CA3256515A1 - Genetic-carbohydrate combinations for enhanced, specific targeting of the liver and kidney - Google Patents

Genetic-carbohydrate combinations for enhanced, specific targeting of the liver and kidney

Info

Publication number
CA3256515A1
CA3256515A1 CA3256515A CA3256515A CA3256515A1 CA 3256515 A1 CA3256515 A1 CA 3256515A1 CA 3256515 A CA3256515 A CA 3256515A CA 3256515 A CA3256515 A CA 3256515A CA 3256515 A1 CA3256515 A1 CA 3256515A1
Authority
CA
Canada
Prior art keywords
conjugate
pna
mir
carbohydrate
genetic sequence
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
CA3256515A
Other languages
English (en)
French (fr)
Inventor
Raman Bahal
Vikas Kumar
Aniket Wahane
Original Assignee
University of Connecticut
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 University of Connecticut filed Critical University of Connecticut
Publication of CA3256515A1 publication Critical patent/CA3256515A1/en
Pending legal-status Critical Current

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3181—Peptide nucleic acid, PNA
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
CA3256515A 2022-04-26 2023-04-26 Genetic-carbohydrate combinations for enhanced, specific targeting of the liver and kidney Pending CA3256515A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202263335174P 2022-04-26 2022-04-26
US63/335,174 2022-04-26
PCT/US2023/019940 WO2023212027A2 (en) 2022-04-26 2023-04-26 Genetic sequence-carbohydrate conjugates for enhanced liver- and kidney-specific targeting

Publications (1)

Publication Number Publication Date
CA3256515A1 true CA3256515A1 (en) 2023-11-02

Family

ID=88519558

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3256515A Pending CA3256515A1 (en) 2022-04-26 2023-04-26 Genetic-carbohydrate combinations for enhanced, specific targeting of the liver and kidney

Country Status (6)

Country Link
US (1) US20250346895A1 (enExample)
EP (1) EP4514970A2 (enExample)
JP (1) JP2025514180A (enExample)
CN (1) CN119213006A (enExample)
CA (1) CA3256515A1 (enExample)
WO (1) WO2023212027A2 (enExample)

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6746868B1 (en) * 1997-09-18 2004-06-08 Isis Pharmaceuticals, Inc. Chemical modification of DNA using peptide nucleic acid conjugates
NZ528966A (en) * 2003-10-17 2006-11-30 Otago Innovation Ltd Peptide nucleic acid conjugates and uses thereof
EP2229186A2 (en) * 2007-12-04 2010-09-22 Alnylam Pharmaceuticals Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
US11833254B2 (en) * 2020-05-27 2023-12-05 University Of Connecticut Discoidal nano universal platform for efficient delivery of PNAs

Also Published As

Publication number Publication date
US20250346895A1 (en) 2025-11-13
WO2023212027A3 (en) 2023-12-07
WO2023212027A2 (en) 2023-11-02
EP4514970A2 (en) 2025-03-05
JP2025514180A (ja) 2025-05-02
CN119213006A (zh) 2024-12-27

Similar Documents

Publication Publication Date Title
JP6715325B2 (ja) siRNA、siRNAを含む医薬組成物及び結合体、並びにそれらの応用
CA2917320C (en) Respiratory disease-related gene specific sirna, double-helical oligo rna structure containing sirna, composition containing same for preventing or treating respiratory disease
EP2844662B1 (en) Tetragalnac and peptide containing conjugates and methods for delivery of oligonucleotides
RU2599449C1 (ru) Новые конъюгаты олигонуклеотидов и их применение
CN115851723A (zh) 一种抑制lpa基因表达的rna抑制剂及其应用
JP2023158214A (ja) Hif2αの遺伝子発現を阻害する組成物及び方法
BR112016000160B1 (pt) estrutura de oligonucleotídeo tipo nanopartícula melhorada tendo alta eficiência e método para a preparação da mesma
KR102746742B1 (ko) 엠피레귤린 유전자 특이적 이중가닥 올리고뉴클레오티드 및 이를 포함하는 섬유증 관련 질환 및 호흡기 관련 질환 예방 및 치료용 조성물
CA3242059A1 (en) Nucleic acid, composition and conjugate containing nucleic acid, preparation method thereof and use thereof
CN107530439B (zh) 用于治疗癌症的对人抗原R表达的siRNA抑制
US20130108686A1 (en) Method for the delivery of oligonucleotides
US20250346895A1 (en) Genetic sequence-carbohydrate conjugates for enhanced liver- and kidney-specific targeting
KR20130068032A (ko) 안정한 안티센스 올리고뉴클레오티드 접합체 및 그 제조방법
US20240409938A1 (en) Lipophilic sirna conjugates for the treatment of central nervous system diseases
JP7709164B2 (ja) 核酸送達促進剤
US20260125681A1 (en) DOUBLE-STRANDED RNAi AGENTS FOR TARGETING AND REGULATING HBV GENE EXPRESSION, AND A USE THEREOF
US20260092085A1 (en) Synthetic triplex peptide nucleic acid-based inhibitors for cancer therapy
WO2025091026A1 (en) Therapeutic compositions containing g-clamp pna and uses thereof
Wang Poly (ethylene) Glycol-Based Bottlebrush Polymers as Nanocarriers for Oligonucleotide Therapeutics: Design, Synthesis, and Applications
WO2026046236A1 (zh) 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
HK40127600A (en) Compositions and methods for inhibiting adenylate cyclase 9 (ac9)
CN119074947A (zh) 一种肝靶向小核酸药物载体、药用组合物及其制备方法和应用
Iwata MALDI-TOF MS: m/z calcd for CgH3ClNNaOgS [M-Na]": 749.13 Found: 749.12.
KR20130080727A (ko) 리간드가 결합된 이중나선 올리고 rna 구조체 및 그 제조방법

Legal Events

Date Code Title Description
A00 Application filed

Free format text: ST27 STATUS EVENT CODE: A-0-1-A10-A00-A101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: APPLICATION RECEIVED - PCT

Effective date: 20241024

A00 Application filed

Free format text: ST27 STATUS EVENT CODE: A-1-1-A10-A00-A102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: COMPLIANCE REQUIREMENTS DETERMINED MET

Effective date: 20250219

A15 Pct application entered into the national or regional phase

Free format text: ST27 STATUS EVENT CODE: A-1-1-A10-A15-X000 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: NATIONAL ENTRY REQUIREMENTS DETERMINED COMPLIANT

Effective date: 20250219

P18 Priority claim added or amended

Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P18-P105 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: PRIORITY CLAIM REQUIREMENTS DETERMINED COMPLIANT

Effective date: 20250219

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REQUEST TO REGISTER A DOCUMENT RECEIVED

Effective date: 20250220

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20250220

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20250221

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 2ND ANNIV.) - STANDARD

Year of fee payment: 2

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20250407

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20250407

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 3RD ANNIV.) - STANDARD

Year of fee payment: 3

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20260422

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20260422

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REGISTER A DOCUMENT REQUIREMENTS DETERMINED COMPLIANT

Effective date: 20260513

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20260601