CA3144063A1 - Ppm1a inhibitors and methods of using same - Google Patents

Ppm1a inhibitors and methods of using same Download PDF

Info

Publication number
CA3144063A1
CA3144063A1 CA3144063A CA3144063A CA3144063A1 CA 3144063 A1 CA3144063 A1 CA 3144063A1 CA 3144063 A CA3144063 A CA 3144063A CA 3144063 A CA3144063 A CA 3144063A CA 3144063 A1 CA3144063 A1 CA 3144063A1
Authority
CA
Canada
Prior art keywords
oligonucleotide
linkage
seq
patient
ppm1a
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
CA3144063A
Other languages
English (en)
French (fr)
Inventor
Sandra HINCKLEY
Duncan Brown
Sudhir Agrawal
Daniel Elbaum
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.)
Quralis Corp
Original Assignee
Quralis Corp
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 Quralis Corp filed Critical Quralis Corp
Publication of CA3144063A1 publication Critical patent/CA3144063A1/en
Pending legal-status Critical Current

Links

Classifications

    • 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
    • C12N15/1137—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
    • A61P25/00—Drugs for disorders of the nervous system
    • 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
    • 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
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
    • Y—GENERAL 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
    • 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

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)
CA3144063A 2019-06-21 2020-06-19 Ppm1a inhibitors and methods of using same Pending CA3144063A1 (en)

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
CA3144063A1 true CA3144063A1 (en) 2022-01-14

Family

ID=74040507

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3144063A Pending CA3144063A1 (en) 2019-06-21 2020-06-19 Ppm1a inhibitors and methods of using same

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=)

Families Citing this family (5)

* 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
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

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
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
JP2022537581A (ja) 2022-08-26

Similar Documents

Publication Publication Date Title
JP7795914B2 (ja) 神経学的疾患を処置するためのオリゴヌクレオチドおよびその使用方法
TWI833770B (zh) 用於減少 lrrk2 表現之化合物及方法
US20220372489A1 (en) Ppm1a inhibitors and methods of using same
US20180312839A1 (en) Methods and compositions for increasing smn expression
US20250051766A1 (en) Treatment of neurological diseases using modulators of unc13a gene transcripts
WO2023102242A9 (en) Splice switcher antisense oligonucleotides with modified backbone chemistries
IL313205A (en) Microbial consortia
US20230235332A1 (en) Treatment of neurological diseases using modulators of gene transcripts
WO2023102188A1 (en) Gapmer antisense oligonucleotides with modified backbone chemistries
WO2023102548A1 (en) Treatment of neurological diseases using modulators of kcnq2 gene transcripts
EP4720294A2 (en) Modified unc13a oligonucleotides
WO2023102227A2 (en) Treatment of neurological diseases using modulators of smn2 gene transcripts
HK40121931A (zh) 具有修饰的主链化学的gapmer反义寡核苷酸
IL313204A (en) Methods for the synthesis of factor D complement inhibitors
HK40097883A (zh) 使用基因转录物调控剂治疗神经学疾病

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20220914