AU2011296882B2 - Antisense nucleic acids - Google Patents

Antisense nucleic acids Download PDF

Info

Publication number
AU2011296882B2
AU2011296882B2 AU2011296882A AU2011296882A AU2011296882B2 AU 2011296882 B2 AU2011296882 B2 AU 2011296882B2 AU 2011296882 A AU2011296882 A AU 2011296882A AU 2011296882 A AU2011296882 A AU 2011296882A AU 2011296882 B2 AU2011296882 B2 AU 2011296882B2
Authority
AU
Australia
Prior art keywords
seq
nucleic acid
exon
synthetic nucleic
skipping
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
AU2011296882A
Other languages
English (en)
Other versions
AU2011296882A1 (en
Inventor
Tetsuya Nagata
Youhei Satou
Shin'ichi Takeda
Naoki Watanabe
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Nippon Shinyaku Co Ltd
National Center of Neurology and Psychiatry
Original Assignee
Nippon Shinyaku Co Ltd
National Center of Neurology and Psychiatry
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=45773054&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=AU2011296882(B2) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Nippon Shinyaku Co Ltd, National Center of Neurology and Psychiatry filed Critical Nippon Shinyaku Co Ltd
Publication of AU2011296882A1 publication Critical patent/AU2011296882A1/en
Application granted granted Critical
Publication of AU2011296882B2 publication Critical patent/AU2011296882B2/en
Priority to AU2015203659A priority Critical patent/AU2015203659A1/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • 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
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707—Muscular dystrophy
    • C07K14/4708—Duchenne dystrophy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
    • C12N2310/3145—Phosphoramidates with the nitrogen in 3' or 5'-position
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Proteomics, Peptides & Aminoacids (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)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Saccharide Compounds (AREA)
AU2011296882A 2010-09-01 2011-08-31 Antisense nucleic acids Active AU2011296882B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2015203659A AU2015203659A1 (en) 2010-09-01 2015-06-30 Antisense nucleic acid

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2010-196032 2010-09-01
JP2010196032 2010-09-01
PCT/JP2011/070318 WO2012029986A1 (ja) 2010-09-01 2011-08-31 アンチセンス核酸

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2015203659A Division AU2015203659A1 (en) 2010-09-01 2015-06-30 Antisense nucleic acid

Publications (2)

Publication Number Publication Date
AU2011296882A1 AU2011296882A1 (en) 2013-04-18
AU2011296882B2 true AU2011296882B2 (en) 2015-04-09

Family

ID=45773054

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2011296882A Active AU2011296882B2 (en) 2010-09-01 2011-08-31 Antisense nucleic acids

Country Status (20)

Country Link
US (16) US9079934B2 (enExample)
EP (6) EP3543341A1 (enExample)
JP (12) JP5363655B2 (enExample)
KR (1) KR101310569B1 (enExample)
CN (1) CN103154245B (enExample)
AU (1) AU2011296882B2 (enExample)
CA (1) CA2809637C (enExample)
CY (2) CY1117367T1 (enExample)
DK (2) DK2612917T3 (enExample)
ES (2) ES2567411T3 (enExample)
HR (2) HRP20160336T1 (enExample)
HU (2) HUE046364T2 (enExample)
LT (1) LT3018211T (enExample)
PL (2) PL3018211T3 (enExample)
PT (1) PT3018211T (enExample)
RS (2) RS54649B1 (enExample)
SI (2) SI3018211T1 (enExample)
SM (2) SMT201900559T1 (enExample)
TW (1) TWI541024B (enExample)
WO (1) WO2012029986A1 (enExample)

