KR20210099090A - Msh3 활성과 연관된 트리뉴클레오티드 반복 확장 장애의 치료 방법 - Google Patents

Msh3 활성과 연관된 트리뉴클레오티드 반복 확장 장애의 치료 방법 Download PDF

Info

Publication number
KR20210099090A
KR20210099090A KR1020217020797A KR20217020797A KR20210099090A KR 20210099090 A KR20210099090 A KR 20210099090A KR 1020217020797 A KR1020217020797 A KR 1020217020797A KR 20217020797 A KR20217020797 A KR 20217020797A KR 20210099090 A KR20210099090 A KR 20210099090A
Authority
KR
South Korea
Prior art keywords
oligonucleotide
msh3
cell
sequence
oligonucleotides
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
KR1020217020797A
Other languages
English (en)
Korean (ko)
Inventor
네선 앤서니 버밍엄
브라이언 알. 베텐코트
피터 에드워드 비알렉
Original Assignee
트리플렛 테라퓨틱스, 인코포레이티드
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 트리플렛 테라퓨틱스, 인코포레이티드 filed Critical 트리플렛 테라퓨틱스, 인코포레이티드
Publication of KR20210099090A publication Critical patent/KR20210099090A/ko
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
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • 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
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • 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
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • 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 ===---===

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurosurgery (AREA)
  • Biotechnology (AREA)
  • Neurology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Psychiatry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Hospice & Palliative Care (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
KR1020217020797A 2018-12-03 2019-12-02 Msh3 활성과 연관된 트리뉴클레오티드 반복 확장 장애의 치료 방법 Pending KR20210099090A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862774791P 2018-12-03 2018-12-03
US62/774,791 2018-12-03
US201962877142P 2019-07-22 2019-07-22
US62/877,142 2019-07-22
PCT/US2019/064054 WO2020117702A1 (en) 2018-12-03 2019-12-02 Methods for the treatment of trinucleotide repeat expansion disorders associated with msh3 activity

Publications (1)

Publication Number Publication Date
KR20210099090A true KR20210099090A (ko) 2021-08-11

Family

ID=70974779

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020217020797A Pending KR20210099090A (ko) 2018-12-03 2019-12-02 Msh3 활성과 연관된 트리뉴클레오티드 반복 확장 장애의 치료 방법

Country Status (7)

Country Link
US (1) US20220072028A1 (https=)
JP (1) JP7714162B6 (https=)
KR (1) KR20210099090A (https=)
AU (1) AU2019394847A1 (https=)
CA (1) CA3121779A1 (https=)
MX (1) MX2021006463A (https=)
WO (2) WO2020117702A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20210355491A1 (en) * 2020-04-20 2021-11-18 University Of Massachusetts Oligonucleotides for msh3 modulation
US20230313185A1 (en) * 2020-05-08 2023-10-05 Triplet Therapeutics, Inc. Methods for the treatment of nucleotide repeat expansion disorders associated with msh3 activity
AR122534A1 (es) 2020-06-03 2022-09-21 Triplet Therapeutics Inc Métodos para el tratamiento de los trastornos de expansión por repetición de nucleótidos asociados con la actividad de msh3
EP4247392A4 (en) * 2020-06-03 2025-10-08 Takeda Pharmaceuticals Usa Inc METHODS FOR THE TREATMENT OF TRINUCLEOTIDE REPEAT EXPANSION DISORDERS ASSOCIATED WITH MSH3 ACTIVITY
WO2021252799A2 (en) * 2020-06-11 2021-12-16 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing msh3 expression
US20240263179A1 (en) * 2021-06-04 2024-08-08 Takeda Pharmaceuticals U.S.A., Inc. Methods for the treatment of nucleotide repeat expansion disorders associated with msh3 activity
WO2023168304A2 (en) * 2022-03-02 2023-09-07 Triplet Therapeutics, Inc. Methods for the treatment of nucleotide repeat expansion disorders associated with msh3 activity
WO2024251211A1 (en) * 2023-06-06 2024-12-12 Revir Therapeutics, Inc. Compositions, methods, and systems for modulating splicing of msh3

