CN119365597A - 诱导dmd外显子44跳跃的抗体寡核苷酸缀合物组合物和方法 - Google Patents

诱导dmd外显子44跳跃的抗体寡核苷酸缀合物组合物和方法 Download PDF

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Publication number
CN119365597A
CN119365597A CN202380042480.5A CN202380042480A CN119365597A CN 119365597 A CN119365597 A CN 119365597A CN 202380042480 A CN202380042480 A CN 202380042480A CN 119365597 A CN119365597 A CN 119365597A
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pmo
cases
antibody
sequence
conjugate
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Inventor
比亚特丽丝·戴安娜·达里蒙特
乌苏·埃查尼兹伊里戈伊恩
文卡塔·拉马纳·多帕拉普迪
迈克尔·卡拉米安·科克伦
艾萨克·马克斯
泰勒·阿尔宾
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Avidity Biosciences Inc
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Avidity Biosciences Inc
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
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    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A61K39/39533—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals
    • A61K39/3955—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals against proteinaceous materials, e.g. enzymes, hormones, lymphokines
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    • A61K47/6801—Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
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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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    • 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
    • A61K47/6811—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates the drug being a protein or peptide, e.g. transferrin or bleomycin
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CN202380042480.5A 2022-04-05 2023-04-05 诱导dmd外显子44跳跃的抗体寡核苷酸缀合物组合物和方法 Pending CN119365597A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202263327725P 2022-04-05 2022-04-05
US63/327,725 2022-04-05
PCT/US2023/017574 WO2023196400A2 (en) 2022-04-05 2023-04-05 Antibody oligonucleotide conjugate compositions and methods of inducing dmd exon 44 skipping

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CN119365597A true CN119365597A (zh) 2025-01-24

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US (3) US12071621B2 (https=)
EP (1) EP4504940A2 (https=)
JP (1) JP2025511714A (https=)
KR (1) KR20240165468A (https=)
CN (1) CN119365597A (https=)
AU (2) AU2023249162B2 (https=)
CA (1) CA3247486A1 (https=)
IL (1) IL315995A (https=)
MX (1) MX2024012268A (https=)
WO (1) WO2023196400A2 (https=)

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MX2019008199A (es) 2017-01-06 2019-11-25 Avidity Biosciences Llc Composiciones de acido nucleico polipeptido y metodos de induccion de la omision de exon.
US20200282074A1 (en) * 2017-09-22 2020-09-10 Avidity Biosciences, Inc. Nucleic acid-polypeptide compositions and methods of inducing exon skipping
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
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
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
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
US12097263B2 (en) 2018-08-02 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US12071621B2 (en) 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping
US20250339553A1 (en) 2022-04-15 2025-11-06 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating myotonic dystrophy
WO2025160275A1 (en) 2024-01-24 2025-07-31 Arrowhead Pharmaceuticals, Inc. Anti-transferrin receptor antibodies, conjugates thereof, and uses thereof

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CN111108201A (zh) * 2017-07-21 2020-05-05 艾伯塔大学校董事会 结合至人类肌养蛋白前体mRNA的外显子51的反义寡核苷酸
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