Extracellular vesicle-aso constructs targeting cebp/beta
AssigneeLonza Sales AG
InventorsDalia BURZYN, Sushrut KAMERKAR
Life of this patent
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Abstract
The present disclosure relates to extracellular vesicles, e.g., exosomes, comprising an antisense oligonucleotide (ASO), wherein the ASO comprises a contiguous nucleotide sequence of 10 to 30 nucleotides in length that is complementary to a nucleic acid sequence within a CEBP/β transcript. Also provided herein are methods for producing the exosomes and methods for using the exosomes to treat and/or prevent diseases or disorders.
Claims143
An extracellular vesicle comprising an antisense oligonucleotide (ASO) which comprises a contiguous nucleotide sequence of 10 to 30 nucleotides in length that is complementary to a nucleic acid sequence within nucleotides 1800-1900 of a CEBP/β transcript corresponding to a nucleotide sequence as set forth in SEQ ID NO:
- 11;
- wherein the extracellular vesicle selectively delivers ASO to myeloid-derived suppressor cells (MDSCs) and other myeloid cells;
- wherein the ASO is capable of down-regulating expression of the CEBP/β mRNA or CEBP/β protein;
- and
- wherein the down-regulating CEBP/β expression can promote the immune-modulation of MDSCs and other myeloid cells to a pro-inflammatory phenotype.
An extracellular vesicle comprising an antisense oligonucleotide (ASO) which comprises a contiguous nucleotide sequence of 10 to 30 nucleotides in length that is complementary to a nucleic acid sequence within nucleotides 1500-2106, nucleotides 1600-2106, nucleotides 1700-2106, nucleotides 1800-2106, nucleotides 1500-2000, nucleotides 1500-1900, nucleotides 1600-2100, nucleotides 1700-2000, or nucleotides 1800-1900 of a CEBP/β transcript corresponding to the nucleic acid sequence set forth in SEQ ID NO:
- 11.
An extracellular vesicle comprising an antisense oligonucleotide (ASO) which comprises a contiguous nucleotide sequence of 10 to 30 nucleotides in length that is complementary to a nucleic acid sequence within nucleotides 995-1014 of a CEBP/β transcript corresponding to a nucleotide sequence as set forth in SEQ ID NO:
- 11.
An antisense oligonucleotide (ASO), comprising a contiguous nucleotide sequence of 10 to 30 nucleotides in length that is complementary to a nucleic acid sequence within nucleotides 1438-2106 of a CEBP/β transcript corresponding to a nucleotide sequence as set forth in SEQ ID NO:
- 11.
An ASO comprising a contiguous nucleotide sequence of 10 to 30 nucleotides in length that is complementary to a nucleic acid sequence within nucleotides 995-1014 of a CEBP/β transcript corresponding to a nucleotide sequence as set forth in SEQ ID NO:
- 11.
A pharmaceutical composition comprising the extracellular vesicle of any one of claims 2 to 94 and 132, the ASO of any one of claims 79 to 113, or the conjugate of claim 114 or 115, and a pharmaceutically acceptable diluent, carrier, salt, or adjuvant.
Dependencies are inferred from the claim text.