LEXINGTON, Mass.--(BUSINESS WIRE)--Translate Bio, a leading messenger RNA (mRNA) therapeutics company developing a new class of potentially transformative medicines to treat diseases caused by protein or gene dysfunction, today announced an oral presentation at the 41st Annual European Cystic Fibrosis Conference taking place in Belgrade, Serbia, from June 7-10, 2018.
Presentation Title: Development of a CFTR mRNA therapy capable
of treating lung disease in all patients with cystic fibrosis
Date
and Time: June 7, 2018 at 5:00 p.m. CET
Session title: Pharmacology
and genetic tools for CF basic research correction
Presenting
Author: Ann Barbier, MD, PhD, Chief Medical Officer
Abstract
number: WS09.1
The full abstract can be found at https://www.ecfs.eu/belgrade2018.
About MRT5005
MRT5005 is the first clinical-stage mRNA
product candidate designed to address the underlying cause of CF by
delivering mRNA encoding fully functional cystic fibrosis transmembrane
conductance regulator (CFTR) protein to the lung epithelial cells
through nebulization. MRT5005 is being developed to treat all patients
with CF, regardless of the underlying genetic mutation. In 2015, the FDA
granted orphan drug designation to MRT5005 for the treatment of CF.
About Cystic Fibrosis
Cystic fibrosis is the most common
fatal inherited disease in the United States, affecting more than 30,000
patients in the U.S. and more than 70,000 patients worldwide. CF is
caused by genetic mutations that result in dysfunctional or absent CFTR
protein. This defect causes mucus buildup in the lungs, pancreas and
other organs. Mortality is primarily driven by a progressive decline in
lung function. According to the Cystic Fibrosis Foundation, the median
age at death for patients with CF was 29.6 years in 2016. There is no
cure for CF. CFTR modulators that are currently marketed or in clinical
development are effective only in patients with specific mutations, and
patients still experience pulmonary exacerbations and a progressive
decline in lung function, which represents a significant unmet need.
About Translate Bio
Translate Bio is a leading mRNA
therapeutics company developing a new class of potentially
transformative medicines to treat diseases caused by protein or gene
dysfunction. The Company’s MRT platform is designed to develop product
candidates that deliver mRNA carrying instructions to produce
intracellular, transmembrane and secreted proteins for therapeutic
benefit. The Company believes that its MRT platform is applicable to a
broad range of diseases caused by insufficient protein production or
where production of proteins can modify disease, including diseases that
affect the lung, liver, eye, central nervous system, lymphatic system
and circulatory system. The Company’s two lead programs are being
developed as treatments for CF and ornithine transcarbamylase (OTC)
deficiency. For more information about the Company, please visit www.translate.bio
or on Twitter at @TranslateBio.