Shire has organized a Research and Development (R&D) Day specially designed to present the company’s latest medications for the treatment of rare diseases and other conditions, including cystic fibrosis (CF), to the investment community. Among the therapies discussed, the company presented ones that are included in their recently announced partnership with the Cystic Fibrosis Foundation Therapeutics Inc. (CFFT).
The non-profit drug discovery and development affiliate of the Cystic Fibrosis Foundation (CFF), the CFFT, recently established a collaboration with Shire, and granted the company up to $15 million to support the development of the company’s Messenger RNA (mRNA) Technology platform for CF. The natural material fabricated by living organisms, mRNA, transports coded genetic data from a gene to the ribosome, which in turn translates it into protein.
Several conditions are related to low levels of functional protein. Therefore, Shire is studying therapeutic alternatives that are able to release mRNA to the places in the body where it can be used by the cellular mechanisms in order to create functional protein copies.
CF patients suffer a mutation in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene, which causes a dysfunction of the normal conduction of lung fluids, provoking thick secretions, lung dysfunction and infections. For the treatment of CF, Shire aims to place mRNA able to code a fully functional (wild type) version of the CFTR protein to the lungs, in order to elevate the production levels of functional CFTR protein and consequently improve lung function and reduce the frequency and severity of infections.
“Our clinical and scientific capabilities in discovering new therapies for rare diseases are focused on new indications and therapeutic areas,” explained Philip J. Vickers, Ph.D., Global Head of Research & Development. “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.”
In addition, the partnership between CFFT and Shire also includes SHP607, which a potential replacement therapy currently being studied for the prevention of Retinopathy of Prematurity (ROP), and that has recently received the Fast Track designation from the U.S. Food and Drug Administration (FDA).
“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,” said the Chief Executive Officer of the company, Flemming Ornskov, M.D., MPH. The lectures presented during the R&D Day were broadcasted, and archives of the event can still be viewed on the company’s website here.