Central Acting HDAC6i

Central Acting HDAC6i is a brain-penetrant small molecule inhibitor of histone deacetylase 6 (HDAC6) currently in the lead optimization phase by Augustine Therapeutics. It is being developed for the treatment of neurodegenerative diseases, specifically Amyotrophic Lateral Sclerosis (ALS) and Alzheimer's Disease. Unlike the company's lead candidate AGT-100216, which is peripherally restricted, this program is specifically engineered to cross the blood-brain barrier to target HDAC6 within the central nervous system. HDAC6 is a cytoplasmic enzyme that regulates critical cellular processes such as axonal transport and protein clearance, both of which are frequently impaired in neurodegeneration. The therapeutic approach utilizes a novel non-hydroxamate, non-hydrazide chemotype designed for high selectivity and suitability for chronic administration. In 2023, the program received a $300,000 grant from the ALS Association to support its preclinical development.

Other names

CNS-penetrant HDAC6 inhibitorbrain-penetrant HDAC6 inhibitorAugustine Therapeutics CNS-penetrant HDAC6 inhibitor