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Mitometin

Development stage: Preclinical
Lead developer: 2N Pharma
Modality: Small Molecules
Administration: Oral

Overview

Mitometin is an orally administered, small molecule drug candidate developed by 2N Pharma. It acts as a reversible inhibitor of carnitine palmitoyl transferase 1 (CPT1), a key enzyme involved in beta-oxidation by facilitating the transport of fatty acids across the mitochondrial membrane. The drug is being developed to restore metabolic balance in affected mitochondria and is under investigation for its potential use in treating amyotrophic lateral sclerosis (ALS)[1][2].

Targets

SLC7A1 (Cationic amino acid transporter 1)

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