing using animal models of human diseases. Pharmacokinetic (PK) and Pharmacodynamic (PD) profiling helps...
