WO2026006477A8 - Compositions oligonucléotidiques et méthodes associées - Google Patents

Compositions oligonucléotidiques et méthodes associées

Info

Publication number
WO2026006477A8
WO2026006477A8 PCT/US2025/035290 US2025035290W WO2026006477A8 WO 2026006477 A8 WO2026006477 A8 WO 2026006477A8 US 2025035290 W US2025035290 W US 2025035290W WO 2026006477 A8 WO2026006477 A8 WO 2026006477A8
Authority
WO
WIPO (PCT)
Prior art keywords
present disclosure
disclosure provides
oligonucleotide compositions
technologies
methods
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
PCT/US2025/035290
Other languages
English (en)
Other versions
WO2026006477A1 (fr
Inventor
Stephen Lister LAKE
Xiao Shelley HU
Jianxin Gao
Yilin Zhang
Philip Ross
Vincent ADUDA
Mamoru Shimizu
Danlin Xu
Keith Andrew BOWMAN
Sarah Diane LAMORE
Michael Angelo PANZARA
Mohammed Rowshon ALAM
Hyun Gyung Jang
Asela Kalyanapriya BANDARA
Joseph Andrew HAEGELE
Mark Richard Hurtt
Varun Goel
Padmakumar NARAYANAN
Anne-Marie Li-Kwai-Cheung
Cynthia Fajardo CARACTA
Anthony TROMBINO
Lanyi XIE
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.)
Wave Life Sciences Pte Ltd
Original Assignee
Wave Life Sciences Pte Ltd
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
Priority claimed from PCT/US2024/040849 external-priority patent/WO2025030155A1/fr
Application filed by Wave Life Sciences Pte Ltd filed Critical Wave Life Sciences Pte Ltd
Publication of WO2026006477A1 publication Critical patent/WO2026006477A1/fr
Publication of WO2026006477A8 publication Critical patent/WO2026006477A8/fr
Pending legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • 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
    • 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
    • C12N2310/33—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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses
    • 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
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Hospice & Palliative Care (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Psychiatry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (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)

Abstract

Entre autres, la présente divulgation concerne diverses technologies comprenant des compositions oligonucléotidiques chiralement régulées et des technologies de fabrication et d'utilisation de telles compositions oligonucléotidiques. Dans certains modes de réalisation, la présente divulgation concerne des technologies utiles pour l'inactivation spécifique d'un allèle de transcrits de huntingtine mutante. Dans certains modes de réalisation, la présente divulgation concerne des technologies utiles pour réduire l'expression, le niveau, la quantité et/ou l'activité de transcrits de huntingtine mutante ou de produits associés. Dans certains modes de réalisation, la présente divulgation concerne des méthodes de traitement de la maladie de Huntington.
PCT/US2025/035290 2024-06-25 2025-06-25 Compositions oligonucléotidiques et méthodes associées Pending WO2026006477A1 (fr)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US202463664150P 2024-06-25 2024-06-25
US63/664,150 2024-06-25
USPCT/US2024/040849 2024-08-02
PCT/US2024/040849 WO2025030155A1 (fr) 2023-08-03 2024-08-02 Compositions d'oligonucléotides et procédés associés
US202463693161P 2024-09-10 2024-09-10
US63/693,161 2024-09-10

Publications (2)

Publication Number Publication Date
WO2026006477A1 WO2026006477A1 (fr) 2026-01-02
WO2026006477A8 true WO2026006477A8 (fr) 2026-04-23

Family

ID=98222997

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2025/035290 Pending WO2026006477A1 (fr) 2024-06-25 2025-06-25 Compositions oligonucléotidiques et méthodes associées

Country Status (1)

Country Link
WO (1) WO2026006477A1 (fr)

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL307462A (en) * 2021-04-14 2023-12-01 Prilenia Neurotherapeutics Ltd Treatment of prodromal huntington disease
AU2023321711A1 (en) * 2022-08-11 2025-02-20 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof

Also Published As

Publication number Publication date
WO2026006477A1 (fr) 2026-01-02

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