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WO2024249328A3 - Compositions de modulation de sod1 et leurs procédés d'utilisation - Google Patents

Compositions de modulation de sod1 et leurs procédés d'utilisation Download PDF

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Publication number
WO2024249328A3
WO2024249328A3 PCT/US2024/031069 US2024031069W WO2024249328A3 WO 2024249328 A3 WO2024249328 A3 WO 2024249328A3 US 2024031069 W US2024031069 W US 2024031069W WO 2024249328 A3 WO2024249328 A3 WO 2024249328A3
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Prior art keywords
methods
sod1
compositions
expression
aspects
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WO2024249328A2 (fr
Inventor
Zhen Li
Rui ZHU
Huey-Jing Huang
Bo Cheng
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AdaRx Pharmaceuticals Inc
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AdaRx Pharmaceuticals Inc
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Priority to AU2024279767A priority Critical patent/AU2024279767A1/en
Publication of WO2024249328A2 publication Critical patent/WO2024249328A2/fr
Publication of WO2024249328A3 publication Critical patent/WO2024249328A3/fr
Anticipated expiration legal-status Critical
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    • C12Y115/00—Oxidoreductases acting on superoxide as acceptor (1.15)
    • C12Y115/01—Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
    • C12Y115/01001—Superoxide dismutase (1.15.1.1)
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    • 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
    • C12N15/1137—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 enzymes
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/312—Phosphonates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/3125—Methylphosphonates
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/351—Conjugate
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide

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  • Biophysics (AREA)
  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Des aspects de la divulgation concernent des composés, des compositions et des procédés pour moduler l'expression ou l'activité de la superoxyde dismutase 1 (SOD1). Selon certains aspects, les composés, les compositions et les procédés de l'invention peuvent être utilisés pour réduire l'expression de l'ARNm de SOD1 chez une cellule ou un animal. Selon certains aspects, les composés, les compositions et les procédés de l'invention peuvent être utilisés pour réduire l'expression de la protéine SOD1 chez une cellule ou un animal.
PCT/US2024/031069 2023-05-26 2024-05-24 Compositions de modulation de sod1 et leurs procédés d'utilisation Pending WO2024249328A2 (fr)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2024279767A AU2024279767A1 (en) 2023-05-26 2024-05-24 Sod1-modulating compositions and methods of use thereof

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US202363504692P 2023-05-26 2023-05-26
US63/504,692 2023-05-26
US202363510090P 2023-06-23 2023-06-23
US63/510,090 2023-06-23
US202363613687P 2023-12-21 2023-12-21
US63/613,687 2023-12-21

Publications (2)

Publication Number Publication Date
WO2024249328A2 WO2024249328A2 (fr) 2024-12-05
WO2024249328A3 true WO2024249328A3 (fr) 2025-01-02

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Country Status (3)

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AU (1) AU2024279767A1 (fr)
TW (1) TW202511483A (fr)
WO (1) WO2024249328A2 (fr)

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WO2022174000A2 (fr) * 2021-02-12 2022-08-18 Alnylam Pharmaceuticals, Inc. Compositions d'arni de superoxyde dismutase 1 (sod1) et procédés d'utilisation correspondants pour traiter ou prévenir des maladies neurodégénératives associées à la superoxyde dismutase 1 (sod1)

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