Augustine Therapeutics, Belgium

Inhibition of an enzyme called histone deacetylase 6, or HDAC6, has been shown in mice to be protective in several types of CMT. However, early HDAC6 inhibitors tested in CMT demonstrated concerning toxicities, especially for long-term use. Augustine Therapeutics has developed a new class of HDAC6 inhibitors that avoids these toxicities and has exhibited exciting potential in a mouse model of CMT1A. This project will expand on these preliminary results, thoroughly evaluating the lead drug candidate’s toxicities, optimal dosage, and efficacy in symptom prevention and reversal in CMT1A mice. Augustine Therapeutics has two additional drug candidates available for testing if the lead candidate fails safety testing. These studies are a necessary step towards the development of a new therapeutic for the treatment of CMT1A.