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Translate Bio Announces Publication of Preclinical Data Demonstrating Efficacy of Systemic mRNA Delivery in Fabry Disease Model in the Journal Molecular Therapy

Telesis Bio, Inc. (TBIO) 
Company Research Source: GlobeNewswire
LEXINGTON, Mass., March 18, 2019 (GLOBE NEWSWIRE) -- Translate Bio (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 publication of preclinical results demonstrating efficacy in a mouse model of Fabry disease using a lipid nanoparticle (LNP) formulation for systemic delivery of mRNA derived from the Company’s mRNA therapeutic (MRT) platform. Fabry disease is a rare inherited disorder caused by a deficiency of a-galactosidase A. The disease is progressive, destructive and potentially life-threatening. The paper, published in the journal Molecular Therapy and titled Improved Efficacy in a Fabry Disease Model Using a Systemic mRNA Liver Depot System as Compared to Enzyme Replacement Therapy, reported the sustained expression of human a-galactosidase (GLA) protein via LNP-formulated mRNA in mice and non-human primates. The Show less Read more
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