# US 2013/0158102 A1 · Compositions and Methods for Inhibiting Expression of a Gene from the JC Virus

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- Number: US 2013/0158102 A1
- Document ID: US20130158102A1
- Status: Published application
- Kind: A1
- Application number: 13/779,116
- Filed: 2013-02-27
- Published: 2013-06-20
- Earliest claimed: 2006-04-28
- Assignees: Alnylam Pharmaceuticals, Inc.
- Inventors: Pamela Tan; Dinah Sah; Birgit Bramlage
- Web page: https://patentgrep.com/patent/US20130158102A1

## CPC

- C12N 15/1131: {against viruses}
- A61K 48/005: {characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered}
- A61P 25/28: for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
- A61P 31/12: Antivirals
- A61P 31/20: for DNA viruses
- C07H 21/02: with ribosyl as saccharide radical
- C12N 2310/14: interfering nucleic acids [NA]
- C12N 2310/315: Phosphorothioates
- C12N 2310/321: 2'-O-R Modification
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)
- 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
- C07H: SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS (derivatives of aldonic or saccharic acids C07C, C07D; aldonic acids, saccharic acids C07C59/105, C07C59/285; cyanohydrins C07C255/16; glycals C07D; compounds of unknown constitution C07G; polysaccharides, derivatives thereof C08B; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification C12N15/00; sugar industry C13)

## Abstract

The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a gene from the JC Virus (JC virus genome), comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of a gene from the JC Virus. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by JC virus expression and the expression of a gene from the JC Virus using the pharmaceutical composition; and methods for inhibiting the expression of a gene from the JC Virus in a cell.

## Claims

### Claim 1

A double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a human JC virus genome in a cell, wherein said dsRNA comprises at least two sequences that are complementary to each other and wherein a sense strand comprises a first sequence and an antisense strand comprises a second sequence comprising a region of complementarity which is substantially complementary to at least a part of a mRNA encoding JC virus, and wherein said region of complementarity is less than 30 nucleotides in length and wherein said dsRNA, upon contact with a cell expressing said JC virus, inhibits expression of said JC virus genome.

### Claim 2 (depends on 1)

The dsRNA of claim 1, wherein said first sequence is selected from the group consisting of Tables 1a and 1b and said second sequence is selected from the group consisting of Tables 1a and 1b.

### Claim 3 (depends on 1)

The dsRNA of claim 1, wherein at least one nucleotide of said first sequence or said second sequence is a modified nucleotide.

### Claim 4 (depends on 2)

The dsRNA of claim 2, wherein at least one nucleotide of said first sequence or said second sequence is a modified nucleotide.

### Claim 5 (depends on 3)

The dsRNA of claim 3, wherein said modified nucleotide is chosen from the group of: a 2′-O-methyl modified nucleotide, a nucleotide comprising a 5′-phosphorothioate group, and a terminal nucleotide linked to a cholesteryl derivative or dodecanoic acid bisdecylamide group.

### Claim 6 (depends on 3)

The dsRNA of claim 3, wherein said modified nucleotide is chosen from the group of: a 2′-deoxy-2′-fluoro modified nucleotide, a 2′-deoxy-modified nucleotide, a locked nucleotide, an abasic nucleotide, 2′-amino-modified nucleotide, 2′-alkyl-modified nucleotide, morpholino nucleotide, a phosphoramidate, and a non-natural base comprising nucleotide.

### Claim 7 (depends on 3)

The dsRNA of claim 3, wherein said first sequence is selected from the group consisting of Tables 1a and 1b and said second sequence is selected from the group consisting of Tables 1a and 1b.

### Claim 8 (depends on 6)

The dsRNA of claim 6, wherein said first sequence is selected from the group consisting of Tables 1a and 1b and said second sequence is selected from the group consisting of Tables 1a and 1b.

### Claim 9 (depends on 1)

A cell comprising the dsRNA of claim 1.

### Claim 10 (depends on 1)

A pharmaceutical composition for inhibiting the expression of a gene from the JC Virus in an organism, comprising the dsRNA of claim 1 and a pharmaceutically acceptable carrier.

### Claim 11 (depends on 10)

The pharmaceutical composition of claim 10, wherein said first sequence of said dsRNA is selected from the group consisting of Tables 1a and 1b and said second sequence of said dsRNA is selected from the group consisting of Tables 1a and 1b.

### Claim 12 (depends on 10)

The pharmaceutical composition of claim 10, wherein the pharmaceutically acceptable carrier comprises a lipid.

### Claim 13 (depends on 1)

A method for inhibiting the expression of a gene from the JC Virus in a cell, the method comprising:
(a) introducing into the cell the dsRNA of claim 1; and
(b) maintaining the cell produced in step (a) for a time sufficient to obtain degradation of the mRNA transcript of a gene from the JC Virus, thereby inhibiting expression of a gene from the JC Virus in the cell.

### Claim 14 (depends on 1)

A method of treating, preventing or managing pathological processes mediated by JC virus expression comprising administering to a patient in need of such treatment, prevention or management a therapeutically or prophylactically effective amount of the dsRNA of claim 1.

### Claim 15 (depends on 1)

A vector for inhibiting the expression of a gene from the JC Virus in a cell, said vector comprising a regulatory sequence operably linked to a nucleotide sequence that encodes at least one strand of the dsRNA of claim 1.

### Claim 16 (depends on 15)

A cell comprising the vector of claim 15.