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Nov 10, 2011

Children's Hospital Boston and Pfizer Join Forces Against Duchenne Muscular Dystrophy

  • Children's Hospital Boston and Pfizer entered a collaboration aimed at identifying therapies for Duchenne muscular dystrophy (DMD). The one-year collaborative research agreement focuses on the work of the laboratory of Louis Kunkel, Ph.D., director of the program in genomics at Children's Hospital Boston and discoverer of the dystrophin gene that underlies the biology of DMD.

    In March 2011, Dr. Kunkel and colleagues announced the results of their efforts to screen 1,200 chemicals already approved for human use in a zebrafish model of DMD for any that might have a restorative effect on muscle tissue. Dr. Kunkel’s team found that several had the desired effect in the zebrafish model, including compounds owned by Pfizer.

    With today’s agreement, Pfizer will provide Dr. Kunkel access to select proprietary compounds as well as relevant data about these compounds. Pfizer is also committing internal resources to the project, such as medicinal chemistry.

    In collaboration with Pfizer’s Carl Morris, Ph.D., Dr. Kunkel’s laboratory will test the compounds provided by Pfizer in the DMD zebrafish model, with an eye toward identifying candidates for further preclinical development.

    “This agreement brings together the best of two worlds, namely Pfizer’s ability to develop promising new compounds and our ability to test them in physiologically relevant models,” Dr. Kunkel notes. “Here at Children’s, we have the expertise in basic science and clinical care but not the crucial chemical and development background that Pfizer has brought to the table.”

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