KR20240042138A - 자가면역 질환의 치료를 위한 ｒｎａ - Google Patents

자가면역 질환의 치료를 위한 ｒｎａ Download PDF

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Publication number
KR20240042138A
KR20240042138A KR1020247008874A KR20247008874A KR20240042138A KR 20240042138 A KR20240042138 A KR 20240042138A KR 1020247008874 A KR1020247008874 A KR 1020247008874A KR 20247008874 A KR20247008874 A KR 20247008874A KR 20240042138 A KR20240042138 A KR 20240042138A
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South Korea
Prior art keywords
uridine
thio
methyl
cells
pseudouridine
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KR1020247008874A
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English (en)
Korean (ko)
Inventor
우르 사힌
세바스찬 크레이터
크리스티나 크리엔케
유타 페트쉬엔카
레나 마르엔 크란츠
무스타파 디켄
Original Assignee
비온테크 에스이
트론-트란슬라셔날레 온콜로기 안 데어 유니버시태츠메디진 데어 요하네스 구텐베르크-유니버시태트 마인츠 게마인뉘치게 게엠베하
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Application filed by 비온테크 에스이, 트론-트란슬라셔날레 온콜로기 안 데어 유니버시태츠메디진 데어 요하네스 구텐베르크-유니버시태트 마인츠 게마인뉘치게 게엠베하 filed Critical 비온테크 에스이
Publication of KR20240042138A publication Critical patent/KR20240042138A/ko
Withdrawn legal-status Critical Current

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/0005—Vertebrate antigens
    • A61K39/0008—Antigens related to auto-immune diseases; Preparations to induce self-tolerance
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0033—Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/1003—Extracting or separating nucleic acids from biological samples, e.g. pure separation or isolation methods; Conditions, buffers or apparatuses therefor
    • C12N15/1006—Extracting or separating nucleic acids from biological samples, e.g. pure separation or isolation methods; Conditions, buffers or apparatuses therefor by means of a solid support carrier, e.g. particles, polymers
    • C12N15/101—Extracting or separating nucleic acids from biological samples, e.g. pure separation or isolation methods; Conditions, buffers or apparatuses therefor by means of a solid support carrier, e.g. particles, polymers by chromatography, e.g. electrophoresis, ion-exchange, reverse phase
    • 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
    • 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
    • C12N15/117—Nucleic acids having immunomodulatory properties, e.g. containing CpG-motifs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/53—DNA (RNA) vaccination
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/555—Medicinal preparations containing antigens or antibodies characterised by a specific combination antigen/adjuvant
    • A61K2039/55511—Organic adjuvants
    • A61K2039/55555—Liposomes; Vesicles, e.g. nanoparticles; Spheres, e.g. nanospheres; Polymers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/60—Medicinal preparations containing antigens or antibodies characteristics by the carrier linked to the antigen
    • A61K2039/6018—Lipids, e.g. in lipopeptides
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/335—Modified T or U

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Immunology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Mycology (AREA)
  • Rheumatology (AREA)
  • Transplantation (AREA)
  • Analytical Chemistry (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Dispersion Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
KR1020247008874A 2017-04-11 2018-04-10 자가면역 질환의 치료를 위한 ｒｎａ Withdrawn KR20240042138A (ko)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
EPPCT/EP2017/058651 2017-04-11
PCT/EP2017/058651 WO2018188730A1 (en) 2017-04-11 2017-04-11 Rna for treatment of autoimmune diseases
PCT/EP2018/059188 WO2018189193A1 (en) 2017-04-11 2018-04-10 Rna for treatment of autoimmune diseases
KR1020197033154A KR102824942B1 (ko) 2017-04-11 2018-04-10 자가면역 질환의 치료를 위한 ｒｎａ

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
KR1020197033154A Division KR102824942B1 (ko) 2017-04-11 2018-04-10 자가면역 질환의 치료를 위한 ｒｎａ

Publications (1)

Publication Number Publication Date
KR20240042138A true KR20240042138A (ko) 2024-04-01

Family

ID=58632945

Family Applications (2)

