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ACY-957 boosts HBG mRNA in primate models
March 2017
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BOSTON—The 58th Annual Meeting of the American Society of Hematology saw Acetylon Pharmaceuticals Inc. present preclinical data comparing the effects of alternative dosing schedules for ACY-957, its selective HDAC1,2 inhibitor, on gamma globin (HBG) protein expression. ACY-957 was found to be well tolerated in a three-week dosing regimen, leading to greater than 50-fold increases of HBG mRNA and protein in non-anemic primates. Acetlyon is developing such inhibitors to treat sickle cell disease and beta-thalassemia. When combined with alpha globin, HBG protein forms fetal hemoglobin (HBF), which can be a substitute for defective adult hemoglobin produced in patients with sickle cell or beta-thalassemia disease. Previous work has demonstrated that ACY-957 induces HBG mRNA and HBF protein in vitro and that five sequential days of oral dosing is enough to induce potentially therapeutic levels of HBG mRNA in non-anemic primates.

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