IP Library Granted Patent US 11,118,170
Granted Patent B2
US 11,118,170 · App. 16/854,551 · Granted Sep 14, 2021

Engineered clostridium botulinum toxin adapted to deliver molecules into selected cells

Inventors: Benjamin J. Pavlik (Lincoln, NE); Paul Blum (Lincoln, NE); Kevin Van Cott (Hickman, NE)
Assignee: The Board of Regents of the University of Nebraska
C12N9/52A61K38/45A61K47/6415A61K49/0056C07K14/33C12N15/62C12Y204/02C12Y304/24069C07K2319/33Y02A50/30
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Quick Facts
Patent No.
US 11,118,170
App. No.
16/854,551
Granted
Sep 14, 2021
Kind
B2
Abstract

An engineered payload-delivery system includes a target cell binding unit, covalently bound to a pore forming unit, and a payload portion adapted with a region capable of non-covalently binding to the pore forming unit. The pore forming unit is derived from a particular sub-serotype of Clostridium toxin, while the payload region is derived from a different sub-serotype of Clostridium toxin. The disclosed chimeric protein-based composition is capable of specifically delivering payload to neural cells.

Claims (19)

1. A polynucleotide encoding a polypeptide having a sequence at least 90% identical to SEQ ID NO 4.

2. The polynucleotide of claim 1 , wherein the encoded polypeptide has a sequence at least 95% identical to SEQ ID NO 4.

3. The polynucleotide of claim 1 , wherein the encoded polypeptide has a sequence at least 99% identical to SEQ ID NO 4.

4. The polynucleotide of claim 1 , wherein the encoded polypeptide has a sequence identical to SEQ ID NO 4.

5. A host cell comprising the polynucleotide of claim 1 .

6. The host cell of claim 5 , wherein the encoded polypeptide has a sequence at least 95% identical to SEQ ID NO 4.

7. The host cell of claim 5 , wherein the encoded polypeptide has a sequence identical to the sequence of SEQ ID NO 4.

8. The host cell of claim 5 , wherein the host cell is a bacterium or a virus.

9. The host cell of claim 5 , wherein the host cell is used to produce the polypeptide encoded by said polynucleotide in vitro.

10. The host cell of claim 7 , wherein the host cell is introduced into a subject for delivery of an agent to a target cell.

11. The host cell of claim 10 , wherein the agent comprises at least one member selected from the group consisting of a therapeutic agent, a diagnostic agent, and combinations thereof.

12. The host cell of claim 7 , wherein the polypeptide produced by the host cell is introduced into a subject for delivery of an agent to a target cell.

13. The host cell of claim 12 , wherein the agent comprises at least one member selected from the group consisting of a therapeutic agent, a diagnostic agent, and combinations thereof.

14. The host cell of claim 12 , wherein the target cell is a neuronal cell.

15. The host cell of claim 14 , wherein the target cell is a member selected from the group consisting of a cell of a brain tumor, a cell of a neuroblastoma, and a cell of a retinoblastoma, peripheral neuron; motor neuron, sensory neuron, and combination thereof.

16. A method of delivering an agent to a target cell, comprising administering a composition comprising the polynucleotide of claim 1 to a subject, wherein the subject comprises the target cell.

17. The method of claim 16 , wherein the target cell is a member selected from the group consisting of a cell of a brain tumor, a cell of a neuroblastoma, and a cell of a retinoblastoma, peripheral neuron; motor neuron, sensory neuron, and combination thereof.

18. A method of delivering an agent to a target cell, comprising administering the host cell of claim 5 to a subject, wherein the subject comprises the target cell.

19. The method of claim 18 , wherein the target cell is a member selected from the group consisting of a cell of a brain tumor, a cell of a neuroblastoma, and a cell of a retinoblastoma, peripheral neuron; motor neuron, sensory neuron, and combination thereof.

Assignments (2)
CONFIRMATORY LICENSE Recorded Jul 20, 2020
From: NEBRASKA, UNIVERSITY OF, LINCOLN
To: DEFENSE THREAT REDUCTION AGENCY, US DOD
Reel/Frame 053249/0361 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Apr 24, 2020
From: PAVLIK, BENJAMIN J.; BLUM, PAUL; VAN COTT, KEVIN
To: THE BOARD OF REGENTS OF THE UNIVERSITY OF NEBRASKA
Reel/Frame 052489/0965 →
Continuity (3)
Division 15574151
Provisional Application 62162582 · May 15, 2015
Related Publication 20200255815A1 · Aug 13, 2020