IP Library › Granted Patent US 11,505,578
Granted Patent B2
US 11,505,578 · App. 17/382,102 · Granted Nov 22, 2022

Endogenous Gag-based capsids and uses thereof

Inventors: Colin Malone (Brooklyn, NY); Ian Peikon (Bethpage, NY); Zachary Gilbert (Brooklyn, NY); Andrey Pisarev (Long Beach Township, NJ); Adam Fraites (Long Beach Township, NJ); Jessica Crisp (Phoenix, AZ)
Assignee: VNV NEWCO INC.
C07K14/005A61K38/00A61K48/0008C07K14/435C07K14/46C07K16/00C12N9/22C12N15/11C12N15/87C12N15/907C12N2310/20
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Quick Facts
Patent No.
US 11,505,578
App. No.
17/382,102
Granted
Nov 22, 2022
Kind
B2
Abstract

Nucleic acids encoding endogenous Gag peptides can be isolated from various organisms. Nucleic acids encoding various endogenous Gag polypeptides can be isolated from human DNA. The nucleic acids can be used to express endogenous Gag polypeptides that can be assembled into capsids. Endogenous Gag polypeptides and capsids can be used package cargo and/or deliver it to cells, for example, to package and/or deliver a nucleic acid molecule for use in gene editing, such as a component involved in a CRISPR-Cas system.

Claims (31)

1. A composition comprising: (a) a capsid that comprises an endogenous Gag polypeptide, and (b) a nucleic acid molecule for use in gene editing; wherein the nucleic acid molecule for use in gene editing is associated with the capsid, and the endogenous Gag polypeptide is not an Arc polypeptide.

2. The composition of claim 1 , wherein the nucleic acid molecule for use in gene editing encodes or is a component involved in a CRISPR-Cas system.

3. The composition of claim 1 , wherein the nucleic acid molecule for use in gene editing encodes a component involved in a Zinc finger nuclease.

4. The composition of claim 1 , wherein the nucleic acid molecule for use in gene editing encodes a component involved in a transcription activator-like effector nuclease.

5. The composition of claim 1 , wherein the endogenous Gag polypeptide is a human endogenous Gag polypeptide.

6. The composition of claim 1 , wherein the endogenous Gag polypeptide is a Paraneoplastic Ma antigen family polypeptide.

7. The composition of claim 1 , wherein the endogenous Gag polypeptide is a retrotransposon Gag-like family polypeptide.

8. The composition of claim 7 , wherein the retrotransposon Gag-like family polypeptide comprises an amino acid sequence that is at least 90% identical to SEQ ID NO: 28.

9. The composition of claim 1 , wherein the 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;

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.

10. The composition of claim 1 , further comprising a delivery component that comprises an extracellular vesicle, microvesicle, liposome, micelle, or viral envelope glycoprotein.

11. The composition of claim 10 , wherein the delivery component comprises the extracellular vesicle.

12. The composition of claim 10 , wherein the delivery component comprises the liposome.

13. The composition of claim 10 , wherein the delivery component comprises the viral envelope glycoprotein.

14. A polynucleotide comprising a nucleotide sequence encoding the endogenous Gag polypeptide of claim 1 and the nucleic acid molecule for use in gene editing of claim 1 .

15. A method of delivering a gene editing system to a cell comprising administering the composition of claim 1 to the cell.

16. The method of claim 15 , wherein the cell is a eukaryotic cell.

17. The method of claim 15 , wherein the cell is a vertebrate cell.

18. The method of claim 15 , wherein the cell is a mammalian cell.

19. The method of claim 15 , wherein the cell is a human cell.

Assignments (3)
CHANGE OF NAME Recorded Jul 22, 2023
From: VNV NEWCO INC.
To: AERA THERAPEUTICS, INC.
Reel/Frame 064347/0692 →
CORRECTIVE ASSIGNMENT TO CORRECT THE THIRD INVENTOR'S FIRST NAME PREVIOUSLY RECORDED AT REEL: 057467 FRAME: 0166. ASSIGNOR(S) HEREBY CONFIRMS THE ASSIGNMENT . Recorded Oct 8, 2021
From: MALONE, COLIN; PEIKON, IAN; GILBERT, ZACHARY; PISAREV, ANDREY; FRAITES, ADAM; CRISP, JESSICA
To: VNV NEWCO INC.
Reel/Frame 057758/0105 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Sep 13, 2021
From: MALONE, COLIN; PEIKON, IAN; GILBERT, ZACH; PISAREV, ANDREY; FRAITES, ADAM; CRISP, JESSICA
To: VNV NEWCO INC.
Reel/Frame 057467/0166 →
Continuity (3)
Continuation 17277119
Provisional Application 62733015 · Sep 18, 2018
Related Publication 20220002358A1 · Jan 6, 2022
Cited By (5)
US 12,319,938 US 12,351,814 US 12,351,815 US 12,404,525 US 12,617,822