# US 12,760,083 B2 · Chimeric receptor therapy

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- Number: US 12,760,083 B2
- Document ID: US12760083B2
- Status: Granted
- Kind: B2
- Application number: 17/572,618
- Filed: 2022-01-10
- Published: 2022-07-14
- Granted: 2026-10-13
- Earliest claimed: 2021-01-11
- Applicants: Precigen, Inc.
- Inventors: Helen Sabzevari; Cheryl G. Bolinger; Rutul R. Shah; ChangHung Chen; Vinodhbabu Kurella; Amy Wesa
- Web page: https://patentgrep.com/patent/US12760083B2

## CPC

- C07K 14/7051: {T-cell receptor (TcR)-CD3 complex}
- 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 40/36: Immune checkpoint inhibitors
- A61K 40/4202: {Receptors, cell surface antigens or cell surface determinants}
- A61K 40/421: {Immunoglobulin superfamily}
- A61K 40/4257: {Mucins, e.g. MUC-1}
- A61P 35/00: Antineoplastic agents
- C07K 16/2803: {against the immunoglobulin superfamily}
- A61K 2039/505: {comprising antibodies}
- A61K 40/50: characterised by the use of allogeneic cells
- A61K 2239/28: Expressing multiple CARs, TCRs or antigens
- A61K 2239/31: characterized by the route of administration
- A61K 2239/38: characterised by the dose, timing or administration schedule
- A61K 2239/48: Blood cells, e.g. leukemia or lymphoma
- A61K 2239/49: Breast
- A61K 2239/54: Pancreas
- A61K 2239/59: Reproductive system, e.g. uterus, ovaries, cervix or testes
- C07K 2317/21: from primates, e.g. man
- C07K 2317/565: Complementarity determining region [CDR]
- C07K 2317/622: Single chain antibody (scFv)
- C07K 2319/02: containing a signal sequence
- C07K 2319/03: containing a transmembrane segment
- C07K 2319/33: fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
- 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

## Abstract

A non-naturally occurring polynucleotide encoding a miRNA that inhibits the expression of an immune checkpoint protein. The polynucleotide may further encode a chimeric receptor, a cytokine, and/or a cell tag. A vector comprising the aforementioned polynucleotide. A modified immune effector cell comprising the aforementioned polynucleotide. Compositions and kits comprising the aforementioned polynucleotide and/or cell. A method for treating a subject suffering from a disease or disorder, comprising administering the aforementioned cell to a subject in need thereof. The use of the aforementioned cell in the manufacture of a medicament for the treatment of a disease or disorder. A method for the detection of a disease or disorder associated with the overexpression of an antigen in a subject. A method for the treatment of a disease or disorder comprising the serial administration of polynucleotides encoding a chimeric antigen receptor or a cell comprising the same.

## Claims

### Claim 1

A non-naturally occurring polynucleotide comprising:
(a) a first nucleic acid sequence comprising SEQ ID NO: 179;
(b) a second nucleic acid sequence comprising SEQ ID NO: 180; and
(c) a third nucleic acid sequence encoding a chimeric antigen receptor (CAR), the CAR comprising:
(i) a variable light chain (VL) domain comprising the amino acid sequence set forth in SEQ ID NO: 387,
(ii) a variable heavy chain (VH) domain comprising the amino acid sequence set forth in SEQ ID NO: 349.

### Claim 2 (depends on 1)

The polynucleotide of claim 1, further encoding a cytokine.

### Claim 3 (depends on 1)

The polynucleotide of claim 1, further encoding a fusion protein comprising: (a) a polypeptide comprising the amino acid sequence set forth in SEQ ID NO: 519; and (b) a polypeptide comprising the amino acid sequence set forth in SEQ ID NO: 521.

### Claim 4 (depends on 1)

The polynucleotide of claim 1, further encoding a cell tag.

### Claim 5 (depends on 4)

The polynucleotide of claim 4, wherein the cell tag comprises: (a) a truncated HER1; and (b) a CD28 transmembrane domain.

### Claim 6 (depends on 1)

The polynucleotide of claim 1, wherein the first and second nucleic acid sequences are contained within the polynucleotide sequence set forth in SEQ ID NO: 267.

### Claim 7 (depends on 1)

The polynucleotide of claim 1, wherein the CAR comprises the amino acid sequence set forth in SEQ ID NO: 465.

### Claim 8 (depends on 1)

The polynucleotide of claim 1, wherein the CAR comprises the amino acid sequence set forth in SEQ ID NO: 623.

### Claim 9 (depends on 1)

A vector comprising the polynucleotide of claim 1.

### Claim 10 (depends on 9)

The vector of claim 9, further comprising a Sleeping Beauty transposon.

### Claim 11 (depends on 1)

An in vitro modified immune effector cell comprising the polynucleotide of claim 1.

### Claim 12 (depends on 1)

A composition comprising the polynucleotide of claim 1.

### Claim 13 (depends on 1)

A kit comprising the polynucleotide of claim 1.
