Please login to the form below

Not currently logged in
Email:
Password:

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

Share

Featured jobs

Subscribe to our email news alerts

PMHub

Add my company
Videum Health

Videum Health is a premium video platform that offers brands innovative engagement strategies to reach targeted healthcare audiences on a...

Latest intelligence

patients
Retaining reader value in plain language summaries of clinical studies
Balancing the risk of misinterpretation with the public’s ability to understand simplified plain-language summaries...
Can we talk about the ego-bias and chemicals influencing your target audience’s behaviour?
Over the Summer, the Page & Page team became fascinated by two books on this very subject. Two books from one author, Dean Burnett, an eminent neuroscientist, lecturing at Cardiff...
Paris
Making Europe a leader in bioscience: boosting trust and opening minds
A vision of Paris as Europe's leading hub for life sciences innovation...

Infographics