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999010369A1 (en) * 1997-08-28 1999-03-04 Thomas Jefferson University Compositions, kits, and methods for effecting adenine nucleotide modulation of dna mismatch recognition proteins
US20050244851A1 (en) * 2004-01-13 2005-11-03 Affymetrix, Inc. Methods of analysis of alternative splicing in human
WO2008157747A1 (en) * 2007-06-21 2008-12-24 The Jackson Laboratory Use of inhibition of exonuclease 1 in methods for therapy and diagnostic of neurodegenerative diseases, eye diseases, and mitochondrial disorders
US10202599B2 (en) * 2011-08-11 2019-02-12 Ionis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
WO2015171918A2 (en) * 2014-05-07 2015-11-12 Louisiana State University And Agricultural And Mechanical College Compositions and uses for treatment thereof
EP4464338A3 (en) * 2014-11-07 2025-02-12 Editas Medicine, Inc. Systems for improving crispr/cas-mediated genome-editing
EP3806850A2 (en) * 2018-06-15 2021-04-21 Ideaya Biosciences, Inc. Methods of inhibiting proliferative cells
AR122534A1 (es) * 2020-06-03 2022-09-21 Triplet Therapeutics Inc Métodos para el tratamiento de los trastornos de expansión por repetición de nucleótidos asociados con la actividad de msh3
US20240263179A1 (en) * 2021-06-04 2024-08-08 Takeda Pharmaceuticals U.S.A., Inc. Methods for the treatment of nucleotide repeat expansion disorders associated with msh3 activity

Also Published As

Publication number Publication date
WO2020117703A1 (en) 2020-06-11
MX2021006463A (es) 2021-09-28
JP2022510041A (ja) 2022-01-25
JP7714162B2 (ja) 2025-07-29
WO2020117702A1 (en) 2020-06-11
CA3121779A1 (en) 2020-06-11
AU2019394847A1 (en) 2021-07-22
US20220072028A1 (en) 2022-03-10
JP7714162B6 (ja) 2025-09-08

Similar Documents

Publication Publication Date Title
CN108368507B (zh) 程序性细胞死亡1配体1(PD-L1)的iRNA组合物及其使用方法
CN108064154B (zh) 用于抑制hao1(羟酸氧化酶1(乙醇酸盐氧化酶))基因表达的组合物及方法
US20240287524A1 (en) GLUCOKINASE (GCK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
KR20230033651A (ko) Serpina1의 adar-매개 편집을 위한 방법 및 조성물
KR20210099090A (ko) Msh3 활성과 연관된 트리뉴클레오티드 반복 확장 장애의 치료 방법
KR20200133373A (ko) 17β-하이드록시스테로이드 데하이드로게나제 유형 13 (HSD17B13) iRNA 조성물 및 그것의 사용 방법
KR20160118346A (ko) 케토헥소키나제(ＫＨＫ) ｉＲＮＡ 조성물 및 그의 사용 방법
CN113631709A (zh) 用于治疗kcnt1相关病症的组合物和方法
US12297432B2 (en) Oligonucleotides for the treatment of nucleotide repeat expansion disorders associated with MSH3 activity
CN117203336A (zh) 亨廷顿蛋白(HTT)iRNA药剂组合物以及其使用方法
US12485136B2 (en) Methods for the treatment of trinucleotide repeat expansion disorders associated with MLH3 activity
US20240263179A1 (en) Methods for the treatment of nucleotide repeat expansion disorders associated with msh3 activity
AU2020394627B2 (en) Methods for the treatment of nucleotide repeat expansion disorders associated with MSH3 activity
US20250002912A1 (en) Compositions and methods for the treatment of pcdh19 related disorders
US20230041178A1 (en) Methods for the treatment of trinucleotide repeat exapnsion disorders associated with ogg1 activity
HK40072587A (en) Programmed cell death 1 ligand 1 (pd-l1) irna compositions and methods of use thereof
HK1255138B (en) Programmed cell death 1 ligand 1 (pd-l1) irna compositions and methods of use thereof

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

A201 Request for examination
PA0201 Request for examination

St.27 status event code: A-1-2-D10-D11-exm-PA0201

E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

St.27 status event code: A-1-2-D10-D21-exm-PE0902

T11-X000 Administrative time limit extension requested

St.27 status event code: U-3-3-T10-T11-oth-X000

E13 Pre-grant limitation requested

Free format text: ST27 STATUS EVENT CODE: A-2-3-E10-E13-LIM-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-NAP-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

PN2301 Change of applicant

St.27 status event code: A-3-3-R10-R11-asn-PN2301

R11 Change to the name of applicant or owner or transfer of ownership requested

Free format text: ST27 STATUS EVENT CODE: A-3-3-R10-R11-ASN-PN2301 (AS PROVIDED BY THE NATIONAL OFFICE)

D22 Grant of ip right intended

Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D22-EXM-PE0701 (AS PROVIDED BY THE NATIONAL OFFICE)

PE0701 Decision of registration

St.27 status event code: A-1-2-D10-D22-exm-PE0701