# US 2013/0156791 A1 · Methods and pharmaceutical compositions for the treatment of hiv-1 infections

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- Number: US 2013/0156791 A1
- Document ID: US20130156791A1
- Status: Published application
- Kind: A1
- Application number: 13/814,954
- Filed: 2011-08-08
- Published: 2013-06-20
- Earliest claimed: 2010-08-09
- Inventors: Jean-luc Perfettini; Guido Kroemer; Marie-Lise Gougeon; Mauro Placentini
- Web page: https://patentgrep.com/patent/US20130156791A1

## CPC

- A61K 39/42: viral
- A61K 31/7088: Compounds having three or more nucleosides or nucleotides
- A61K 31/7105: Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
- A61K 31/713: Double-stranded nucleic acids or oligonucleotides
- A61K 38/46: Hydrolases (3)
- A61P 31/18: for HIV
- A61P 43/00: Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
- C12N 15/1138: {against receptors or cell surface proteins}
- C12Q 1/18: Testing for antimicrobial activity of a material
- C12Y 306/01005: Apyrase (3.6.1.5), i.e. ATP diphosphohydrolase
- G01N 33/5041: {involving analysis of members of signalling pathways}
- G01N 33/56988: {HIV or HTLV}
- 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)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)
- C12Q: MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS (immunoassay G01N33/53); COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Y: ENZYMES
- G01N: INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES (measuring or testing apparatus or processes other than immunoassay, involving enzymes or microorganisms C12M, C12Q)

## Abstract

The present invention provides methods and compositions (such as pharmaceutical compositions) for treating or preventing human immunodeficiency virus type 1 (HIV-1) infection. More particularly, the present invention relates to a blocking agent of a component of the purinergic signalling pathway selected from the group consisting of pannexin 1, ATP or P2Y2 receptor for use in the treatment or prevention of HIV-1 infection.

## Claims

### Claim at position 1

1-8. (canceled)

### Claim 9

A method for screening a drug for the treatment or prevention of HIV-1 infection comprising the steps of:
a) providing a candidate compound, and
b) determining whether said candidate compound is a P2Y2 antagonist or a pannexin 1 antagonist.

### Claim 10

(canceled)

### Claim 11

A method of treating or preventing HIV-1 infection in a subject in need thereof, comprising
administering to the subject a therapeutically effective amount of a blocking agent that blocks a component of the purinergic signaling pathway, wherein the component is selected from the group consisting of pannexin 1, ATP and P2Y2 receptor.

### Claim 12 (depends on 11)

The method of claim 11, wherein said blocking agent is selected from the group consisting of P2Y2 receptor antagonists, pannexin 1 antagonists and ATP-degrading agents.

### Claim 13 (depends on 12)

The method of claim 12 wherein said P2Y2 receptor antagonists and pannexin 1 antagonists are selected from the group consisting of small organic molecules, antibodies and aptamers.

### Claim 14 (depends on 13)

The method of claim 13, wherein said ATP degrading agent is a pyrase.

### Claim 15 (depends on 14)

The method of claim 14, wherein said blocking agent is an inhibitor of expression selected from the group consisting of antisense RNA molecules, antisense DNA molecules, small inhibitory RNAs (siRNAs), short hairpin RNA and ribozymes.

### Claim 16

A method of determining whether or not a subject is at high risk of contracting HIV-1, comprising
obtaining a biological sample from the subject,
detecting in the biological sample the presence of a mutation in the P2Y2 receptor or in pannexin 11, wherein the mutation leads to a high risk of contracting HIV-1
and, if the mutation is present in the P2Y2 receptor or in pannexin 11, then concluding that the subject is at high risk of contracting HIV-1; or
if the mutation is not present in the P2Y2 receptor or pannexin 11, then concluding that the subject is not at high risk of contracting HIV-1.

### Claim 17 (depends on 16)

The method of claim 16, wherein said step of detecting is carried out by a technique selected from the group consisting of: analysis of restriction fragment length polymorphism, hybridisation techniques, DNA sequencing, exonuclease resistance assays, microsequencing, solid phase extension using ddNTPs, extension in solution using ddNTPs, oligonucleotide assays, detecting single nucleotide polymorphisms, ligation chain reactions, mini-sequencing, the use of DNA “chips”, and allele-specific oligonucleotide hybridisation with single or dual-labeled probes merged with PCR or with molecular beacons.

### Claim 18 (depends on 17)

The method of claim 17, wherein said technique of detecting single nucleotide polymorphism is carried out by dynamic allele-specific hybridization.