Families Citing this family (73)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HUE028632T2 (en) 2004-06-28 2016-12-28 Univ Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
USRE48960E1 (en) 2004-06-28 2022-03-08 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
CA2596506C (en) 2005-02-09 2021-04-06 Avi Biopharma, Inc. Antisense composition and method for treating muscle atrophy
NZ584793A (en) 2007-10-26 2012-05-25 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
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
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
IL313162A (en) 2008-10-24 2024-07-01 Sarepta Therapeutics Inc Antisense compounds for inducing axon termination and preparations containing them for the treatment of muscular dystrophy
NZ626359A (en) 2009-11-12 2016-02-26 Univ Western Australia Antisense molecules and methods for treating pathologies
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
EP4043039A1 (en) * 2012-01-27 2022-08-17 BioMarin Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
HK1216902A1 (zh) * 2012-12-20 2016-12-09 Sarepta Therapeutics, Inc. 用作治疗肌肉萎缩的改进外显子跳跃组合物
BR112015023038A2 (pt) * 2013-03-14 2017-11-14 Sarepta Therapeutics Inc composições para salto de exon para o tratamento da distrofia muscular
AU2014236140B2 (en) 2013-03-14 2019-10-03 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
EP3662912B1 (en) 2013-03-15 2025-12-03 Sarepta Therapeutics, Inc. Improved dosages of eteplirsen for treating duchenne muscular dystrophy
RS58573B1 (sr) * 2014-03-12 2019-05-31 Nippon Shinyaku Co Ltd Antisensna nukleinska kiselina
DK3146970T3 (da) 2014-05-19 2022-12-05 Knc Laboratories Co Ltd Nukleinsyrelægemiddel til induktion af overspringning af en exonvariant af CD44-genet og øgning af ekspression af CD44-mRNA af normal type
PT3159409T (pt) 2014-06-17 2020-01-21 Nippon Shinyaku Co Ltd Ácido nucleico anti-sentido para utilização no tratamento da distrofia muscular de duchenne
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
RS60493B1 (sr) * 2015-09-15 2020-08-31 Nippon Shinyaku Co Ltd Antismisaona nukleinska kiselina
US10563199B2 (en) 2015-09-16 2020-02-18 Nippon Shinyaku Co., Ltd. Antisense nucleic acid for treating amyotrophy
US20190127733A1 (en) 2015-10-09 2019-05-02 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
BR112018007066A2 (pt) 2015-10-09 2018-10-23 Sarepta Therapeutics Inc composições e métodos para tratamento da distrofia muscular de duchene e distúrbios relacionados
FR3044926B1 (fr) 2015-12-09 2020-01-31 Genethon Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine
KR101686379B1 (ko) 2016-02-05 2016-12-13 박정열 휴대용 구이기
US11472824B2 (en) 2016-05-24 2022-10-18 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
MD3464305T2 (ro) 2016-05-24 2025-01-31 Sarepta Therapeutics Inc Procedee de preparare de oligomeri
MA45183A (fr) 2016-05-24 2019-04-10 Sarepta Therapeutics Inc Procédés de préparation d'oligomères morpholino de phosphorodiamidate
EP3464306B1 (en) * 2016-05-24 2024-03-27 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
MA45362A (fr) 2016-05-24 2019-04-10 Sarepta Therapeutics Inc Procédés de préparation d'oligomères morpholino de phosphorodiamidate
MX2018014160A (es) * 2016-06-30 2019-04-01 Sarepta Therapeutics Inc Procesos para preparar oligomeros de morfolino fosforodiamidato.
CA3025575A1 (en) 2016-06-30 2018-01-04 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystrophy
BR112019000061A2 (pt) * 2016-07-05 2019-11-26 Biomarin Technologies B.V. comutação e modulação de splicing de pré-mrna compreendendo porções de arcabouço bicíclico, com características melhoradas para o tratamento de distúrbios genéticos
KR101715572B1 (ko) 2016-10-11 2017-03-22 박종찬 구이대 승강구조를 갖는 양방향 구이기
AU2017382741B2 (en) 2016-12-19 2025-01-30 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
AU2017382723B2 (en) * 2016-12-19 2025-01-30 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
KR102646318B1 (ko) 2016-12-19 2024-03-12 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
WO2018223056A1 (en) 2017-06-02 2018-12-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
AU2018326780A1 (en) * 2017-08-31 2020-02-27 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy
EA201991450A1 (ru) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
US20200254002A1 (en) 2017-09-28 2020-08-13 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
US20210102205A1 (en) * 2018-04-26 2021-04-08 Sarepta Therapeutics, Inc. Exon skipping oligomers and oligomer conjugates for muscular dystrophy
JP7427608B2 (ja) * 2018-05-11 2024-02-05 ウェイブ ライフ サイエンシズ リミテッド オリゴヌクレオチド組成物及びその使用方法
US10758629B2 (en) 2018-05-29 2020-09-01 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
MY209148A (en) 2018-06-26 2025-06-24 Nippon Shinyaku Co Ltd Composition comprising antisense oligonucleotide and use thereof for treatment of duchenne 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
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
SG11202100934PA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating dystrophinopathies
WO2020028864A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
GB201812972D0 (en) 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
GB201812980D0 (en) 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
CA3122200A1 (en) 2018-12-07 2020-06-11 Oxford University Innovation Limited Linkers
US20220144902A1 (en) * 2018-12-25 2022-05-12 National Center Of Neurology And Psychiatry Method for inducing muscular cells using cells in spot urine
GB201821269D0 (en) * 2018-12-28 2019-02-13 Nippon Shinyaku Co Ltd Myostatin signal inhibitor
CA3165961A1 (en) 2019-12-26 2021-07-01 Nippon Shinyaku Co., Ltd. Antisense nucleic acid that induces skipping of exon 50
MX2022010545A (es) 2020-02-28 2022-09-21 Nippon Shinyaku Co Ltd Acidos nucleicos antisentido que inducen la omision del exon 51.
CA3175125A1 (en) 2020-03-11 2021-09-16 Biocomber Co., Ltd. Single-stranded nucleic acid molecule for inducing -1 frameshift and composition
WO2022059754A1 (ja) * 2020-09-18 2022-03-24 天野エンザイム株式会社 加工ひよこ豆ミルクの製造方法
AU2021427595A1 (en) * 2021-02-12 2023-09-28 Oxford University Innovation Limited Cell-penetrating peptide conjugates and methods of their use
KR20240004609A (ko) 2021-04-30 2024-01-11 사렙타 쎄러퓨틱스 인코퍼레이티드 근이영양증에 대한 치료 방법
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
CA3226366A1 (en) 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating dystrophinopathies
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
EP4389893A4 (en) 2021-08-21 2025-09-10 Takeda Pharmaceuticals Co PEPTIDE-DRUG CONJUGATE BINDING HUMAN TRANSFERRIN RECEPTOR
CN118201966A (zh) 2021-08-24 2024-06-14 肽梦想株式会社 人转铁蛋白受体结合抗体-肽缀合物
JPWO2023127918A1 (enExample) 2021-12-27 2023-07-06
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
EP4486386A1 (en) 2022-03-03 2025-01-08 Yale University Compositions and methods for delivering therapeutic polynucleotides for exon skipping
JP2025079347A (ja) * 2022-03-11 2025-05-22 日本新薬株式会社 キャリアペプチドが連結された核酸
US20250195667A1 (en) 2022-03-17 2025-06-19 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer conjugates
US20250297253A1 (en) 2022-05-06 2025-09-25 Josho Gakuen Educational Foundation Artificial rna molecule
WO2025153061A1 (zh) * 2024-01-19 2025-07-24 云合智药（苏州）生物科技有限公司 用于抑制激活素iib型受体基因表达的核酸及其用途

