Discovery of a novel warhead against beta-secretase through fragment-based lead generation - PubMed (original) (raw)

. 2007 Nov 29;50(24):5903-11.

doi: 10.1021/jm070825k. Epub 2007 Nov 7.

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Discovery of a novel warhead against beta-secretase through fragment-based lead generation

Stefan Geschwindner et al. J Med Chem. 2007.

Abstract

Fragment-based lead generation was applied to find novel small-molecule inhibitors of beta-secretase (BACE-1), a key target for the treatment of Alzheimer's disease. Fragment hits coming from a 1D NMR screen were characterized by BIAcore, and the most promising compounds were soaked into protein crystals to help the rational design of more potent hit analogues. Problems arising due to our inability to grow BACE-1 crystals at the biologically relevant pH at which the screen was run were overcome by using endothiapepsin as a surrogate aspartyl protease. Among others, we identified 6-substituted isocytosines as a novel warhead against BACE-1, and the accompanying paper in this journal describes how these were optimized to a lead series of nanomolar inhibitors.1.

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