CN115297868B - 用于编码核糖核酸的器官保护性表达和调节的组合物及方法 - Google Patents

用于编码核糖核酸的器官保护性表达和调节的组合物及方法 Download PDF

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CN115297868B
CN115297868B CN202180016068.7A CN202180016068A CN115297868B CN 115297868 B CN115297868 B CN 115297868B CN 202180016068 A CN202180016068 A CN 202180016068A CN 115297868 B CN115297868 B CN 115297868B
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mirna
cells
mrna
sequences
cell
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CN115297868A (zh
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罗曼·米科尔
瓦莱丽·杜瓦尔
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United Therapeutics Corp
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • 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
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
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    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
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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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
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  • General Health & Medical Sciences (AREA)
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  • Biotechnology (AREA)
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  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
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  • Immunology (AREA)
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  • Communicable Diseases (AREA)
  • Mycology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicinal Preparation (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
CN202180016068.7A 2020-02-21 2021-02-22 用于编码核糖核酸的器官保护性表达和调节的组合物及方法 Active CN115297868B (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202062979619P 2020-02-21 2020-02-21
US62/979,619 2020-02-21
US202063059458P 2020-07-31 2020-07-31
US63/059,458 2020-07-31
PCT/US2021/019028 WO2021168405A1 (en) 2020-02-21 2021-02-22 Compositions and methods for organ-protective expression and modulation of coding ribonucleic acids

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CN115297868A CN115297868A (zh) 2022-11-04
CN115297868B true CN115297868B (zh) 2024-04-12

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US (4) US11596685B2 (https=)
EP (1) EP4106770A4 (https=)
JP (1) JP7539723B2 (https=)
KR (2) KR102838785B1 (https=)
CN (1) CN115297868B (https=)
AU (2) AU2021224990B2 (https=)
CA (1) CA3168048A1 (https=)
IL (1) IL295724A (https=)
MX (1) MX2022010084A (https=)
WO (1) WO2021168405A1 (https=)
ZA (1) ZA202208795B (https=)

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GB201714430D0 (en) * 2017-09-07 2017-10-25 Micol Romain Compositions and processes for targeted delivery and expression and modulation of therapeutic components in tissue
EP4065600A1 (en) 2019-11-25 2022-10-05 Cornell University Apoe gene therapy
CN113583977B (zh) * 2020-04-30 2025-06-06 杭州康万达医药科技有限公司 可受微小rna调控的分离的重组溶瘤痘病毒及其应用
MX2023001187A (es) * 2020-07-31 2023-04-04 Combined Therapeutics Inc Composiciones y métodos para una vacunación mejorada.
CN116445492A (zh) * 2022-01-10 2023-07-18 北京锦篮基因科技有限公司 用于x染色体连锁肾上腺脑白质营养不良的基因治疗药物和方法
CN114874999B (zh) * 2022-04-22 2023-04-21 中国医学科学院病原生物学研究所 一种基于痘苗病毒载体的新型冠状病毒病毒样颗粒疫苗
CN116004628B (zh) * 2023-01-03 2024-07-19 苏州大学 一种预防或治疗慢性疼痛的miRNA靶点及其应用
CN117530932B (zh) * 2024-01-08 2024-04-26 中国药科大学 一种抑制肿瘤生长及肺转移的人工外泌体及其应用
WO2025251079A1 (en) * 2024-05-31 2025-12-04 University Of Massachusetts Rna compositions and methods of use thereof in the treatment of cancer

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US7683036B2 (en) 2003-07-31 2010-03-23 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
WO2009012263A2 (en) 2007-07-18 2009-01-22 The Trustees Of Columbia University In The City Of New York Tissue-specific micrornas and compositions and uses thereof
US7993831B2 (en) * 2007-09-14 2011-08-09 Asuragen, Inc. Methods of normalization in microRNA detection assays
WO2010126766A1 (en) * 2009-04-30 2010-11-04 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Inducible interleukin-12
CA3018046A1 (en) 2011-12-16 2013-06-20 Moderna Therapeutics, Inc. Modified nucleoside, nucleotide, and nucleic acid compositions
CN108474003A (zh) 2015-11-20 2018-08-31 俄勒冈健康与科学大学 包含微小rna识别元件的cmv载体
SI3458083T1 (sl) * 2016-05-18 2023-03-31 Modernatx, Inc. Polinukleotidi, ki kodirajo interlevkin-12 (IL12) in njihova uporaba
CN110402145A (zh) * 2016-10-26 2019-11-01 莫得纳特斯公司 用于增强免疫应答的信使核糖核酸及其使用方法
WO2018115527A2 (en) * 2016-12-23 2018-06-28 Curevac Ag Mers coronavirus vaccine
GB201714430D0 (en) 2017-09-07 2017-10-25 Micol Romain Compositions and processes for targeted delivery and expression and modulation of therapeutic components in tissue
EP3986447A4 (en) * 2019-06-18 2023-08-16 Citranvi Biosciences, LLC RATIONALLY MANIPULATED CARRIER PROTEINS FOR VACCINES
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Publication number Publication date
US20230241206A1 (en) 2023-08-03
US11931409B2 (en) 2024-03-19
AU2021224990B2 (en) 2025-08-28
JP2023514594A (ja) 2023-04-06
US11596685B2 (en) 2023-03-07
ZA202208795B (en) 2023-11-29
IL295724A (en) 2022-10-01
WO2021168405A1 (en) 2021-08-26
EP4106770A4 (en) 2024-05-15
KR20250114450A (ko) 2025-07-29
EP4106770A1 (en) 2022-12-28
AU2025271119A1 (en) 2025-12-18
US20240252618A1 (en) 2024-08-01
MX2022010084A (es) 2022-12-07
US20220202932A1 (en) 2022-06-30
KR20220144831A (ko) 2022-10-27
KR102838785B1 (ko) 2025-07-28
AU2021224990A1 (en) 2022-09-15
JP7539723B2 (ja) 2024-08-26
US12220456B2 (en) 2025-02-11
US20250170233A1 (en) 2025-05-29
CN115297868A (zh) 2022-11-04
CA3168048A1 (en) 2021-08-26

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