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

“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…


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