CN114729355A - Ppm1a抑制剂以及使用其的方法 - Google Patents

Ppm1a抑制剂以及使用其的方法 Download PDF

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Publication number
CN114729355A
CN114729355A CN202080057853.2A CN202080057853A CN114729355A CN 114729355 A CN114729355 A CN 114729355A CN 202080057853 A CN202080057853 A CN 202080057853A CN 114729355 A CN114729355 A CN 114729355A
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China
Prior art keywords
oligonucleotide
ppm1a
seq
patient
disease
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Pending
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CN202080057853.2A
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English (en)
Chinese (zh)
Inventor
S.辛克利
D.布朗
S.阿加瓦尔
D.埃尔鲍姆
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Quelis Co
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Quelis Co
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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
    • C12N15/1137—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 enzymes
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
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    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

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  • Chemical & Material Sciences (AREA)
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  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
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  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Neurosurgery (AREA)
  • Biochemistry (AREA)
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  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Psychology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN202080057853.2A 2019-06-21 2020-06-19 Ppm1a抑制剂以及使用其的方法 Pending CN114729355A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962864988P 2019-06-21 2019-06-21
US62/864,988 2019-06-21
US201962871356P 2019-07-08 2019-07-08
US62/871,356 2019-07-08
PCT/US2020/038703 WO2020257631A2 (en) 2019-06-21 2020-06-19 Ppm1a inhibitors and methods of using same

Publications (1)

Publication Number Publication Date
CN114729355A true CN114729355A (zh) 2022-07-08

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CN202080057853.2A Pending CN114729355A (zh) 2019-06-21 2020-06-19 Ppm1a抑制剂以及使用其的方法

Country Status (9)

Country Link
US (1) US20220372489A1 (https=)
EP (1) EP3987030A4 (https=)
JP (1) JP2022537581A (https=)
KR (1) KR20220035137A (https=)
CN (1) CN114729355A (https=)
AU (1) AU2020296104A1 (https=)
CA (1) CA3144063A1 (https=)
IL (1) IL289127A (https=)
WO (1) WO2020257631A2 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN117031014A (zh) * 2023-08-14 2023-11-10 陕西脉元生物科技有限公司 抗matr3自身抗体的试剂在制备检测神经系统自身免疫疾病的产品中的应用及试剂盒

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4392562A4 (en) * 2021-08-27 2025-09-24 Ionis Pharmaceuticals Inc COMPOUNDS AND METHODS FOR MODULATING SCN1A EXPRESSION
WO2023102188A1 (en) * 2021-12-03 2023-06-08 Quralis Corporation Gapmer antisense oligonucleotides with modified backbone chemistries
KR102856378B1 (ko) * 2022-09-30 2025-09-08 주식회사 젬백스앤카엘 4r 타우병증의 치료 또는 예방용 펩티드
KR102775989B1 (ko) * 2022-11-01 2025-03-04 윤주호 콜치신과 메트포르민을 유효성분으로 하는 퇴행성 신경계 질환의 예방 또는 치료용 조성물

Citations (4)

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Publication number Priority date Publication date Assignee Title
WO1997010796A2 (en) * 1995-09-01 1997-03-27 Ramot-University Authority For Applied Research And Industrial Development Ltd. MANIPULATION AND DETECTION OF PROTEIN PHOSPHATASE 2C - PP2Cα - EXPRESSION IN TUMOR CELLS FOR CANCER THERAPY, PREVENTION AND DETECTION
US20040102397A1 (en) * 2002-11-22 2004-05-27 Isis Pharmaceuticals Inc. Modulation of PPM1B expression
WO2011073903A1 (en) * 2009-12-14 2011-06-23 Koninklijke Philips Electronics N.V. Novel tumor markers
US20140206852A1 (en) * 2013-01-17 2014-07-24 Moderna Therapeutics, Inc. Signal-sensor polynucleotides

Family Cites Families (7)

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Publication number Priority date Publication date Assignee Title
CA2227306A1 (en) * 1995-09-01 1997-03-27 Ramot-University Authority For Applied Research And Industrial Developme Nt Ltd. Manipulation and detection of protein phosphatase 2c - pp2c.alpha. - expression in tumor cells for cancer therapy, prevention and detection
US20030050270A1 (en) * 1998-11-20 2003-03-13 Monia Brett P. Antisense modulation of Inhibitor-kappa B Kinase-beta expression
WO2003014321A2 (en) * 2001-08-10 2003-02-20 The Rockefeller University Compositions and methods for modulation of darpp-32 phosphorylation
GB0207251D0 (en) * 2002-03-27 2002-05-08 Isis Innovation Lymphoma-associated antigens
AU2003270900A1 (en) * 2002-09-25 2004-04-19 Pharmacia Corporation Antisense modulation of microsomal prostaglandin e2 synthase expression
AU2003271651A1 (en) * 2002-10-04 2004-04-23 Bayer Healthcare Ag Regulation of human pp2c-like protein phosphatase
US11096956B2 (en) * 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof

Patent Citations (4)

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Publication number Priority date Publication date Assignee Title
WO1997010796A2 (en) * 1995-09-01 1997-03-27 Ramot-University Authority For Applied Research And Industrial Development Ltd. MANIPULATION AND DETECTION OF PROTEIN PHOSPHATASE 2C - PP2Cα - EXPRESSION IN TUMOR CELLS FOR CANCER THERAPY, PREVENTION AND DETECTION
US20040102397A1 (en) * 2002-11-22 2004-05-27 Isis Pharmaceuticals Inc. Modulation of PPM1B expression
WO2011073903A1 (en) * 2009-12-14 2011-06-23 Koninklijke Philips Electronics N.V. Novel tumor markers
US20140206852A1 (en) * 2013-01-17 2014-07-24 Moderna Therapeutics, Inc. Signal-sensor polynucleotides

Non-Patent Citations (3)

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Title
AKHLAQ A. FAROOQUI: "Lipid Mediators and Their Metabolism in the Nucleus: Implications for Alzheimer’s Disease", JOURNAL OF ALZHEIMER\'S DISEASE, vol. 30, pages 163 *
J.-H. HU等: "Protein kinase and protein phosphatase expression in amyotrophic lateral sclerosis spinal cord", JOURNAL OF NEUROCHEMISTRY, vol. 85, pages 432 - 442, XP002592836 *
REGINA AUGUSTIN等: "Bioinformatics Identification ofModules of Transcription Factor Binding Sites in Alzheimer’s Disease-Related Genes by In Silico Promoter Analysis andMicroarrays", INTERNATIONAL JOURNAL OF ALZHEIMER’S DISEASE, vol. 2011, pages 1 - 13 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN117031014A (zh) * 2023-08-14 2023-11-10 陕西脉元生物科技有限公司 抗matr3自身抗体的试剂在制备检测神经系统自身免疫疾病的产品中的应用及试剂盒

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Publication number Publication date
WO2020257631A3 (en) 2021-01-21
US20220372489A1 (en) 2022-11-24
IL289127A (en) 2022-02-01
KR20220035137A (ko) 2022-03-21
EP3987030A4 (en) 2023-10-18
AU2020296104A1 (en) 2022-01-27
EP3987030A2 (en) 2022-04-27
WO2020257631A2 (en) 2020-12-24
CA3144063A1 (en) 2022-01-14
JP2022537581A (ja) 2022-08-26

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Application publication date: 20220708