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DUN'040

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

Overview

DUN'040 is a first-in-class, brain-penetrant, covalent small molecule inhibitor of the lipid kinase PIKfyve. Developed by Dunad Therapeutics, it is currently in IND-enabling studies for the treatment of amyotrophic lateral sclerosis (ALS), including both sporadic and familial forms. The compound is designed to address TDP-43 proteinopathy, a pathological hallmark found in the majority of ALS patients, by modulating the endolysosomal system. By inhibiting PIKfyve, DUN'040 aims to enhance the clearance of toxic protein aggregates and restore cellular homeostasis in motor neurons.

Other names: DUN'040

Targets

PIKFYVE (Phosphoinositide kinase, FYVE-type zinc finger containing enzyme)

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