2. Academic Validation
  3. 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.
PMID: 21456623 DOI: 10.1021/jm200135z
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.

Figures
Products