JP2024543782A - Ｃａｇリピート疾患を治療するための組成物および方法 - Google Patents

Ｃａｇリピート疾患を治療するための組成物および方法 Download PDF

Info

Publication number
JP2024543782A
JP2024543782A JP2024521180A JP2024521180A JP2024543782A JP 2024543782 A JP2024543782 A JP 2024543782A JP 2024521180 A JP2024521180 A JP 2024521180A JP 2024521180 A JP2024521180 A JP 2024521180A JP 2024543782 A JP2024543782 A JP 2024543782A
Authority
JP
Japan
Prior art keywords
seq
mismatch
substitution
strand
double
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
JP2024521180A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024543782A5 (https=
Inventor
フィリップ、 ジェイ． ジェンシク、
キース、 ティー． ギャグノン、
デビッド コレイ、
クラーク、 エレオノラ デ
メーガン ブルウェット、
メリッサ エイ． コッターマン、
Original Assignee
アイリス メディスン，インク．
ボード・オブ・トラスティーズ、サザン・イリノイ・ユニバーシティ
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 アイリス メディスン，インク．, ボード・オブ・トラスティーズ、サザン・イリノイ・ユニバーシティ filed Critical アイリス メディスン，インク．
Publication of JP2024543782A publication Critical patent/JP2024543782A/ja
Publication of JP2024543782A5 publication Critical patent/JP2024543782A5/ja
Pending legal-status Critical Current

Links

Images

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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • 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
    • A61P25/28—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
    • C12N15/63—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
    • C12N15/86—Viral vectors
    • 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
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/531—Stem-loop; Hairpin
    • 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
    • C12N2320/50—Methods for regulating/modulating their activity
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Psychology (AREA)
  • Virology (AREA)
  • Dermatology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2024521180A 2021-10-06 2022-10-05 Ｃａｇリピート疾患を治療するための組成物および方法 Pending JP2024543782A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163253070P 2021-10-06 2021-10-06
US63/253,070 2021-10-06
US202263339363P 2022-05-06 2022-05-06
US63/339,363 2022-05-06
PCT/US2022/077572 WO2023060104A1 (en) 2021-10-06 2022-10-05 Compositions and methods for treating cag repeat diseases

Publications (2)

Publication Number Publication Date
JP2024543782A true JP2024543782A (ja) 2024-11-26
JP2024543782A5 JP2024543782A5 (https=) 2025-10-14

Family

ID=85804745

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024521180A Pending JP2024543782A (ja) 2021-10-06 2022-10-05 Ｃａｇリピート疾患を治療するための組成物および方法

Country Status (9)

Country Link
US (1) US20240425856A1 (https=)
EP (1) EP4413137A4 (https=)
JP (1) JP2024543782A (https=)
AU (1) AU2022358732A1 (https=)
CA (1) CA3232737A1 (https=)
IL (1) IL311638A (https=)
MX (1) MX2024004119A (https=)
TW (1) TW202329985A (https=)
WO (1) WO2023060104A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2024253981A1 (en) * 2023-04-05 2025-11-06 Board Of Trustees Of Southern Illinois University Compositions and methods for treating cag repeat diseases
US20240360443A1 (en) * 2023-04-09 2024-10-31 Dystrogen Gene Therapies Inc. Nucleic acid molecule and use of the nucleic acid molecule in therapy of diseases induced by expansion of trinucleotide cag repeats
AU2024332679A1 (en) * 2023-08-31 2026-04-16 Iris Medicine, Inc. Methods and compositions for treating ggggcc repeat expansion diseases
WO2025137623A1 (en) * 2023-12-22 2025-06-26 Iris Medicine, Inc. Methods and compositions for treating ctg repeat expansion diseases
WO2025213123A1 (en) * 2024-04-05 2025-10-09 Encoded Therapeutics, Inc. Methods and compositions for increasing expression of ube3a

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL243776B1 (pl) * 2013-09-02 2023-10-09 Inst Chemii Bioorganicznej Polskiej Akademii Nauk Cząsteczka kwasu nukleinowego, kaseta ekspresyjna, wektor ekspresyjny, komórka eukariotycznego gospodarza i zastosowanie cząsteczki kwasu nukleinowego w terapii chorób wywoływanych ekspansją trójnukleotydowych powtórzeń typu CAG
EP3359667A1 (en) * 2015-10-05 2018-08-15 ProQR Therapeutics II B.V. Use of single-stranded antisense oligonucleotide in prevention or treatment of genetic diseases involving a trinucleotide repeat expansion
WO2018152523A1 (en) * 2017-02-20 2018-08-23 Northwestern University Use of trinucleotide repeat rnas to treat cancer

Also Published As

Publication number Publication date
MX2024004119A (es) 2024-12-06
EP4413137A1 (en) 2024-08-14
US20240425856A1 (en) 2024-12-26
CA3232737A1 (en) 2023-04-13
IL311638A (en) 2024-05-01
WO2023060104A1 (en) 2023-04-13
TW202329985A (zh) 2023-08-01
AU2022358732A1 (en) 2024-04-11
EP4413137A4 (en) 2026-02-11

Similar Documents

Publication Publication Date Title
US20240425856A1 (en) Compositions and Methods for Treating Cag Repeat Diseases
KR20190051020A (ko) 안 질환 치료용 안티센스 올리고뉴클레오타이드
JP7565218B2 (ja) 筋萎縮性側索硬化症および脊髄に関連する障害の治療
US20180237775A1 (en) Antisense oligonucleotides and uses thereof
CN111108198A (zh) 治疗亨廷顿病的组合物和方法
BR112018073472A2 (pt) composições e métodos de tratamento da doença de huntington
AU2018346102A2 (en) Gene therapies for lysosomal disorders
WO2016196507A1 (en) Methods of delivery of transgenes for treating brain diseases
EP2627339A1 (en) A modified human u1snrna molecule, a gene encoding for the modified human u1snrna molecule, an expression vector including the gene, and the use thereof in gene therapy
WO2009086428A2 (en) Methods and compositions for increasing gene expression
US20200199625A1 (en) RNAi induced reduction of ataxin-3 for the treatment of Spinocerebellar ataxia type 3
JP2023532864A (ja) 導入遺伝子発現系
JP2023512836A (ja) 神経変性疾患を処置するための組成物および方法
JP2025513066A (ja) Ａｐｏｅの核酸制御
WO2025038430A1 (en) Aav capsid variants and uses thereof
AU2022345874A1 (en) Liver-specific expression cassettes, vectors and uses thereof for expressing therapeutic proteins
JP7787094B2 (ja) 筋萎縮性側索硬化症を治療するためのアンチセンス配列
TW202504620A (zh) 用於治療ｃａｇ重複疾病之組成物和方法
CA3272944A1 (en) MAPPING COMPOSITIONS AND REGULATION METHODS
WO2024078345A1 (zh) snRNA核酸分子及其应用
WO2025213123A1 (en) Methods and compositions for increasing expression of ube3a
CA2999192C (en) Antisense oligonucleotides hybridizing with a key element of the polyadenylation region of a dux4 pre-mrna and uses thereof
AU2024265701A1 (en) A modular system to convert therapeutic microrna expression cassettes from polymerase iii-based to polymerase ii-based promoters
WO2025007937A1 (zh) 靶向USH2A pre-mRNA假外显子PE40的snRNA及其应用
EP4118436A1 (en) Diagnostic methods using sirt1 expression

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20251003

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20251003