Translate Bio to Present Preclinical Data at the 41st Annual European Cystic Fibrosis Conference
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.
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.
Source: Translate Bio