# US 11,306,294 B2 · Virus-like particles which can be used in the treatment of autoimmune diseases

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- Number: US 11,306,294 B2
- Document ID: US11306294B2
- Status: Granted
- Kind: B2
- Application number: 16/759,125
- Filed: 2018-10-26
- Published: 2020-11-05
- Granted: 2022-04-19
- Earliest claimed: 2017-10-26
- Assignees: Sorbonne Universite; Inserm (Institut National De La Sante Et De La Recherche Medicale); Assistance Publique-Hopitaux de Paris
- Applicants: Sorbonne Universite; INSERM (Institut National de la Santé et de la Recherche Médicale); Assistance Publique—Hôpitaux de Paris
- Inventors: Bertrand Bellier; David Klatzmann
- Web page: https://patentgrep.com/patent/US11306294B2

## CPC

- C12N 7/00: Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof (preparing medicinal viral antigen or antibody compositions, e.g. virus vaccines, A61K39/00)
- A61K 35/76: Viruses; Subviral particles; Bacteriophages
- A61K 39/0008: {Antigens related to auto-immune diseases; Preparations to induce self-tolerance}
- A61P 29/00: Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
- A61P 37/08: Antiallergic agents (antiasthmatic agents A61P11/06; ophthalmic antiallergics A61P27/14)
- C07K 14/005: from viruses
- C07K 14/70521: {CD28, CD152}
- C07K 14/70578: {NGF-receptor/TNF-receptor superfamily, e.g. CD27, CD30, CD40, CD95 (NGF-receptor C07K14/71, TNF-receptor C07K14/7151)}
- C12N 15/86: Viral vectors
- A61K 2035/122: {for inducing tolerance or supression of immune responses}
- A61K 2039/55505: {Inorganic adjuvants}
- A61K 2039/55533: {IL-2}
- C12N 2740/13023: Virus like particles [VLP]
- C12N 2740/16023: Virus like particles [VLP]
- C12N 2740/16043: viral genome or elements thereof as genetic vector
- 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)
- 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)

## Abstract

The invention relates to a virus-like particle comprising an autoantigen and an immunoregulatory molecule exposed on its surface. The invention also relates to the use of said particle in the treatment of an autoimmune disease.

## Claims

### Claim 1

A virus-like particle comprising an antigen and an immunoregulatory molecule exposed on the surface of the particle, wherein the antigen is an autoantigen, the immunoregulatory molecule is linked to the transmembrane glycoprotein domain of vesicular stomatitis virus (VSV-G) or to the glycophosphatidylinositol (GPI) anchoring domain of the CD59 glycoprotein, and the virus-like particle is a synthetic retroviral particle.

### Claim 2 (depends on 1)

The virus-like particle as claimed in claim 1, wherein the immunoregulatory molecule is an immune checkpoint receptor.

### Claim 3 (depends on 2)

The virus-like particle as claimed in claim 2, wherein the immunoregulatory molecule comprises the extracellular domain of said immune checkpoint receptor.

### Claim 4 (depends on 1)

The virus-like particle as claimed in claim 1, wherein the immunoregulatory molecule is selected from the group consisting of IL-2, IL-10 and TGF-beta.

### Claim 5 (depends on 1)

The virus-like particle as claimed in claim 1 for use in the treatment of an autoimmune disease.

### Claim 6 (depends on 5)

The virus-like particle for use as claimed in claim 5, wherein the autoimmune disease is multiple sclerosis, type 1 diabetes, lupus, an autoimmune thyroid, Crohn's disease, rheumatoid arthritis, celiac disease, myasthenia gravis, autoimmune atrophic gastritis, or autoimmune hepatitis (AIH).

### Claim 7 (depends on 5)

The virus-like particle for use as claimed in claim 5, said particle being in the form of a pharmaceutical composition further comprising a pharmaceutical excipient.

### Claim 8 (depends on 1)

A plasmid or set of plasmid(s) comprising nucleic acid sequence(s) encoding one or more modified viral protein(s) of the virus-like particle as defined in claim 1.

### Claim 9 (depends on 8)

The plasmid or set of plasmid(s) as claimed in claim 8 for use in the treatment of an autoimmune disease.

### Claim 10 (depends on 9)

The plasmid or set of plasmid(s) for use as claimed in claim 9, wherein the autoimmune disease is multiple sclerosis, type 1 diabetes, lupus, autoimmune thyroid, Crohn's disease, rheumatoid arthritis, celiac disease, myasthenia gravis, autoimmune atrophic gastritis or autoimmune hepatitis (AIH).

### Claim 11 (depends on 2)

The virus-like particle as claimed in claim 2, wherein the immunoregulatory molecule is selected from the group consisting of IL-2, IL-10 and TGF-beta.

### Claim 12 (depends on 2)

The virus-like particle as claimed in claim 2 for use in the treatment of an autoimmune disease.

### Claim 13 (depends on 2)

The virus-like particle as claimed in claim 2, wherein the immune checkpoint receptor is selected from the group consisting of CTLA4, PD-1, Tim-3, LAG-3, TIGIT, and OX40.