CN118103516A - 修饰的可复制rna和相关组合物及其用途 - Google Patents

修饰的可复制rna和相关组合物及其用途 Download PDF

Info

Publication number
CN118103516A
CN118103516A CN202280068980.1A CN202280068980A CN118103516A CN 118103516 A CN118103516 A CN 118103516A CN 202280068980 A CN202280068980 A CN 202280068980A CN 118103516 A CN118103516 A CN 118103516A
Authority
CN
China
Prior art keywords
molecule
rna
uridine
sequence
alphavirus
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
CN202280068980.1A
Other languages
English (en)
Chinese (zh)
Inventor
M·佩尔科维奇
T·拜塞特
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.)
Translationale Onkologie An Der Universitatsmedizin Der Johannes Gutenberg-Univers
Biotechnology Europe Inc
Original Assignee
Translationale Onkologie An Der Universitatsmedizin Der Johannes Gutenberg-Univers
Biotechnology Europe Inc
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 Translationale Onkologie An Der Universitatsmedizin Der Johannes Gutenberg-Univers, Biotechnology Europe Inc filed Critical Translationale Onkologie An Der Universitatsmedizin Der Johannes Gutenberg-Univers
Publication of CN118103516A publication Critical patent/CN118103516A/zh
Pending legal-status Critical Current

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/14Antivirals for RNA viruses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2770/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses positive-sense
    • C12N2770/00011Details
    • C12N2770/36011Togaviridae
    • C12N2770/36111Alphavirus, e.g. Sindbis virus, VEE, EEE, WEE, Semliki
    • C12N2770/36121Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2770/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses positive-sense
    • C12N2770/00011Details
    • C12N2770/36011Togaviridae
    • C12N2770/36111Alphavirus, e.g. Sindbis virus, VEE, EEE, WEE, Semliki
    • C12N2770/36134Use of virus or viral component as vaccine, e.g. live-attenuated or inactivated virus, VLP, viral protein
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2770/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses positive-sense
    • C12N2770/00011Details
    • C12N2770/36011Togaviridae
    • C12N2770/36111Alphavirus, e.g. Sindbis virus, VEE, EEE, WEE, Semliki
    • C12N2770/36141Use of virus, viral particle or viral elements as a vector
    • C12N2770/36143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Virology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN202280068980.1A 2021-10-18 2022-10-17 修饰的可复制rna和相关组合物及其用途 Pending CN118103516A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP2021078830 2021-10-18
EPPCT/EP2021/078830 2021-10-18
PCT/EP2022/078870 WO2023066875A1 (en) 2021-10-18 2022-10-17 Modified replicable rna and related compositions and their use

Publications (1)

Publication Number Publication Date
CN118103516A true CN118103516A (zh) 2024-05-28

Family

ID=78483248

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202280068980.1A Pending CN118103516A (zh) 2021-10-18 2022-10-17 修饰的可复制rna和相关组合物及其用途

Country Status (7)

Country Link
US (1) US20250236883A1 (https=)
EP (1) EP4419695A1 (https=)
JP (1) JP2024539087A (https=)
CN (1) CN118103516A (https=)
AU (1) AU2022369342A1 (https=)
CA (1) CA3234396A1 (https=)
WO (1) WO2023066875A1 (https=)