Application Number Title Priority Date Filing Date
KR1020247008874A Withdrawn KR20240042138A (ko) 2017-04-11 2018-04-10 자가면역 질환의 치료를 위한 ｒｎａ
KR1020197033154A Active KR102824942B1 (ko) 2017-04-11 2018-04-10 자가면역 질환의 치료를 위한 ｒｎａ

Family Applications After (1)

Application Number Title Priority Date Filing Date
KR1020197033154A Active KR102824942B1 (ko) 2017-04-11 2018-04-10 자가면역 질환의 치료를 위한 ｒｎａ

Country Status (23)

Country Link
US (2) US11701413B2 (https=)
EP (2) EP3609529B1 (https=)
JP (3) JP7078641B2 (https=)
KR (2) KR20240042138A (https=)
CN (2) CN110719787B (https=)
AU (1) AU2018253352B2 (https=)
BR (1) BR112019020667A8 (https=)
CA (1) CA3059505A1 (https=)
CY (1) CY1124767T1 (https=)
DK (1) DK3609529T3 (https=)
ES (1) ES2893451T3 (https=)
HR (1) HRP20211559T1 (https=)
HU (1) HUE056871T2 (https=)
IL (2) IL269793B (https=)
LT (1) LT3609529T (https=)
MX (1) MX2019012144A (https=)
PL (1) PL3609529T3 (https=)
PT (1) PT3609529T (https=)
RS (1) RS62582B1 (https=)
SG (2) SG11201909520VA (https=)
SI (1) SI3609529T1 (https=)
SM (1) SMT202100572T1 (https=)
WO (2) WO2018188730A1 (https=)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018188730A1 (en) * 2017-04-11 2018-10-18 Biontech Rna Pharmaceuticals Gmbh Rna for treatment of autoimmune diseases
CA3119449A1 (en) * 2018-11-12 2020-05-22 Translate Bio, Inc. Methods for inducing immune tolerance
IL297419B2 (en) * 2020-04-22 2025-02-01 BioNTech SE Coronavirus vaccine
CN116710115A (zh) 2020-11-20 2023-09-05 思维疗法股份有限公司 用于优化的肽疫苗的组合物和方法
US11058751B1 (en) 2020-11-20 2021-07-13 Think Therapeutics, Inc. Compositions for optimized RAS peptide vaccines
US11161892B1 (en) 2020-12-07 2021-11-02 Think Therapeutics, Inc. Method of compact peptide vaccines using residue optimization
US11421015B2 (en) 2020-12-07 2022-08-23 Think Therapeutics, Inc. Method of compact peptide vaccines using residue optimization
CN115232825B (zh) * 2021-04-22 2023-09-26 中国人民解放军军事科学院军事医学研究院 一种基于1083骨架的新冠病毒疫苗
US11464842B1 (en) 2021-04-28 2022-10-11 Think Therapeutics, Inc. Compositions and method for optimized peptide vaccines using residue optimization
CN117750971A (zh) 2021-05-26 2024-03-22 易姆赛斯股份公司 治疗或预防自身免疫病的方法
AR126654A1 (es) 2021-06-29 2023-11-01 Imcyse Sa Péptidos y métodos para el tratamiento de neuromielitis óptica
CN113336862B (zh) * 2021-07-05 2022-03-01 广东省科学院动物研究所 一种抗多发性硬化的重组蛋白及其制备方法和用途
WO2023030635A1 (en) * 2021-09-02 2023-03-09 BioNTech SE Potency assay for therapeutic potential of coding nucleic acid
CN118369335A (zh) * 2022-01-06 2024-07-19 上海吉量医药工程有限公司 N1位修饰假尿嘧啶核苷及其在mRNA合成中的应用

Family Cites Families (17)

