# US 10,765,760 B2 · Exon skipping oligomer conjugates for muscular dystrophy

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- Number: US 10,765,760 B2
- Document ID: US10765760B2
- Status: Granted
- Kind: B2
- Application number: 15/993,287
- Filed: 2018-05-30
- Published: 2019-12-05
- Granted: 2020-09-08
- Earliest claimed: 2018-05-29
- Assignees: Sarepta Therapeutics, Inc.
- Applicants: Sarepta Therapeutics, Inc.
- Inventors: Gunnar J. Hanson; Marco A. Passini; Frederick Joseph Schnell
- Web page: https://patentgrep.com/patent/US10765760B2

## CPC

- A61K 47/6807: {the drug or compound being a sugar, nucleoside, nucleotide, nucleic acid, e.g. RNA antisense}
- A61K 9/0019: {Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner (non-active ingredients are additionally classified in A61K47/00)}
- A61K 47/6455: {Polycationic oligopeptides, polypeptides or polyamino acids, e.g. for complexing nucleic acids}
- C07K 14/4707: {Muscular dystrophy}
- C12N 15/111: {General methods applicable to biologically active non-coding nucleic acids}
- 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)}
- C12N 15/62: DNA sequences coding for fusion proteins
- C12N 2310/11: Antisense
- C12N 2310/3233: Morpholino-type ring
- C12N 2310/3513: Protein; Peptide
- C12N 2320/33: Alteration of splicing
- 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)

## Abstract

Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 52 skipping are described.

## Claims

### Claim 1

An antisense oligomer conjugate of Formula (IV):
or a pharmaceutically acceptable salt thereof.

### Claim 2 (depends on 1)

The antisense oligomer conjugate of claim 1, wherein the antisense oligomer is of Formula (IVA):

### Claim 3 (depends on 1)

A pharmaceutical composition, comprising an antisense oligomer conjugate of claim 1, or a pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable carrier.

### Claim 4 (depends on 2)

A pharmaceutical composition, comprising an antisense oligomer conjugate of claim 2, or a pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable carrier.