US2022088224A1PendingUtilityA1

Arc-based capsids and uses thereof

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Assignee: VNV NEWCO INCPriority: Sep 18, 2018Filed: Sep 18, 2019Published: Mar 24, 2022
Est. expirySep 18, 2038(~12.2 yrs left)· nominal 20-yr term from priority
Inventors:Colin MaloneIan PeikonZachary GilbertAndrey PisarevAdam FraitesJessica Crisp
A61K 2039/55555A61K 9/5184A61K 9/5169A61K 48/0008C07K 14/46C12N 15/87C07K 14/435C12N 15/11C12N 9/22A61K 38/00C12N 2310/20C12N 15/86C12N 15/907C07K 14/005C07K 16/00
63
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Claims

Abstract

Disclosed herein, in certain embodiments, are recombinant Arc and endogenous Gag polypeptides, and methods of using recombinant Arc and endogenous Gag polypeptides.

Claims

exact text as granted — not AI-modified
1 . A capsid comprising a recombinant endogenous Gag polypeptide and a therapeutic agent. 
     
     
         2 . The capsid of  claim 1 , wherein the therapeutic agent is a nucleic acid. 
     
     
         3 . The capsid of  claim 2 , wherein the nucleic acid is an RNA. 
     
     
         4 . (canceled) 
     
     
         5 . (canceled) 
     
     
         6 . The capsid of any of  claim 1 , wherein the recombinant endogenous Gag polypeptide is a human endogenous Gag polypeptide. 
     
     
         7 . The capsid of  claim 6 , wherein the recombinant human endogenous Gag polypeptide is an endogenous Gag polypeptide comprising:
 a) an amino acid sequence that is SEQ ID NO: 16 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 16;   b) an amino acid sequence that is SEQ ID NO: 17 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 17;   c) an amino acid sequence that is SEQ ID NO: 18 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 18;   d) an amino acid sequence that is SEQ ID NO: 19 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 19;   e) an amino acid sequence that is SEQ ID NO: 20 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 20;   f) an amino acid sequence that is SEQ ID NO: 21 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 21;   g) an amino acid sequence that is SEQ ID NO: 22 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 22;   h) an amino acid sequence that is SEQ ID NO: 23 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 23;   i) an amino acid sequence that is SEQ ID NO: 24 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 24;   j) an amino acid sequence that is SEQ ID NO: 25 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 25; or   k) an amino acid sequence that is SEQ ID NO: 26 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 26; or   l) an amino acid sequence that is SEQ ID NO: 27 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 27; or   m) an amino acid sequence that is SEQ ID NO: 28 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 28.   
     
     
         8 . A capsid comprising a recombinant Arc polypeptide or a recombinant endogenous Gag polypeptide,
 wherein the recombinant Arc polypeptide is not a rat Arc polypeptide or a  Drosophila melanogaster  Arc polypeptide.   
     
     
         9 . The capsid of  claim 8 , further comprising a cargo. 
     
     
         10 . The capsid of  claim 9 , wherein the cargo is a nucleic acid. 
     
     
         11 . The capsid of  claim 10 , wherein the cargo is an RNA. 
     
     
         12 . The capsid of  claim 9 , wherein the cargo is a therapeutic agent. 
     
     
         13 . The capsid of  claim 8 , wherein the recombinant Arc polypeptide is an Arc polypeptide comprising:
 a) an amino acid sequence that is SEQ ID NO: 2 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 2;   b) an amino acid sequence that is SEQ ID NO: 3 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 3;   c) an amino acid sequence that is SEQ ID NO: 4 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 4;   d) an amino acid sequence that is SEQ ID NO: 5 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 5;   e) an amino acid sequence that is SEQ ID NO: 6 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 6;   f) an amino acid sequence that is SEQ ID NO: 7 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 7;   g) an amino acid sequence that is SEQ ID NO: 8 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 8;   h) an amino acid sequence that is SEQ ID NO: 9 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 9;   i) an amino acid sequence that is SEQ ID NO: 10 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 10; or   j) an amino acid sequence that is SEQ ID NO: 11 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 11; or   k) an amino acid sequence that is SEQ ID NO: 12 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 12; or   l) an amino acid sequence that is SEQ ID NO: 13 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 13; or   m) an amino acid sequence that is SEQ ID NO: 14 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 14; or   n) an amino acid sequence that is SEQ ID NO: 15 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 15.   
     
     
         14 . The capsid of any of  claim 8 , wherein the recombinant endogenous Gag polypeptide is an endogenous Gag polypeptide comprising:
 a) an amino acid sequence that is SEQ ID NO: 16 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 16;   b) an amino acid sequence that is SEQ ID NO: 17 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 17;   c) an amino acid sequence that is SEQ ID NO: 18 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 18;   d) an amino acid sequence that is SEQ ID NO: 19 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 19;   e) an amino acid sequence that is SEQ ID NO: 20 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 20;   f) an amino acid sequence that is SEQ ID NO: 21 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 21; or   g) an amino acid sequence that is SEQ ID NO: 22 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 22; or   h) an amino acid sequence that is SEQ ID NO: 23 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 23; or   i) an amino acid sequence that is SEQ ID NO: 24 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 24; or   j) an amino acid sequence that is SEQ ID NO: 25 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 25; or   k) an amino acid sequence that is SEQ ID NO: 26 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 26; or   l) an amino acid sequence that is SEQ ID NO: 27 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 27; or   m) an amino acid sequence that is SEQ ID NO: 28 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 28.   
     
     
         15 . A vector comprising DNA encoding the recombinant Arc polypeptide or the recombinant endogenous Gag polypeptide of  claim 8 . 
     
     
         16 . A method of delivering a cargo to a cell comprising administering the capsid  claim 9  to the cell. 
     
     
         17 . The method of  claim 16 , wherein the cell is a eukaryotic cell. 
     
     
         18 . The method of  claim 16 , wherein the cell is a vertebrate cell. 
     
     
         19 . The method of  claim 16 , wherein the cell is a mammalian cell. 
     
     
         20 . The method of  claim 16 , wherein the cell is a human cell. 
     
     
         21 . The method of  claim 16 , wherein the cargo is a nucleic acid. 
     
     
         22 . The method of  claim 21 , wherein the cell expresses a gene encoded by the nucleic acid. 
     
     
         23 . The method of  claim 16 , wherein the cargo is a therapeutic agent. 
     
     
         24 . A method of transfecting a nucleic acid into a cell comprising administering the capsid of  claim 10  to the cell. 
     
     
         25 . The capsid of  claim 8 , wherein the recombinant Arc polypeptide is an Arc polypeptide comprising an amino acid sequence that is SEQ ID NO: 1 or an amino acid sequence that is at least 90% identical to the SEQ ID NO: 1.

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