Galapagos wins grant for cystic fibrosis programme

By Mike Nagle

- Last updated on GMT

Related tags: Cystic fibrosis

The search for novel drug targets for cystic fibrosis received a
boost after Dutch biotechnology company Galapagos announced the
extension of its alliance with Cystic Fibrosis Foundation
Therapeutics (CFFT), the non-profit drug discovery and development
affiliate of the Cystic Fibrosis Foundation.

The search for novel drug targets for cystic fibrosis received a boost this week when Dutch biotechnology company Galapagos announced the extension of its alliance with Cystic Fibrosis Foundation Therapeutics (CFFT), the non-profit drug discovery and development affiliate of the Cystic Fibrosis Foundation. There is still a pressing need for new treatments for cystic fibrosis. Despite nearly two decades of research since the discovery of the defective gene underlying the disease, most patients still rely on daily physiotherapy and antibiotics to manage the disease, and as yet there is no treatment that addresses the underlying pathology. BioFocus DPI, a service division of Galapagos, will receive over €800,000 in research fees through this 15 month extension to the two year collaboration that began in April 2005. Robert Beall, president and chief executive of the Cystic Fibrosis Foundation, is confident that the alliance will prove successful. He said: "BioFocus DPI's innovative target discovery platform is poised to deliver promising targets for the treatment of cystic fibrosis."​ Cystic fibrosis (CF) affects approximately 100,000 people worldwide. It is caused by a mutation in the cystic fibrosis transmembrane conductance regulator gene (CFTR) which is responsible for producing sweat, digestion juices and mucus. The disease develops when neither of the body's two copies of the gene functions properly due to a misfold in the chloride channel. The defective genes cause abnormally thick, sticky mucus to be formed. The body can't cough out the mucus and airways become blocked which leads to lung infections. Since the collaboration began, BioFocus DPI has used high throughput screening techniques to search for novel drug targets to help promote the restoration of the misfolded channel. The scientists will use the extension to validate hits from this stage using smaller scale, medium throughput assays. The additional time will also enable them to run more in depth assays that they hadn't thought possible earlier. Otto van de Stolpe, the CEO at Galapagos said: "It is particularly gratifying that our target discovery engine is being applied to the unmet medical needs addressed by patient foundations such as the Cystic Fibrosis Foundation."​ The first drug developed specifically for cystic fibrosis, Roche/Genentech's Pulmozyme (dornase alfa), was approved in the mid-1990s as a mucus thinning treatment. However, it wasn't until this year that the first drug aimed at restoring the function of the misfolded gene, VX-770, entered Phase I clinical trials. The drug was developed by Vertex Pharmaceuticals, Massachusetts.

Related topics: Preclinical Research, Preclinical

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