Drug intelligence / Profile preview

CC-15965

Development stage
Unknown
Lead developer
Bristol Myers Squibb
Modality
Small Molecules
Administration
Oral
01

Overview

CC-15965 is a potent, selective, and orally bioavailable small molecule inhibitor of **Leucine-Rich Repeat Kinase 2 (LRRK2)**. Developed by Celgene (now a subsidiary of Bristol Myers Squibb), it was designed for the treatment of Parkinson's disease. LRRK2 is a complex protein with both kinase and GTPase activities, and mutations in the LRRK2 gene—most notably the G2019S mutation—are a leading genetic cause of both familial and sporadic Parkinson's disease. These mutations typically result in increased kinase activity, which is associated with lysosomal dysfunction, impaired vesicular trafficking, and neurodegeneration. By inhibiting LRRK2 kinase activity, CC-15965 aims to reduce the pathological phosphorylation of downstream targets, such as Rab GTPases, thereby potentially slowing the progression of the disease and preserving neuronal function.

02

Targets

LRRK2 (Leucine-rich repeat serine/threonine-protein kinase 2)

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