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WO2024263891A3 - Delivery of rna therapeutics using ring-shaped nucleic acids - Google Patents

Delivery of rna therapeutics using ring-shaped nucleic acids Download PDF

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Publication number
WO2024263891A3
WO2024263891A3 PCT/US2024/034985 US2024034985W WO2024263891A3 WO 2024263891 A3 WO2024263891 A3 WO 2024263891A3 US 2024034985 W US2024034985 W US 2024034985W WO 2024263891 A3 WO2024263891 A3 WO 2024263891A3
Authority
WO
WIPO (PCT)
Prior art keywords
oligonucleotide
ring
delivery
nucleic acids
shaped nucleic
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
PCT/US2024/034985
Other languages
French (fr)
Other versions
WO2024263891A2 (en
Inventor
Sudhir Agrawal
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Arnay Sciences LLC
Original Assignee
Arnay Sciences LLC
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Arnay Sciences LLC filed Critical Arnay Sciences LLC
Publication of WO2024263891A2 publication Critical patent/WO2024263891A2/en
Publication of WO2024263891A3 publication Critical patent/WO2024263891A3/en
Anticipated expiration legal-status Critical
Pending legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/17—Immunomodulatory nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/532—Closed or circular

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

The present invention provides oligonucleotides referred to as ring-shaped oligonucleotides ("RSO") as described herein, compositions comprising same, and methods of using same. In certain embodiments, it comprises two oligonucleotides, wherein the first oligonucleotide comprises a functional oligonucleotide and the second oligonucleotide comprises a complementary oligonucleotide, wherein the first oligonucleotide and the second oligonucleotide hybridize to form two double-stranded sections, and wherein the 5' end of the first oligonucleotide is brought into proximity with the 3' end of the first oligonucleotide thereby creating a ring-shape.
PCT/US2024/034985 2023-06-23 2024-06-21 Delivery of rna therapeutics using ring-shaped nucleic acids Pending WO2024263891A2 (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202363522878P 2023-06-23 2023-06-23
US63/522,878 2023-06-23
US202363524009P 2023-06-29 2023-06-29
US63/524,009 2023-06-29

Publications (2)

Publication Number Publication Date
WO2024263891A2 WO2024263891A2 (en) 2024-12-26
WO2024263891A3 true WO2024263891A3 (en) 2025-04-03

Family

ID=93936278

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2024/034985 Pending WO2024263891A2 (en) 2023-06-23 2024-06-21 Delivery of rna therapeutics using ring-shaped nucleic acids

Country Status (1)

Country Link
WO (1) WO2024263891A2 (en)

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6369038B1 (en) * 1991-04-25 2002-04-09 Genset Closed antisense and sense oligonucleotides and their applications
WO2023049275A2 (en) * 2021-09-23 2023-03-30 Arnay Sciences, Llc Cyclic structured oligonucleotides as therapeutic agents

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6369038B1 (en) * 1991-04-25 2002-04-09 Genset Closed antisense and sense oligonucleotides and their applications
WO2023049275A2 (en) * 2021-09-23 2023-03-30 Arnay Sciences, Llc Cyclic structured oligonucleotides as therapeutic agents

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
AGRAWAL SUDHIR: "The Evolution of Antisense Oligonucleotide Chemistry—A Personal Journey", BIOMEDICINES, vol. 9, no. 5, pages 503, XP093014250, DOI: 10.3390/biomedicines9050503 *

Also Published As

Publication number Publication date
WO2024263891A2 (en) 2024-12-26

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