TW202126808A - 用於治療血小板減少症的核酸分子及其應用 - Google Patents

用於治療血小板減少症的核酸分子及其應用 Download PDF

Info

Publication number
TW202126808A
TW202126808A TW109132616A TW109132616A TW202126808A TW 202126808 A TW202126808 A TW 202126808A TW 109132616 A TW109132616 A TW 109132616A TW 109132616 A TW109132616 A TW 109132616A TW 202126808 A TW202126808 A TW 202126808A
Authority
TW
Taiwan
Prior art keywords
nucleic acid
thpo
acid molecule
thrombocytopenia
small
Prior art date
Application number
TW109132616A
Other languages
English (en)
Chinese (zh)
Inventor
龍承 李
姜武林
Original Assignee
大陸商中美瑞康核酸技術(南通)研究院有限公司
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 大陸商中美瑞康核酸技術(南通)研究院有限公司 filed Critical 大陸商中美瑞康核酸技術(南通)研究院有限公司
Publication of TW202126808A publication Critical patent/TW202126808A/zh

Links

Images

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic 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
    • 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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1136Non-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 against growth factors, growth regulators, cytokines, lymphokines or hormones
    • 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
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/10Applications; Uses in screening processes
    • C12N2320/11Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • 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
    • C12N2830/00Vector systems having a special element relevant for transcription

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Endocrinology (AREA)
  • Epidemiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
TW109132616A 2019-09-20 2020-09-21 用於治療血小板減少症的核酸分子及其應用 TW202126808A (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
CN201910891508.8 2019-09-20
CN201910891508 2019-09-20

Publications (1)

Publication Number Publication Date
TW202126808A true TW202126808A (zh) 2021-07-16

Family

ID=74883124

Family Applications (1)

Application Number Title Priority Date Filing Date
TW109132616A TW202126808A (zh) 2019-09-20 2020-09-21 用於治療血小板減少症的核酸分子及其應用

Country Status (12)

Country Link
US (1) US20230212565A1 (https=)
EP (1) EP4036232A4 (https=)
JP (1) JP2022552940A (https=)
KR (1) KR20220070206A (https=)
CN (1) CN114585738A (https=)
AU (1) AU2020349991A1 (https=)
BR (1) BR112022005028A2 (https=)
CA (1) CA3148158A1 (https=)
CO (1) CO2022004857A2 (https=)
PE (1) PE20220604A1 (https=)
TW (1) TW202126808A (https=)
WO (1) WO2021052470A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN111849968A (zh) 2019-04-30 2020-10-30 中美瑞康核酸技术(南通)研究院有限公司 寡核苷酸分子及其在急性间歇性卟啉症治疗中的应用
WO2024216109A1 (en) * 2023-04-12 2024-10-17 Alnylam Pharmaceuticals, Inc. Formulations for oligonucleotide delivery

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6270989B1 (en) * 1991-11-05 2001-08-07 Transkaryotic Therapies, Inc. Protein production and delivery
GB2285446B (en) * 1994-01-03 1999-07-28 Genentech Inc Thrombopoietin
EP1871426B1 (en) * 2005-04-15 2017-06-07 The Regents of The University of California Small activating rna molecules and their use
WO2007086990A2 (en) * 2005-11-17 2007-08-02 Board Of Regents, The University Of Texas System Modulation of gene expression by oligomers targeted to chromosomal dna
KR101766408B1 (ko) 2009-06-10 2017-08-10 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
GB201010557D0 (en) * 2010-06-23 2010-08-11 Mina Therapeutics Ltd RNA molecules and uses thereof
KR102195514B1 (ko) * 2011-06-21 2020-12-29 미나 테라퓨틱스 리미티드 알부민 생산 및 세포 증식
CN106032532A (zh) * 2015-03-17 2016-10-19 中国医学科学院北京协和医院 一种小激活rna及其制备方法和应用
KR102599712B1 (ko) * 2015-04-22 2023-11-09 미나 테라퓨틱스 리미티드 C/EBP 알파 saRNA 조성물 및 사용 방법
US20180305689A1 (en) * 2015-04-22 2018-10-25 Mina Therapeutics Limited Sarna compositions and methods of use
JP2019500345A (ja) * 2015-12-14 2019-01-10 コールド スプリング ハーバー ラボラトリー 肝臓病の処置のための組成物および方法
CN106480028B (zh) * 2016-10-12 2019-04-05 上海市第七人民医院 TPO基因的saRNA分子及其应用

Also Published As

Publication number Publication date
KR20220070206A (ko) 2022-05-30
BR112022005028A2 (pt) 2022-07-05
AU2020349991A1 (en) 2022-04-07
WO2021052470A1 (zh) 2021-03-25
EP4036232A1 (en) 2022-08-03
CN114585738A (zh) 2022-06-03
EP4036232A4 (en) 2023-01-11
CO2022004857A2 (es) 2022-07-08
CA3148158A1 (en) 2021-03-25
PE20220604A1 (es) 2022-04-25
US20230212565A1 (en) 2023-07-06
JP2022552940A (ja) 2022-12-21

Similar Documents

Publication Publication Date Title
WO2023051822A1 (zh) 用于治疗与pcsk9相关疾病的靶向寡核苷酸
TW202126808A (zh) 用於治療血小板減少症的核酸分子及其應用
CN116685333A (zh) 一种治疗肝纤维化的siRNA及其递送制剂
CN119405823B (zh) 一种基于g四链体rna的蛋白降解靶向嵌合体及其制备方法与应用
CN118685411B (zh) 抑制agt基因表达的双链寡核苷酸、其缀合物及用途
KR102757180B1 (ko) Ctgf 유전자 특이적 이중가닥 올리고뉴클레오티드 및 이를 포함하는 섬유증 관련 질환 및 호흡기 관련 질환 예방 및 치료용 조성물
CN102028947B (zh) Fam3b基因的抑制剂和组合物及抑制方法以及脂肪肝的疗法和抑制剂的制药用途
JP2024028895A (ja) オリゴヌクレオチド分子及び腫瘍治療におけるその応用
WO2024212954A1 (zh) 抑制lpa基因表达的化合物、药物组合物及其用途
EP4653447A1 (en) Modified nucleoside monomer and double-stranded ribonucleic acid
HK40073799A (en) Nucleic acid molecule for treating thrombocytopenia and application thereof
US20240141347A1 (en) Rna delivery system for treatment of huntington's disease
WO2012145743A1 (en) Microrna-140-5p as a tumor suppressor and sensitizing agent for chemotherapy
WO2025026124A1 (zh) C4b基因抑制剂组合物及其应用
CN115137835A (zh) 一种用于治疗结肠炎的rna递送系统
WO2025185730A1 (zh) 靶向INHBE的RNAi剂
CN121844047A (zh) 抑制c3基因表达的双链寡核苷酸、缀合物以及它们的用途
TW202535429A (zh) 包含5'utr-3'utr組合的多核苷酸及其用途
CN120053691A (zh) 含hiHeps来源的外泌体的药物组合物及其应用
WO2025044868A1 (zh) 双链寡核苷酸及其在抑制血管紧张素原表达中的应用
WO2025216277A1 (ja) Rsv-a rna配列に基づく新規二本鎖rna及びその利用
WO2026028971A1 (ja) Rsv-b rna配列に基づく新規二本鎖rna及びその利用
TW202604550A (zh) 抑制angptl3基因表達的核酸分子
WO2026083945A1 (ja) Rsv nタンパク質 rna配列に基づく新規二本鎖rna及びその利用
CN118480115A (zh) 一种ApoA1来源的多肽及其用途