JP7576542B2 - 筋標的化複合体およびそれらの使用 - Google Patents

筋標的化複合体およびそれらの使用 Download PDF

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Publication number
JP7576542B2
JP7576542B2 JP2021529259A JP2021529259A JP7576542B2 JP 7576542 B2 JP7576542 B2 JP 7576542B2 JP 2021529259 A JP2021529259 A JP 2021529259A JP 2021529259 A JP2021529259 A JP 2021529259A JP 7576542 B2 JP7576542 B2 JP 7576542B2
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muscle
antibody
oligonucleotide
transferrin receptor
cdr
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JPWO2020028857A5 (enExample
JP2021533198A (ja
Inventor
スブラマニアン，ロメシ，アール．
カタナニ，モハメド，ティー．
ウィーデン，ティモシー
ロードス，ジェイソン，ピー．
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Dyne Therapeutics Inc
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Dyne Therapeutics Inc
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Priority to JP2024184975A priority Critical patent/JP7708957B2/ja
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Priority to JP2025112731A priority patent/JP2025175995A/ja
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • 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
    • A61K47/54—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 organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
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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
    • 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
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    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2881—Immunoglobulins [IGs], 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
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    • C12N15/09—Recombinant DNA-technology
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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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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
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JP2021529259A 2018-08-02 2019-08-02 筋標的化複合体およびそれらの使用 Active JP7576542B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2024184975A JP7708957B2 (ja) 2018-08-02 2024-10-21 筋標的化複合体およびそれらの使用
JP2025112731A JP2025175995A (ja) 2018-08-02 2025-07-03 筋標的化複合体およびそれらの使用

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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2025026842A (ja) * 2018-08-02 2025-02-26 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それらの使用

Families Citing this family (36)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
IL319835A (en) 2017-12-06 2025-05-01 Avidity Biosciences Inc Compositions and methods for treating muscular dystrophy and myotonic dystrophy
WO2020028832A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
CA3108386A1 (en) * 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating friedreich's ataxia
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
EA202190416A1 (ru) 2018-08-02 2021-06-23 Дайн Терапьютикс, Инк. Мышечно-специфические комлексы и их применение для лечения плече-лопаточно-лицевой мышечной дистрофии
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
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
EP4087653A4 (en) * 2020-01-10 2024-06-19 Dyne Therapeutics, Inc. MUSCLE TARGETING COMPLEXES AND RELATED USES FOR THE TREATMENT OF MYOTONIC DYSTROPHY
WO2021195469A1 (en) 2020-03-27 2021-09-30 Avidity Biosciences, Inc. Compositions and methods of treating muscle dystrophy
US20230330246A1 (en) * 2020-05-05 2023-10-19 Avidity Biosciences, Inc. Compositions and methods of treating pompe disease
WO2021226454A1 (en) * 2020-05-08 2021-11-11 Mpeg La, L.L.C. Linker compounds
JP2023535074A (ja) * 2020-07-23 2023-08-15 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それらの使用
KR20230042713A (ko) * 2020-07-23 2023-03-29 다인 세라퓨틱스, 인크. 근육 표적화 복합체 및 근긴장성 이영양증을 치료하기 위한 그의 용도
CN116744943A (zh) * 2020-12-04 2023-09-12 达因疗法公司 抗体-寡核苷酸复合物及其用途
IL304048A (en) * 2020-12-31 2023-08-01 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating myotonic dystrophy
WO2022197554A1 (en) * 2021-03-16 2022-09-22 University Of Massachusetts Photocaged citrulline analogs and methods for site-specific incorporation of citrulline into proteins
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular 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
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US11672872B2 (en) 2021-07-09 2023-06-13 Dyne Therapeutics, Inc. Anti-transferrin receptor antibody and uses thereof
US11633498B2 (en) 2021-07-09 2023-04-25 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
WO2023023031A2 (en) * 2021-08-17 2023-02-23 Blaze Bioscience, Inc. Transferrin receptor targeting peptide oligonucleotide complexes and methods of use thereof
CA3230796A1 (en) 2021-09-01 2023-03-09 Biogen Ma Inc. Anti-transferrin receptor antibodies and uses thereof
US20240382605A1 (en) * 2021-09-29 2024-11-21 Ionis Pharmaceuticals, Inc. Conjugated oligonucleotides and uses thereof
US20250082759A1 (en) 2021-12-22 2025-03-13 Sapreme Technologies B.V. Compositions comprising therapeutic nucleic acid and targeted saponin for the treatment of muscle-wasting disorders
PT4452331T (pt) 2021-12-22 2025-10-28 Sapreme Tech Bv Conjugados alvo de saponina-ácido nucleico para o tratamento de distúrbios de atrofia muscular
EP4452281A1 (en) 2021-12-22 2024-10-30 Sapreme Technologies B.V. Compositions comprising therapeutic nucleic acid and saponin for the treatment of muscle-wasting disorders
CA3242927A1 (en) * 2022-01-07 2023-07-13 Genzyme Corporation TISSUE-SPECIFIC IMAGING AND THERAPEUTIC AGENTS TARGETING PROTEINS EXPRESSED ON MUSCLE CELL SURFACE
CN119183457A (zh) 2022-04-15 2024-12-24 达因疗法公司 用于治疗强直性肌营养不良的肌肉靶向复合物和制剂
US20240182561A1 (en) * 2022-11-04 2024-06-06 Regeneron Pharmaceuticals, Inc. Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle
EP4480964A1 (en) * 2023-06-22 2024-12-25 Vect-Horus Transferrin receptor-binding molecules, conjugates thereof and their uses to prevent or treat muscular diseases
WO2024261332A2 (en) * 2023-06-22 2024-12-26 Vect-Horus Transferrin receptor-binding molecules, conjugates thereof and their uses to prevent or treat muscular diseases

