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SEL-148,742

Development stage: Preclinical
Lead developer: Sharp Therapeutics
Modality: Small Molecules
Administration: Oral

Overview

SEL-148,742 is an orally available, brain-penetrant small molecule progranulin (PGRN) modulator being developed by Sharp Therapeutics for the treatment of neurodegenerative diseases, including familial frontotemporal dementia (fFTD), amyotrophic lateral sclerosis (ALS), and Alzheimer's disease. The drug is designed to block sortilin (SORT1)-mediated endocytosis and lysosomal degradation of progranulin, thereby increasing extracellular progranulin levels in the brain. By restoring progranulin levels, SEL-148,742 aims to alleviate neuroinflammation and promote neuronal survival and repair. It is currently in preclinical development.

Targets

GRN (Progranulin)

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