# US 11,026,990 B2 · PUM 1 protein as target for virus inhibition

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- Number: US 11,026,990 B2
- Document ID: US11026990B2
- Status: Granted
- Kind: B2
- Application number: 16/477,406
- Filed: 2017-04-12
- Published: 2020-05-28
- Granted: 2021-06-08
- Earliest claimed: 2017-04-12
- Assignees: Shenzhen International Institute for Biomedical Research
- Applicants: Shenzhen International Institute for Biomedical Research
- Inventors: Grace Zhou; Bernard Roizman; Yonghong Liu
- Web page: https://patentgrep.com/patent/US11026990B2

## CPC

- A61K 38/02: Peptides of undefined number of amino acids; Derivatives thereof
- A61K 31/7088: Compounds having three or more nucleosides or nucleotides
- C07K 14/4702: {Regulators; Modulating activity}
- C12N 9/14: Hydrolases (3)
- C12N 15/113: Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; {Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing (when used in plants C12N15/8218)}
- C12Y 306/04013: RNA helicase (3.6.4.13)
- C12N 2310/11: Antisense
- C12N 2310/14: interfering nucleic acids [NA]
- A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D)
- C07K: PEPTIDES (peptides in foodstuffs A23; obtaining protein compositions for foodstuffs, working-up proteins for foodstuffs A23J; preparations for medicinal purposes A61K; peptides containing beta-lactam rings C07D; cyclic dipeptides not having in their molecule any other peptide link than those which form their ring, e.g. piperazine-2,5-diones, C07D; ergot alkaloids of the cyclic peptide type C07D519/02; macromolecular compounds having statistically distributed amino acid units in their molecules, i.e. when the preparation does not provide for a specific; but for a random sequence of the amino acid units, homopolyamides and block copolyamides derived from amino acids C08G69/00; macromolecular products derived from proteins C08H1/00; preparation of glue or gelatine C09H; single cell proteins, enzymes C12N; genetic engineering processes for obtaining peptides C12N15/00; compositions for measuring or testing processes involving enzymes C12Q; investigation or analysis of biological material G01N33/00)
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)
- C12Y: ENZYMES

## Abstract

Disclosed is a method for prophylaxis or treatment of infection of a virus, or for modulating innate immunity, in a subject comprising administering to the subject a therapeutically effective amount of a PUM1 inhibitor. A pharmaceutical composition comprising the PUM1 inhibitor is also disclosed.

## Claims

### Claim 1

A method for treatment of infection of a virus in a subject, comprising administering to the subject a therapeutically effective amount of a PUM1 inhibitor, wherein the PUM1 inhibitor is an antisense oligomer that targets a nucleic acid molecule encoding PUM1 to block the synthesis of PUM1 wherein the virus is Hepres Simplex Virus type 1 or type 2.

### Claim 2 (depends on 1)

The method of claim 1, wherein the virus is an interferon sensitive virus.

### Claim 3 (depends on 1)

The method of claim 1, wherein the antisense oligomer is delivered by a vesicle.

### Claim 4 (depends on 1)

The method of claim 1, wherein the nucleic acid molecule encoding PUM1 is selected from DNA encoding PUM1, RNA transcribed from the DNA, and cDNA derived from such RNA.

### Claim 5 (depends on 1)

The method of claim 1, wherein the antisense oligomer is selected from a DNA oligonucleotide, an RNA oligonucleotide, and a chimeric oligonucleotide.

### Claim 6 (depends on 5)

The method of claim 5, wherein the antisense oligomer is selected from dsRNA, siRNA, and shRNA.

### Claim 7 (depends on 3)

The method of claim 3, wherein the vesicle is an exosome.

### Claim 8 (depends on 1)

The method of claim 1, wherein the administration of the PUM1 inhibitor induces upregulated expressions of interferon and interferon stimulated genes.

### Claim 9 (depends on 8)

The method of claim 8, wherein the interferon stimulated genes is selected from LGP2, CXCL10, IL6, PKR, RIG-I, MADS, SP100, IFIT1, PML and STING.

### Claim 10 (depends on 1)

The method of claim 1, wherein the subject is a mammal.

### Claim 11 (depends on 10)

The method of claim 10, wherein the mammal is a human being.

### Claim 12 (depends on 1)

The method of claim 1, wherein the antisense oligomer comprises the sequence of 5′-gcugcuuacuaugaccaaatt-3′ (SEQ ID NO: 1).

### Claim 13 (depends on 1)

The method of claim 1, wherein the virus is an interferon β sensitive virus.