oral ELOVL1 inhibitor for ALD, CNS penetrant

toxicities observed in higher species

internal HTS and LBDD

Journal of Medicinal Chemistry

Vertex Pharmaceuticals Inc.

Chemical structure of molecule compound 27 Vertex ELOVL1 inhibitor

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…


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