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Sep. 18, 2021

Compound 19: A Brain Penetrant UGT8 Galactosyltransferase Inhibitor

"Compound 19"

brain pen. UGT8 galactosyltransferase inh. excellent oral PK/PD in brain model from 30k cmpd cellular MTS and optimization J. Med. Chem., Jun. 26, 2020 Sanofi R&D, Waltham, MA

Drug Hunter Team

“Compound 19” is a selective brain-penetrant inhibitor of the ceramide galactosyltransferase UGT8, intended for use in substrate reduction therapy for rare lysosomal storage disorders. The compound has good oral exposure in higher species (%F = 52 in dog) and is active in models looking at enzymatic activity using isotopically-labeled [...]



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