EP4422645A4 - Compositions oligonucléotidiques et leurs procédés d'utilisation - Google Patents

Compositions oligonucléotidiques et leurs procédés d'utilisation

Info

Publication number
EP4422645A4
EP4422645A4 EP22888111.6A EP22888111A EP4422645A4 EP 4422645 A4 EP4422645 A4 EP 4422645A4 EP 22888111 A EP22888111 A EP 22888111A EP 4422645 A4 EP4422645 A4 EP 4422645A4
Authority
EP
European Patent Office
Prior art keywords
oligonucleotide compositions
oligonucleotide
compositions
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
EP22888111.6A
Other languages
German (de)
English (en)
Other versions
EP4422645A2 (fr
Inventor
Michael John Byrne
Vinod Vathipadiekal
Naoki Iwamoto
Chandra Vargeese
Lankai Guo
Andrew Guzior Hoss
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 EP4422645A2 publication Critical patent/EP4422645A2/fr
Publication of EP4422645A4 publication Critical patent/EP4422645A4/fr
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
    • C12N15/1138—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 receptors or cell surface proteins
    • 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
    • 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
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0016—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • C12N2310/314—Phosphoramidates
    • 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
    • C12N2310/33—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

Landscapes

  • Genetics & Genomics (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Saccharide Compounds (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
EP22888111.6A 2021-10-27 2022-10-26 Compositions oligonucléotidiques et leurs procédés d'utilisation Pending EP4422645A4 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163272612P 2021-10-27 2021-10-27
PCT/US2022/047846 WO2023076352A2 (fr) 2021-10-27 2022-10-26 Compositions oligonucléotidiques et leurs procédés d'utilisation

Publications (2)

Publication Number Publication Date
EP4422645A2 EP4422645A2 (fr) 2024-09-04
EP4422645A4 true EP4422645A4 (fr) 2025-12-03

Family

ID=86158742

Family Applications (1)

Application Number Title Priority Date Filing Date
EP22888111.6A Pending EP4422645A4 (fr) 2021-10-27 2022-10-26 Compositions oligonucléotidiques et leurs procédés d'utilisation

Country Status (2)

Country Link
EP (1) EP4422645A4 (fr)
WO (1) WO2023076352A2 (fr)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11201500239VA (en) 2012-07-13 2015-03-30 Wave Life Sciences Japan Asymmetric auxiliary group
ES2917473T3 (es) 2014-01-16 2022-07-08 Wave Life Sciences Ltd Diseño quiral
CN108135921B (zh) 2015-07-22 2023-10-17 波涛生命科学有限公司 寡核苷酸组合物及其方法
CN109562122A (zh) 2016-06-03 2019-04-02 波涛生命科学有限公司 寡核苷酸、组合物及其方法
MA46905A (fr) 2016-11-23 2019-10-02 Wave Life Sciences Ltd Compositions et procédés de synthèse de phosphoramidites et d'oligonucléotides
US11718638B2 (en) 2017-06-21 2023-08-08 Wave Life Sciences Ltd. Compounds, compositions and methods for synthesis
CN111108096A (zh) 2017-09-18 2020-05-05 波涛生命科学有限公司 寡核苷酸制备技术
IL277889B2 (en) 2018-04-12 2025-01-01 Wave Life Sciences Ltd Oligonucleotide preparations and methods of using them
EP3790596A4 (fr) 2018-05-11 2022-04-06 Wave Life Sciences Ltd. Compositions d'oligonucléotides et leurs procédés d'utilisation
CN113748116A (zh) 2019-03-20 2021-12-03 波涛生命科学有限公司 可用于寡核苷酸制备的技术

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016138353A1 (fr) * 2015-02-26 2016-09-01 Ionis Pharmaceuticals, Inc. Modulateurs spécifiques alléliques de la rhodopsine p23h

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3137741A1 (fr) * 2019-04-25 2020-10-29 Wave Life Sciences Ltd. Compositions d'oligonucleotides et leurs methodes d'utilisation

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016138353A1 (fr) * 2015-02-26 2016-09-01 Ionis Pharmaceuticals, Inc. Modulateurs spécifiques alléliques de la rhodopsine p23h

Non-Patent Citations (6)

* Cited by examiner, † Cited by third party
Title
BYRNE MICHAEL ET AL: "Allele-selective Reduction Of Rho P23H-mutant Rhodopsin Rescues Phenotype Associated With Retinitis Pigmentosa In Preclinical Models", 29 September 2021 (2021-09-29), XP093313966, Retrieved from the Internet <URL:https://ir.wavelifesciences.com/static-files/bb37c918-38ba-4026-ab03-fbf8da4dd22c> *
MENG DA ET AL: "Therapy in Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa", MOLECULAR THERAPY, vol. 28, no. 10, 25 August 2020 (2020-08-25), United States, pages 2139 - 2149, XP055941895, ISSN: 1525-0016, Retrieved from the Internet <URL:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7545001/pdf/main.pdf> DOI: 10.1016/j.ymthe.2020.08.012 *
MICHAEL BYRNE ET AL: "Allele-selective reduction of P23H-mutant rhodopsin with stereopure oligonucleotides rescues phenotype associated with retinitis pigmentosa in preclinical models | IOVS | ARVO Journals", INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, June 2021 (2021-06-01), XP093314004, Retrieved from the Internet <URL:https://iovs.arvojournals.org/article.aspx?articleid=2773032> *
SUE F MURRAY ET AL: "Prevention of photoreceptor cell degeneration in P23H rats after allele-specific knockdown of mutant Rhodopsin RNA expression using antisense oligonucleotide (ASO) treatment | IOVS | ARVO Journals", INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE APRIL 2014, VOL.55, 1258, April 2014 (2014-04-01), XP055511300, Retrieved from the Internet <URL:https://iovs.arvojournals.org/article.aspx?articleid=2266473> [retrieved on 20181001] *
SUSAN F. MURRAY ET AL: "Allele-Specific Inhibition of Rhodopsin With an Antisense Oligonucleotide Slows Photoreceptor Cell Degeneration", INVESTIGATIVE OPTHALMOLOGY & VISUAL SCIENCE, vol. 56, no. 11, 5 October 2015 (2015-10-05), US, pages 6362, XP055373895, ISSN: 1552-5783, DOI: 10.1167/iovs.15-16400 *
VINOD VATHIPADIEKAL ET AL: "Stereopure oligonucleotides achieve allele-selective reduction of P23H mutant rhodopsin | IOVS | ARVO Journals", INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, June 2020 (2020-06-01), XP093314023, Retrieved from the Internet <URL:https://iovs.arvojournals.org/article.aspx?articleid=2767274> *

Also Published As

Publication number Publication date
EP4422645A2 (fr) 2024-09-04
WO2023076352A2 (fr) 2023-05-04
WO2023076352A3 (fr) 2023-09-21

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