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100130591A1 (en) * 2008-10-24 2010-05-27 Peter Sazani Multiple exon skipping compositions for dmd
US20100168212A1 (en) * 2008-09-11 2010-07-01 Royal Holloway, University Of London Oligomers

Family Cites Families (60)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU655164B2 (en) 1989-12-20 1994-12-08 Antivirals Inc. Uncharged morpholino-based polymers having phosphorous-containing chiral intersubunit linkages
JP2924179B2 (ja) 1993-02-19 1999-07-26 日本新薬株式会社 グリセロール誘導体，デバイス及び医薬組成物
US7321828B2 (en) 1998-04-13 2008-01-22 Isis Pharmaceuticals, Inc. System of components for preparing oligonucleotides
JP2000325085A (ja) 1999-05-21 2000-11-28 Masafumi Matsuo デュシェンヌ型筋ジストロフィー治療剤
US6653467B1 (en) 2000-04-26 2003-11-25 Jcr Pharmaceutical Co., Ltd. Medicament for treatment of Duchenne muscular dystrophy
WO2001083740A2 (en) 2000-05-04 2001-11-08 Avi Biopharma, Inc. Splice-region antisense composition and method
JP4836366B2 (ja) 2000-08-25 2011-12-14 雅文 松尾 デュシェンヌ型筋ジストロフィー治療剤
US6727355B2 (en) 2000-08-25 2004-04-27 Jcr Pharmaceuticals Co., Ltd. Pharmaceutical composition for treatment of Duchenne muscular dystrophy
EP1191097A1 (en) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
ITRM20020253A1 (it) 2002-05-08 2003-11-10 Univ Roma Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche.
ES2509140T3 (es) 2002-11-25 2014-10-17 Masafumi Matsuo Fármacos de ácido nucleico ENA que modifican el corte y empalme en precursores de ARNm
WO2004083432A1 (en) 2003-03-21 2004-09-30 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
HUE028632T2 (en) 2004-06-28 2016-12-28 Univ Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
JP2006038608A (ja) 2004-07-27 2006-02-09 Tokyo Electric Power Co Inc:The 超音波検査装置及び方法
US20110046200A1 (en) 2004-08-03 2011-02-24 Michael T Howard Use of antisense oligonucleotides to effect translation modulation
FR2874384B1 (fr) 2004-08-17 2010-07-30 Genethon Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables
JPWO2006038608A1 (ja) 2004-10-05 2008-05-15 日本新薬株式会社 オリゴ二本鎖ｒｎａ及び医薬組成物
JP2006129594A (ja) 2004-10-28 2006-05-18 Hitachi Ltd 船舶用電気推進装置の制御方法及びその装置
JP2008538500A (ja) 2005-04-22 2008-10-30 アカデミス ツィーケンホイス ライデン ＳＲタンパク質の結合に対する干渉とＲＮＡ二次構造に対する干渉による、ｍＲＮＡ前駆体におけるエクソン認識の調節
US20090069260A1 (en) 2005-05-30 2009-03-12 Nippon Shinyaku Co., Ltd Method for producing a nucleic-acid-containing complex preparation
EP2548560B1 (en) * 2005-06-23 2015-06-03 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of SMN2 splicing
EP2024499B1 (en) * 2006-05-10 2017-10-25 Sarepta Therapeutics, Inc. Oligonucleotide analogs having cationic intersubunit linkages
EP1857548A1 (en) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
JP4816396B2 (ja) 2006-10-12 2011-11-16 富士ゼロックス株式会社 画像形成装置
WO2008096690A1 (ja) 2007-02-05 2008-08-14 Nippon Shinyaku Co., Ltd. ポリエチレングリコール誘導体
NZ584793A (en) 2007-10-26 2012-05-25 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
RU2606627C2 (ru) 2007-11-15 2017-01-10 Серепта Терапьютикс,Инк. Способ синтеза морфолиновых олигомеров
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
SI2607484T1 (sl) 2008-10-27 2016-11-30 Biomarin Technologies B.V. Metode in sredstva za učinkovito preskakovanje eksona 45 v pre-mRNA Duchennove mišične distrofije
JP2010196032A (ja) 2009-01-29 2010-09-09 Fujifilm Corp 水不溶性色材の分散体及びこの製造方法、これを用いた記録液、インクセット、印画物、画像形成方法、及び画像形成装置
