JP2022552940A - 血小板減少症を治療するための核酸分子及びその応用 - Google Patents

血小板減少症を治療するための核酸分子及びその応用 Download PDF

Info

Publication number
JP2022552940A
JP2022552940A JP2022517729A JP2022517729A JP2022552940A JP 2022552940 A JP2022552940 A JP 2022552940A JP 2022517729 A JP2022517729 A JP 2022517729A JP 2022517729 A JP2022517729 A JP 2022517729A JP 2022552940 A JP2022552940 A JP 2022552940A
Authority
JP
Japan
Prior art keywords
nucleic acid
thpo
small molecule
thrombocytopenia
activating
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.)
Pending
Application number
JP2022517729A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022552940A5 (https=
Inventor
ロンチョン リ
ムーリン カン
Original Assignee
ラクティゲン セラピューティクス
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 ラクティゲン セラピューティクス filed Critical ラクティゲン セラピューティクス
Publication of JP2022552940A publication Critical patent/JP2022552940A/ja
Publication of JP2022552940A5 publication Critical patent/JP2022552940A5/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • 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
    • 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
    • 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)
JP2022517729A 2019-09-20 2020-09-18 血小板減少症を治療するための核酸分子及びその応用 Pending JP2022552940A (ja)

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 (2)

Publication Number Publication Date
JP2022552940A true JP2022552940A (ja) 2022-12-21
JP2022552940A5 JP2022552940A5 (https=) 2023-09-25

Family

ID=74883124

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022517729A Pending JP2022552940A (ja) 2019-09-20 2020-09-18 血小板減少症を治療するための核酸分子及びその応用

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

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2008538896A (ja) * 2005-04-15 2008-11-13 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 小分子活性化ｒｎａ分子及び使用方法
JP2009516512A (ja) * 2005-11-17 2009-04-23 ボード・オブ・リージエンツ，ザ・ユニバーシテイ・オブ・テキサス・システム 染色体ｄｎａに標的化されるオリゴマーによる遺伝子発現の調節
JP2013532973A (ja) * 2010-06-23 2013-08-22 ミナ セラピューティクス リミテッド Ｒｎａ分子およびその使用
JP2018512876A (ja) * 2015-04-22 2018-05-24 ミナ セラピューティクス リミテッド ｓａＲＮＡ組成物および使用方法
JP2018516545A (ja) * 2015-04-22 2018-06-28 ミナ セラピューティクス リミテッド Ｃ／ＥＢＰアルファｓａＲＮＡ組成物および使用方法

Family Cites Families (7)

* 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
KR101766408B1 (ko) 2009-06-10 2017-08-10 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
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
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

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2008538896A (ja) * 2005-04-15 2008-11-13 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 小分子活性化ｒｎａ分子及び使用方法
JP2009516512A (ja) * 2005-11-17 2009-04-23 ボード・オブ・リージエンツ，ザ・ユニバーシテイ・オブ・テキサス・システム 染色体ｄｎａに標的化されるオリゴマーによる遺伝子発現の調節
JP2013532973A (ja) * 2010-06-23 2013-08-22 ミナ セラピューティクス リミテッド Ｒｎａ分子およびその使用
JP2018512876A (ja) * 2015-04-22 2018-05-24 ミナ セラピューティクス リミテッド ｓａＲＮＡ組成物および使用方法
JP2018516545A (ja) * 2015-04-22 2018-06-28 ミナ セラピューティクス リミテッド Ｃ／ＥＢＰアルファｓａＲＮＡ組成物および使用方法

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
ACS SYNTH. BIOL., vol. 6, JPN6024045523, 2017, pages 628 - 637, ISSN: 0005587069 *
BLOOD, vol. Vol.100(10), JPN6024045521, 2002, pages 3457 - 3469, ISSN: 0005587067 *
BLOOD, vol. Vol.130(9), JPN6024045522, 2017, pages 1097 - 1103, ISSN: 0005587068 *
BRITISH JOURNAL OF HAEMATOLOGY, vol. pp.682-711, JPN6024045524, 2018, ISSN: 0005587070 *
日内会誌, vol. 98, JPN6024045520, 2009, pages 1662 - 1668, ISSN: 0005587066 *

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
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

Similar Documents

Publication Publication Date Title
US12409226B2 (en) Delivery and formulation of engineered nucleic acids
JP2022008404A (ja) Ｃ／ＥＢＰα小分子活性化ＲＮＡ組成物
IL194419A (en) Dsrna to inhibit the expression of a 5eg gene in a human cell, a pharmaceutical compound containing it, a vector method
CN108064313A (zh) 用于抑制因子xii的基因表达的组合物和方法
JP2022552940A (ja) 血小板減少症を治療するための核酸分子及びその応用
CN118685411B (zh) 抑制agt基因表达的双链寡核苷酸、其缀合物及用途
WO2022166814A1 (zh) 一种化学修饰的小激活rna
KR102757180B1 (ko) Ctgf 유전자 특이적 이중가닥 올리고뉴클레오티드 및 이를 포함하는 섬유증 관련 질환 및 호흡기 관련 질환 예방 및 치료용 조성물
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
KR20230022167A (ko) Mitf 유전자를 표적으로 하는 핵산 분자 및 이의 사용
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
EP4119665A1 (en) Sirna based on rna sequence of sars-cov-2 and use thereof
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
WO2026019837A1 (en) Compositions and methods for overcoming t-cell exhaustion
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
CN115137835A (zh) 一种用于治疗结肠炎的rna递送系统
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
CN119074947A (zh) 一种肝靶向小核酸药物载体、药用组合物及其制备方法和应用
CN121775152A (zh) 一种药物靶向递送系统及其在制备抗肿瘤药物中的应用
CN121909024A (zh) 包封有核酸的配体修饰脂质纳米颗粒的制造方法

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230914

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20230914

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20241112

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20250507