KR20220035137A - Ppm1a 억제제 및 그의 사용 방법 - Google Patents

Ppm1a 억제제 및 그의 사용 방법 Download PDF

Info

Publication number
KR20220035137A
KR20220035137A KR1020227002134A KR20227002134A KR20220035137A KR 20220035137 A KR20220035137 A KR 20220035137A KR 1020227002134 A KR1020227002134 A KR 1020227002134A KR 20227002134 A KR20227002134 A KR 20227002134A KR 20220035137 A KR20220035137 A KR 20220035137A
Authority
KR
South Korea
Prior art keywords
ppm1a
oligonucleotide
seq
patient
disease
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Withdrawn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
KR1020227002134A
Other languages
English (en)
Korean (ko)
Inventor
샌드라 힝클리
던컨 브라운
수드히르 아그라왈
다니엘 엘바움
Original Assignee
큐랄리스 코포레이션
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 큐랄리스 코포레이션 filed Critical 큐랄리스 코포레이션
Publication of KR20220035137A publication Critical patent/KR20220035137A/ko
Withdrawn legal-status Critical Current

Links

Images

Classifications

    • 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
    • C12N15/1137Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • 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
    • C12N2310/315Phosphorothioates
    • 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
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
    • 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
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3515Lipophilic moiety, e.g. cholesterol
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/31Combination therapy
    • YGENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02ATECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Neurology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Neurosurgery (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • 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)
KR1020227002134A 2019-06-21 2020-06-19 Ppm1a 억제제 및 그의 사용 방법 Withdrawn KR20220035137A (ko)

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
KR20220035137A true KR20220035137A (ko) 2022-03-21

Family

ID=74040507

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020227002134A Withdrawn KR20220035137A (ko) 2019-06-21 2020-06-19 Ppm1a 억제제 및 그의 사용 방법

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
KR20240062036A (ko) * 2022-11-01 2024-05-08 윤주호 콜치신과 메트포르민을 유효성분으로 하는 퇴행성 신경계 질환의 예방 또는 치료용 조성물

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
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

Family Cites Families (11)

* Cited by examiner, † Cited by third party
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
JPH11512294A (ja) * 1995-09-01 1999-10-26 ラモット ユニヴァーシティー オーソリティ フォア アップライド リサーチ アンド インダストリアル ディヴェロプメント リミテッド 癌の治療、予防および発見を目的とする腫瘍細胞での蛋白ホスファターゼ2C−PP2Cα−発現の操作と検出
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
US20040102397A1 (en) * 2002-11-22 2004-05-27 Isis Pharmaceuticals Inc. Modulation of PPM1B expression
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
WO2011073903A1 (en) * 2009-12-14 2011-06-23 Koninklijke Philips Electronics N.V. Novel tumor markers
US20140200261A1 (en) * 2013-01-17 2014-07-17 Moderna Therapeutics, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes
US11096956B2 (en) * 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20240062036A (ko) * 2022-11-01 2024-05-08 윤주호 콜치신과 메트포르민을 유효성분으로 하는 퇴행성 신경계 질환의 예방 또는 치료용 조성물
WO2024096461A3 (ko) * 2022-11-01 2024-11-07 윤주호 콜치신과 메트포르민을 유효성분으로 하는 퇴행성 신경계 질환의 예방 또는 치료용 조성물

Also Published As

Publication number Publication date
CN114729355A (zh) 2022-07-08
WO2020257631A3 (en) 2021-01-21
US20220372489A1 (en) 2022-11-24
IL289127A (en) 2022-02-01
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

Similar Documents

Publication Publication Date Title
RU2730677C2 (ru) Соединение для модуляции экспрессии гена c9orf72 и его применение
KR102482890B1 (ko) 아포지질단백질 (a) 발현을 조절하는 조성물 및 방법
KR102285629B1 (ko) Sod-1 발현을 조절하기 위한 조성물
KR102258516B1 (ko) C9orf72 발현을 조절하기 위한 조성물
AU2018409843B2 (en) Compounds and methods for reducing SNCA expression
TWI834177B (zh) 用於減少tau表現之組合物及方法
AU2017336093B2 (en) Compounds and methods for reducing Tau expression
AU2019384181B2 (en) Compounds and methods for reducing prion expression
AU2018203564A1 (en) Antisense modulation of gccr expression
KR20220035137A (ko) Ppm1a 억제제 및 그의 사용 방법
KR20210038589A (ko) Atxn2 발현 감소용 화합물 및 방법
KR20210008497A (ko) Atxn3 발현 감소용 화합물 및 방법
KR20220033472A (ko) 신경계 질환을 치료하기 위한 올리고뉴클레오티드 및 사용 방법
KR20180016970A (ko) 타우 안티센스 올리고머와 그 용도
KR20190076025A (ko) Atxn3 발현을 감소시키기 위한 화합물 및 방법
KR20200140853A (ko) B형 간염 바이러스 감염을 치료하기 위한 fubp1 억제제의 용도
KR20230043914A (ko) App 발현을 감소시키기 위한 화합물 및 방법
KR101839177B1 (ko) Ptpib 발현의 안티센스 조절
US6448080B1 (en) Antisense modulation of WRN expression
CA3241316A1 (en) Gapmer antisense oligonucleotides with modified backbone chemistries
RU2858547C2 (ru) Композиции для модулирования экспрессии sod-1

Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20220120

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
PC1202 Submission of document of withdrawal before decision of registration

Comment text: [Withdrawal of Procedure relating to Patent, etc.] Withdrawal (Abandonment)

Patent event code: PC12021R01D

Patent event date: 20231120

WITB Written withdrawal of application