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1ST-104 is a preclinical-stage small molecule dual kinase inhibitor being developed by First Biotherapeutics for the treatment of Parkinson's disease. The compound is designed to target both Leucine-rich repeat kinase 2 (LRRK2) and c-Abl (Abelson murine leukemia viral oncogene homolog 1), two enzymes that play critical roles in the pathogenesis of neurodegenerative disorders. LRRK2 is a well-validated genetic target in Parkinson's, where its overactivity is linked to lysosomal dysfunction and protein aggregation. c-Abl is a tyrosine kinase that, when activated by oxidative stress, promotes the phosphorylation and aggregation of alpha-synuclein and inhibits the clearance of toxic proteins. By inhibiting both LRRK2 and c-Abl, 1ST-104 aims to provide a multi-pronged approach to neuroprotection and disease modification.
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