US2021403907A1PendingUtilityA1

Arc-based capsids and uses thereof

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Assignee: VNV NEWCO INCPriority: Sep 18, 2018Filed: Sep 13, 2021Published: Dec 30, 2021
Est. expirySep 18, 2038(~12.2 yrs left)· nominal 20-yr term from priority
Inventors:Colin MaloneIan PeikonZachary GilbertAndrey PisarevAdam FraitesJessica Crisp
C07K 14/161C07K 14/16C12N 15/111C07K 14/15A61K 39/0011C12N 2740/10022C07K 14/005C12N 2320/32A61K 2039/55555A61K 2039/542A61K 2039/51A61P 35/00A61K 9/5184A61K 9/127A61K 9/5169C07K 2319/00A61K 9/1075C12N 15/85A61K 9/1658A61K 39/39A61K 2039/80C12N 2310/20C12N 2310/531C12N 2310/12C12N 2310/141C12N 2310/3519C12N 15/907C12N 15/87C12N 15/11C12N 9/22C07K 16/00C07K 14/46C07K 14/435A61K 48/0008A61K 38/00
67
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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
What is claimed is: 
     
         1 . A composition comprising: (a) a capsid that comprises an endogenous Gag polypeptide and a heterologous cargo, wherein the endogenous Gag polypeptide is not an Arc polypeptide; and (b) a delivery component. 
     
     
         2 . The composition of  claim 1 , wherein the delivery component comprises a microvesicle or a microparticle. 
     
     
         3 . The composition of  claim 1 , wherein the delivery component further comprises a fusogenic molecule. 
     
     
         4 . The composition of  claim 1 , wherein the delivery component further comprises a cell-specific binding protein or an engineered protein that binds to an antigen or cell surface molecule. 
     
     
         5 . The composition of  claim 1 , further comprising a second polypeptide that is fused to the endogenous Gag polypeptide, wherein the second polypeptide binds to a target receptor, antigen, or cell surface molecule. 
     
     
         6 . The composition of  claim 5 , wherein the second polypeptide is an antibody or antigen-binding fragment thereof, a human protein, a viral protein, or an engineered protein. 
     
     
         7 . The composition of  claim 1 , wherein the endogenous Gag polypeptide is a Paraneoplastic Ma antigen family polypeptide. 
     
     
         8 . The composition of  claim 1 , wherein the endogenous Gag polypeptide is a retrotransposon Gag-like family polypeptide. 
     
     
         9 . The composition of  claim 8 , wherein the retrotransposon Gag-like family polypeptide is a PEG10 polypeptide. 
     
     
         10 . The composition of  claim 1 , wherein the heterologous cargo comprises a nucleic acid molecule that comprises or encodes a component of a CRISPR-Cas system, zinc finger nuclease (ZFN) system, or transcription activator-like effector nuclease (TALEN) system. 
     
     
         11 . A composition comprising: (a) a capsid that comprises an endogenous Gag polypeptide, wherein the endogenous Gag polypeptide is not an Arc polypeptide; and (b) a delivery component that comprises a liposome or a micelle. 
     
     
         12 . The composition of  claim 11 , wherein the delivery component comprises the liposome. 
     
     
         13 . The composition of  claim 12 , wherein the liposome comprises a surface presented lipopeptide. 
     
     
         14 . The composition of  claim 11 , wherein the delivery component comprises the micelle. 
     
     
         15 . The composition of  claim 14 , wherein the micelle comprises a hydrophilic polymer, a hydrophilic copolymer, a pH-sensitive polymer, or a pH-sensitive copolymer. 
     
     
         16 . The composition of  claim 11 , further comprising a fusogenic molecule. 
     
     
         17 . The composition of  claim 11 , further comprising a cell-specific binding protein or an engineered protein that binds to an antigen or cell surface molecule. 
     
     
         18 . The composition of  claim 11 , wherein the endogenous Gag polypeptide is a Paraneoplastic Ma antigen family polypeptide. 
     
     
         19 . The composition of  claim 11 , wherein the endogenous Gag polypeptide is a retrotransposon Gag-like family polypeptide. 
     
     
         20 . The composition of  claim 19 , wherein the retrotransposon Gag-like family polypeptide is a PEG10 polypeptide. 
     
     
         21 . The composition of  claim 11 , further comprising a nucleic acid molecule that comprises or encodes a component of a CRISPR-Cas system, zinc finger nuclease (ZFN) system, or transcription activator-like effector nuclease (TALEN) system. 
     
     
         22 . A composition comprising: (a) a capsid that comprises a recombinant endogenous Gag polypeptide and a cargo, wherein the recombinant endogenous Gag polypeptide is not an Arc polypeptide; and (b) a delivery component. 
     
     
         23 . The composition of  claim 22 , wherein the delivery component comprises a microvesicle or a microparticle. 
     
     
         24 . The composition of  claim 22 , wherein the delivery component comprises a fusogenic molecule. 
     
     
         25 . The composition of  claim 22 , wherein the delivery component comprises a cell-specific binding protein or an engineered protein that binds to an antigen or cell surface molecule. 
     
     
         26 . The composition of  claim 22 , wherein the recombinant endogenous Gag polypeptide is a Paraneoplastic Ma antigen family polypeptide. 
     
     
         27 . The composition of  claim 22 , wherein the recombinant endogenous Gag polypeptide is a retrotransposon Gag-like family polypeptide. 
     
     
         28 . The composition of  claim 27 , wherein the retrotransposon Gag-like family polypeptide is a PEG10 polypeptide. 
     
     
         29 . The composition of  claim 22 , wherein the cargo comprises a nucleic acid molecule that comprises or encodes a component of a CRISPR-Cas system, zinc finger nuclease (ZFN) system, or transcription activator-like effector nuclease (TALEN) system. 
     
     
         30 . A method of preparing a composition for suitable for delivery of a cargo, the method comprising:
 (a) isolating an endogenous Gag polypeptide, wherein the endogenous Gag polypeptide is not an Arc polypeptide;   (b) incubating the endogenous Gag polypeptide with the cargo in conditions suitable for capsid formation, thereby packaging the cargo in a capsid that comprises the endogenous Gag polypeptide; and   (c) formulating the capsid with a delivery component that comprises a liposome or a micelle.

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