# US 8,772,471 B2 · Targeted delivery of siRNA

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- Number: US 8,772,471 B2
- Document ID: US8772471B2
- Status: Granted
- Kind: B2
- Application number: 12/524,536
- Filed: 2008-01-25
- Published: 2010-08-19
- Granted: 2014-07-08
- Earliest claimed: 2007-01-26
- Assignees: Industry-University Cooperation Foundation Hanyang University; Immune Disease Institute, Inc.
- Applicants: Premlata Shankar; Sang-Kyung Lee; Manjunath Narasimhaswamy; Priti Kumar; Haoquan Wu; Hong-Seok Ban
- Inventors: Premlata Shankar; Sang-Kyung Lee; Manjunath Narasimhaswamy; Priti Kumar; Haoquan Wu; Hong-Seok Ban
- Web page: https://patentgrep.com/patent/US8772471B2

## CPC

- C12N 15/111: {General methods applicable to biologically active non-coding nucleic acids}
- A61P 31/12: Antivirals
- A61P 31/18: for HIV
- A61P 35/00: Antineoplastic agents
- C12N 15/1135: {against oncogenes or tumor suppressor genes}
- C12N 15/1136: {against growth factors, growth regulators, cytokines, lymphokines or hormones}
- C12N 15/1138: {against receptors or cell surface proteins}
- C12N 15/115: Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith {; Nucleic acids binding to non-nucleic acids, e.g. aptamers}
- A61K 48/00: Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- C12N 2310/14: interfering nucleic acids [NA]
- C12N 2310/3513: Protein; Peptide
- C12N 2320/32: Special delivery means, e.g. tissue-specific
- 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
- 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)

## Abstract

The present invention provides a method of delivering RNA interference molecules to a cell or a cell in a subject, which comprises contacting the cell with a protein-double stranded RNA complex, the complex comprising the double stranded RNA segment containing a double stranded RNA of interest and a protein, the protein comprising (1) a targeting moiety, which will specifically bind to a site on a target cell, and (2) a binding moiety linked thereto, which will bind to the double stranded RNA, wherein the double stranded RNA segment is delivered to a cell and effects RNA interference of the target RNA in the cell.

## Claims

### Claim 1

A composition comprising a targeting moiety associated with a binding moiety, wherein a double stranded RNA segment is associated with the binding moiety, wherein the targeting moiety is an antibody or antigen binding fragment thereof which binds to CD7, and the binding moiety is an oligo-9-peptide (9R), wherein the oligo-9-peptide comprises 9 consecutive arginines.

### Claim 2 (depends on 1)

The composition of claim 1, wherein the targeting moiety specifically binds to a cell-surface antigen on a target cell, wherein the cell surface antigen internalizes when the targeting moiety binds the cell surface antigen.

### Claim 3 (depends on 2)

The composition of claim 2, wherein the target cell is a T-cell.

### Claim 4 (depends on 1)

The composition of claim 1, wherein the targeting moiety and binding moiety are comprised as a fusion protein, wherein the binding moiety is fused to the carboxy portion of the targeting moiety.

### Claim 5 (depends on 2)

The composition of claim 2, wherein the cell is selected from the group consisting of a cultured cell, cell that is part of an organ, a cell that is part of a subject and an embryonic stem cell.

### Claim 6 (depends on 5)

The composition of claim 5, wherein the subject is a human.