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news.Cystic Fibrosis Foundation Therapeutics, the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation, announced a $15m research agreement with Shire to support the development of a new cystic fibrosis treatment targeting the underlying cause of the disease.
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The multi-year research program will evaluate a novel approach to improve the function of the defective protein associated with cystic fibrosis, called CFTR. The program is investigating a potential therapy that would be delivered to the lungs in an aerosolized form, with the goal of boosting lung function and decreasing the number and severity of lung infections in people with the disease.
In people with cystic fibrosis, mutations in the CF gene prevent the production of normal CFTR protein, causing a cascade of debilitating and life-threatening symptoms.
Shire is working to develop a unique technology to deliver normal messenger RNA for CFTR directly to the lungs, where it can be used by the body’s cellular mechanisms to produce working copies of the protein. Messenger RNA is a conveyor of genetic code critical to proper function of the body’s proteins.
If successful, the potential messenger RNA therapy could benefit all people with cystic fibrosis, regardless of an individual’s mutations.
"The Cystic Fibrosis Foundation is pursuing many exciting research avenues to speed the development of new treatments for all people with cystic fibrosis," said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. "Shire brings a promising new approach and significant research capabilities to our efforts to find a cure for this devastating disease."
The first phase of Shire’s CF research program is focused on evaluating the safety and dosing of the potential therapy in the laboratory. With operational headquarters in Lexington, Mass., Shire develops and markets specialty medicines in the fields of rare diseases, neuroscience, gastrointestinal, and internal medicine.
"As a company focused on the development of innovative medicines to treat rare diseases and other specialty conditions, Shire is pleased to enter into this research collaboration with the Foundation, which joins together leaders in industry and patient advocacy to advance pioneering research for the benefit of people with CF," said Philip J. Vickers, Ph.D., global head of research and development at Shire.
The Foundation has built a robust pipeline of potential therapies that target cystic fibrosis from every angle. Currently, the Foundation has research collaborations with leading biotechnology and pharmaceutical companies such as Pfizer, Genzyme and Vertex to discover and develop drugs to treat the basic genetic defect in CF.