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MX2020010871A - Compuestos de 5'-caperuza-trinucleótidos u oligonucleótidos superiores y sus usos en estabilizar arn, que expresan proteínas y en la terapia. - Google Patents

Compuestos de 5'-caperuza-trinucleótidos u oligonucleótidos superiores y sus usos en estabilizar arn, que expresan proteínas y en la terapia.

Info

Publication number
MX2020010871A
MX2020010871A MX2020010871A MX2020010871A MX2020010871A MX 2020010871 A MX2020010871 A MX 2020010871A MX 2020010871 A MX2020010871 A MX 2020010871A MX 2020010871 A MX2020010871 A MX 2020010871A MX 2020010871 A MX2020010871 A MX 2020010871A
Authority
MX
Mexico
Prior art keywords
rna
cap
therapy
cells
trinucleotide
Prior art date
Application number
MX2020010871A
Other languages
English (en)
Inventor
Ugur Sahin
Stephanie Fesser
Andreas Kuhn
Hiromi Muramatsu
Katalin Kariko
Original Assignee
BioNTech SE
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 BioNTech SE filed Critical BioNTech SE
Publication of MX2020010871A publication Critical patent/MX2020010871A/es

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02Compounds 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered

Landscapes

  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (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)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)

Abstract

La presente invención se refiere a compuestos de 5'-caperuza, en particular la estabilización de ARN por estos compuestos de 5'-caperuza, y proporciona composiciones, tal como composiciones farmacéuticas, y células que comprende un ARN que se modifica con este compuesto de 5'-caperuza, así como métodos para producir un péptido o proteína de interés usando la composiciones o células de acuerdo con la presente invención. Además, la presente invención proporciona el ARN, composiciones, o células para el uso en la terapia, en particular para uso en un método para tratar una enfermedad o trastorno por terapia de reemplazo de proteínas, ingeniería del genoma, reprogramación genética, e inmunoterapia; un método para incrementar la estabilidad del ARN en las células; un método para incrementar la expresión del ARN en las células; y un método para proporcionar un ARN con una estructura 5'-caperuza.
MX2020010871A 2018-03-15 2019-03-14 Compuestos de 5'-caperuza-trinucleótidos u oligonucleótidos superiores y sus usos en estabilizar arn, que expresan proteínas y en la terapia. MX2020010871A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP2018056595 2018-03-15
PCT/EP2019/056502 WO2019175356A1 (en) 2018-03-15 2019-03-14 5'-cap-trinucleotide- or higher oligonucleotide compounds and their uses in stabilizing rna, expressing proteins and in therapy

Publications (1)

Publication Number Publication Date
MX2020010871A true MX2020010871A (es) 2020-11-09

Family

ID=65802103

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020010871A MX2020010871A (es) 2018-03-15 2019-03-14 Compuestos de 5'-caperuza-trinucleótidos u oligonucleótidos superiores y sus usos en estabilizar arn, que expresan proteínas y en la terapia.

Country Status (11)

Country Link
US (2) US20210363172A1 (es)
EP (1) EP3765477A1 (es)
JP (1) JP7416705B2 (es)
KR (1) KR20200143391A (es)
CN (1) CN112119084A (es)
AU (1) AU2019235424B2 (es)
BR (1) BR112020026125A8 (es)
CA (1) CA3093509A1 (es)
IL (1) IL278058A (es)
MX (1) MX2020010871A (es)
WO (1) WO2019175356A1 (es)

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EP4208549A1 (en) 2020-09-04 2023-07-12 Verve Therapeutics, Inc. Compositions and methods for capping rnas
TW202245808A (zh) * 2020-12-21 2022-12-01 德商拜恩迪克公司 用於治療癌症之治療性rna
CN115197327A (zh) * 2021-04-08 2022-10-18 上海细胞治疗集团有限公司 Rna修饰嵌合蛋白及其应用
WO2022228827A1 (en) 2021-04-26 2022-11-03 Institut Pasteur Human neutralizing monoclonal antibodies against sars-cov-2 and uses thereof
CN113603739B (zh) * 2021-08-27 2024-08-23 上海兆维科技发展有限公司 一种加帽类似物及其应用
JP7642260B2 (ja) * 2022-02-28 2025-03-10 ▲広▼州市恒▲諾▼康医▲薬▼科技有限公司 Rnaキャッピング用化合物及びその使用
WO2023191342A1 (ko) * 2022-03-31 2023-10-05 한미정밀화학 주식회사 mRNA 캡 유사체 및 이의 용도
WO2023193892A1 (en) 2022-04-05 2023-10-12 BioNTech SE Nucleic acid compositions comprising an inorganic polyphosphate and methods for preparing, storing and using the same
CN117430652A (zh) * 2022-07-22 2024-01-23 广州市恒诺康医药科技有限公司 用于rna加帽的环状取代化合物及其应用
WO2024161314A1 (en) * 2023-01-31 2024-08-08 Seqirus Inc. Capping assay
WO2024193827A1 (en) 2023-03-23 2024-09-26 BioNTech SE Stabilized nucleic acid compositions and methods for preparing, storing and using the same
EP4442276A1 (en) 2023-04-07 2024-10-09 Institut Pasteur Combined antibodies against sarbecoviruses and uses thereof
WO2024236192A1 (en) 2023-05-17 2024-11-21 Institut Pasteur Heterodimer of poxvirus a16 and g9 proteins as an immunogen
US20250026779A1 (en) * 2023-06-29 2025-01-23 Nutcracker Therapeutics, Inc. Ethyl modified rna caps and methods of use
CN117534719B (zh) * 2024-01-09 2024-05-14 北京悦康科创医药科技股份有限公司 一种c6'取代锁核酸修饰加帽类似物及其应用

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Also Published As

Publication number Publication date
BR112020026125A8 (pt) 2022-10-18
CA3093509A1 (en) 2019-09-19
JP2021515577A (ja) 2021-06-24
AU2019235424B2 (en) 2023-12-21
RU2020133704A (ru) 2022-04-15
KR20200143391A (ko) 2020-12-23
CN112119084A (zh) 2020-12-22
EP3765477A1 (en) 2021-01-20
IL278058A (en) 2020-11-30
US20210363172A1 (en) 2021-11-25
WO2019175356A1 (en) 2019-09-19
AU2019235424A1 (en) 2020-09-03
JP7416705B2 (ja) 2024-01-17
BR112020026125A2 (pt) 2021-03-16
US20210340170A1 (en) 2021-11-04

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