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CTD-09 is a pharmaceutical formulation of valproic acid (VPA) developed by CureTeq for the treatment of Spinal Muscular Atrophy (SMA). As a broad-spectrum histone deacetylase (HDAC) inhibitor, valproic acid modulates gene expression by increasing the acetylation of histone proteins, which leads to a more relaxed chromatin structure and enhanced transcriptional activity. In SMA patients, CTD-09 is intended to upregulate the expression of the *SMN2* gene, which serves as a backup for the defective or missing *SMN1* gene, thereby increasing the levels of functional Survival Motor Neuron (SMN) protein. This mechanism aims to mitigate the progressive loss of lower motor neurons and subsequent muscle atrophy characteristic of the disease. CTD-09 is part of CureTeq's 'Value Added Medicines' pipeline, which focuses on optimizing established molecules for new therapeutic indications or improved patient outcomes.
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