Acetylon's HDAC drug fights sickle cell disease, beta thalassemia in blood cell studies


In a preclinical study in human blood cells, Acetylon Pharmaceuticals' selective histone deacetylase (HDAC) 1/2 inhibitor was able to trigger production of fetal hemoglobin, a working form of hemoglobin that is suppressed in adults. This could lead to a treatment for sickle cell disease and beta thalassemia, both diseases in need of safer and more effective treatments. The results were presented at the 54th annual meeting of the American Society of Hematology (ASH). HDACs are members of a group of enzymes that help regulate gene expression. Acetylon's lead drug, the HDAC6 inhibitor ACY-1215, is in a U.S.-based Phase I/II study for the treatment of multiple myeloma, funded by a $27 million round of venture cash raised in 2011, and a $15 million strategic investment from Celgene in 2012. Other companies working on HDAC inhibitors include MEI Pharma ($MEIP) and 4SC, and gene therapy can flip the switch to produce fetal hemoglobin. Press release | Abstract