WO2023086935A3 - Aptamer-based small ribonucleic acid delivery platform and uses thereof - Google Patents

Aptamer-based small ribonucleic acid delivery platform and uses thereof Download PDF

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Publication number
WO2023086935A3
WO2023086935A3 PCT/US2022/079711 US2022079711W WO2023086935A3 WO 2023086935 A3 WO2023086935 A3 WO 2023086935A3 US 2022079711 W US2022079711 W US 2022079711W WO 2023086935 A3 WO2023086935 A3 WO 2023086935A3
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WIPO (PCT)
Prior art keywords
rna
aptamer
oligonucleotides
delivery platform
based small
Prior art date
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PCT/US2022/079711
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French (fr)
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WO2023086935A2 (en
Inventor
Toshihiko TANNO
Yang Liu
Pan Zheng
Martin DEVENPORT
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OncoC4, Inc.
University Of Maryland, Baltimore
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Application filed by OncoC4, Inc., University Of Maryland, Baltimore filed Critical OncoC4, Inc.
Publication of WO2023086935A2 publication Critical patent/WO2023086935A2/en
Publication of WO2023086935A3 publication Critical patent/WO2023086935A3/en

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    • C—CHEMISTRY; METALLURGY
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C12N2310/141—MicroRNAs, miRNAs
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/16—Aptamers
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Genetics & Genomics (AREA)
  • Health & Medical Sciences (AREA)
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  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
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Abstract

Provided herein is a composition comprising an aptamer-based small RNA delivery platform comprising: an aptamer portion comprising a DNA aptamer linked to a first passenger RNA portion; a guide strand RNA of a microRNA (miRNA); and, a second passenger RNA portion conjugated to a cholesterol, wherein the three oligonucleotides assemble through complementary base pair annealing and the hydrophobic cholesterol forms a self-assembled micelle-like nanoparticle with hydrophilic RNA oligonucleotides linked with DNA aptamers on its surface. The aptamer binds to a cell surface molecule for targeted delivery of RNA therapeutics to specific cell surface markers, and once inside the cell, the nanoparticles induce lysosomal leakage, resulting in release of the RNA oligonucleotides into the cytosol, thereby achieving gene silencing.
PCT/US2022/079711 2021-11-12 2022-11-11 Aptamer-based small ribonucleic acid delivery platform and uses thereof WO2023086935A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163278868P 2021-11-12 2021-11-12
US63/278,868 2021-11-12

Publications (2)

Publication Number Publication Date
WO2023086935A2 WO2023086935A2 (en) 2023-05-19
WO2023086935A3 true WO2023086935A3 (en) 2024-03-28

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PCT/US2022/079711 WO2023086935A2 (en) 2021-11-12 2022-11-11 Aptamer-based small ribonucleic acid delivery platform and uses thereof

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WO (1) WO2023086935A2 (en)

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160208245A1 (en) * 2015-01-21 2016-07-21 Korea Institute Of Science And Technology RNA/DNA nanoparticle for siRNA target-specific delivery and vehicle including the same

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160208245A1 (en) * 2015-01-21 2016-07-21 Korea Institute Of Science And Technology RNA/DNA nanoparticle for siRNA target-specific delivery and vehicle including the same

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
BYEONGGEOL MUN,EUNJI JANG, SEUNGMIN HAN, HYE YOUNG SON, YUNA CHOI, YONG-MIN HUH, SEUNGJOO HAAM: "Efficient Self-Assembled MicroRNA Delivery System Consisting of Cholesterol-Conjugated MicroRNA and PEGylated Polycationic Polymer for Tumor Treatment", ACS APPLIED BIO MATERIALS, vol. 2, no. 5, 20 May 2019 (2019-05-20), US , pages 2219 - 2228, XP093155206, ISSN: 2576-6422, DOI: 10.1021/acsabm.9b00186 *
TOSHIHIKO TANNO: "A novel aptamer-based small RNA delivery platform and its application to cancer therapy", GENES & DISEASES, vol. 10, no. 3, 1 May 2023 (2023-05-01), NL , pages 1075 - 1089, XP093155209, ISSN: 2352-3042, DOI: 10.1016/j.gendis.2022.05.004 *
Y. WU, K. SEFAH, H. LIU, R. WANG, W. TAN: "DNA aptamer-micelle as an efficient detection/delivery vehicle toward cancer cells", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES, vol. 107, no. 1, 5 January 2010 (2010-01-05), pages 5 - 10, XP055423110, ISSN: 0027-8424, DOI: 10.1073/pnas.0909611107 *

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