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Dec. 20, 2021

 Compound 27: a Potent CNS-Penetrant ELOVL1 Inhibitor

"compound 27"

oral ELOVL1 inhibitor for ALD, CNS penetrant toxicities observed in higher species internal HTS and LBDD Journal of Medicinal Chemistry Vertex Pharmaceuticals Inc.

Drug Hunter Team

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 be pathological in the rare disease adrenoleukodystrophy (ALD). The starting point came from an HTS on compounds from an internal collection. The molecule demonstrated [...]



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