# US 2022/0033461 A1 · Antigen receptors and uses thereof

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- Number: US 2022/0033461 A1
- Document ID: US20220033461A1
- Status: Published application
- Kind: A1
- Application number: 17/478,906
- Filed: 2021-09-18
- Published: 2022-02-03
- Earliest claimed: 2015-10-07
- Applicants: Biontech Cell & Gene Therapies GmbH; Tron - Translationale Onkologie An Der Universitatsmedizin Der Johannes Gutenberg-Universitat Mainz
- Inventors: Ralf Holger VOSS; Ugur SAHIN; Matthias THEOBALD; Petra SIMON; Matthias BIRTEL
- Web page: https://patentgrep.com/patent/US20220033461A1

## CPC

- C07K 14/7051: {T-cell receptor (TcR)-CD3 complex}
- A61K 35/17: Lymphocytes; B-cells; T-cells; Natural killer cells; Interferon-activated or cytokine-activated lymphocytes (when activated by a specific antigen A61K39/00)
- A61K 40/11: T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cells; Lymphokine-activated killer [LAK] cells
- A61K 40/31: Chimeric antigen receptors [CAR]
- A61K 40/42: Cancer antigens
- A61P 35/00: Antineoplastic agents
- A61P 43/00: Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
- C07K 16/28: against receptors, cell surface antigens or cell surface determinants
- C07K 16/30: from tumour cells
- C07K 16/32: against translation products of oncogenes
- C12N 5/0636: {T lymphocytes}
- A61K 2039/585: {wherein the target is cancer}
- A61K 2239/13: Antibody-based
- A61K 2239/59: Reproductive system, e.g. uterus, ovaries, cervix or testes
- C07K 2317/35: Valency
- C07K 2317/52: Constant or Fc region; Isotype
- C07K 2317/524: CH2 domain
- C07K 2317/526: CH3 domain
- C07K 2317/56: variable (Fv) region, i.e. VH and/or VL
- C07K 2317/622: Single chain antibody (scFv)
- C07K 2317/74: Inducing cell proliferation
- C07K 2319/33: fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
- C12N 2510/00: Genetically modified cells
- 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)
- 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
- 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 generally embraces the treatment of diseases by targeting cells expressing an antigen on the cell surface. In particular the invention relates to recombinant antigen receptors and uses thereof. T cells engineered to express such antigen receptors are useful in the treatment of diseases characterized by expression of one or more antigens bound by the antigen receptors.

## Claims

### Claim at position 1

1-60. (canceled)

### Claim 61

An antigen receptor, which receptor comprises a first peptide chain and a second peptide chain, wherein
the first peptide chain comprises at least a first and a second domain, and an immunoreceptor signal transmission domain;
the second peptide chain comprises at least a first and a second domain, and an immunoreceptor signal transmission domain;
wherein the immunoreceptor signal transmission domain is preferably of human origin,
wherein the first domain from the first peptide chain forms together with one of the domains from the second peptide chain a first antigen binding site, and
wherein the second domain from the first peptide chain forms together with the other domain from the second peptide chain a second antigen binding site.

### Claim 62 (depends on 61)

The receptor of claim 61, wherein the immunoreceptor signal transmission domain comprises a constant or invariant region of a T cell receptor chain or a constant or invariant region of an immune cell Fc receptor chain or a portion of the constant or invariant region.

### Claim 63 (depends on 61)

The receptor of claim 61, wherein the first and/or second peptide chains further comprise a linker between the first and second domains and/or between the first and second domains and the immunoreceptor signal transmission domain.

### Claim 64 (depends on 61)

The receptor of claim 61, wherein the first and/or second domains each comprise a variable region of an immunoglobulin chain or a variable region of a T cell receptor chain or a portion of the variable region.

### Claim 65 (depends on 61)

The receptor of claim 61, wherein
(i) the first peptide chain comprises a constant region of a T cell receptor alpha chain or a portion thereof and the second peptide chain comprises a constant region of a T cell receptor beta chain or a portion thereof, or
(ii) the first peptide chain comprises a constant region of a T cell receptor beta chain or a portion thereof and the second peptide chain comprises a constant region of a T cell receptor alpha chain or a portion thereof.