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015534996A (ja) 2012-10-23 2015-12-07 シンアフィックス ビー．ブイ． 修飾抗体、抗体コンジュゲート及びそれらを調製する方法
WO2017221883A1 (ja) 2016-06-20 2017-12-28 武田薬品工業株式会社 抗体－薬物コンジュゲート
WO2018129384A1 (en) 2017-01-06 2018-07-12 Avidity Biosciences Llc Nucleic acid-polypeptide compositions and methods of inducing exon skipping

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040063654A1 (en) * 2001-11-02 2004-04-01 Davis Mark E. Methods and compositions for therapeutic use of RNA interference
US20120122801A1 (en) * 2005-01-05 2012-05-17 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
DK3252068T3 (da) * 2009-10-12 2025-08-25 Larry J Smith Metoder og sammensætninger til modulering af genekspression ved anvendelse af oligonukleotidbaserede lægemidler administreret in vivo eller in vitro
JP2014523884A (ja) * 2011-06-24 2014-09-18 セントローズ， エルエルシー 細胞外標的化薬物複合体
JP6322652B2 (ja) * 2013-01-07 2018-05-09 オムニオクス， インコーポレイテッド Ｈ−ｎｏｘタンパク質の重合体形態
GB201507926D0 (en) * 2015-05-08 2015-06-24 Proqr Therapeutics N V Improved treatments using oligonucleotides
IL314102B2 (en) 2018-08-02 2025-10-01 Dyne Therapeutics Inc Transferrin receptor-targeting conjugates and their uses

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015534996A (ja) 2012-10-23 2015-12-07 シンアフィックス ビー．ブイ． 修飾抗体、抗体コンジュゲート及びそれらを調製する方法
WO2017221883A1 (ja) 2016-06-20 2017-12-28 武田薬品工業株式会社 抗体－薬物コンジュゲート
WO2018129384A1 (en) 2017-01-06 2018-07-12 Avidity Biosciences Llc Nucleic acid-polypeptide compositions and methods of inducing exon skipping

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2025026842A (ja) * 2018-08-02 2025-02-26 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それらの使用
JP7708957B2 (ja) 2018-08-02 2025-07-15 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それらの使用

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