# US 10,370,663 B2 · Delivery of therapeutic agent

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- Number: US 10,370,663 B2
- Document ID: US10370663B2
- Status: Granted
- Kind: B2
- Application number: 15/824,793
- Filed: 2017-11-28
- Published: 2018-05-17
- Granted: 2019-08-06
- Earliest claimed: 2012-11-13
- Assignees: Codiak BioSciences, Inc.
- Applicants: Codiak BioSciences, Inc.
- Inventors: Jan Lötvall; Jonas Andrej Nilsson
- Web page: https://patentgrep.com/patent/US10370663B2

## CPC

- C12N 15/1135: {against oncogenes or tumor suppressor genes}
- A61P 1/04: for ulcers, gastritis or reflux esophagitis, e.g. antacids, inhibitors of acid secretion, mucosal protectants
- A61P 1/16: for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
- A61P 11/00: Drugs for disorders of the respiratory system
- A61P 11/02: Nasal agents, e.g. decongestants
- A61P 13/08: of the prostate
- A61P 13/12: of the kidneys
- A61P 15/00: Drugs for genital or sexual disorders (for disorders of sex hormones A61P5/24); Contraceptives
- A61P 17/00: Drugs for dermatological disorders
- A61P 21/00: Drugs for disorders of the muscular or neuromuscular system
- A61P 25/00: Drugs for disorders of the nervous system
- A61P 35/00: Antineoplastic agents
- A61P 35/02: specific for leukemia
- A61P 43/00: Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
- C12N 15/111: {General methods applicable to biologically active non-coding nucleic acids}
- C12N 15/85: for animal cells
- C12N 2310/14: interfering nucleic acids [NA]
- C12N 2320/30: Special therapeutic applications
- 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

## Abstract

A method of producing nanovesicles comprising an oligonucleotide inhibitor to an oncogene or a proto-oncogene or the gene product thereof, said method comprises a) introducing a DNA sequence encoding an oligonucleotide capable of inhibiting a human oncogenic or proto-oncogenic transcription factor, into a mammalian cell; b) allowing the cell to express said inhibitor oligonucleotide; and c) obtaining nanovesicles containing said inhibitor oligonucleotide from said cell. Nanovesicles produced by the claimed method can be effectively and specifically targeted to e.g. cancer cells to deliver the inhibitor oligonucleotide.

## Claims

### Claim 1

An isolated nanovesicle comprising a recombinantly expressed c-Myc inhibitor oligonucleotide, and further comprising a targeting molecule on the surface of the nanovesicle, wherein said targeting molecule is an antibody or fab-fragment thereof, and wherein the nanovesicle is produced by a producer cell recombinantly expressing L-myc.

### Claim 2 (depends on 1)

An isolated nanovesicle according to claim 1, wherein the isolated nanovesicle further comprises recombinant L-myc.

### Claim 3 (depends on 1)

An isolated nanovesicle of claim 1, wherein said oligonucleotide is an RNA molecule.

### Claim 4 (depends on 3)

An isolated nanovesicle of claim 3, wherein said oligonucleotide is an RNAi molecule.

### Claim 5 (depends on 4)

An isolated nanovesicle of claim 4, wherein said RNA is shRNA or siRNA comprising a sequence selected from the group consisting of SEQ ID NO: 93-102.

### Claim 6 (depends on 1)

An isolated nanovesicle of claim 1 which is an artificial nanovesicle produced by serial extrusions of cells through microfilters and/or nanofilters.

### Claim 7 (depends on 1)

An isolated nanovesicle of claim 1 and a pharmaceutically acceptable carrier.