Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), the non-profit drug discovery and development affiliate of the Cystic Fibrosis Foundation (CFF), and Shire, a specialty biopharmaceutical company, have established a partnership for the development of a technology designed to improve lung function and decrease infections in patients who suffer from cystic fibrosis (CF).
The agreement between the company and the CFFT, which will donate $15 million to the initiative, is meant to accelerate the development of Shire’s Messenger RNA (mRNA) Technology platform for CF. The therapy is based on a natural material produced by living organisms to convey coded genetic information from a gene (DNA) to the ribosome, with the purpose of translating coded genetic information into protein.
“Our clinical and scientific capabilities in discovering new therapies for rare diseases are focused on new indications and therapeutic areas,” said the Global Head of Research & Development at Shire, Philip J. Vickers, Ph.D. “We have a number of significant clinical milestones anticipated over the next 18 months, and aim to accelerate delivery of therapies to patients from a highly productive internal pipeline, complemented by the acquisition of external assets and innovative collaborations.”
Among its recent R&D efforts, Shire is currently researching new therapeutics in its preclinical pipeline that they believe can deliver mRNA into the body, leading to the production of normal working copies of the protein. CF patients who have the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene mutation suffer a deregulation of the normal function of lung fluids, causing secretions to thicken.
Given the serious consequences that can be caused by thickening secretions, including restricted lung function and lung infections, Shire aims to deliver mRNA that is able to regulate the full functioning of the CFTR protein in the lungs in order to improve the symptoms of CF patients. By elevating the levels of functional CFTR protein, the mRNA Technology platform may be able to improve lung function, and consequently reduce both frequency and severity of lung infections.
“Shire’s clear and focused strategy has enabled us to transform our pipeline with 22 programs in the clinic, the most in Shire’s history,” added the Chief Executive Officer of the company, Flemming Ornskov, M.D., MPH.