oral ELOVL1 inhibitor for ALD, CNS penetrant
toxicities observed in higher species
internal HTS and LBDD
Journal of Medicinal Chemistry
Vertex Pharmaceuticals Inc.
8. The Vertex ELOVL1 inhibitor, compound 27, is a potent (13 nM) CNS-penetrant compound that reduces very long chain fatty acid (VLCFA) levels in the CNS, which are believed to…