US2016130567A1PendingUtilityA1

Messenger una molecules and uses thereof

43
Assignee: ARCTURUS THERAPEUTICS INCPriority: Nov 2, 2014Filed: Nov 1, 2015Published: May 12, 2016
Est. expiryNov 2, 2034(~8.3 yrs left)· nominal 20-yr term from priority
Inventors:Padmanabh ChivukulaLuigi WarrenKiyoshi TachikawaJoseph E. Payne
A61P 43/00A61P 37/04A61P 35/00A61P 1/16C07K 14/805C07K 14/4712C12Y 304/21022C07K 14/8125C12N 9/0071C12N 9/1018C07K 14/4717C12Y 207/0104C07K 14/505C12Y 302/01033C12N 9/88C12N 9/2451C07K 14/445C12N 9/1205C12N 9/644C07K 14/745C12N 2840/60C07K 14/47A61K 48/00C12Y 201/03003C07K 14/705C07K 14/575C07K 14/524C12Y 204/01025C12N 9/1051C07K 14/463C12N 2840/105C12Y 114/16001C12Y 403/02001C12N 2740/16222C12N 9/2402C07K 14/005A61K 39/00
43
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Claims

Abstract

This invention provides a range of translatable messenger UNA (mUNA) molecules. The mUNA molecules can be translated in vitro and in vivo to provide an active polypeptide or protein, or to provide an immunization agent or vaccine component. The mUNA molecules can be used as an active agent to express an active polypeptide or protein in cells or subjects. Among other things, the mUNA molecules are useful in methods for treating rare diseases.

Claims

exact text as granted — not AI-modified
What is claimed is: 
     
         1 . A mUNA molecule, comprising one or more UNA monomers, and comprising nucleic acid monomers, wherein the mUNA molecule is translatable to express a polypeptide or protein. 
     
     
         2 . The molecule of  claim 1 , wherein the molecule comprises from 200 to 12,000 monomers. 
     
     
         3 . The molecule of  claim 1 , wherein the molecule comprises from 200 to 4,000 monomers. 
     
     
         4 . The molecule of  claim 1 , wherein the molecule comprises from 1 to 8,000 UNA monomers. 
     
     
         5 . The molecule of  claim 1 , wherein the molecule comprises from 1 to 100 UNA monomers. 
     
     
         6 . The molecule of  claim 1 , wherein the molecule comprises from 1 to 20 UNA monomers. 
     
     
         7 . The molecule of  claim 1 , wherein the molecule comprises one or more modified nucleic acid nucleotides, or one or more chemically-modified nucleic acid nucleotides. 
     
     
         8 . The molecule of  claim 1 , wherein the molecule comprises a 5′ cap, a 5′ untranslated region of monomers, a coding region of monomers, a 3′ untranslated region of monomers, and a tail region of monomers. 
     
     
         9 . The molecule of  claim 8 , wherein the molecule comprises a translation enhancer in a 5′ or 3′ untranslated region. 
     
     
         10 . The molecule of  claim 1 , wherein the molecule is translatable in vivo. 
     
     
         11 . The molecule of  claim 1 , wherein the molecule is translatable in vitro. 
     
     
         12 . The molecule of  claim 1 , wherein the molecule is translatable in a mammalian cell. 
     
     
         13 . The molecule of  claim 1 , wherein the molecule is translatable in a human in vivo. 
     
     
         14 . The molecule of  claim 1 , wherein a translation product of the molecule is an active peptide or protein. 
     
     
         15 . The molecule of  claim 1 , wherein a translation product of the molecule is human EPO, human Factor IX, human alpha-1-antitrypsin, human CFTR, human ASL, human PAH, human NIS, or human hepcidin. 
     
     
         16 . The molecule of  claim 1 , wherein the molecule exhibits at least 2-fold increased translation efficiency in vivo as compared to a native mRNA that encodes the same translation product. 
     
     
         17 . The molecule of  claim 1 , wherein the molecule exhibits at least 3-fold increased translation efficiency in vivo as compared to a native mRNA that encodes the same translation product. 
     
     
         18 . The molecule of  claim 1 , wherein the molecule exhibits at least 5-fold increased translation efficiency in vivo as compared to a native mRNA that encodes the same translation product. 
     
     
         19 . The molecule of  claim 1 , wherein the molecule exhibits at least 10-fold increased translation efficiency in vivo as compared to a native mRNA that encodes the same translation product. 
     
     
         20 . The molecule of  claim 1 , wherein the molecule has a cytoplasmic half-life in a cell at least 2-fold greater than a native mRNA of the cell that encodes the same translation product. 
     
     
         21 . The molecule of  claim 1 , wherein the molecule is a therapeutic agent for a rare disease, a liver disease, or a cancer. 
     
     
         22 . The molecule of  claim 1 , wherein the molecule is an immunization agent or vaccine component for a rare disease, a liver disease, or a cancer. 
     
     
         23 . The molecule of  claim 1 , wherein the molecule comprises a sequence selected from SEQ ID NOs: 1-164. 
     
     
         24 . A composition comprising a mUNA molecule of  claim 1  and a pharmaceutically acceptable carrier. 
     
     
         25 . A vaccine or immunization composition comprising a mUNA molecule of  claim 1 . 
     
     
         26 . The composition of  claim 24 , wherein the carrier is a nanoparticle or liposome. 
     
     
         27 . A method for ameliorating, preventing or treating a disease or condition in a subject comprising administering to the subject a composition of  claim 24 . 
     
     
         28 . The method of  claim 27 , wherein the disease or condition is a rare disease, liver disease, or cancer. 
     
     
         29 . The method of  claim 27 , wherein the disease or condition is described in Table 1. 
     
     
         30 . A method for producing a polypeptide or protein in vivo, the method comprising administering to a mammal a composition of  claim 24 . 
     
     
         31 . The method of  claim 30 , wherein the polypeptide or protein is deficient in a disease or condition described in Table 1. 
     
     
         32 . The method of  claim 30 , wherein the protein is human EPO, human Factor IX, human alpha-1-antitrypsin, human CFTR, human ASL, human PAH, human NIS, or human hepcidin. 
     
     
         33 . A method for producing a polypeptide or protein in vitro, the method comprising transfecting a cell with a mUNA molecule of  claim 1 . 
     
     
         34 . The method of  claim 33 , wherein the transfecting is done with a transfection reagent. 
     
     
         35 . The method of  claim 33 , wherein the polypeptide or protein is deficient in a disease or condition described in Table 1. 
     
     
         36 . The method of  claim 33 , wherein the protein is human EPO, human Factor IX, human alpha-1-antitrypsin, human CFTR, human ASL, human PAH, human NIS, or human hepcidin. 
     
     
         37 . A method for ameliorating, preventing or treating a rare disease or condition in a subject associated with a deficiency in a peptide or protein, the method comprising administering to the subject a mUNA molecule encoding the peptide or protein. 
     
     
         38 . The method of  claim 37 , wherein the disease or condition described in Table 1. 
     
     
         39 . A method for producing a polypeptide or protein in vivo, the method comprising administering to a mammal a mUNA molecule encoding the polypeptide or protein. 
     
     
         40 . The method of  claim 39 , wherein the polypeptide or protein is human EPO, human Factor IX, human alpha-1-antitrypsin, human CFTR, human ASL, human PAH, human NIS, or human hepcidin.

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