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AR133304A1 - Agentes oligonucleotídicos de cadena doble y sus usos - Google Patents

Agentes oligonucleotídicos de cadena doble y sus usos

Info

Publication number
AR133304A1
AR133304A1 ARP240101891A ARP240101891A AR133304A1 AR 133304 A1 AR133304 A1 AR 133304A1 AR P240101891 A ARP240101891 A AR P240101891A AR P240101891 A ARP240101891 A AR P240101891A AR 133304 A1 AR133304 A1 AR 133304A1
Authority
AR
Argentina
Prior art keywords
double
stranded oligonucleotide
oligonucleotide agent
extension
antisense strand
Prior art date
Application number
ARP240101891A
Other languages
English (en)
Inventor
Xiaoming Zou
Jianyu Lu
Qingfeng Li
Cuicui Gao
Qionger He
Hongkuan Yang
Mingjie Wang
Original Assignee
Visirna Therapeutics Suzhou Co Ltd
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 Visirna Therapeutics Suzhou Co Ltd filed Critical Visirna Therapeutics Suzhou Co Ltd
Publication of AR133304A1 publication Critical patent/AR133304A1/es

Links

Classifications

    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • 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
    • C12N2310/312—Phosphonates
    • C12N2310/3125—Methylphosphonates
    • 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
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • 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

Landscapes

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

Abstract

En la presente se describe un agente oligonucleotídico de cadena doble y sus usos, que comprende un sitio de escisión específico para una endonucleasa, donde el agente oligonucleotídico de cadena doble escindido es capaz de inhibir la expresión del gene gen diana por medio de ARN de interferencia (ARNi). Reivindicación 1: Un agente oligonucleotídico de cadena doble que comprende una cadena sentido y una cadena antisentido, donde: la cadena sentido y la cadena antisentido forman una porción de cadena doble de 15 a 27 pares de bases de longitud y una extensión 5’ en la cadena antisentido; la extensión 5’ tiene una longitud de al menos tres nucleótidos y es escindible del nucleótido más 3’ de la extensión 5’, y el agente oligonucleotídico de cadena doble escindido es capaz de silenciar un ARN diana o inhibir la expresión de un gen diana por medio de ARN de interferencia. Reivindicación 52: Una composición farmacéutica que comprende un agente oligonucleotídico de cadena doble de acuerdo con las reivindicaciones precedentes, y un portador farmacéuticamente aceptable.
ARP240101891A 2023-07-21 2024-07-19 Agentes oligonucleotídicos de cadena doble y sus usos AR133304A1 (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
CN202310904516 2023-07-21
CN202310900474 2023-07-21
CN202311532296 2023-11-16
CN202410396475 2024-04-02

Publications (1)

Publication Number Publication Date
AR133304A1 true AR133304A1 (es) 2025-09-17

Family

ID=94374144

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP240101891A AR133304A1 (es) 2023-07-21 2024-07-19 Agentes oligonucleotídicos de cadena doble y sus usos

Country Status (3)

Country Link
AR (1) AR133304A1 (es)
TW (1) TW202519663A (es)
WO (1) WO2025021034A1 (es)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN120118909A (zh) * 2025-05-14 2025-06-10 北京悦康科创医药科技股份有限公司 靶向调控LPA基因表达的siRNA双链体及其在预防和治疗心脑血管相关疾病中的用途

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1318586C (zh) * 2001-11-05 2007-05-30 詹森药业有限公司 短的双链RNAs的体外合成方法
US20130236968A1 (en) * 2010-06-21 2013-09-12 Alnylam Pharmaceuticals, Inc. Multifunctional copolymers for nucleic acid delivery
FI3366775T4 (fi) * 2011-11-18 2025-11-14 Alnylam Pharmaceuticals Inc Modifioituja rnai-agensseja
EP3549610A1 (en) * 2018-04-05 2019-10-09 Silence Therapeutics GmbH Nucleic acid conjugates
CN117858946A (zh) * 2021-05-29 2024-04-09 强新科技国际研究院 作为新型基因沉默技术的非对称短双链体dna及其应用
US20240254490A1 (en) * 2021-05-29 2024-08-01 1Globe Health Institute Llc Short Duplex DNA as a Novel Gene Silencing Technology and Use Thereof

Also Published As

Publication number Publication date
TW202519663A (zh) 2025-05-16
WO2025021034A1 (en) 2025-01-30

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