# US 12,600,947 B2 · Composition for reprogramming cells into plasmacytoid dendritic cells or interferon type I-producing cells, methods and uses thereof

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- Number: US 12,600,947 B2
- Document ID: US12600947B2
- Status: Granted
- Kind: B2
- Application number: 17/766,369
- Filed: 2020-10-09
- Published: 2024-02-15
- Granted: 2026-04-14
- Earliest claimed: 2019-10-10
- Assignees: Asgard Therapeutics AB
- Applicants: Asgard Therapeutics AB
- Inventors: Carlos Filipe Ribeiro Lemos Pereira; Cristiana Ferreira Pires; Fábio Fiúza Rosa; Abigail Altman
- Web page: https://patentgrep.com/patent/US12600947B2

## CPC

- C07K 14/4702: {Regulators; Modulating activity}
- C12N 5/0639: {Dendritic cells, e.g. Langherhans cells in the epidermis}
- A61K 38/1709: {from mammals}
- A61K 40/19: Dendritic cells
- A61K 40/22: Immunosuppressive or immunotolerising
- A61K 40/24: Antigen-presenting cells [APC]
- A61K 40/416: {Antigens related to auto-immune diseases; Preparations to induce self-tolerance}
- A61K 40/42: Cancer antigens
- A61K 40/46: Viral antigens
- C12N 15/86: Viral vectors
- C12N 2501/60: Transcription factors
- C12N 2506/00: Differentiation of animal cells from one lineage to another; Differentiation of pluripotent 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)
- 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)

## Abstract

The present disclosure relates to compositions, constructs and vectors for reprogramming cells into plasmacytoid dendritic cells or interferon type I-producing cells, methods and uses thereof. The present disclosure relates to the development of methods for making plasmacytoid dendritic cells or interferon type I-producing cells that promote antiviral and anti-tumoral immune responses from differentiated, multipotent or pluripotent stem cells by introducing and expressing isolated/synthetic transcription factors. More particularly, the disclosure provides methods for obtain plasmacytoid dendritic cells or interferon type I-producing cells by direct cellular reprogramming with the surprisingly use of combinations of specific transcription factors.

## Claims

### Claim 1

A construct or a vector encoding a combination of at least three transcription factors, wherein the combination of transcription factors comprises IRF8, SPIB, and IKZF2, wherein said construct or vector is a viral vector, a plasmid, or an RNA vector.

### Claim 2 (depends on 1)

The construct or vector according to claim 1, wherein the vector is a viral vector.

### Claim 3 (depends on 2)

The construct or vector according to claim 2, wherein the viral vector is selected from the group consisting of: a retroviral, adenoviral, lentiviral, herpes viral, pox viral, paramyxoviral, rhabdoviral, alphaviral, flaviral and adeno-associated viral vector.

### Claim 4 (depends on 1)

The construct or vector according to claim 1, wherein the construct or vector is synthetic mRNA, naked alphavirus RNA replicons or naked flavivirus RNA replicons.

### Claim 5 (depends on 1)

The construct or vector according to claim 1, wherein the construct or vector further codes for one or more other transcription factors selected from the group consisting of: ARID5A, BCL11A, CBFA2T3, CREB3L2, ETS1, IKZF1, STAT1, TCF4, TCF12, and TSC22D1.

### Claim 6 (depends on 5)

The construct or vector according to claim 5, wherein the transcription factors are selected from:
a. IRF8 comprising a sequence having at least 95% identity with SEQ. ID. NO. 1 or SEQ. ID. NO. 2,
b. SPIB comprising a sequence having at least 95% identity with SEQ. ID. NO. 3 or SEQ. ID. NO. 4,
c. ARID5A comprising a sequence having at least 95% identity with SEQ. ID. NO. 5 or SEQ. ID. NO. 6,
d. BCL11A comprising a sequence having at least 95% identity with SEQ. ID. NO. 7 or SEQ. ID. NO. 8,
e. CBFA2T3 comprising a sequence having at least 95% identity with SEQ. ID. NO. 9 or SEQ. ID. NO. 10,
f. CREB3L2 comprising a sequence having at least 95% identity with SEQ. ID. NO. 11 or SEQ. ID. NO. 12,
g. ETS1 comprising a sequence having at least 95% identity with SEQ. ID. NO. 13 or SEQ. ID. NO. 14,
h. IKZF1 comprising a sequence having at least 95% identity with SEQ. ID. NO. 23 or SEQ. ID. NO. 24,
i. IKZF2 comprising a sequence having at least 95% identity with SEQ ID NO. 25 or SEQ ID NO. 26,
j. TCF4 comprising a sequence having at least 95% identity with SEQ. ID. NO. 41 or SEQ. ID. NO. 42,
k. TCF12 comprising a sequence having at least 95% identity with SEQ. ID. NO. 43 or SEQ. ID. NO. 44,
l. STAT1 comprising a sequence having at least 95% identity with SEQ. ID. NO. 45 or SEQ. ID. NO. 46, or
m. TSC22D1 comprising a sequence having at least 95% identity with SEQ. ID. NO. 47 or SEQ. ID. NO. 48.