EP2421971B1 (en) 2009-04-24 2016-07-06 BioMarin Technologies B.V. Oligonucleotide comprising an inosine for treating dmd
CN115227710A (zh) * 2009-06-17 2022-10-25 冷泉港实验室 用于在对象中调节smn2剪接的组合物和方法
NZ626359A (en) * 2009-11-12 2016-02-26 Univ Western Australia Antisense molecules and methods for treating pathologies
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
BR112013020273A2 (pt) 2011-02-08 2016-10-18 Charlotte Mecklenburg Hospital oligonucleotídeos antissenso
KR102229650B1 (ko) 2011-05-05 2021-03-19 사렙타 쎄러퓨틱스, 인코퍼레이티드 펩타이드 올리고뉴클레오타이드 접합체
EP4043039A1 (en) 2012-01-27 2022-08-17 BioMarin Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
EP3608407A1 (en) 2012-07-03 2020-02-12 BioMarin Technologies B.V. Oligonucleotide for the treatment of muscular dystrophy patients
HK1216902A1 (zh) 2012-12-20 2016-12-09 Sarepta Therapeutics, Inc. 用作治疗肌肉萎缩的改进外显子跳跃组合物
BR112015023038A2 (pt) 2013-03-14 2017-11-14 Sarepta Therapeutics Inc composições para salto de exon para o tratamento da distrofia muscular
AU2014236140B2 (en) 2013-03-14 2019-10-03 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
EP3662912B1 (en) 2013-03-15 2025-12-03 Sarepta Therapeutics, Inc. Improved dosages of eteplirsen for treating duchenne muscular dystrophy
JP6655011B2 (ja) 2014-06-23 2020-02-26 東亞合成株式会社 細胞の多核化を誘導するペプチドおよびその利用
AU2015301978C1 (en) 2014-08-09 2022-06-09 Research Institute At Nationwide Children's Hospital Methods and materials for activating an internal ribosome entry site in exon 5 of the DMD gene
BR112018006445A2 (pt) 2015-09-30 2018-12-11 Sarepta Therapeutics Inc métodos para tratar distrofia muscular
BR112018007066A2 (pt) 2015-10-09 2018-10-23 Sarepta Therapeutics Inc composições e métodos para tratamento da distrofia muscular de duchene e distúrbios relacionados
EP3464306B1 (en) 2016-05-24 2024-03-27 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
MD3464305T2 (ro) 2016-05-24 2025-01-31 Sarepta Therapeutics Inc Procedee de preparare de oligomeri
MA45183A (fr) 2016-05-24 2019-04-10 Sarepta Therapeutics Inc Procédés de préparation d'oligomères morpholino de phosphorodiamidate
WO2017213854A1 (en) 2016-05-24 2017-12-14 Sarepta Therapeutics, Inc. Pharmaceutical composition comprising eteplirsen
CA3025575A1 (en) 2016-06-30 2018-01-04 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystrophy
MX2018014160A (es) 2016-06-30 2019-04-01 Sarepta Therapeutics Inc Procesos para preparar oligomeros de morfolino fosforodiamidato.
ES3038159T3 (en) 2016-11-16 2025-10-09 Academisch Ziekenhuis Leiden Substances for targeting various selected organs or tissues
KR102646318B1 (ko) 2016-12-19 2024-03-12 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
AU2017382741B2 (en) 2016-12-19 2025-01-30 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
AU2017382723B2 (en) 2016-12-19 2025-01-30 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
AU2018326780A1 (en) 2017-08-31 2020-02-27 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy
EP3687577A1 (en) 2017-09-28 2020-08-05 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
US20200254002A1 (en) 2017-09-28 2020-08-13 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
US20200248178A1 (en) 2017-09-28 2020-08-06 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100168212A1 (en) * 2008-09-11 2010-07-01 Royal Holloway, University Of London Oligomers
US20100130591A1 (en) * 2008-10-24 2010-05-27 Peter Sazani Multiple exon skipping compositions for dmd

