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Children's National Medical Center Researcher Developing Proteomic Assay for Summit's DMD Drug

NEW YORK (GenomeWeb News) – Drug firm Summit and the Children's National Medical Center in Washington today announced a collaboration for the development of a proteomic assay to measure biomarkers in support of Summit's candidate drug for Duchenne Muscular Dystrophy (DMD).

Under the terms of the deal, Yetrib Hathout, a principal investigator at CNMC's Center for Genetic Medicine Research, will develop a mass spectrometry-based assay to measure levels of a protein called utrophin in DMD muscle that may prove useful in treating the disease.

DMD is caused by genetic mutations that prevent the generation of dystrophin, a structural protein, in patients. As a result, DMD patients experience progressive muscle deterioration which ultimately leads to death.

Utrophin is a protein expressed in fetal and regenerating muscle that is functionally similar to dystrophin and which may potentially restore and maintain healthy muscle function.UK-based Summit has created a program aimed at developing utrophin modulators, and its lead utrophin modulator drug is called SMT C1100.

In late 2012 the company completed a Phase 1 clinical trial for SMT C1100 that indicated the drug is safe and well-tolerated. Developing biomarkers that can quantify utrophin protein levels in DMD muscles "will play an important role in providing evidence for the potential effectiveness of Summit's utrophin modulator drugs in future patient clinical trials," Summit said in a statement.

The Foundation to Eradicate Duchenne is providing funding to support the collaboration. Terms of the deal were not disclosed.