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MRNA therapy for Fabry disease

  • US 9,308,281 B2
  • Filed: 06/18/2014
  • Issued: 04/12/2016
  • Est. Priority Date: 06/08/2011
  • Status: Active Grant
First Claim
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1. A method of treating Fabry disease, comprising administering to a human subject in need of treatment an mRNA encoding a human alpha-galactosidase (hGLA) protein such that the hGLA protein is secreted extracellularly after expression and systemically distributed, and such that serum hGLA protein level is increased for at least 72 hours as compared to the baseline serum hGLA level before treatment,wherein the mRNA is administered via intravenous or pulmonary administration, andwherein the Fabry disease is treated.

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