Is a
Patent attributes
Patent Jurisdiction
Patent Number
Patent Inventor Names
Iain Mcfadyen0
Ellalahewage Sathyajith Kumarasinghe0
Paolo Martini0
Stephen Hoge0
Vladimir Presnyak0
Kerry Benenato0
Ding An0
Date of Patent
October 29, 2024
0Patent Application Number
163026070
Date Filed
May 18, 2017
0Patent Citations
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Patent Primary Examiner
Patent abstract
The invention relates to mRNA therapy for the treatment of Alagille syndrome (ALGS), mRNAs for use in the invention, when administered in vivo, encode JAGGED 1 (JAG1), isoforms thereof functional fragments thereof, and fusion proteins comprising JAG1, mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of JAG1 expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient JAG1 activity in subjects.
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