BioMarin, a California-based biopharmaceutical company, announced today that it has licensed for development compounds that were developed with funding from the Cystic Fibrosis Foundation. The compounds, which improve the function of the key protein associated with cystic fibrosis (known as CFTR), were discovered by Alan Verkman, M.D., Ph.D., director of the Cystic Fibrosis Research Development Program at the University of California, San Francisco. With funding from Cystic Fibrosis Foundation Therapeutics, Dr. Verkman and his team developed a high-throughput screening system, allowing them to screen thousands of chemical compounds that could restore CFTR activity.
"This is one more exciting example of how a CF Foundation investment can bring promising therapies forward," said Robert J. Beall, Ph.D., President and CEO of the Cystic Fibrosis Foundation. "The CF community should be proud that their ongoing combined efforts make these kinds of investments possible."
BioMarin, which already has FDA-approval on other products, expects the lead compounds to undergo additional animal testing and optimization with the goal of filing an FDA application in 2009 that will allow initial clinical testing in humans.
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