We continue our series in the Biologics campaign – now it is about the Leucine-rich repeat kinase 2 (LRRK2).
LRRK2 is a large, ubiquitous protein with an underexplored function. Pathogenic mutations in the gene encoding LRRK2 are the most common genetic cause of familial and sporadic Parkinson's disease (PD) cases. Now LRRK2 is a promising target for disease-modifying PD treatment because patients with LRRK2-associated PD (LRRK2-PD) compose an intimate subgroup that shares underlying pathophysiology.
You can find more about challenges in LRRK2-associated PD clinical trials here.
The latter investigations show that small-molecule LRRK2 kinase inhibitors can be neuroprotective in preclinical models of PD. LRRK2 kinase activity inhibition represents a genetically maintained, chemically tractable, and potentially disease-modifying mechanism to treat Parkinson’s disease. Here authors describe the example of the optimization of a novel series of potent, selective, CNS-penetrant ATP-competitive LRRK2 inhibitors. These drug candidates are a result of integrating CNS drug-like properties using structure-based drug design and parallel medicinal chemistry technologies.
More information about this research you can find here.
