Selective inhibitors of tau tubulin kinase 1 (TTBK1), an enzyme mainly expressed in CNS, may be potential drug candidates for different neurodegenerative diseases. Their ability to decrease aberrant hyperphosphorylation of TAU and TDP-43 proteins, attenuate the deleterious effects of such proteinopathies for neuronal cells. This fact is especially relevant in the field of frontotemporal dementia (FTD) therapy where both TAU and TDP-43-pathies are present in the brain of the  patients.

The present project born with the aim to provide the final proof of concept in two different animal models of a selective TTBK1 inhibitor. Data here obtained will try to support further development to clinical trials in FTD patients. Our main goal is to provide new drug candidates that can fight against FTD, a rare disease that is the second prevalent dementia all over the world.