# US 10,864,284 B2 · RNA transcription vector and uses thereof

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- Number: US 10,864,284 B2
- Document ID: US10864284B2
- Status: Granted
- Kind: B2
- Application number: 16/106,912
- Filed: 2018-08-21
- Published: 2018-12-13
- Granted: 2020-12-15
- Earliest claimed: 2013-11-12
- Assignees: Vrije Universiteit Brussel
- Applicants: Vrije Universiteit Brussel
- Inventors: Carlo Heirman; Kristiaan Thielemans
- Web page: https://patentgrep.com/patent/US10864284B2

## CPC

- A61K 48/0066: {Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid}
- A61K 31/7105: Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
- A61K 39/0011: {Cancer antigens}
- A61P 1/16: for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
- A61P 31/04: Antibacterial agents
- A61P 31/10: Antimycotics
- A61P 31/12: Antivirals
- A61P 31/18: for HIV
- A61P 35/00: Antineoplastic agents
- A61P 37/08: Antiallergic agents (antiasthmatic agents A61P11/06; ophthalmic antiallergics A61P27/14)
- C07K 14/70575: {NGF/TNF-superfamily, e.g. CD70, CD95L, CD153, CD154 (NGF C07K14/48, TNF C07K14/525)}
- C07K 14/70596: {Molecules with a "CD"-designation not provided for elsewhere}
- C12N 15/85: for animal cells
- A61K 2039/53: {DNA (RNA) vaccination}
- A61K 2039/54: {characterised by the route of administration}
- A61K 2039/55516: {Proteins; Peptides}
- C12N 2320/52: modulating the physical stability, e.g. GC-content
- C12N 2810/10: Vectors comprising a non-peptidic targeting moiety
- C12N 2830/50: regulating RNA stability, not being an intron, e.g. poly A signal
- C12N 2840/105: enhancing translation
- 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)
- 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)
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)

## Abstract

The present invention in general relates to an improved RNA transcription vector, which is very suitable for the production of mRNA for in vivo therapeutic purposes. The improvements in the vector in particular reside in the presence of a transcription enhancer and a nuclear retention element.

## Claims

### Claim 1

An RNA molecule comprising a translation enhancer (TE) which is 89% identical to the nucleotide sequence set forth in SEQ ID NO: 1, a translatable nucleic acid sequence, and a nuclear retention sequence which is identical to the nucleotide sequence set forth in SEQ ID NO: 4.

### Claim 2 (depends on 1)

The RNA molecule according to claim 1, further comprising a poly-A tail.

### Claim 3 (depends on 1)

The RNA molecule according to claim 1, wherein the translatable nucleic acid sequence is selected from the group consisting of mRNA encoding CD40L, mRNA encoding CD70, mRNA encoding caTLR4, and antigen/disease specific mRNA.

### Claim 4 (depends on 1)

The RNA molecule according to claim 1, wherein the translation enhancer is identical to the nucleotide sequence set forth in SEQ ID NO: 1.

### Claim 5 (depends on 1)

A composition comprising one or more of the RNA molecules as claimed in claim 1.

### Claim 6 (depends on 5)

The composition according to claim 5, wherein the one or more RNA molecules represent mRNA molecules which encode CD40L, CD70, caTLR4 or antigen/disease specific mRNA.

### Claim 7 (depends on 1)

A kit comprising one or more of the RNA molecules according to claim 1.

### Claim 8 (depends on 5)

A kit comprising the composition according to claim 5.