Family Cites Families (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008119827A1 (en) 2007-04-02 2008-10-09 Fit Biotech Oy Transreplicase constructs
CN104072561B (zh) 2007-06-19 2017-12-22 路易斯安那州州立大学及农业机械学院管理委员会 信使rna帽的抗‑反向硫代磷酸类似物的合成和用途
ES2840725T3 (es) 2007-08-20 2021-07-07 Glaxo Group Ltd Procedimiento de producción
PL215513B1 (pl) 2008-06-06 2013-12-31 Univ Warszawski Nowe boranofosforanowe analogi dinukleotydów, ich zastosowanie, czasteczka RNA, sposób otrzymywania RNA oraz sposób otrzymywania peptydów lub bialka
DE102008061522A1 (de) 2008-12-10 2010-06-17 Biontech Ag Verwendung von Flt3-Ligand zur Verstärkung von Immunreaktionen bei RNA-Immunisierung
EP2281579A1 (en) 2009-08-05 2011-02-09 BioNTech AG Vaccine composition comprising 5'-Cap modified RNA
NZ606591A (en) 2010-07-06 2015-02-27 Novartis Ag Cationic oil-in-water emulsions
ES2557382T3 (es) 2010-07-06 2016-01-25 Glaxosmithkline Biologicals Sa Liposomas con lípidos que tienen un valor de pKa ventajoso para el suministro de ARN
MX2013002332A (es) 2010-08-31 2013-03-18 Novartis Ag Lipidos apropiados para suministro liposomal del arn que codifica la proteina.
SI4043040T1 (sl) 2010-08-31 2023-04-28 Glaxosmithkline Biologicals Sa Mali liposomi za dostavo imunogen-kodirajoče RNA
LT4226941T (lt) 2010-08-31 2025-01-10 Glaxosmithkline Biologicals Sa Pegilintos liposomos, skirtos imunogeną koduojančios rnr pristatymui
EP4115875A1 (en) 2011-07-06 2023-01-11 GlaxoSmithKline Biologicals S.A. Liposomes having useful n:p ratio for delivery of rna molecules
JP6059220B2 (ja) 2011-07-06 2017-01-18 ノバルティス アーゲー 核酸を含む水中油型エマルジョン
MX350258B (es) 2011-07-06 2017-08-31 Novartis Ag Emulsiones cationicas de aceite en agua.
MX366055B (es) 2011-08-31 2019-06-26 Novartis Ag Liposomas pegilados para admistracion de acido ribonucleico (arn) que codifica para inmunogeno.
WO2013143555A1 (en) 2012-03-26 2013-10-03 Biontech Ag Rna formulation for immunotherapy
WO2014071963A1 (en) 2012-11-09 2014-05-15 Biontech Ag Method for cellular rna expression
WO2016005004A1 (en) 2014-07-11 2016-01-14 Biontech Rna Pharmaceuticals Gmbh Stabilization of poly(a) sequence encoding dna sequences
WO2017162266A1 (en) 2016-03-21 2017-09-28 Biontech Rna Pharmaceuticals Gmbh Rna replicon for versatile and efficient gene expression
US20220054611A1 (en) * 2018-12-17 2022-02-24 Immune Design Corp. Pathogen-associated molecular pattern molecules and rna immunogenic compositions and methods of using the compositions for treating cancer
WO2020144295A1 (en) * 2019-01-10 2020-07-16 Biontech Rna Pharmaceuticals Gmbh Localized administration of rna molecules for therapy

Also Published As

Publication number Publication date
WO2023066875A1 (en) 2023-04-27
JP2024539087A (ja) 2024-10-28
US20250236883A1 (en) 2025-07-24
AU2022369342A1 (en) 2024-03-14
CA3234396A1 (en) 2023-04-27
EP4419695A1 (en) 2024-08-28

Similar Documents

Publication Publication Date Title
JP7465310B2 (ja) 多用途性且つ効率的な遺伝子発現のためのrnaレプリコン
RU2752580C2 (ru) Транс-реплицирующая рнк
JP7769644B2 (ja) 多用途かつ効率的な遺伝子発現のためのrnaレプリコン
US20250250626A1 (en) Methods for determining mutations for increasing modified replicable rna function and related compositions and their use
US20250281602A1 (en) Replicon Compositions and Methods of Using Same for the Treatment of Diseases
CN118103516A (zh) 修饰的可复制rna和相关组合物及其用途
HK40094277A (zh) 用於多用途和有效的基因表达的rna复制子
TW202526021A (zh) 包括高度活性反式擴增複製酶之系統及組合物
HK40059264A (en) Trans-replicating rna

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination