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WO2025064660A3 - Compositions d'arni de récepteur de type 1c de l'activine a (acvr1c) et leurs procédés d'utilisation - Google Patents

Compositions d'arni de récepteur de type 1c de l'activine a (acvr1c) et leurs procédés d'utilisation Download PDF

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Publication number
WO2025064660A3
WO2025064660A3 PCT/US2024/047464 US2024047464W WO2025064660A3 WO 2025064660 A3 WO2025064660 A3 WO 2025064660A3 US 2024047464 W US2024047464 W US 2024047464W WO 2025064660 A3 WO2025064660 A3 WO 2025064660A3
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WIPO (PCT)
Prior art keywords
acvr1c
methods
activin
receptor type
irna compositions
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Pending
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PCT/US2024/047464
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WO2025064660A2 (fr
Inventor
Elane FISHILEVICH
Aditi Dubey
James D. MCININCH
Mark K. SCHLEGEL
Adam CASTORENO
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Alnylam Pharmaceuticals Inc
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Alnylam Pharmaceuticals Inc
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Publication of WO2025064660A2 publication Critical patent/WO2025064660A2/fr
Publication of WO2025064660A3 publication Critical patent/WO2025064660A3/fr
Pending legal-status Critical Current
Anticipated expiration legal-status Critical

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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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • 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
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol

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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)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

La présente divulgation concerne des agents d'ARNi, par exemple des agents d'ARN double brin (ARNdb), ciblant un gène de récepteur de type 1C de l''activine A (ACVR1C). L'invention concerne également des procédés d'utilisation de tels agents d'ARNi pour inhiber l'expression d'un gène ACVR1C et des procédés de prévention et de traitement d'un trouble associé à l'ACVRIC, par exemple, un trouble métabolique, par exemple, le syndrome métabolique.
PCT/US2024/047464 2023-09-21 2024-09-19 Compositions d'arni de récepteur de type 1c de l'activine a (acvr1c) et leurs procédés d'utilisation Pending WO2025064660A2 (fr)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202363539682P 2023-09-21 2023-09-21
US63/539,682 2023-09-21
US202463694377P 2024-09-13 2024-09-13
US63/694,377 2024-09-13

Publications (2)

Publication Number Publication Date
WO2025064660A2 WO2025064660A2 (fr) 2025-03-27
WO2025064660A3 true WO2025064660A3 (fr) 2025-05-30

Family

ID=93117301

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2024/047464 Pending WO2025064660A2 (fr) 2023-09-21 2024-09-19 Compositions d'arni de récepteur de type 1c de l'activine a (acvr1c) et leurs procédés d'utilisation

Country Status (2)

Country Link
TW (1) TW202535433A (fr)
WO (1) WO2025064660A2 (fr)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025137384A1 (fr) 2023-12-20 2025-06-26 Arrowhead Pharmaceuticals, Inc. Procédés et réactifs pour une synthèse améliorée d'oligonucléotides

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011112671A2 (fr) * 2010-03-09 2011-09-15 President And Fellows Of Harvard College Conversion de cellules endothéliales vasculaires en cellules de type cellules souches multipotentes
WO2023003922A1 (fr) * 2021-07-21 2023-01-26 Alnylam Pharmaceuticals, Inc. Compositions d'arni de gène cible associé à un trouble métabolique et leurs méthodes d'utilisation
WO2023064530A1 (fr) * 2021-10-15 2023-04-20 Alnylam Pharmaceuticals, Inc. Compositions d'arni à administration extra-hépatique et leurs procédés d'utilisation

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