# US 10,479,996 B2 · Antisense antiviral compound and method for treating ss/RNA viral infection

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- Number: US 10,479,996 B2
- Document ID: US10479996B2
- Status: Granted
- Kind: B2
- Application number: 14/535,092
- Filed: 2014-11-06
- Published: 2015-09-24
- Granted: 2019-11-19
- Earliest claimed: 2004-09-16
- Assignees: Sarepta Therapeutics, Inc.
- Applicants: Sarepta Therapeutics, Inc.
- Inventors: Patrick L. Iversen; David A. Stein; Dwight D. Weller
- Web page: https://patentgrep.com/patent/US10479996B2

## CPC

- C12N 15/1131: {against viruses}
- A61P 31/12: Antivirals
- A61P 31/14: for RNA viruses
- C07K 19/00: Hybrid peptides {, i.e. peptides covalently bound to nucleic acids, or non-covalently bound protein-protein complexes}
- C12N 15/111: {General methods applicable to biologically active non-coding nucleic acids}
- C12N 2310/11: Antisense
- C12N 2310/314: Phosphoramidates
- C12N 2310/3233: Morpholino-type ring
- C12N 2310/3513: Protein; Peptide
- C12N 2310/531: Stem-loop; Hairpin
- C12N 2320/11: for the determination of target sites, i.e. of active nucleic acids
- C12N 2770/00011: Details
- C12N 2770/10011: Arteriviridae
- C12N 2770/12011: Astroviridae
- C12N 2770/16011: Caliciviridae
- C12N 2770/20011: Coronaviridae
- C12N 2770/24011: Flaviviridae
- C12N 2770/28011: Hepeviridae
- C12N 2770/32011: Picornaviridae
- C12N 2770/36011: Togaviridae
- Y02A 50/30: Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- 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)
- Y02A: TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE

## Abstract

The invention provides antisense antiviral compounds and methods of their use and production in inhibition of growth of viruses of the Flaviviridae, Picornaviridae, Caliciviridae, Togaviridae, Arteriviridae, Coronaviridae, Astroviridae and Hepeviridae families in the treatment of a viral infection. The antisense antiviral compounds are substantially uncharged morpholino oligonucleotides having a sequence of 12-40 subunits, including at least 12 subunits having a targeting sequence that is complementary to a region associated with stem-loop secondary structure within the 5′-terminal end 40 bases of the positive-sense RNA strand of the virus.

## Claims

### Claim 1

A morpholino subunit of formula:
or a salt or isomer thereof, wherein:
B is a base-pairing moiety;
Ri is C1-C12 alkylcarbonyl;
R2 is C7-C30 aralkyl; and
Z is halo.

### Claim 2 (depends on 1)

The morpholino subunit of claim 1, wherein Ri is trifluoroacetyl.

### Claim 3 (depends on 1)

The morpholino subunit of claim 1, wherein R2 is trityl.

### Claim 4 (depends on 1)

The morpholino subunit of claim 1, wherein R2 is 4-methoxytrityl.

### Claim 5 (depends on 1)

The morpholino subunit of claim 1, wherein Z is chloro.

### Claim 6 (depends on 1)

The morpholino subunit of claim 1, wherein B is adenine, cytosine, guanine, uracil, thymine, or hypoxanthine.

### Claim 7 (depends on 1)

The morpholino subunit of claim 1, wherein B comprises one or more protecting groups.

### Claim 8 (depends on 1)

The morpholino subunit of claim 1, wherein B is adenine, cytosine, guanine, uracil, thymine or hypoxanthine, each of which comprise at least one protecting group.

### Claim 9 (depends on 1)

The morpholino subunit of claim 1, wherein the morpholino subunit is of formula:
or a salt or isomer thereof, wherein B is a base-pairing moiety.