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STL-182

Development stage: Preclinical
Lead developer: Spotlight Innovation
Modality: Small Molecules

Overview

**STL-182** is a small molecule drug developed for the treatment of **spinal muscular atrophy (SMA)**, a genetic neuromuscular disorder caused by mutations in the SMN1 gene leading to deficient survival motor neuron (SMN) protein levels. It functions by stabilizing and increasing levels of the endogenous SMN protein produced from the SMN2 gene, a homolog that partially compensates for SMN1 loss. In preclinical mouse models of SMA, STL-182 has demonstrated potential to restore neuromuscular function.[1][4][7]

Targets

SMN (Survival motor neuron protein)

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