Translate Bio announced the publication of preclinical results demonstrating efficacy in a mouse model of Fabry disease using a lipid nanoparticle, or LNP, formulation for systemic delivery of mRNA derived from the company’s mRNA therapeutic, or 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, reported the sustained expression of human a-galactosidase, or GLA, protein via LNP-formulated mRNA in mice and non-human primates. The results also demonstrate the efficacy of this approach through reduction of a clinically relevant biomarker in a mouse model of Fabry disease. The article was published online and will appear in the April 10 issue of the journal. In the study, multi-component lipid nanoparticles were formulated for delivery of mRNA encoding human GLA protein. Upon delivery of human GLA mRNA to mice, serum GLA protein levels reached as high as 1,330-fold over normal physiological values. Additionally, treatment with mRNA therapy was compared with conventional enzyme replacement therapy, or ERT, in the mouse model of Fabry disease. Results demonstrated increased protein levels in the key organs affected by the disease with mRNA therapy compared to ERT. These higher protein levels translated into a greater reduction in two clinically relevant biomarkers using mRNA therapy.
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