Compounds discovered at the University of California, San Francisco, enhance CFTR protein functionality.

BioMarin Pharmaceutical licensed intellectual property covering compounds for the treatment of cystic fibrosis from the University of California, San Francisco. “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,” informs Emil Kakkis, M.D., Ph.D., CMO.

The licensed drug candidates reportedly improve cystic fibrosis transmembrane conductance regulator (CFTR) protein functionality. CFTR is a transmembrane protein that functions primarily as a chloride channel in the plasma membrane of epithelial cells. The disease occurs due to mutations in the CFTR gene that cause  the protein to misfold and leads to mucus buildup and, ultimately, organ dysfunction and severe infections in the lungs.

BioMarin says that the compounds will undergo additional animal testing and optimization and expects to file an IND in 2009. “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,” comments Jean-Jacques Bienaime, CEO. “It has a clear clinical and regulatory path and will allow us to leverage our commercial infrastructure targeting specialists.”

BioMarin has two marketed products that are both orphan drugs. Additionally, two out of three candidates in its pipeline have also received orphan drug status. These include a Phase III agent and preclinical compound for phenylketonuria.

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