US2022054602A1PendingUtilityA1

mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES

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Assignee: TRANSLATE BIO INCPriority: Nov 30, 2010Filed: Sep 2, 2021Published: Feb 24, 2022
Est. expiryNov 30, 2030(~4.3 yrs left)· nominal 20-yr term from priority
Inventors:Braydon Charles GuildMichael HeartleinFrank Derosa
C12N 9/2465C07K 14/505A61K 38/27C12Y 201/03003C12N 15/67A61K 38/45C12Y 302/01022A61K 38/47C12N 9/1018A61K 38/1816A61K 48/005C07K 14/61
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Claims

Abstract

Compositions for modulating the expression of a protein in a target cell comprising at least one RNA molecule which comprises at least one modification 5 conferring stability to the RNA, as well as related methods, are disclosed.

Claims

exact text as granted — not AI-modified
What is claimed is: 
     
         1 . A composition for modulating the expression of a protein in a target cell, wherein said composition comprises at least one RNA molecule and a transfer vehicle and wherein the RNA comprises at least one modification which confers stability to the RNA. 
     
     
         2 . The composition of  claim 1 , wherein the RNA molecule is selected from the group consisting of mRNA, miRNA, snRNA, and snoRNA. 
     
     
         3 . The composition of  claim 1 , wherein the RNA molecule comprises more than one modification which confers stability to the RNA molecule. 
     
     
         4 . The composition of  claim 1 , wherein the RNA molecule comprises a modification of the 5′ untranslated region of said RNA molecule. 
     
     
         5 . The composition of  claim 4 , wherein said modification comprises a partial sequence of a CMV immediate-early 1 (IE 1) gene. 
     
     
         6 . The composition of  claim 5 , wherein said partial sequence of the CMV immediate-early 1 (IEI) gene comprises SEQ ID NO: 2 or SEQ ID NO: 1. 
     
     
         7 . The composition of  claim 4 , wherein said modification comprises the inclusion of a poly A tail. 
     
     
         8 . The composition of  claim 4 , wherein said modification comprises the inclusion of a Cap 1 structure. 
     
     
         9 . The composition of  claim 1 , wherein the RNA molecule comprises a modification of the 3′ untranslated of said RNA molecule. 
     
     
         10 . The composition of  claim 9 , wherein said modification comprises the inclusion of a sequence encoding human growth hormone (hGH). 
     
     
         11 . The composition of  claim 10 , wherein said sequence encoding human growth hormone (hGH) comprises SEQ ID NO: 3. 
     
     
         12 . The composition of  claim 9 , wherein said modification comprises the inclusion of a poly A tail. 
     
     
         13 . The composition of  claim 1 , wherein the RNA encodes ornithine carbamoyl transferase. 
     
     
         14 . The composition of  claim 1 , wherein the RNA encodes alpha galactosidase. 
     
     
         15 . The composition of  claim 1 , wherein the RNA encodes erythropoietin. 
     
     
         16 . A method of treating a subject deficient in a protein, comprising administering a composition comprising an mRNA and a transfer vehicle, wherein the mRNA encodes a functional protein corresponding to the protein which is deficient in the subject, and wherein the mRNA comprises at least one modification which confers stability to the administered mRNA. 
     
     
         17 . The method  claim 16 , wherein following expression of said mRNA by a target cell a functional protein is produced. 
     
     
         18 . The method of  claim 17 , wherein said functional protein is secreted from said target cell. 
     
     
         19 . The method of  claim 16 , wherein said mRNA encodes ornithine carbamoyltransferase. 
     
     
         20 . The method of  claim 16 , wherein said mRNA encodes alpha galactosidase.

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