# US 2014/0194359 A1 · Interfering rna delivery system and uses thereof

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- Number: US 2014/0194359 A1
- Document ID: US20140194359A1
- Status: Published application
- Kind: A1
- Application number: 14/156,227
- Filed: 2014-01-15
- Published: 2014-07-10
- Earliest claimed: 2012-01-06
- Assignees: Alcon Research, Ltd.
- Applicants: Alcon Research, Ltd.
- Inventors: Ying Yu; Jon E. Chatterton
- Web page: https://patentgrep.com/patent/US20140194359A1

## CPC

- 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)}
- A61K 31/7088: Compounds having three or more nucleosides or nucleotides
- A61K 47/645: {Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT}
- A61K 48/0025: {wherein the non-active part clearly interacts with the delivered nucleic acid}
- A61K 48/0033: {the non-active part being non-polymeric}
- C12N 15/111: {General methods applicable to biologically active non-coding nucleic acids}
- C12N 15/87: Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N 15/1137: {against enzymes (viral enzymes C12N15/1131; receptors C12N15/1138)}
- C12N 2310/14: interfering nucleic acids [NA]
- C12N 2310/3513: Protein; Peptide
- C12N 2320/32: Special delivery means, e.g. tissue-specific
- C12Y 502/01008: Peptidylprolyl isomerase (5.2.1.8), i.e. cyclophilin
- 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)
- C12Y: ENZYMES

## Abstract

The invention provides a delivery system comprising a cell penetrating peptide, 10 histidines, and an interfering RNA molecule. The system can be used for delivering interfering RNA molecules into a cell in vivo or in vitro. Therapeutic uses for the delivery system are also provided.

## Claims

### Claim 1

An interfering RNA delivery system comprising an interfering RNA molecule linked to a YM3-10H peptide.

### Claim 2 (depends on 1)

The delivery system of claim 1, wherein the interfering RNA molecule is linked to the YM3-10H peptide via a covalent bond.

### Claim 3 (depends on 1)

The delivery system of claim 1, wherein the YM3-10H peptide comprises:
(SEQ ID NO: 2)
CHHHHHTHRLPRRRRRRHHHHHC;
(SEQ ID NO: 3)
HHHHHTHRLPRRRRRRHHHHHC;
(SEQ ID NO: 4)
THRLPRRRRRRHHHHHHHHHHC;
(SEQ ID NO: 5)
GGTHRLPRRRRRRHHHHHHHHHHC;
(SEQ ID NO: 6)
THRLPRRRRRRGGHHHHHHHHHHC;
(SEQ ID NO: 7)
THRLPRRRRRRHHHHHGGHHHHHC;
(SEQ ID NO: 8)
THRLPRRRRRRHHHHHHHHHHC;
or
(SEQ ID NO: 9)
CTHRLPRRRRRRHHHHHHHHHHC.

### Claim 4 (depends on 1)

The delivery system of claim 1, wherein the interfering RNA molecule is a siRNA, miRNA, or shRNA.

### Claim 5 (depends on 1)

A pharmaceutical composition comprising the delivery system of claim 1 and a pharmaceutically acceptable carrier.

### Claim 6 (depends on 1)

A method of treating or preventing an ocular disorder in a patient, comprising administering to the patient the interfering RNA delivery system of claim 1, wherein the interfering RNA molecule can attenuate expression of a gene associated with the ocular disorder.

### Claim 7 (depends on 6)

The method of claim 6, wherein the ocular disorder is associated with ocular angiogenesis, dry eye, ocular inflammatory conditions, ocular hypertension, or glaucoma.

### Claim 8 (depends on 7)

The method of claim 7, wherein the system is administered by intraocular injection, ocular topical application, subconjunctival injection, intravitreal injection, anterior or posterior juxtascleral injection, intravenous injection, oral administration, intramuscular injection, intraperitoneal injection, transdermal application, intranasal application, or transmucosal application.

### Claim 9

A method of delivering an interfering RNA molecule into a cell, comprising:
(a) attaching an interfering RNA molecule to a YM3-10H peptide, thereby forming an interfering RNA delivery system; and
(b) administering the interfering RNA delivery system to the cell under conditions suitable for the system to enter the cell.

### Claim 10 (depends on 9)

The method of claim 9, wherein the interfering RNA molecule is linked to the YM3-10H peptide via a covalent bond.

### Claim 11 (depends on 13)

The method of claim 13, wherein the interfering RNA molecule is a siRNA, miRNA, or shRNA.

### Claim 12 (depends on 9)

The method of claim 9, wherein the YM3-10H peptide comprises:
(SEQ ID NO: 2)
CHHHHHTHRLPRRRRRRHHHHHC;
(SEQ ID NO: 3)
HHHHHTHRLPRRRRRRHHHHHC;
(SEQ ID NO: 4)
THRLPRRRRRRHHHHHHHHHHC;
(SEQ ID NO: 5)
GGTHRLPRRRRRRHHHHHHHHHHC;
(SEQ ID NO: 6)
THRLPRRRRRRGGHHHHHHHHHHC;
(SEQ ID NO: 7)
THRLPRRRRRRHHHHHGGHHHHHC;
(SEQ ID NO: 8)
THRLPRRRRRRHHHHHHHHHHC;
or
(SEQ ID NO: 9)
CTHRLPRRRRRRHHHHHHHHHHC.