Cystic Fibrosis Foundation, Shire Partner On Cystic Fibrosis Treatments
By Cyndi Root
The Cystic Fibrosis Foundation has partnered with Shire on new treatments for cystic fibrosis (CF). The Foundation announced the collaboration in a press release, stating that its non-profit drug discovery and development affiliate, the Cystic Fibrosis Foundation Therapeutics (CFFT), is participating in the $15 million research agreement. The focus of investigations is the CFTR (Cystic Fibrosis Transmembrane Conductance Regulator), a defective protein, and an aerosol lung therapy.
Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation, said, "The Cystic Fibrosis Foundation is pursuing many exciting research avenues to speed the development of new treatments for all people with cystic fibrosis. Shire brings a promising new approach and significant research capabilities to our efforts to find a cure for this devastating disease."
Cystic Fibrosis Foundation and Shire Agreement
The Cystic Fibrosis Foundation is working with other partners on its Messenger RNA (mRNA) platform and other therapies for cystic fibrosis. Current partners include Pfizer, Genzyme, and Vertex. In Shire’s announcement of the deal with CFFT, it stated that the CFFT committed to up to $15 million to support the multi-year research program. Investigators intend to use Shire’s MRNA technology platform in research efforts. A therapy of interest is an aerosol delivered to the lungs, aiding lung function and limiting the severity of lung infections in people with cystic fibrosis.
The CFFT states that Shire will proceed with the research strategy in phases, the first of which is safety and dosing. Those studies will be conducted at Shire’s operational headquarters in Lexington, MA, and the partners intend to include other leaders in industry and patient advocacy groups in the research effort.
Shire’s mRNA Platform
In CF, CFTR gene mutations disrupt fluid regulation and cause thickened secretions and deficits in lung function. Shire’s mRNA technology is a method and a product, wherein mRNA is delivered to sites in the body so the cellular mechanisms can produce working copies of defective proteins. Restoring sufficient levels of functional proteins can address the underlying causes of disease in cystic fibrosis and other diseases, according to Shire.
The technology shows promise, according to other pharmaceutical companies and experts. The MIT Technology Review reports that Messenger RNA molecules could become the “next big class of biopharmaceutical drug.” AstraZeneca has already made the investment, paying Moderna Therapeutics $240 million for rights to its mRNA technology.