Translate Bio Announces Publication of Preclinical Data Demonstrating Efficacy of Systemic mRNA Delivery in Fabry Disease Model in the Journal Molecular Therapy
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 α-galactosidase (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
“This research demonstrates the ability of our mRNA, delivered via LNP, to target the liver directly, resulting in the potential for an improved therapeutic profile compared with conventional treatment for this disease,” said
Heartlein continued, “The sustained expression of the GLA enzyme that we observed further supports our belief that the liver is a factory for the production of protein and the prolonged expression of that protein.”
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 (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.
About Fabry Disease
Fabry disease is a rare inherited lysosomal storage disorder caused by deficiency of α-galactosidase A, an enzyme needed to break down a fatty substance in the body called globotriaosylceramide. When this substance accumulates in the body’s cells, the resulting cell damage can cause a range of mild to severe symptoms including life-threatening complications such as kidney failure, heart attacks and strokes, often at a young age. Fabry disease occurs in all racial and ethnic populations, though males are typically more severely affected than females.
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include, but are not limited to, those regarding: the ability of Translate Bio’s mRNA to target the liver directly; the potential benefits of mRNA therapy, including in the treatment of Fabry disease; Translate Bio’s beliefs regarding the liver’s ability to produce the GLA protein; Translate Bio’s beliefs regarding the broad applicability of its MRT platform; and Translate Bio’s plans, strategies and prospects for its business, including its lead development programs. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from current expectations and beliefs, including but not limited to: Translate Bio’s ability to advance the development of its platform and programs under the timelines it projects, demonstrate the requisite safety and efficacy of its product candidates and replicate in clinical trials any positive findings from preclinical studies; the content and timing of decisions made by the
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Source: Translate Bio, Inc.