### Claim 66 (depends on 61)

The receptor of claim 61, wherein the first domain from the first peptide chain comprises a variable region of a heavy chain of an immunoglobulin with a specificity for an antigen or a portion thereof and the domain from the second peptide chain forming an antigen binding site with the first domain from the first peptide chain comprises a variable region of a light chain of an immunoglobulin with a specificity for the antigen or a portion thereof.

### Claim 67 (depends on 61)

The receptor of claim 61, wherein the second domain from the first peptide chain comprises a variable region of a heavy chain of an immunoglobulin with a specificity for an antigen or a portion thereof and the domain from the second peptide chain forming an antigen binding site with the second domain from the first peptide chain comprises a variable region of a light chain of an immunoglobulin with a specificity for the antigen or a portion thereof.

### Claim 68 (depends on 61)

The receptor of claim 61, wherein the first and second antigen binding sites bind to the same antigen or different antigens.

### Claim 69 (depends on 61)

The receptor of claim 61, wherein
the first and the second domains from the first peptide chain each comprise a variable region of a heavy chain of an immunoglobulin or a portion thereof; and
the first and the second domains from the second peptide chain each comprise a variable region of a light chain of an immunoglobulin or a portion thereof.

### Claim 70 (depends on 61)

The receptor of claim 61, wherein
the N-terminal domain from the first peptide chain forms together with the N-terminal domain from the second peptide chain an antigen binding site; and
the C-terminal domain from the first peptide chain forms together with the C-terminal domain from the second peptide chain an antigen binding site.

### Claim 71

A peptide chain comprising a first and a second domain which each comprise a variable region of a heavy chain of an immunoglobulin or each comprise a variable region of a light chain of an immunoglobulin and wherein the peptide chain further comprises an immunoreceptor signal transmission domain.

### Claim 72 (depends on 61)

A recombinant cell expressing both the first and second peptide chains defined in claim 61.

### Claim 73 (depends on 71)

A recombinant cell expressing the peptide chain of claim 71.

### Claim 74

An ex vivo method for producing a cell expressing an antigen receptor which receptor comprises a first peptide chain and a second peptide chain, the method comprising:
(a) providing a cell, preferably a human cell;
(b) providing a first genetic construct encoding the first peptide chain comprising at least a first and a second domain, and an immunoreceptor signal transmission domain;
(c) providing a second genetic construct encoding the second peptide chain comprising at least a first and a second domain and an immunoreceptor signal transmission domain;
(d) introducing the first and second genetic constructs into the cell; and
(e) allowing the constructs to be expressed in the cell;
wherein the cell is preferably a T cell,
wherein the first domain from the first peptide chain is able to form together with one of the domains from the second peptide chain a first antigen binding site, and
wherein the second domain from the first peptide chain is able to form together with the other domain from the second peptide chain a second antigen binding site,
wherein the first peptide chain and the second peptide chain are preferably provided on a single genetic construct.

### Claim 75 (depends on 61)

A nucleic acid encoding both the first and second peptide chains defined in claim 61, wherein the nucleic acid is preferably DNA or RNA.

### Claim 76 (depends on 71)

A nucleic acid encoding the peptide chain of claim 71, wherein the nucleic acid is preferably DNA or RNA.

### Claim 77 (depends on 61, 72, 75)

A pharmaceutical composition comprising
(a) the antigen receptor of claim 61,
(b) the recombinant cell of claim 72,
(c) a nucleic acid of claim 75, or
(d) a nucleic acid encoding the first peptide chain and a nucleic acid encoding the second peptide chain, said chains being defined as in claim 61;
and a pharmaceutically acceptable carrier, preferably for use as a medicament.

### Claim 78 (depends on 61, 72, 75)

A pharmaceutical composition comprising the antigen receptor of claim 61, the recombinant cell of claim 72, or the nucleic acid of claim 75; and a pharmaceutically acceptable carrier for use in the treatment of a disease characterized by expression of at least one antigen which is bound by the antigen receptor, wherein the disease is preferably a cancer disease.
