Regenacy’s proprietary, isoform selective HDAC1,2 inhibitors are differentiated from pan-Class I inhibitors by superior preclinical safety and tolerability profiles, and they include both blood-brain barrier penetrant and non-penetrant preclinical candidates formulated for oral administration as tablets, with excellent pharmacokinetic properties.

Regenacy’s HDAC1,2 inhibitors have demonstrated preclinical proof-of-concept for the treatment of Acute myelogenous leukemia (AML), and Diffuse Large B-Cell Lymphoma (DL-BCL), as well as sickle cell disease, beta-thalassemia and cognitive dysfunction. HDAC1,2 inhibitors can work in combination with standard of care and emerging cancer therapies to kill cancer cells. In addition, HDAC1,2 inhibition can increase the production of fetal hemoglobin (HbF) protein in primates. Elevated levels of normal HbF can reduce disease severity and restore normal function in patients with sickle cell disease (SCD) or beta-thalassemia (bT) by replacing missing or defective adult hemoglobin. Recent studies in mouse models of Alzheimer’s Disease have shown that HDAC1,2 inhibition improved performance in spatial learning.

Additional opportunities: Cognitive dysfunction and leukemia (AML/MDS).