EA202190417A1 - Мышечно-специфические комплексы и их применение для лечения болезни помпе - Google Patents

Мышечно-специфические комплексы и их применение для лечения болезни помпе

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Publication number
EA202190417A1
EA202190417A1 EA202190417A EA202190417A EA202190417A1 EA 202190417 A1 EA202190417 A1 EA 202190417A1 EA 202190417 A EA202190417 A EA 202190417A EA 202190417 A EA202190417 A EA 202190417A EA 202190417 A1 EA202190417 A1 EA 202190417A1
Authority
EA
Eurasian Patent Office
Prior art keywords
muscle
disease
pump
treatment
application
Prior art date
Application number
EA202190417A
Other languages
English (en)
Russian (ru)
Inventor
Ромеш Р. Субраманиян
Мохаммед Т. Катанани
Тимоти Уиден
Original Assignee
Дайн Терапьютикс, Инк.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Дайн Терапьютикс, Инк. filed Critical Дайн Терапьютикс, Инк.
Publication of EA202190417A1 publication Critical patent/EA202190417A1/ru

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    • C07—ORGANIC CHEMISTRY
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    • 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
    • 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
    • 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
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    • 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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    • A61K47/6889—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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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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EA202190417A 2018-08-02 2019-08-02 Мышечно-специфические комплексы и их применение для лечения болезни помпе EA202190417A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862713959P 2018-08-02 2018-08-02
PCT/US2019/044960 WO2020028841A1 (en) 2018-08-02 2019-08-02 Muscle targeting complexes and uses thereof for treating pompe disease

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EA202190417A1 true EA202190417A1 (ru) 2021-06-23

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US (1) US20210317226A1 (https=)
EP (1) EP3830127A4 (https=)
JP (2) JP2021533197A (https=)
KR (1) KR20210054513A (https=)
CN (2) CN118662653A (https=)
CA (1) CA3108285A1 (https=)
EA (1) EA202190417A1 (https=)
IL (1) IL280483A (https=)
WO (1) WO2020028841A1 (https=)

Families Citing this family (21)

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Publication number Priority date Publication date Assignee Title
FI3635009T3 (fi) 2017-06-07 2026-04-07 Regeneron Pharma Koostumuksia ja menetelmiä entsyymien internalisoimiseksi
JP7779653B2 (ja) 2018-02-07 2025-12-03 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 治療用タンパク質送達のための方法および組成物
EP3793591A1 (en) 2018-05-17 2021-03-24 Regeneron Pharmaceuticals, Inc. Anti-cd63 antibodies, conjugates, and uses thereof
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
US11911484B2 (en) 2018-08-02 2024-02-27 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
US12097263B2 (en) 2018-08-02 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
EP3829595A4 (en) 2018-08-02 2022-08-24 Dyne Therapeutics, Inc. MUSCLE-DIRECTED COMPLEXES AND USES THEREOF IN THE TREATMENT OF DYSTROPHINOPATHIES
US20220193250A1 (en) 2018-08-02 2022-06-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US20230330246A1 (en) * 2020-05-05 2023-10-19 Avidity Biosciences, Inc. Compositions and methods of treating pompe disease
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
IL309936A (en) 2021-07-09 2024-03-01 Dyne Therapeutics Inc Muscle targeting complexes and formulations for the treatment of dystrophinopathy
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11672872B2 (en) 2021-07-09 2023-06-13 Dyne Therapeutics, Inc. Anti-transferrin receptor antibody and uses thereof
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
EP4396231A1 (en) 2021-09-01 2024-07-10 Biogen MA Inc. Anti-transferrin receptor antibodies and uses thereof
US20250339553A1 (en) 2022-04-15 2025-11-06 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating myotonic dystrophy
CA3263494A1 (en) * 2022-07-29 2024-02-01 Regeneron Pharmaceuticals, Inc. ANTI-TFR FUSIONS: PAYLOAD AND THEIR METHODS OF USE
WO2025160275A1 (en) 2024-01-24 2025-07-31 Arrowhead Pharmaceuticals, Inc. Anti-transferrin receptor antibodies, conjugates thereof, and uses thereof

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004094647A2 (en) * 2003-04-18 2004-11-04 Cytovia, Inc. Methods of treating diseases responsive to induction of apoptosis and screening assays
US20170182189A1 (en) * 2014-05-23 2017-06-29 Genzyme Corporation Inhibiting or downregulating glycogen synthase by creating premature stop codons using antisense oligonucleotides
AU2016365834B2 (en) * 2015-12-08 2023-09-28 Regeneron Pharmaceuticals, Inc. Compositions and methods for internalizing enzymes
CA3021267A1 (en) * 2016-04-18 2017-10-26 Sarepta Therapeutics, Inc. Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
CN109310765A (zh) * 2016-06-20 2019-02-05 领先基因生物技术股份有限公司 抗体-药物偶联物

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