WO2023122316A2 - Molécules d'oligonucléotides modulant l'épissage (sso) pour cibler la bêta-caténine et leurs utilisations - Google Patents

Molécules d'oligonucléotides modulant l'épissage (sso) pour cibler la bêta-caténine et leurs utilisations Download PDF

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Publication number
WO2023122316A2
WO2023122316A2 PCT/US2022/053895 US2022053895W WO2023122316A2 WO 2023122316 A2 WO2023122316 A2 WO 2023122316A2 US 2022053895 W US2022053895 W US 2022053895W WO 2023122316 A2 WO2023122316 A2 WO 2023122316A2
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Prior art keywords
sso
catenin
exon
seq
less
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PCT/US2022/053895
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English (en)
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WO2023122316A3 (fr
Inventor
Jin Hong
Leonid Beigelman
Aneerban BHATTACHARYA
Laxman Eltepu
Vera HUANG
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Aligos Therapeutics, Inc.
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Application filed by Aligos Therapeutics, Inc. filed Critical Aligos Therapeutics, Inc.
Publication of WO2023122316A2 publication Critical patent/WO2023122316A2/fr
Publication of WO2023122316A3 publication Critical patent/WO2023122316A3/fr

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    • 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
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • 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
    • C12N2320/31—Combination therapy
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

La présente invention se rapporte au domaine de composés et de préparations pharmaceutiques et à leur méthode d'utilisation dans le traitement d'une maladie. L'invention concerne des molécules d'oligonucléotide modulant l'épissage (SSO) pour cibler la bêta-caténine, des compositions les contenant, et leurs utilisations pour traiter ou prévenir des maladies ou des troubles associés à la bêta-caténine. En particulier, la présente invention concerne le domaine des molécules SSO qui ciblent la bêta-caténine pour le traitement du cancer.
PCT/US2022/053895 2021-12-23 2022-12-22 Molécules d'oligonucléotides modulant l'épissage (sso) pour cibler la bêta-caténine et leurs utilisations WO2023122316A2 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163293557P 2021-12-23 2021-12-23
US63/293,557 2021-12-23

Publications (2)

Publication Number Publication Date
WO2023122316A2 true WO2023122316A2 (fr) 2023-06-29
WO2023122316A3 WO2023122316A3 (fr) 2023-08-03

Family

ID=85172415

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2022/053895 WO2023122316A2 (fr) 2021-12-23 2022-12-22 Molécules d'oligonucléotides modulant l'épissage (sso) pour cibler la bêta-caténine et leurs utilisations

Country Status (1)

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WO (1) WO2023122316A2 (fr)

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017160983A1 (fr) * 2016-03-16 2017-09-21 Dicerna Pharmaceuticals, Inc. Compositions et méthodes de traitement de maladies et de troubles associés à la bêta-caténine
AU2018350983A1 (en) * 2017-10-18 2020-04-09 Dicerna Pharmaceuticals, Inc. Beta catenin nucleic acid inhibitor molecule
WO2021207641A1 (fr) * 2020-04-10 2021-10-14 Aligos Therapeutics, Inc. Molécules d'oligonucléotide antisens (aso) et leurs utilisations pour des maladies à coronavirus

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
"Genbank", Database accession no. NM_001904.3
MARTIN: "Remington's Pharmaceutical Sciences", 1975, MACK PUBL. CO.
MASAHIKO HORIBATAKAO YAMAGUCHISATOSHI OBIKA, JOURNAL OF ORGANIC CHEMISTRY, vol. 81, 2016, pages 11000 - 11008
TAKAO YAMAGUCHIMASAHIKO HORIBASATOSHI OBIKA, CHEM. COMMUN., vol. 51, 2015, pages 9737 - 9740
VONBRULL ET AL., MOLECULAR BIOTECHNOLOGY, vol. 60, 2018, pages 339 - 349

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WO2023122316A3 (fr) 2023-08-03

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