Acetylon announces results from selective HDAC 1/2 inhibitor preclinical study on SCD and bT

Published on December 11, 2012 at 1:35 AM · No Comments

Acetylon Pharmaceuticals Inc., a leader in targeted epigenetic drug discovery and development for enhanced therapeutic outcomes, today announced results from a preclinical study of a selective histone deacetylase (HDAC) 1/2 inhibitor for the treatment of sickle cell disease (SCD) and beta-thalassemia (bT) at the 54th Annual Meeting of the American Society of Hematology (ASH), taking place in Atlanta, Georgia. The study results suggest that selective inhibition of the epigenetic regulators of gene expression, HDACs 1 and 2, could represent a refined and targeted approach for the treatment of SCD and bT through the induction of disease-corrective fetal hemoglobin (HbF) in human red blood cell progenitors.

"The data presented at ASH demonstrate the potential for selective HDAC1/2 inhibition in hemoglobinopathies, including the two most prevalent severe human genetic diseases, sickle cell disease and beta thalassemia, for which treatment options are severely limited," said Walter C. Ogier, President and Chief Executive Officer and co-founder of Acetylon. "This program adds to our broadening pipeline of targeted epigenetic drugs and is yet another example of the wide array of human disease indications for which selective HDAC inhibitors hold promise."

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