JP6949131B2 - 治療用ｒｎａ - Google Patents

治療用ｒｎａ Download PDF

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JP6949131B2
JP6949131B2 JP2019546924A JP2019546924A JP6949131B2 JP 6949131 B2 JP6949131 B2 JP 6949131B2 JP 2019546924 A JP2019546924 A JP 2019546924A JP 2019546924 A JP2019546924 A JP 2019546924A JP 6949131 B2 JP6949131 B2 JP 6949131B2
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seq
rna
identity
protein
nucleotide
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JP2020509016A5 (enExample
JP2020509016A (ja
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フリーデリーケ・ギーゼケ
ウグル・サヒン
ドミトリ・ジー・ウィーダーシャイン
ティモシー・アール・ワーゲナー
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Sanofi SA
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Sanofi SA
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Priority to JP2022203863A priority patent/JP7461449B2/ja
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/52—Cytokines; Lymphokines; Interferons
    • C07K14/53—Colony-stimulating factor [CSF]
    • C07K14/535—Granulocyte CSF; Granulocyte-macrophage CSF
    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/19—Cytokines; Lymphokines; Interferons
    • A61K38/193—Colony stimulating factors [CSF]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/19—Cytokines; Lymphokines; Interferons
    • A61K38/20—Interleukins [IL]
    • A61K38/208—IL-12
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/19—Cytokines; Lymphokines; Interferons
    • A61K38/20—Interleukins [IL]
    • A61K38/2086—IL-13 to IL-16
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/19—Cytokines; Lymphokines; Interferons
    • A61K38/21—Interferons [IFN]
    • A61K38/212—IFN-alpha
    • 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
    • A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A61K39/39533—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals
    • A61K39/3955—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals against proteinaceous materials, e.g. enzymes, hormones, lymphokines
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • 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
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/52—Cytokines; Lymphokines; Interferons
    • C07K14/54—Interleukins [IL]
    • C07K14/5434—IL-12
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/52—Cytokines; Lymphokines; Interferons
    • C07K14/54—Interleukins [IL]
    • C07K14/5443—IL-15
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/52—Cytokines; Lymphokines; Interferons
    • C07K14/555—Interferons [IFN]
    • C07K14/56—IFN-alpha
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/67—General methods for enhancing the expression
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
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    • 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)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Immunology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Toxicology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Dermatology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Mycology (AREA)
  • Endocrinology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2019546924A 2017-02-28 2018-02-27 治療用ｒｎａ Active JP6949131B2 (ja)

Priority Applications (3)

Application Number Priority Date Filing Date Title
JP2021037923A JP7201725B2 (ja) 2017-02-28 2021-03-10 治療用ｒｎａ
JP2022203863A JP7461449B2 (ja) 2017-02-28 2022-12-21 治療用ｒｎａ
JP2024045866A JP2024073650A (ja) 2017-02-28 2024-03-22 治療用ｒｎａ

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201762464981P 2017-02-28 2017-02-28
US62/464,981 2017-02-28
EP17306089 2017-08-23
EP17306089.8 2017-08-23
US201762597527P 2017-12-12 2017-12-12
US62/597,527 2017-12-12
PCT/US2018/019878 WO2018160540A1 (en) 2017-02-28 2018-02-27 Therapeutic rna

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2021037923A Division JP7201725B2 (ja) 2017-02-28 2021-03-10 治療用ｒｎａ

Publications (3)

Publication Number Publication Date
JP2020509016A JP2020509016A (ja) 2020-03-26
JP2020509016A5 JP2020509016A5 (enExample) 2021-07-29
JP6949131B2 true JP6949131B2 (ja) 2021-10-13

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JP2019546924A Active JP6949131B2 (ja) 2017-02-28 2018-02-27 治療用ｒｎａ
JP2021037923A Active JP7201725B2 (ja) 2017-02-28 2021-03-10 治療用ｒｎａ
JP2022203863A Active JP7461449B2 (ja) 2017-02-28 2022-12-21 治療用ｒｎａ
JP2024045866A Pending JP2024073650A (ja) 2017-02-28 2024-03-22 治療用ｒｎａ

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JP2021037923A Active JP7201725B2 (ja) 2017-02-28 2021-03-10 治療用ｒｎａ
JP2022203863A Active JP7461449B2 (ja) 2017-02-28 2022-12-21 治療用ｒｎａ
JP2024045866A Pending JP2024073650A (ja) 2017-02-28 2024-03-22 治療用ｒｎａ

Country Status (21)

