CA3236136A1 - Compositions d'oligonucleotides et leurs methodes d'utilisation - Google Patents

Compositions d'oligonucleotides et leurs methodes d'utilisation Download PDF

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Publication number
CA3236136A1
CA3236136A1 CA3236136A CA3236136A CA3236136A1 CA 3236136 A1 CA3236136 A1 CA 3236136A1 CA 3236136 A CA3236136 A CA 3236136A CA 3236136 A CA3236136 A CA 3236136A CA 3236136 A1 CA3236136 A1 CA 3236136A1
Authority
CA
Canada
Prior art keywords
oligonucleotide
rho
linkage
oligonucleotides
sequence
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
CA3236136A
Other languages
English (en)
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 CA3236136A1 publication Critical patent/CA3236136A1/fr
Pending legal-status Critical Current

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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
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===

Abstract

La présente invention concerne, entre autres, des oligonucléotides RHO, des compositions et des méthodes. Selon certains modes de réalisation, les oligonucléotides selon l'invention comprennent des modifications de nucléobases, des modifications de sucres, des modifications de liaisons internucléotidiques et/ou des motifs associés, et ont des propriétés, activités et/ou sélectivités améliorées. Selon certains autres modes de réalisation, la présente invention concerne des oligonucléotides RHO, des compositions et des méthodes de prévention et/ou de traitement d'états, de troubles ou de maladies liés à RHO, tels que la rétinopathie (par exemple, la dégénérescence rétinienne, la maladie dégénérative de la rétine, le trouble dégénératif de la rétine, le trouble dégénératif de la rétine héréditaire, la rétinite pigmentaire, la rétinite pigmentaire dominante autosomique, etc.).
CA3236136A 2021-10-27 2021-10-27 Compositions d'oligonucleotides et leurs methodes d'utilisation Pending CA3236136A1 (fr)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
PCT/US2021/056900 WO2023075766A1 (fr) 2021-10-27 2021-10-27 Compositions d'oligonucléotides et leurs méthodes d'utilisation

Publications (1)

Publication Number Publication Date
CA3236136A1 true CA3236136A1 (fr) 2023-05-04

Family

ID=86158394

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3236136A Pending CA3236136A1 (fr) 2021-10-27 2021-10-27 Compositions d'oligonucleotides et leurs methodes d'utilisation

Country Status (3)

Country Link
AU (1) AU2021471586A1 (fr)
CA (1) CA3236136A1 (fr)
WO (1) WO2023075766A1 (fr)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110088113A (zh) 2016-11-23 2019-08-02 波涛生命科学有限公司 用于亚磷酰胺和寡核苷酸合成的组合物和方法

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2019218987A1 (en) * 2018-02-12 2020-07-23 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof
BR112021021214A2 (pt) * 2019-04-25 2022-01-04 Wave Life Sciences Ltd Oligonucleotídeo; composição de oligonucleotídeo quiralmente controlada; composição farmacêutica; método para prevenir, tratar ou melhorar uma afecção, distúrbio ou doença relacionada a rho em um indivíduo suscetível à mesma ou que sofre da mesma; método para diminuir a atividade, expressão e/ou nível de um gene-alvo rho ou seu produto de gene em uma célula; método para supressão de uma transcrição de uma sequência-alvo rho; e composto, oligonucleotídeo, composição ou método
US20220145300A1 (en) * 2019-05-09 2022-05-12 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof

Also Published As

Publication number Publication date
AU2021471586A1 (en) 2024-05-09
WO2023075766A1 (fr) 2023-05-04

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