IP Library Granted Patent US 8,822,213
Granted Patent B2
US 8,822,213 · App. 13/127,968 · Granted Sep 2, 2014

Bispecific intracellular delivery vehicles

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Quick Facts
Patent No.
US 8,822,213
App. No.
13/127,968
Granted
Sep 2, 2014
Kind
B2
Abstract

A composition for delivering an agent to a cell, comprising a bispecific affinity reagent and a pH-responsive, membrane destabilizing polymer. The bispecific affinity reagent may include a first affinity reagent covalently linked to a second affinity reagent, wherein the first affinity reagent binds to a molecule on the surface of a cell, and the second affinity reagent binds to an intracellular target.

Claims (41)

1. A composition for delivering an agent to a cell, comprising:

a bispecific affinity reagent, comprising a first affinity reagent covalently linked to a second affinity reagent, wherein the first affinity reagent binds to a molecule on the surface of a cell, and the second affinity reagent binds to an intracellular target, and

wherein the bispecific affinity reagent comprises an antibody, antibody fragment, antibody-like molecule, a single protein, or a specific scFv; and

a pH-responsive, membrane destabilizing polymer.

2. The composition of claim 1 , wherein the first affinity reagent and the second affinity reagent are included in a single polypeptide chain.

3. A composition for delivering an agent to a cell, comprising:

a bispecific affinity reagent, comprising a first affinity reagent covalently linked to a second affinity reagent, wherein the first affinity reagent binds to a molecule on the surface of a cell, and the second affinity reagent binds to an intracellular target, and

wherein the first affinity reagent is an antibody, antibody fragment, antibody-like molecule, a single protein, peptide, oligosaccharide, or aptamer; and

a pH-responsive, membrane destabilizing polymer.

4. The composition of claim 3 wherein the first affinity reagent is a small molecule selected from the group consisting of folate or a ligand for the asialoglycoprotein receptor.

5. The composition of claim 3 , wherein the peptide is an RGD peptide.

6. The composition of claim 1 , wherein the first affinity reagent comprises a protein transduction domain.

7. The composition of claim 6 , wherein the protein transduction domain comprises antennapedia, TAT, VP22, transportan, penetratin, polyarginine, fragments thereof, or combinations thereof.

8. A composition for delivering an agent to a cell, comprising:

a bispecific affinity reagent, comprising a first affinity reagent covalently linked to a second affinity reagent, wherein the first affinity reagent binds to a molecule on the surface of a cell, and the second affinity reagent binds to an intracellular target, and

wherein the second affinity reagent is an antibody, antibody fragment, antibody-like molecule, peptide, or aptamer; and

a pH-responsive, membrane destabilizing polymer.

9. The composition of claim 8 , wherein the peptide is BH3 or BAK-BH3.

10. A composition for delivering an agent to a cell, comprising:

a bispecific affinity reagent, comprising a first affinity reagent covalently linked to a second affinity reagent, wherein the first affinity reagent binds to a molecule on the surface of a cell, and the second affinity reagent binds to an intracellular target, and

wherein the intracellular target is a DNA binding protein, a cytoplasmic domain of a membrane receptor, a kinase, a GTPase, a phosphatase, or a protein in the Bel family of proteins; and

a pH-responsive, membrane destabilizing polymer.

11. The composition of claim 10 , wherein the DNA binding protein is p53.

12. The composition of claim 10 , wherein the GTPase is ras.

13. The composition of claim 10 , wherein the polymer is a copolymer comprising:

a membrane destabilizing segment comprising a plurality of anionic species that are anionic at about neutral pH, and a plurality of hydrophobic species, and, optionally a plurality of cationic species that are cationic at about neutral pH.

14. The composition of claim 13 , wherein the copolymer further comprises:

an amphiphilic or hydrophilic segment.

15. A composition for delivering an agent to a cell, comprising:

a bispecific affinity reagent, comprising a first affinity reagent covalently linked to a second affinity reagent through a cleavable linker, wherein the first affinity reagent binds to a molecule on the surface of a cell, and the second affinity reagent binds to an intracellular target, and

a pH-responsive, membrane destabilizing polymer.

16. A composition for delivering an agent to a cell, comprising:

a bispecific affinity reagent, comprising a first affinity reagent covalently linked to a second affinity reagent, wherein the first affinity reagent binds to a molecule on the surface of a cell, and the second affinity reagent binds to an intracellular target, and

a pH-responsive, membrane destabilizing polymer, wherein the pH-responsive, membrane destabilizing polymer comprises a plurality of pendant linking groups, wherein the first affinity reagent is linked to the polymer and wherein the second affinity reagent is linked to the polymer.

17. The composition of claim 16 , wherein the first affinity reagent is linked to an end group of the polymer.

18. The composition of claim 16 , wherein the second affinity reagent is linked to one of the plurality of pendant linking groups.

19. The composition of claim 16 , further comprising a plurality of second affinity reagents, wherein the plurality of second affinity reagents is linked to the polymer via the pendant linking groups.

20. The composition of claim 16 , further comprising a plurality of first affinity reagents, wherein the plurality of first affinity reagents is linked to the polymer via the pendant linking groups.

21. The composition of claim 1 , further comprising a therapeutic or diagnostic agent.

22. The composition claim 16 , wherein the second affinity reagent is cleavably linked to the polymer.

23. The composition of claim 16 , wherein the second affinity reagent is an antibody, antibody fragment, antibody-like molecule, or peptide.

Assignments (8)
INTELLECTUAL PROPERTY SECURITY AGREEMENT Recorded Apr 3, 2020
From: GENEVANT SCIENCES LTD.
To: ROIVANT SCIENCES LTD.
Reel/Frame 052312/0930 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Oct 12, 2018
From: PHASERX, INC.
To: ROIVANT HEPATOLOGY GMBH
Reel/Frame 047649/0829 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Oct 12, 2018
From: ROIVANT HEPATOLOGY GMBH
To: GENEVANT SCIENCES GMBH
Reel/Frame 047689/0406 →
CONFIRMATORY LICENSE Recorded Dec 30, 2014
From: UNIVERSITY OF WASHINGTON / CENTER FOR COMMERCIALIZATION
To: NATIONAL INSTITUTES OF HEALTH (NIH), U.S. DEPT. OF HEALTH AND HUMAN SERVICES (DHHS), U.S. GOVERNMENT
Reel/Frame 034714/0238 →
CONFIRMATORY LICENSE Recorded Jul 6, 2012
From: UNIVERSITY OF WASHINGTON / CENTER FOR COMMERCIALIZATION
To: NATIONAL INSTITUTES OF HEALTH (NIH), U.S. DEPT. OF HEALTH AND HUMAN SERVICES (DHHS), U.S. GOVERNMENT
Reel/Frame 028503/0359 →
CONFIRMATORY LICENSE Recorded Jan 30, 2012
From: UNIVERSITY OF WASHINGTON / CENTER FOR COMMERCIALIZATION
To: NATIONAL INSTITUTES OF HEALTH (NIH), U.S. DEPT. OF HEALTH AND HUMAN SERVICES (DHHS), U.S. GOVERNMENT
Reel/Frame 027614/0593 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Aug 3, 2011
From: OVERELL, ROBERT; JOHNSON, PAUL H.
To: PHASERX, INC.
Reel/Frame 026693/0160 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Aug 3, 2011
From: STAYTON, PATRICK S.; HOFFMAN, ALLAN S.; CONVERTINE, ANTHONY J.; DUVALL, CRAIG L.
To: WASHINGTON, UNIVERSITY OF
Reel/Frame 026693/0021 →