— MRT5201 was efficiently delivered to the liver of a mouse model of OTC deficiency —
— Preclinical data demonstrated a single IV dose of MRT5201 was sufficient to protect against hyperammonemia for up to 4 weeks —
Translate Bio, Inc. (Nasdaq: TBIO), a clinical-stage messenger RNA (mRNA) therapeutics company developing a new class of potentially transformative medicines to treat diseases caused by protein or gene dysfunction, today announced the presentation of preclinical data for MRT5201, an mRNA therapeutic designed to treat patients with ornithine transcarbamylase (OTC) deficiency, the most common urea cycle disorder. The poster presentation, entitled “Treatment of Ornithine Transcarbamylase Deficiency with a Targeted mRNA Therapeutic (MRT),” includes data demonstrating that a functional OTC mRNA encapsulated in a lipid nanoparticle (LNP) was successfully delivered to the liver in a preclinical mouse model of OTC deficiency via intravenous (IV) administration. These data were featured in a poster presentation at the Society for Inherited Metabolic Disorders (SIMD) Annual Meeting on April 7, 2019 in Bellevue, Washington.
“MRT5201 is designed to treat patients with OTC deficiency by intravenous delivery of mRNA encoding fully functional OTC enzyme to the liver to enable hepatocytes to produce the normal OTC enzyme,” said Dr. Ann Barbier, chief medical officer of Translate Bio. “These data demonstrate MRT5201’s ability to prevent hyperammonemia in a relevant disease model and support further study of MRT5201 as a possible treatment for OTC deficiency.”
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