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US7030098B2 (en) * 1999-03-12 2006-04-18 The Board Of Trustees Of The Leland Stanford Junior University DNA vaccination for treatment of autoimmune disease
AU2002952834A0 (en) 2002-09-16 2002-12-05 The Walter And Eliza Hall Institute Of Medical Research A method of treating an autoimmune disease
US9012219B2 (en) * 2005-08-23 2015-04-21 The Trustees Of The University Of Pennsylvania RNA preparations comprising purified modified RNA for reprogramming cells
PL2578685T3 (pl) 2005-08-23 2020-01-31 The Trustees Of The University Of Pennsylvania Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania
WO2009030254A1 (en) * 2007-09-04 2009-03-12 Curevac Gmbh Complexes of rna and cationic peptides for transfection and for immunostimulation
EP2050814A1 (en) 2007-10-17 2009-04-22 Txcell Compositions for treating multiple sclerosis
US8808982B2 (en) 2009-12-07 2014-08-19 Cellscript, Llc Compositions and methods for reprogramming eukaryotic cells
WO2012154933A1 (en) * 2011-05-10 2012-11-15 The Brigham And Women's Hospital, Inc. Detecting inclusion body myositis
CN103998055B (zh) * 2011-09-30 2017-10-24 北京艾棣维欣生物技术有限公司 预防和治疗自身免疫性疾病的联合促进剂、抗原和dna疫苗
EP3144389B1 (en) 2011-12-30 2018-05-09 Cellscript, Llc Making and using in vitro-synthesized ssrna for introducing into mammalian cells to induce a biological or biochemical effect
WO2013120498A1 (en) * 2012-02-15 2013-08-22 Curevac Gmbh Nucleic acid comprising or coding for a histone stem-loop and a poly(a) sequence or a polyadenylation signal for increasing the expression of an encoded allergenic antigen or an autoimmune self-antigen
US20160032316A1 (en) 2013-03-14 2016-02-04 The Trustees Of The University Of Pennsylvania Purification and Purity Assessment of RNA Molecules Synthesized with Modified Nucleosides
AU2016253972B2 (en) * 2015-04-27 2020-01-02 Acuitas Therapeutics Inc. Nucleoside-modified RNA for inducing an adaptive immune response
CN106395552B (zh) 2015-08-03 2020-03-17 奥的斯电梯公司 无绳电梯系统导轨组件
US10808242B2 (en) * 2015-08-28 2020-10-20 Biontech Rna Pharmaceuticals Gmbh Method for reducing immunogenicity of RNA
SI3445850T1 (sl) * 2016-04-22 2021-12-31 BioNTech SE Postopki za zagotavljanje enoverižne RNA
WO2018188730A1 (en) * 2017-04-11 2018-10-18 Biontech Rna Pharmaceuticals Gmbh Rna for treatment of autoimmune diseases

Also Published As

Publication number Publication date
US20200061166A1 (en) 2020-02-27
DK3609529T3 (da) 2021-10-11
CY1124767T1 (el) 2022-11-25
WO2018188730A1 (en) 2018-10-18
US20230330198A1 (en) 2023-10-19
LT3609529T (lt) 2021-10-25
KR102824942B1 (ko) 2025-06-25
CN110719787B (zh) 2024-03-15
IL269793A (en) 2019-11-28
SI3609529T1 (sl) 2021-11-30
MX2019012144A (es) 2019-11-21
IL290980A (en) 2022-05-01
EP3981424A1 (en) 2022-04-13
CN118161602A (zh) 2024-06-11
RU2019135808A3 (https=) 2021-08-10
SMT202100572T1 (it) 2021-11-12
HRP20211559T1 (hr) 2022-01-21
SG11201909520VA (en) 2019-11-28
EP3609529B1 (en) 2021-09-08
CN110719787A (zh) 2020-01-21
JP2020516658A (ja) 2020-06-11
AU2018253352B2 (en) 2025-04-10
IL269793B (en) 2022-04-01
PL3609529T3 (pl) 2021-12-13
KR20190134765A (ko) 2019-12-04
US11701413B2 (en) 2023-07-18
RS62582B1 (sr) 2021-12-31
JP7444924B2 (ja) 2024-03-06
PT3609529T (pt) 2021-10-14
RU2019135808A (ru) 2021-05-11
CA3059505A1 (en) 2018-10-18
HUE056871T2 (hu) 2022-04-28
EP3609529A1 (en) 2020-02-19
ES2893451T3 (es) 2022-02-09
JP2022110127A (ja) 2022-07-28
BR112019020667A8 (pt) 2022-08-16
WO2018189193A1 (en) 2018-10-18
JP2024059794A (ja) 2024-05-01
BR112019020667A2 (pt) 2020-05-05
SG10202111163VA (en) 2021-11-29
JP7078641B2 (ja) 2022-05-31
AU2018253352A1 (en) 2019-10-17

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