KR102805405B1 - 핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법 - Google Patents

핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법 Download PDF

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KR102805405B1
KR102805405B1 KR1020207011734A KR20207011734A KR102805405B1 KR 102805405 B1 KR102805405 B1 KR 102805405B1 KR 1020207011734 A KR1020207011734 A KR 1020207011734A KR 20207011734 A KR20207011734 A KR 20207011734A KR 102805405 B1 KR102805405 B1 KR 102805405B1
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polynucleic acid
acid molecule
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exon
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KR20200060443A (ko
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아서 에이 레빈
앤드루 존 길
마이클 캐러미언 코크런
한후아 후앙
벤카타 라마나 도팔라푸디
롭 버크
비어트리스 다이애나 다리몬트
윤유 시
레이첼 존스
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어비디티 바이오사이언시스 인크.
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A61K47/62—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 a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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
    • A61K47/6807—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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    • A61K47/6801—Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
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    • 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
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
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    • A61K47/6843—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 the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site the antibody targeting a material from animals or humans
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    • A61K47/69—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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6949—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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit inclusion complexes, e.g. clathrates, cavitates or fullerenes
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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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  • Proteomics, Peptides & Aminoacids (AREA)
  • Orthopedic Medicine & Surgery (AREA)
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  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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KR1020207011734A 2017-09-22 2018-09-21 핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법 Active KR102805405B1 (ko)

Priority Applications (1)

Application Number Priority Date Filing Date Title
KR1020257014783A KR20250065737A (ko) 2017-09-22 2018-09-21 핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762561939P 2017-09-22 2017-09-22
US62/561,939 2017-09-22
US201862696766P 2018-07-11 2018-07-11
US62/696,766 2018-07-11
PCT/US2018/052289 WO2019060775A1 (en) 2017-09-22 2018-09-21 NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND EXON JUMP INDUCTION METHODS

Related Child Applications (1)

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KR1020257014783A Division KR20250065737A (ko) 2017-09-22 2018-09-21 핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법

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KR20200060443A KR20200060443A (ko) 2020-05-29
KR102805405B1 true KR102805405B1 (ko) 2025-05-12

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KR1020257014783A Pending KR20250065737A (ko) 2017-09-22 2018-09-21 핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법
KR1020207011734A Active KR102805405B1 (ko) 2017-09-22 2018-09-21 핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법

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US (3) US20200282074A1 (enExample)
EP (1) EP3684376A4 (enExample)
JP (3) JP2020537497A (enExample)
KR (2) KR20250065737A (enExample)
CN (1) CN111770757A (enExample)
AU (2) AU2018335880B2 (enExample)
CA (1) CA3075425A1 (enExample)
IL (1) IL273429A (enExample)
MA (1) MA50269A (enExample)
MX (1) MX2020003130A (enExample)
SG (1) SG11202002517RA (enExample)
WO (1) WO2019060775A1 (enExample)

Families Citing this family (43)

