CA3226361A1 - Muscle targeting complexes and uses thereof for modulation of genes associated with muscle health - Google Patents

Muscle targeting complexes and uses thereof for modulation of genes associated with muscle health Download PDF

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Publication number
CA3226361A1
CA3226361A1 CA3226361A CA3226361A CA3226361A1 CA 3226361 A1 CA3226361 A1 CA 3226361A1 CA 3226361 A CA3226361 A CA 3226361A CA 3226361 A CA3226361 A CA 3226361A CA 3226361 A1 CA3226361 A1 CA 3226361A1
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Prior art keywords
seq
amino acid
acid sequence
optionally
complex
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Pending
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CA3226361A
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English (en)
French (fr)
Inventor
Romesh R. Subramanian
Mohammed T. QATANANI
Cody A. Desjardins
Duncan Brown
Victor Kotelianski
Timothy Weeden
Brendan QUINN
John NAJIM
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Dyne Therapeutics Inc
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Dyne Therapeutics Inc
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Publication of CA3226361A1 publication Critical patent/CA3226361A1/en
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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    • 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
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    • 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
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    • 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
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    • C12Y204/02—Pentosyltransferases (2.4.2)
    • C12Y204/02008—Hypoxanthine phosphoribosyltransferase (2.4.2.8)
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CA3226361A 2021-07-09 2022-07-01 Muscle targeting complexes and uses thereof for modulation of genes associated with muscle health Pending CA3226361A1 (en)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US202163220071P 2021-07-09 2021-07-09
US202163220039P 2021-07-09 2021-07-09
US202163220085P 2021-07-09 2021-07-09
US202163220056P 2021-07-09 2021-07-09
US202163220050P 2021-07-09 2021-07-09
US63/220,071 2021-07-09
US63/220,050 2021-07-09
US63/220,039 2021-07-09
US63/220,056 2021-07-09
US63/220,085 2021-07-09
PCT/US2022/073362 WO2023283531A2 (en) 2021-07-09 2022-07-01 Muscle targeting complexes and uses thereof for modulation of genes associated with muscle health

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CA3226361A1 true CA3226361A1 (en) 2023-01-12

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CA3226361A Pending CA3226361A1 (en) 2021-07-09 2022-07-01 Muscle targeting complexes and uses thereof for modulation of genes associated with muscle health

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US (1) US20240382513A1 (enExample)
EP (1) EP4366774A4 (enExample)
JP (1) JP2024525614A (enExample)
CA (1) CA3226361A1 (enExample)
WO (1) WO2023283531A2 (enExample)

Families Citing this family (18)

* Cited by examiner, † Cited by third party
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EP4169576A1 (en) 2018-03-23 2023-04-26 University of Massachusetts Gene therapeutics for treating bone disorders
CA3108282A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US12097263B2 (en) 2018-08-02 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
SG11202100928QA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US12370264B1 (en) 2018-08-02 2025-07-29 Dyne Therapeutics, Inc. Complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of delivering oligonucleotide to a subject
EP4185320A4 (en) * 2020-07-23 2025-10-08 Dyne Therapeutics Inc MUSCLE TARGETING COMPLEXES AND THEIR USES IN THE TREATMENT OF MUSCLE ATROPHY
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11633498B2 (en) 2021-07-09 2023-04-25 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
JP2024525608A (ja) 2021-07-09 2024-07-12 ダイン セラピューティクス，インコーポレーテッド ジストロフィン異常症を処置するための筋標的化複合体および製剤
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
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