Drug intelligence / Profile preview

CTD-09

Development stage
Preclinical
Lead developer
Cureteq
Modality
Small Molecules
Administration
Oral
01

Overview

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.

Other names
valproic acidsodium valproate
02

Targets

HDAC9HDAC1 (Histone Deacetylase 1)HDAC8 (Histone Deacetylase 8)HDAC7HDAC4HDAC5 (Histone deacetylase 5)

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