US2014206753A1PendingUtilityA1

Lipid nanoparticle compositions and methods for mrna delivery

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Assignee: GUILD BRAYDON CHARLESPriority: Jun 8, 2011Filed: Jun 8, 2012Published: Jul 24, 2014
Est. expiryJun 8, 2031(~4.9 yrs left)· nominal 20-yr term from priority
Inventors:Braydon Charles GuildFrank DerosaMichael Heartlein
A61P 7/06A61P 5/00A61P 3/00A61P 1/16A61K 48/0075A61K 38/57C12N 9/644A61K 9/0073A61K 9/1272C12N 15/52C12Y 302/01022C07K 14/8125C12N 9/2465A61K 38/4846A61K 9/0019A61K 9/5123C12Y 304/21022A61K 9/1271A61K 38/1816A61K 48/00A61K 31/713A61K 38/47C07K 14/505C12N 15/85A61K 48/0091A61K 48/0008A61K 48/0083A61K 48/005A61K 48/0041C12N 15/88A61P 43/00
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Claims

Abstract

Disclosed herein are compositions and methods for modulating the production of a protein in a target cell. The compositions and methods disclosed herein are capable of ameliorating diseases associated with protein or enzyme deficiencies.

Claims

exact text as granted — not AI-modified
1 . A composition comprising (a) at least one mRNA molecule at least a portion of which encodes a functional secreted polypeptide; and (b) a transfer vehicle comprising a lipid nanoparticle. 
     
     
         2 - 19 . (canceled) 
     
     
         20 . A method of treating a subject having a deficiency in a functional polypeptide, comprising administering a composition comprising (a) at least one mRNA at least a portion of which encodes the functional secreted polypeptide; and (b) a transfer vehicle comprising a lipid nanoparticle, wherein following administration of said composition said mRNA is expressed in a target cell to produce said functional secreted polypeptide. 
     
     
         21 - 38 . (canceled) 
     
     
         39 . A method of treating a subject having a deficiency in a functional secreted polypeptide, comprising administering a composition comprising (a) at least one mRNA at least a portion of which encodes the functional secreted polypeptide; and (b) a transfer vehicle comprising a lipid nanoparticle, wherein following administration of said composition said mRNA is translated in a target cell to produce the functional polypeptide in said target cell at at least a minimum therapeutic level more than one hour after administration. 
     
     
         40 . A method of producing a functional secreted polypeptide in a target cell, comprising administering a composition comprising (a) at least one mRNA at least a portion of which encodes the functional secreted polypeptide; and (b) a transfer vehicle comprising a lipid nanoparticle, wherein following administration of said composition said mRNA is translated in a target cell to produce a functional secreted polypeptide at at least a minimum therapeutic level more than one hour after administration.

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