WO2026073043A2 - Compositions oligonucléotidiques et méthodes associées - Google Patents

Compositions oligonucléotidiques et méthodes associées

Info

Publication number
WO2026073043A2
WO2026073043A2 PCT/US2025/048309 US2025048309W WO2026073043A2 WO 2026073043 A2 WO2026073043 A2 WO 2026073043A2 US 2025048309 W US2025048309 W US 2025048309W WO 2026073043 A2 WO2026073043 A2 WO 2026073043A2
Authority
WO
WIPO (PCT)
Prior art keywords
fl2r
ssp
oligonucleotide
composition
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
PCT/US2025/048309
Other languages
English (en)
Other versions
WO2026073043A3 (fr
Inventor
Elizabeth Katherine WAGNER
Abbie Madeline MAGUIRE
Chandra Vargeese
Hsiu-Chiung Yang
Pachamuthu Kandasamy
Sruthi Madhukumar NAIR
Chikdu Shakti SHIVALILA
Ann Fiegen DURBIN
Naoki Iwamoto
Michael John Byrne
Jayakanthan Kumarasamy
Sarah Diane LAMORE
Sharon Lee CHICKLAS
Jigar Desai
Fengjiao ZHANG
Jeonghoon Choi
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
Application filed by Wave Life Sciences Pte Ltd filed Critical Wave Life Sciences Pte Ltd
Publication of WO2026073043A2 publication Critical patent/WO2026073043A2/fr
Publication of WO2026073043A3 publication Critical patent/WO2026073043A3/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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • C12N2310/321—2'-O-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
    • C12N2310/32—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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)

Abstract

Entre autres, la présente invention concerne des compositions oligonucléotidiques et des procédés associés. Dans certains modes de réalisation, des oligonucléotides sont pourvus de diverses modifications chimiques de sucres, de nucléobases et/ou de liaisons internucléotidiques et de motifs associés et sont utiles pour le saut d'exon. Selon certains modes de réalisation, la présente invention concerne des techniques utiles pour sauter l'exon 45 des transcrits de la dystrophine (DMD). Dans certains modes de réalisation, la présente invention concerne des technologies utiles pour moduler l'épissage de transcrit de DMD. Dans certains modes de réalisation, la présente divulgation concerne des méthodes de prévention ou de traitement d'états, de troubles ou de maladies comprenant la dystrophie musculaire de Duchenne.
PCT/US2025/048309 2023-09-28 2025-09-26 Compositions oligonucléotidiques et méthodes associées Pending WO2026073043A2 (fr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202363586371P 2023-09-28 2023-09-28
USPCT/US2024/049139 2024-09-27
PCT/US2024/049139 WO2025072886A1 (fr) 2023-09-28 2024-09-27 Compositions d'oligonucléotides et procédés associés

Publications (2)

Publication Number Publication Date
WO2026073043A2 true WO2026073043A2 (fr) 2026-04-02
WO2026073043A3 WO2026073043A3 (fr) 2026-05-07

Family

ID=95202294

Family Applications (2)

Application Number Title Priority Date Filing Date
PCT/US2024/049139 Pending WO2025072886A1 (fr) 2023-09-28 2024-09-27 Compositions d'oligonucléotides et procédés associés
PCT/US2025/048309 Pending WO2026073043A2 (fr) 2023-09-28 2025-09-26 Compositions oligonucléotidiques et méthodes associées

Family Applications Before (1)

Application Number Title Priority Date Filing Date
PCT/US2024/049139 Pending WO2025072886A1 (fr) 2023-09-28 2024-09-27 Compositions d'oligonucléotides et procédés associés

Country Status (2)

Country Link
AU (1) AU2024348542A1 (fr)
WO (2) WO2025072886A1 (fr)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL2872485T3 (pl) 2012-07-13 2021-05-31 Wave Life Sciences Ltd. Asymetryczna grupa pomocnicza

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR122020021379B1 (pt) * 2008-10-24 2021-05-11 Sarepta Therapeutics, Inc. oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular
SG11202009877XA (en) * 2018-04-12 2020-11-27 Wave Life Sciences Ltd Oligonucleotide compositions and methods of use thereof
CA3163608A1 (fr) * 2020-01-10 2021-07-15 Romesh R. Subramanian Complexes de ciblage de muscle et leurs utilisation
AU2021277407A1 (en) * 2020-05-22 2022-11-17 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof

Also Published As

Publication number Publication date
AU2024348542A1 (en) 2026-04-16
WO2025072886A1 (fr) 2025-04-03
WO2025072886A8 (fr) 2025-06-05
WO2025072886A9 (fr) 2025-05-15

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