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BioMarin licenses cystic fibrosis technology from UCSF

US-headquartered BioMarin Pharmaceutical has licensed from the University of California, San Francisco (UCSF) intellectual property covering compounds demonstrated to improve cystic fibrosis transmembrane conductance regulator (CFTR) protein functionality.

US-headquartered BioMarin Pharmaceutical has licensed from the University of California, San Francisco (UCSF) intellectual property covering compounds demonstrated to improve cystic fibrosis transmembrane conductance regulator (CFTR) protein functionality.

BioMarin expects the lead compounds to undergo additional animal testing and optimisation, with an expected IND filing for 2009.

CFTR is a transmembrane protein that functions primarily as a chloride channel in the plasma membrane of epithelial cells. The most common mutation in cystic fibrosis (CF) causes deformity to specific protein, which leads to a build up of mucous and severe infections in the lungs.

Jean-Jacques Bienaime, BioMarin's CEO, said: "We are pleased to add this programme to our growing product development pipeline. The cystic fibrosis indication aligns well with our existing product portfolio and core competencies, as it is a well-defined and relatively large orphan disease. It has a clear clinical and regulatory path and will allow us to leverage our commercial infrastructure targeting specialists."

CF affects an estimated 30,000 patients in the US and around 40,000 patients outside the US. The median age of survival of a CF patient is 36.5 years. Datamonitor identified 15 ongoing CF-related projects most of which were in phase II clinical trials.

24th August 2007

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