Polycystic kidney disease (PKD) is a genetic disorder with a prevalence up to 1:3000 in which fluid filled cysts develop within the kidneys, causing the kidneys to enlarge and loose function over time. PKD can cause serious complications due to the disturbed urinary clearance, including high blood pressure and ultimately kidney failure.
PKD patients experience a steady decline in renal function throughout their lives, which forms a serious threat that also accounts for a significant amount of end stage renal failure. Medical costs and discomfort for the patient associated with PKD are significant. Interventions that can slow the progression of the disease have the potential to lead to substantial reductions in costs for the treatment of PKD and will improve the quality of life.
For this purpose InnoSer is involved in a scientific consortium that focuses on a proof-of-concept to deliver novel treatments for PKD. In this consortium, InnoSer performs key translational studies by investigating therapeutic efficacy of lead compounds in unique screening platform for PKD. These platforms include PKD1 -/- mouse cells in a 3D environment and cysts cultured from primary human PKD tissue, enabling the recapitulation of the pre-clinical disease phenotype and allows therapeutic efficacy testing of selected lead compounds. Finally, successful lead compounds will be tested in unique PKD animal models to validate their therapeutic efficacy in vivo.