AU2022345098B2 - Compositions and methods of treating facioscapulohumeral muscular dystrophy - Google Patents

Compositions and methods of treating facioscapulohumeral muscular dystrophy

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Publication number
AU2022345098B2
AU2022345098B2 AU2022345098A AU2022345098A AU2022345098B2 AU 2022345098 B2 AU2022345098 B2 AU 2022345098B2 AU 2022345098 A AU2022345098 A AU 2022345098A AU 2022345098 A AU2022345098 A AU 2022345098A AU 2022345098 B2 AU2022345098 B2 AU 2022345098B2
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acid molecule
polynucleic acid
instances
seq
sequence
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AU2022345098A1 (en
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Rob BURKE
Beatrice Diana DARIMONT
Barbora MALECOVA
David SALA CANO
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Avidity Biosciences Inc
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Avidity Biosciences Inc
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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2881—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against CD71
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/68—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • A61K47/6801—Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
    • A61K47/6803—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
    • A61K47/6807—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates the drug or compound being a sugar, nucleoside, nucleotide, nucleic acid, e.g. RNA antisense
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/3513—Protein; Peptide
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    • C12N2310/35—Nature of the modification
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    • C12N2310/3521—Methyl
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    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Peptides Or Proteins (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
AU2022345098A 2021-09-16 2022-09-15 Compositions and methods of treating facioscapulohumeral muscular dystrophy Active AU2022345098B2 (en)

Applications Claiming Priority (3)

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US202163245123P 2021-09-16 2021-09-16
US63/245,123 2021-09-16
PCT/US2022/043705 WO2023043953A1 (en) 2021-09-16 2022-09-15 Compositions and methods of treating facioscapulohumeral muscular dystrophy

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AU2022345098A1 AU2022345098A1 (en) 2024-04-04
AU2022345098B2 true AU2022345098B2 (en) 2026-02-12

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US (5) US11912779B2 (enExample)
EP (1) EP4401792A4 (enExample)
JP (2) JP7742485B2 (enExample)
KR (1) KR20240055874A (enExample)
CN (1) CN118265544A (enExample)
AU (1) AU2022345098B2 (enExample)
CA (1) CA3231330A1 (enExample)
IL (2) IL326535A (enExample)
MX (1) MX2024003266A (enExample)
WO (1) WO2023043953A1 (enExample)

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JP2019513371A (ja) 2016-04-01 2019-05-30 アビディティー バイオサイエンシーズ エルエルシー 核酸ポリペプチド組成物とその使用
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WO2021188390A1 (en) * 2020-03-19 2021-09-23 Avidity Biosciences, Inc. Compositions and methods of treating facioscapulohumeral muscular dystrophy
CN118265544A (zh) 2021-09-16 2024-06-28 艾维迪提生物科学公司 治疗面肩肱型肌营养不良的组合物和方法
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WO2025259607A1 (en) * 2024-06-10 2025-12-18 Avidity Biosciences, Inc. Novel biomarker for determining prognosis of facioscapulohumeral muscular dystrophy
EP4663240A1 (en) * 2024-06-12 2025-12-17 Ospedale San Raffaele S.r.l. Ifn- pathway inhibitors for use in the treatment of fshd

Citations (2)

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