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SIR-X56

Development stage: Preclinical
Lead developer: Sironax
Modality: Small Molecules
Administration: Oral

Overview

SIR-X56 is a novel, orally bioavailable small-molecule agonist of nicotinamide phosphoribosyltransferase (NAMPT), a rate-limiting enzyme in the nicotinamide adenine dinucleotide (NAD+) salvage pathway. By activating NAMPT, SIR-X56 increases intracellular NAD+ levels, which are essential for energy metabolism, redox homeostasis, and mitochondrial function. Preclinical studies have demonstrated that SIR-X56 restores muscle NAD+ levels and improves motor function in models of Duchenne muscular dystrophy (DMD) and aging, while also reducing cardiac injury and extending lifespan in models of Friedreich ataxia.

Targets

NAMPT

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