CN110337305A - 治疗性rna - Google Patents

治疗性rna Download PDF

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Publication number
CN110337305A
CN110337305A CN201880013857.3A CN201880013857A CN110337305A CN 110337305 A CN110337305 A CN 110337305A CN 201880013857 A CN201880013857 A CN 201880013857A CN 110337305 A CN110337305 A CN 110337305A
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China
Prior art keywords
rna
seq
protein
composition
tumor
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CN201880013857.3A
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English (en)
Chinese (zh)
Inventor
F·吉塞克
U·萨欣
D·G·威德沙因
T·R·瓦格纳尔
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Debiotech SA
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Sanofi Aventis France
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Publication of CN110337305A publication Critical patent/CN110337305A/zh
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    • 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
    • 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]
    • A61K38/2086—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
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    • 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
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    • 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
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    • 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
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    • 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
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    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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)
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  • General Health & Medical Sciences (AREA)
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  • Pharmacology & Pharmacy (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
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  • Biophysics (AREA)
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  • Biotechnology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • 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)
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CN201880013857.3A 2017-02-28 2018-02-27 治疗性rna Pending CN110337305A (zh)

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

Publications (1)

Publication Number Publication Date
CN110337305A true CN110337305A (zh) 2019-10-15

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CN201880013857.3A Pending CN110337305A (zh) 2017-02-28 2018-02-27 治疗性rna

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US (3) US20200147176A1 (cg-RX-API-DMAC10.html)
EP (1) EP3589308A1 (cg-RX-API-DMAC10.html)
JP (4) JP6949131B2 (cg-RX-API-DMAC10.html)
KR (2) KR20240144261A (cg-RX-API-DMAC10.html)
CN (1) CN110337305A (cg-RX-API-DMAC10.html)
AU (1) AU2018229278A1 (cg-RX-API-DMAC10.html)
BR (1) BR112019017743A2 (cg-RX-API-DMAC10.html)
CA (1) CA3054733A1 (cg-RX-API-DMAC10.html)
CL (1) CL2019002461A1 (cg-RX-API-DMAC10.html)
CO (1) CO2019010355A2 (cg-RX-API-DMAC10.html)
CR (1) CR20190444A (cg-RX-API-DMAC10.html)
EC (1) ECSP19070336A (cg-RX-API-DMAC10.html)
IL (1) IL268894A (cg-RX-API-DMAC10.html)
MA (1) MA47680A (cg-RX-API-DMAC10.html)
MX (1) MX2019010269A (cg-RX-API-DMAC10.html)
PE (1) PE20200735A1 (cg-RX-API-DMAC10.html)
PH (1) PH12019501959A1 (cg-RX-API-DMAC10.html)
SG (1) SG11201907846VA (cg-RX-API-DMAC10.html)
TW (2) TW201842921A (cg-RX-API-DMAC10.html)
UY (1) UY37621A (cg-RX-API-DMAC10.html)
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CN113557060A (zh) * 2019-01-21 2021-10-26 赛诺菲 用于晚期实体瘤癌症的治疗性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
WO2023056915A1 (en) * 2021-10-08 2023-04-13 Suzhou Abogen Biosciences Co., Ltd. Polynucleotides encoding interleukin-12 (il-12) and related composition and methods 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
CN116438306A (zh) * 2020-10-20 2023-07-14 St制药株式会社 用于5'-加帽的rna合成的寡核苷酸
CN116585464A (zh) * 2020-04-22 2023-08-15 生物技术欧洲股份公司 Rna构建体及其用途
WO2023246938A1 (zh) * 2022-06-24 2023-12-28 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

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US11629340B2 (en) 2017-03-03 2023-04-18 Obsidian Therapeutics, Inc. DHFR tunable protein regulation
EP4253544A3 (en) 2017-05-18 2023-12-20 ModernaTX, Inc. Modified messenger rna comprising functional rna elements
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TW202026423A (zh) * 2018-08-24 2020-07-16 法商賽諾菲公司 用於實體瘤癌症的治療性ｒｎａ
CN115120716A (zh) * 2019-01-14 2022-09-30 健泰科生物技术公司 用pd-1轴结合拮抗剂和rna疫苗治疗癌症的方法
AU2020210614A1 (en) * 2019-01-21 2021-08-26 Sanofi Therapeutic RNA and anti-PD1 antibodies for advanced stage solid tumor cancers
JP7740988B2 (ja) * 2019-03-12 2025-09-17 バイオエヌテック エスエー 前立腺癌のための治療用ｒｎａ
WO2020200481A1 (en) * 2019-04-05 2020-10-08 Biontech Rna Pharmaceuticals Gmbh Treatment involving interleukin-2 (il2) and interferon (ifn)
EP4529928A3 (en) * 2019-10-09 2025-05-28 Translate Bio, Inc. Compositions, methods and uses of messenger rna
US20220380720A1 (en) * 2019-11-12 2022-12-01 Actym Therapeutics, Inc. Immunostimulatory bacteria delivery platforms and their use for delivery of therapeutic products
BR112022015077A2 (pt) * 2020-01-31 2022-10-04 Genentech Inc Métodos para induzir células t cd8+ específicas de neoepítopos em um indivíduo com um tumor e para induzir o tráfico de células t cd8+, vacinas de rna, vacina de rna para uso e antagonistas de ligação
JP2023535225A (ja) * 2020-07-24 2023-08-16 ストランド セラピューティクス インコーポレイテッド 改変ヌクレオチドを含む脂質ナノ粒子
EP4240396B1 (en) * 2020-11-04 2025-01-01 Ethris GmbH Ifn-lambda mrna for treating viral infections
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
KR20230129479A (ko) 2021-01-08 2023-09-08 스트랜드 세러퓨틱스 인코포레이티드 발현 구성체 및 이의 용도
CA3170747A1 (en) * 2021-01-27 2022-08-04 Moritz THRAN Method of reducing the immunostimulatory properties of in vitro transcribed rna
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
AU2023262576A1 (en) 2022-04-26 2024-10-10 Strand Therapeutics Inc. Lipid nanoparticles comprising venezuelan equine encephalitis (vee) replicon and uses thereof
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
US11878055B1 (en) 2022-06-26 2024-01-23 BioNTech SE Coronavirus vaccine
CA3262766A1 (en) 2022-07-28 2024-02-01 Stemcell Technologies Canada Inc. Polynucleotides encoding bound antigens and their uses
CN120129702A (zh) * 2022-10-06 2025-06-10 生物技术公司 靶向密蛋白-18.2的rna组合物
WO2024074211A1 (en) * 2022-10-06 2024-04-11 BioNTech SE Rna compositions targeting claudin-18.2
KR20260003656A (ko) 2023-04-12 2026-01-07 스트랜드 세러퓨틱스 인코포레이티드 합성 회로 및 이의 용도
KR20260042228A (ko) 2023-07-24 2026-03-30 스트랜드 세러퓨틱스 인코포레이티드 체외 세포 요법을 위한 조작된 면역 세포를 생산하기 위한 rna 기반 합성 회로
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
TW202532107A (zh) 2023-11-14 2025-08-16 大陸商上海瑞宏迪醫藥有限公司 一種含核酸構建體的醫藥組成物及其醫藥用途
WO2025217591A1 (en) 2024-04-12 2025-10-16 Strand Therapeutics Inc. Human-derived synthetic regulators and uses thereof
WO2026165118A1 (en) 2025-01-29 2026-08-06 Strand Therapeutics Inc. Polynucleotides comprising detargeting sensors and uses thereof

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