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”
“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…