Shire And Cystic Fibrosis Foundation: Rare Disease Drug Collaboration

Shire and the Cystic Fibrosis Foundation Therapeutics Inc. (CFFT), the nonprofit drug development affiliate of the Cystic Fibrosis Foundation (CFF), announce on December 10^th, a $15 million multi-year research agreement to support Shire’s Messenger RNA (mRNA) Technology platform for Cystic Fibrosis (CF). The potential treatment is designed with the goal of “boosting lung function and decreasing the number and severity of lung infections”.

Per the CFF news release :

“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 first phase of Shire’s CF program is focusing on evaluating the dosing and safety of the therapy in the lab.

Just last month in November, Royalty Pharma announces its acquisition of royalties on Vertex Pharmaceutials’ CF treatments owned by CFFT for a cash payment of $3.3 billion. CFF plans on reinvesting the money into new research efforts and expanding patient programs. According to a Bloomberg online article :

“The transaction is part of a growing trend in philanthropy called mission investing. In mission investing, instead of giving research grants as charity, philanthropies act more like business partners and expect a share of profits stemming from their gift, according to Aaron Dorfman, executive director of the National Committee for Responsive Philanthropy.”

Please Note: “Chemlab2” by Tahoenathan (Own work) [CC-BY-SA-3.0] | via Wikimedia Commons.

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