1. Academic Validation
  2. Discovery of 2-arylbenzoxazoles as upregulators of utrophin production for the treatment of Duchenne muscular dystrophy

Discovery of 2-arylbenzoxazoles as upregulators of utrophin production for the treatment of Duchenne muscular dystrophy

  • J Med Chem. 2011 May 12;54(9):3241-50. doi: 10.1021/jm200135z.
Daniel R Chancellor 1 Kay E Davies Olivier De Moor Colin R Dorgan Peter D Johnson Adam G Lambert Daniel Lawrence Cristina Lecci Carole Maillol Penny J Middleton Gary Nugent Séverine D Poignant Allyson C Potter Paul D Price Richard J Pye Richard Storer Jonathon M Tinsley Renate van Well Richard Vickers Julia Vile Fraser J Wilkes Francis X Wilson Stephen P Wren Graham M Wynne
Affiliations

Affiliation

  • 1 Summit plc , 91 Milton Park, Abingdon, Oxfordshire, OX14 4RY, United Kingdom.
Abstract

A series of novel 2-arylbenzoxazoles that upregulate the production of utrophin in murine H2K cells, as assessed using a luciferase reporter linked assay, have been identified. This compound class appears to hold considerable promise as a potential treatment for Duchenne muscular dystrophy. Following the delineation of structure-activity relationships in the series, a number of potent upregulators were identified, and preliminary ADME evaluation is described. These studies have resulted in the identification of 1, a compound that has been progressed to clinical trials.

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