* 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
KR20190104381A (ko) 2017-01-06 2019-09-09 어비디티 바이오사이언시스 엘엘씨 핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
JP7506601B2 (ja) * 2017-12-06 2024-06-26 アビディティー バイオサイエンシーズ，インク． 筋萎縮症と筋緊張性ジストロフィーを処置するための組成物および方法
SG11202100928QA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular 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
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
SG11202100934PA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US12097263B2 (en) 2018-08-02 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
AU2020263487A1 (en) 2019-04-25 2021-12-16 Avidity Biosciences, Inc. Nucleic acid compositions and methods of multi-exon skipping
US20220282247A1 (en) * 2019-08-02 2022-09-08 Research Institute At Nationwide Children's Hospital Exon 44-targeted nucleic acids and recombinant adeno-associated virus comprising said nucleic acids for treatment of dystrophin-based myopathies
JP7560544B2 (ja) * 2019-09-19 2024-10-02 アルネー サイエンシズ、エルエルシー 遺伝子スプライシングを調節するために有用な化合物および方法
MX2022007491A (es) * 2019-12-19 2022-08-15 Nippon Shinyaku Co Ltd Acido nucleico antisentido que permite la omision de exones.
BR112022013603A2 (pt) * 2020-01-10 2022-09-13 Dyne Therapeutics Inc Complexos de direcionamento a músculo e usos dos mesmos para tratar distrofia muscular facioescapuloumeral
CA3172111A1 (en) 2020-03-19 2021-09-23 Barbora MALECOVA Compositions and methods of treating facioscapulohumeral muscular dystrophy
EP4656242A2 (en) 2020-03-27 2025-12-03 Avidity Biosciences, Inc. Compositions and methods of treating muscle dystrophy
BR112023001125A2 (pt) * 2020-07-23 2023-02-14 Dyne Therapeutics Inc Complexos de direcionamento muscular e usos dos mesmos para tratar distrofinopatias
WO2022120132A1 (en) * 2020-12-04 2022-06-09 Dyne Therapeutics, Inc. Antibody-oligonucleotide complexes and uses thereof
IL307937A (en) 2021-04-30 2023-12-01 Sarepta Therapeutics Inc Treatment methods for muscular dystrophy
IL308418A (en) * 2021-05-28 2024-01-01 Novo Nordisk As Compositions and methods for inhibiting expression of mitochondrial amidoxime reducing component 1 (MARC1)
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
MX2024000489A (es) 2021-07-09 2024-04-09 Dyne Therapeutics Inc Complejos dirigidos al musculo y formulaciones para el tratamiento de las distrofinopatías.
US11633498B2 (en) 2021-07-09 2023-04-25 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US20240318176A1 (en) * 2021-07-09 2024-09-26 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
EP4366741A2 (en) * 2021-07-09 2024-05-15 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
WO2023026994A1 (ja) * 2021-08-21 2023-03-02 武田薬品工業株式会社 ヒトトランスフェリンレセプター結合ペプチド－薬物コンジュゲート
CN118265544A (zh) 2021-09-16 2024-06-28 艾维迪提生物科学公司 治疗面肩肱型肌营养不良的组合物和方法
KR20240125036A (ko) * 2021-12-23 2024-08-19 마이레큘, 인크. 폴리뉴클레오타이드의 전달을 위한 조성물
JP2025503098A (ja) * 2022-01-20 2025-01-30 ボールデン セラピューティクス， インコーポレイテッド ＭｕＳＫ標的化オリゴヌクレオチド
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
US20250195667A1 (en) 2022-03-17 2025-06-19 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer conjugates
US12071621B2 (en) 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping
CA3255934A1 (en) 2022-04-15 2023-10-19 Dyne Therapeutics, Inc. MUSCLE TARGETTING COMPLEXES AND FORMULATIONS FOR THE TREATMENT OF MYOTONIC DYSTROPHY
WO2024101349A1 (ja) 2022-11-07 2024-05-16 株式会社日本触媒 可溶化ポリマーの製造方法および吸水性樹脂の製造方法
WO2024197302A1 (en) * 2023-03-23 2024-09-26 Yale University Compositions and methods for delivering antibody oligonucleotide conjugates for exon skipping
CN116381125A (zh) * 2023-06-05 2023-07-04 迦进生物医药(上海)有限公司 评估与蛋白偶联的核酸稳定性的方法及试剂盒
WO2025072246A1 (en) * 2023-09-26 2025-04-03 Entrada Therapeutics, Inc. Compounds and methods for skipping exon 50 in duchenne muscular dystrophy
WO2025212707A2 (en) * 2024-04-03 2025-10-09 Avidity Biosciences, Inc. Antibody-oligonucleotide conjugate compositions and methods of inducing dmd exon 52 skipping

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014144978A2 (en) * 2013-03-15 2014-09-18 Sarepta Therapeutics, Inc. Improved compositions for treating muscular dystrophy

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HUE028662T2 (en) * 2007-10-26 2016-12-28 Academisch Ziekenhuis Leiden Preparations and methods for controlling muscle disorders
EP2274425A2 (en) * 2008-04-11 2011-01-19 Alnylam Pharmaceuticals Inc. Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
EP2119783A1 (en) * 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
US20110130346A1 (en) * 2008-05-30 2011-06-02 Isis Innovation Limited Peptide conjugates for delvery of biologically active compounds
TR201902952T4 (tr) * 2008-10-24 2019-03-21 Sarepta Therapeutics Inc Dmd için ekson atlama bileşimleri.
SG193324A1 (en) * 2011-04-01 2013-10-30 Wyeth Llc Antibody-drug conjugates
UA121117C2 (uk) * 2014-06-17 2020-04-10 Ніппон Шин'Яку Ко., Лтд. Антисенсова нуклеїнова кислота
MX388743B (es) * 2015-05-19 2025-03-20 Sarepta Therapeutics Inc Conjugados oligonucleótido-péptido.
KR20190104381A (ko) * 2017-01-06 2019-09-09 어비디티 바이오사이언시스 엘엘씨 핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법
US12071621B2 (en) * 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014144978A2 (en) * 2013-03-15 2014-09-18 Sarepta Therapeutics, Inc. Improved compositions for treating muscular dystrophy

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
T. Sugo et al. Journal of Controlled Release. 2016, vol.237, pp.1-13*

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