A pipeline of next-generation brain-targeting therapies
Our pipeline
Abbreviations: FIH, first in human; FKBP12, FK506-binding protein 12; GLP, good laboratory practice; IND, investigational new drug; LRRK2, leucine-rich repeat kinase 2.
About Parkinson’s Disease and LRRK2
Parkinson’s disease (PD) is the second most common neurodegenerative disorder among the elderly. While most PD cases occur sporadically, observation of inherited mutations revealed potential molecular pathways underlying neuronal degeneration, offering insights for developing treatments that could change the course of the disease. For example, genome-wide association studies have identified a group of associated risk loci, including the gene encoding leucine-rich repeat kinase 2 (LRRK2). Mutations in the LRRK2 gene have been recognized as genetic risk factors for both familial and sporadic forms of PD, and aberrant kinase activity of LRRK2 mutants has been associated with pathological features of PD. Clinical studies with LRRK2 inhibitors from unique chemical scaffolds have yielded similar safety findings, including lung abnormalities, which suggest on-target effects in the periphery. As a result, there is growing interest in identifying brain-selective LRRK2 inhibitors.
