IP Library › Granted Patent US 12,275,949
Granted Patent B2
US 12,275,949 · App. 18/359,013 · Granted Apr 15, 2025

High-transducing HSV vectors

Inventors: David M. Krisky (Pittsburgh, PA); James B. Wechuck (Pittsburgh, PA); James R. Goss (Pittsburgh, PA)
Assignee: PeriphaGen, Inc.
C12N15/86A61K48/0025C12N7/00C12N2710/16621C12N2710/16643C12N2710/16662C12N2830/008
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Quick Facts
Patent No.
US 12,275,949
App. No.
18/359,013
Granted
Apr 15, 2025
Kind
B2
Abstract

Disclosed herein are high transducing replication defective herpes simplex virus (HSV) vectors of McKrae strain.

Claims (14)

1. A gene therapy vector comprising a replication-defective variant of herpes simplex virus (HSV) McKrae strain whose genome contains an alteration such that the variant fails to express a functional ICP4 protein comprising SEQ ID NO: 16, wherein the gene therapy vector is capable of expressing a payload in a cell susceptible to McKrae strain infection for at least 18 days.

2. The gene therapy vector of claim 1 , wherein the gene therapy vector comprises a neuron specific promoter.

3. The gene therapy vector of claim 2 , wherein the neuron specific promoter is a calcitonin gene-related peptide (CGRP) promoter.

4. The gene therapy vector of claim 1 , wherein the gene therapy vector comprises a human cytomegalovirus (HCMV) enhancer.

5. The gene therapy vector of claim 1 , wherein the gene therapy vector comprises a bovine growth hormone (BGH) polyadenylation signal.

6. The gene therapy vector of claim 1 , further comprising a nucleic acid that encodes a therapeutic polypeptide.

7. The gene therapy vector of claim 1 , wherein the functional ICP4 protein has an amino acid sequence of SEQ ID NO: 2.

8. The gene therapy vector of claim 1 , wherein the gene therapy vector is capable of expressing a payload in a cell susceptible to McKrae strain infection for at least 50 days.

9. The gene therapy vector of claim 1 , wherein the gene therapy vector is capable of expressing a payload in a cell susceptible to McKrae strain infection for at least 100 days.

10. The gene therapy vector of claim 1 , wherein the gene therapy vector is capable of producing in neurons a mean genome copy number of greater than 5,000 transcripts.

11. The gene therapy vector of claim 1 , wherein the gene therapy vector is capable of producing in neurons a mean genome copy number of greater than 10,000 transcripts.

12. The gene therapy vector of claim 1 , wherein the gene therapy vector is capable of producing in neurons a mean genome copy number of greater than 15,000 transcripts.

13. A cell transduced with a gene therapy vector according to claim 1 .

14. A pharmaceutical composition comprising a gene therapy vector according to claim 1 and a pharmaceutically acceptable carrier.

Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Feb 29, 2024
From: KRISKY, DAVID M.; WECHUCK, JAMES B.; GOSS, JAMES R.
To: PERIPHAGEN, INC.
Reel/Frame 066605/0724 →
Continuity (3)
Division 16088393
Provisional Application 62313391 · Mar 25, 2016
Related Publication 20240158809A1 · May 16, 2024
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