BioMarin Licenses Technology From Leading Cystic Fibrosis Research Laboratory at the University of California, San...( Molecular Chaperones Target Underl...)

Molecular Chaperones Target Underlying Defect in Cystic Fibrosis; CFTR

Folding and Transport

NOVATO, Calif., Aug. 22 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) announced today that it 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 optimization, with the goal of filing an IND in 2009.

Emil Kakkis, M.D., Ph.D., Chief Medical Officer of BioMarin commented. "Dr. Verkman's laboratory has performed pioneering work in the field of CFTR modulation. The compounds licensed have been developed through a rigorous high-throughput screening process and have demonstrated a high degree of potency and specificity for the folding and activation of the abnormal CFTR protein. We look forward to accelerating the lead optimization activities in collaboration with Dr. Verkman and his colleagues so that we can take the very best of these molecules into clinical development."

"Over the last decade, considerable efforts and funding from the Cystic Fibrosis Foundation and the biotechnology industry have resulted in the development of therapeutics that have significantly extended and improved the lives of cystic fibrosis patients," said Dr. Alan Verkman, Professor of Medicine and Physiology and Director of the Cystic Fibrosis Research Development Program at the University of California, San Francisco. "Despite this progress, there is no therapeutic approved that addresses the underlying molecular defect in cystic fibrosis, and most patients succumb to respiratory failure as young adults. BioMarin has a strong track record of aggressively moving disease modifying therapeutics through clinical development, and I am very pleased that they have made a commitment to fur
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