CA3148158A1 - Nucleic acid molecule for treating thrombocytopenia and use thereof - Google Patents

Nucleic acid molecule for treating thrombocytopenia and use thereof Download PDF

Info

Publication number
CA3148158A1
CA3148158A1 CA3148158A CA3148158A CA3148158A1 CA 3148158 A1 CA3148158 A1 CA 3148158A1 CA 3148158 A CA3148158 A CA 3148158A CA 3148158 A CA3148158 A CA 3148158A CA 3148158 A1 CA3148158 A1 CA 3148158A1
Authority
CA
Canada
Prior art keywords
seq
nucleic acid
thpo
acid molecule
small 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
CA3148158A
Other languages
English (en)
French (fr)
Inventor
Longcheng Li
Moorim KANG
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Ractigen Therapeutics
Original Assignee
Ractigen Therapeutics
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 Ractigen Therapeutics filed Critical Ractigen Therapeutics
Publication of CA3148158A1 publication Critical patent/CA3148158A1/en
Pending legal-status Critical Current

Links

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)
CA3148158A 2019-09-20 2020-09-18 Nucleic acid molecule for treating thrombocytopenia and use thereof Pending CA3148158A1 (en)

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
CA3148158A1 true CA3148158A1 (en) 2021-03-25

Family

ID=74883124

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3148158A Pending CA3148158A1 (en) 2019-09-20 2020-09-18 Nucleic acid molecule for treating thrombocytopenia and use thereof

Country Status (12)

Country Link
US (1) US20230212565A1 (cg-RX-API-DMAC10.html)
EP (1) EP4036232A4 (cg-RX-API-DMAC10.html)
JP (1) JP2022552940A (cg-RX-API-DMAC10.html)
KR (1) KR20220070206A (cg-RX-API-DMAC10.html)
CN (1) CN114585738A (cg-RX-API-DMAC10.html)
AU (1) AU2020349991A1 (cg-RX-API-DMAC10.html)
BR (1) BR112022005028A2 (cg-RX-API-DMAC10.html)
CA (1) CA3148158A1 (cg-RX-API-DMAC10.html)
CO (1) CO2022004857A2 (cg-RX-API-DMAC10.html)
PE (1) PE20220604A1 (cg-RX-API-DMAC10.html)
TW (1) TW202126808A (cg-RX-API-DMAC10.html)
WO (1) WO2021052470A1 (cg-RX-API-DMAC10.html)

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
AU2024255186A1 (en) * 2023-04-12 2025-10-30 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
JP5362350B2 (ja) * 2005-04-15 2013-12-11 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
CA2629664A1 (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
KR20220038506A (ko) 2009-06-10 2022-03-28 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
GB201010557D0 (en) * 2010-06-23 2010-08-11 Mina Therapeutics Ltd RNA molecules and uses thereof
WO2012175958A1 (en) * 2011-06-21 2012-12-27 Mina Therapeutics Limited Albumin production and cell proliferation
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
CN108291228A (zh) * 2015-04-22 2018-07-17 米纳治疗有限公司 C/EBPαSARNA组合物和使用方法
EP3286318A2 (en) * 2015-04-22 2018-02-28 Mina Therapeutics Limited Sarna compositions and methods of use
WO2017106283A1 (en) * 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of liver diseases
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
AU2020349991A1 (en) 2022-04-07
US20230212565A1 (en) 2023-07-06
PE20220604A1 (es) 2022-04-25
TW202126808A (zh) 2021-07-16
CO2022004857A2 (es) 2022-07-08
EP4036232A1 (en) 2022-08-03
EP4036232A4 (en) 2023-01-11
BR112022005028A2 (pt) 2022-07-05
JP2022552940A (ja) 2022-12-21
KR20220070206A (ko) 2022-05-30
WO2021052470A1 (zh) 2021-03-25
CN114585738A (zh) 2022-06-03

Similar Documents

Publication Publication Date Title
CN113164509B (zh) 用于抑制17β-HSD 13型(HSD17B13)表达的RNAi试剂、其组合物和使用方法
JP2023156455A (ja) 第ｘｉｉ因子の遺伝子発現を阻害するための組成物と方法
EP4036232A1 (en) Nucleic acid molecule for treating immune thrombocytopenia and application thereof
JP2026503499A (ja) 修飾されたヌクレオシド単量体及び二本鎖リボ核酸
WO2025223537A1 (zh) 一种dsRNA、其应用及制备方法
TWI915897B (zh) Cfb抑制劑組合物及其應用
US20260009029A1 (en) Compound for inhibiting expression of lpa gene, pharmaceutical composition and use thereof
TW202613309A (zh) 抑制ｖｅｇｆａ基因和ａｎｇ－２基因表現的雙靶核酸分子
WO2025119212A1 (zh) 一种新型的双链siRNA、其缀合物及其用途
KR20260061212A (ko) 인히빈 서브유닛 베타 E (INHBE)의 발현을 억제하기 위한 RNAi 작용제, 그의 제약 조성물, 및 사용 방법
KR20260040626A (ko) 안지오텐시노겐을 표적으로 하는 올리고뉴클레오티드 및 이의 용도
WO2024212918A1 (zh) 抑制c3基因表达的化合物、药物组合物及其用途
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
HK40073799A (en) Nucleic acid molecule for treating thrombocytopenia and application thereof
CN114127292A (zh) 减轻反义核酸的毒性的方法
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
EP4317434A1 (en) Rna delivery system for treatment of huntington's disease
CN118995719B (zh) 抑制lpa基因表达的双链寡核苷酸及其应用
WO2025026124A1 (zh) C4b基因抑制剂组合物及其应用
CN119563025A (zh) 一种抑制LP(a)基因表达的dsRNA分子及其应用
TW202604550A (zh) 抑制ａｎｇｐｔｌ３基因表達的核酸分子
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
WO2026028971A1 (ja) Ｒｓｖ－ｂ ｒｎａ配列に基づく新規二本鎖ｒｎａ及びその利用
WO2024240080A1 (zh) 双链寡核苷酸及其缀合物和用途
WO2026012477A1 (zh) 一种dsRNA、其应用及制备方法

Legal Events

Date Code Title Description
MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 4TH ANNIV.) - STANDARD

Year of fee payment: 4

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20240712

D11 Substantive examination requested

Free format text: ST27 STATUS EVENT CODE: A-1-1-D10-D11-D117 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REQUEST FOR EXAMINATION RECEIVED

Effective date: 20240916

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P11-P101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT RECEIVED - VOLUNTARY AMENDMENT

Effective date: 20240916

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT DETERMINED COMPLIANT

Effective date: 20241019

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20241019

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20241202

D00 Search and/or examination requested or commenced

Free format text: ST27 STATUS EVENT CODE: A-1-1-D10-D00-D118 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REQUEST FOR EXAMINATION REQUIREMENTS DETERMINED COMPLIANT

Effective date: 20241203

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P11-P102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT DETERMINED COMPLIANT

Effective date: 20241203

P13 Application amended

Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P13-X000 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: APPLICATION AMENDED

Effective date: 20241203

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20241203

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20250415

D11 Substantive examination requested

Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D11-D155 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: ALL REQUIREMENTS FOR EXAMINATION DETERMINED COMPLIANT

Effective date: 20250507

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20260318

U13 Renewal or maintenance fee not paid

Free format text: ST27 STATUS EVENT CODE: N-2-6-U10-U13-U300 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: DEEMED ABANDONED - FAILURE TO RESPOND TO MAINTENANCE FEE NOTICE

Effective date: 20260519