KR20220070206A - 혈소판 감소증 치료용 핵산 분자 및 그의 이용 - Google Patents

혈소판 감소증 치료용 핵산 분자 및 그의 이용 Download PDF

Info

Publication number
KR20220070206A
KR20220070206A KR1020227008451A KR20227008451A KR20220070206A KR 20220070206 A KR20220070206 A KR 20220070206A KR 1020227008451 A KR1020227008451 A KR 1020227008451A KR 20227008451 A KR20227008451 A KR 20227008451A KR 20220070206 A KR20220070206 A KR 20220070206A
Authority
KR
South Korea
Prior art keywords
dna
nucleic acid
sequence
artificial sequence
thpo
Prior art date
Legal status (The legal status 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 status listed.)
Withdrawn
Application number
KR1020227008451A
Other languages
English (en)
Korean (ko)
Inventor
롱청 리
무림 강
Original Assignee
락티젠 세러퓨틱스
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 락티젠 세러퓨틱스 filed Critical 락티젠 세러퓨틱스
Publication of KR20220070206A publication Critical patent/KR20220070206A/ko
Withdrawn legal-status Critical Current

Links

Images

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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
    • 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
    • C12N15/113—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
    • 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
    • C12N15/113—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
    • C12N15/1136—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 against growth factors, growth regulators, cytokines, lymphokines or hormones
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • 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
    • C12N2830/00—Vector 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)
KR1020227008451A 2019-09-20 2020-09-18 혈소판 감소증 치료용 핵산 분자 및 그의 이용 Withdrawn KR20220070206A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
CN201910891508.8 2019-09-20
CN201910891508 2019-09-20
PCT/CN2020/116227 WO2021052470A1 (zh) 2019-09-20 2020-09-18 用于治疗血小板减少症的核酸分子及其应用

Publications (1)

Publication Number Publication Date
KR20220070206A true KR20220070206A (ko) 2022-05-30

Family

ID=74883124

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020227008451A Withdrawn KR20220070206A (ko) 2019-09-20 2020-09-18 혈소판 감소증 치료용 핵산 분자 및 그의 이용

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
BR112022005028A2 (pt) 2022-07-05
AU2020349991A1 (en) 2022-04-07
WO2021052470A1 (zh) 2021-03-25
EP4036232A1 (en) 2022-08-03
TW202126808A (zh) 2021-07-16
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
KR102769565B1 (ko) 17베타-HSD 유형 13- (HSD17B13)의 발현을 억제하기 위한 RNAi 작용제, 그의 조성물, 및 사용 방법
IL194419A (en) Dsrna to inhibit the expression of a 5eg gene in a human cell, a pharmaceutical compound containing it, a vector method
US8153605B2 (en) Modulation of toll-like receptor 3 expression by antisense oligonucleotides
WO2023051822A1 (zh) 用于治疗与pcsk9相关疾病的靶向寡核苷酸
CN114585633A (zh) 寡核苷酸缀合物组合物和使用方法
WO2015099122A1 (ja) 遺伝子発現制御のための人工ミミックｍｉＲＮＡおよびその用途
EP4650446A1 (en) Sirna for inhibiting expression of programmed cell death 1 ligand 1 gene, conjugate thereof, pharmaceutical composition thereof, and use thereof
KR101390966B1 (ko) Ｈｉｆｌａ의 발현을 저해하는 ｓｉＲＮＡ 및 이를 포함하는 항암 조성물
TW202405174A (zh) 用於抑制超氧化物歧化酶1(SOD1)表現之RNAi藥劑、其組合物及使用方法
KR20220070206A (ko) 혈소판 감소증 치료용 핵산 분자 및 그의 이용
KR20110039381A (ko) 안티센스 올리고누클레오티드에 의한 톨-유사 수용체 7 발현의 조절
KR101168726B1 (ko) 암 치료용 약학 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
CN110551722A (zh) 一种核酸复合物及其制备方法和应用
CN117821468A (zh) 肺靶向可溶性PD-L1 mRNA脂质纳米颗粒及其应用
US11155819B2 (en) Double-stranded RNA molecule targeting CKIP-1 and use thereof
HK40073799A (en) Nucleic acid molecule for treating thrombocytopenia and application thereof
EP4317434A1 (en) Rna delivery system for treatment of huntington's disease
WO2025247332A1 (zh) 一种抑制vegfa基因和ang-2基因表达的双靶核酸分子
TW202613309A (zh) 抑制ｖｅｇｆａ基因和ａｎｇ－２基因表現的雙靶核酸分子
WO2025026124A1 (zh) C4b基因抑制剂组合物及其应用
WO2024169907A1 (zh) 一种调节补体C3表达的siRNA、其缀合物和药物组合物及用途
WO2025043720A1 (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
JP2026510866A (ja) ミトコンドリアアミドキシム還元成分１（ＭＡＲＣ１）の発現を阻害するためのＲＮＡｉ剤、その医薬組成物、及び使用方法

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

PC1203 Withdrawal of no request for examination

St.27 status event code: N-1-6-B10-B12-nap-PC1203