CN115335401A - 肌肉靶向复合物及其用于治疗肌养蛋白病的用途 - Google Patents

肌肉靶向复合物及其用于治疗肌养蛋白病的用途 Download PDF

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Publication number
CN115335401A
CN115335401A CN202180019467.9A CN202180019467A CN115335401A CN 115335401 A CN115335401 A CN 115335401A CN 202180019467 A CN202180019467 A CN 202180019467A CN 115335401 A CN115335401 A CN 115335401A
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cdr
amino acid
seq
acid sequence
antibody
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罗梅什·R·苏布拉马尼亚
穆罕默德·T·卡塔纳尼
蒂莫西·威登
科迪·A·德雅尔丹
布伦丹·奎因
贾森·P·罗兹
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Dyne Therapeutics Inc
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Dyne Therapeutics Inc
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    • C—CHEMISTRY; METALLURGY
    • 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
    • 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
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K47/6889—Conjugates wherein the antibody being the modifying agent and wherein the linker, binder or spacer confers particular properties to the conjugates, e.g. peptidic enzyme-labile linkers or acid-labile linkers, providing for an acid-labile immuno conjugate wherein the drug may be released from its antibody conjugated part in an acidic, e.g. tumoural or environment
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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
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CN202180019467.9A 2020-01-10 2021-01-08 肌肉靶向复合物及其用于治疗肌养蛋白病的用途 Pending CN115335401A (zh)

Applications Claiming Priority (15)

Application Number Priority Date Filing Date Title
US202062959796P 2020-01-10 2020-01-10
US62/959,796 2020-01-10
US202062965748P 2020-01-24 2020-01-24
US62/965,748 2020-01-24
US202062968443P 2020-01-31 2020-01-31
US62/968,443 2020-01-31
US202062980874P 2020-02-24 2020-02-24
US62/980,874 2020-02-24
US202063055537P 2020-07-23 2020-07-23
US63/055,537 2020-07-23
US202063069066P 2020-08-23 2020-08-23
US63/069,066 2020-08-23
US202063132929P 2020-12-31 2020-12-31
US63/132,929 2020-12-31
PCT/US2021/012756 WO2021142307A1 (en) 2020-01-10 2021-01-08 Muscle targeting complexes and uses thereof for treating dystrophinopathies

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US (1) US20230111212A1 (https=)
EP (1) EP4087878A4 (https=)
JP (2) JP7780691B2 (https=)
KR (1) KR20220125802A (https=)
CN (1) CN115335401A (https=)
AU (1) AU2021205346A1 (https=)
BR (1) BR112022013572A2 (https=)
IL (1) IL294478A (https=)
MX (1) MX2022008540A (https=)
WO (1) WO2021142307A1 (https=)

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JP2024525611A (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 筋標的化複合体およびジストロフィノパチーを処置するためのその使用
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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
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IL316143A (en) 2022-04-15 2024-12-01 Dyne Therapeutics Inc Muscle targeting complexes and formulations for the treatment of myotonic dystrophy
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