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US (3) US20200147176A1 (enExample)
EP (1) EP3589308A1 (enExample)
JP (4) JP6949131B2 (enExample)
KR (2) KR20240144261A (enExample)
CN (1) CN110337305A (enExample)
AU (1) AU2018229278A1 (enExample)
BR (1) BR112019017743A2 (enExample)
CA (1) CA3054733A1 (enExample)
CL (1) CL2019002461A1 (enExample)
CO (1) CO2019010355A2 (enExample)
CR (1) CR20190444A (enExample)
EC (1) ECSP19070336A (enExample)
IL (1) IL268894A (enExample)
MA (1) MA47680A (enExample)
MX (1) MX2019010269A (enExample)
PE (1) PE20200735A1 (enExample)
PH (1) PH12019501959A1 (enExample)
SG (1) SG11201907846VA (enExample)
TW (2) TW202428301A (enExample)
UY (1) UY37621A (enExample)
WO (1) WO2018160540A1 (enExample)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021098735A (ja) * 2017-02-28 2021-07-01 サノフイＳａｎｏｆｉ 治療用ｒｎａ
JP2022518236A (ja) * 2019-01-21 2022-03-14 サノフイ 進行期固形腫瘍がんに対する治療用ｒｎａおよび抗ｐｄ１抗体

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KR20220136378A (ko) * 2020-01-31 2022-10-07 제넨테크, 인크. Pd-1 축 결합 길항제 및 rna 백신을 이용하여 네오에피토프-특이적 t 세포를 유도하는 방법
CN113521269A (zh) * 2020-04-22 2021-10-22 生物技术Rna制药有限公司 冠状病毒疫苗
CN116710079A (zh) 2020-07-24 2023-09-05 斯特兰德生物科技公司 包含经修饰的核苷酸的脂质纳米颗粒
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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
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EP4087938A2 (en) * 2021-01-27 2022-11-16 CureVac AG Method of reducing the immunostimulatory properties of in vitro transcribed rna
CN115702940A (zh) * 2021-08-05 2023-02-17 苏州科锐迈德生物医药科技有限公司 包含环状rna分子的制剂在制备治疗肿瘤的药物中的应用
CN116829578B (zh) * 2021-10-08 2025-03-21 苏州艾博生物科技有限公司 编码白细胞介素-12(il-12)的多核苷酸以及其相关组合物和方法
US12186387B2 (en) 2021-11-29 2025-01-07 BioNTech SE Coronavirus vaccine
CN116179549A (zh) 2021-12-08 2023-05-30 南京吉迈生物技术有限公司 通过5’utr序列变体修饰基因转录和翻译效率
IL316186A (en) 2022-04-26 2024-12-01 Strand Therapeutics Inc Lipid nanoparticles containing Venezuelan equine encephalitis (VEE) replicon and uses thereof
CN115960906A (zh) * 2022-05-07 2023-04-14 苏州科锐迈德生物医药科技有限公司 用于抗肿瘤免疫治疗的rna组合物、rna制剂及用途
TW202400784A (zh) 2022-05-13 2024-01-01 大陸商上海瑞宏迪醫藥有限公司 包含utr的核酸構建體及其應用
CN117327709A (zh) * 2022-06-24 2024-01-02 深圳瑞吉生物科技有限公司 用于实体肿瘤的治疗性mRNA及其应用
WO2024002985A1 (en) 2022-06-26 2024-01-04 BioNTech SE Coronavirus vaccine
CN119866224A (zh) 2022-07-28 2025-04-22 加拿大干细胞技术公司 编码连接抗原的多核苷酸以及其用途
WO2024074211A1 (en) * 2022-10-06 2024-04-11 BioNTech SE Rna compositions targeting claudin-18.2
AU2023357320A1 (en) * 2022-10-06 2025-04-17 BioNTech SE Rna compositions targeting claudin-18.2
JP2026513742A (ja) 2023-04-12 2026-05-01 ストランド セラピューティクス インコーポレイテッド 合成回路及びその使用
WO2025024559A1 (en) 2023-07-24 2025-01-30 Strand Therapeutics Inc. Rna-based synthetic circuit for producing engineered immune cells for an extracorporeal cell therapy
KR20260046366A (ko) 2023-07-25 2026-04-07 스트랜드 세러퓨틱스 인코포레이티드 마이크로 rna 비표적화 센서를 포함하는 폴리뉴클레오타이드 및 이의 용도
KR102935773B1 (ko) * 2023-09-01 2026-03-09 한양대학교 산학협력단 증강된 유전자 발현을 위한 5‘ 비번역 영역을 포함하는 뉴클레오타이드를 포함하는 조성물 및 방법
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