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The HDAX HDAC6 inhibitor is a small molecule therapeutic being developed by HDAX Therapeutics in partnership with the Charcot-Marie-Tooth Research Foundation (CMTRF). This drug candidate is designed to selectively inhibit Histone Deacetylase 6 (HDAC6), a cytoplasmic enzyme that deacetylates non-histone proteins such as alpha-tubulin. In Charcot-Marie-Tooth (CMT) disease, the overactivity of HDAC6 leads to the deacetylation of microtubules, which impairs axonal transport and mitochondrial movement in peripheral nerves. By inhibiting HDAC6, the drug aims to restore microtubule stability and axonal health, potentially offering a disease-modifying treatment for various forms of CMT. The program is currently in the discovery phase.
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