Also Published As

Publication number Publication date
RS54649B1 (sr) 2016-08-31
RU2567664C2 (ru) 2015-11-10
SI2612917T1 (sl) 2016-05-31
EP4400168A2 (en) 2024-07-17
HUE046364T2 (hu) 2020-02-28
EP2612917A4 (en) 2014-12-31
US20210340171A1 (en) 2021-11-04
JP6867636B1 (ja) 2021-04-28
ES2750748T3 (es) 2020-03-27
JP2024170458A (ja) 2024-12-10
CN103154245A (zh) 2013-06-12
EP4400168A3 (en) 2024-10-23
JP5363655B2 (ja) 2013-12-11
JP6647430B2 (ja) 2020-02-14
JP2021072822A (ja) 2021-05-13
HUE027321T2 (en) 2016-10-28
US20130211062A1 (en) 2013-08-15
US20250042933A1 (en) 2025-02-06
US10683322B2 (en) 2020-06-16
JP2018027083A (ja) 2018-02-22
US20170320903A1 (en) 2017-11-09
US20200109162A1 (en) 2020-04-09
HRP20191770T1 (hr) 2019-12-27
JP6141728B2 (ja) 2017-06-07
TWI541024B (zh) 2016-07-11
US20230151050A1 (en) 2023-05-18
EP2612917B1 (en) 2016-02-24
CY1122167T1 (el) 2020-11-25
US10647741B2 (en) 2020-05-12
US20150166995A1 (en) 2015-06-18
JP2014054250A (ja) 2014-03-27
JP6193343B2 (ja) 2017-09-06
EP3543341A1 (en) 2019-09-25
US20190218244A1 (en) 2019-07-18
EP3018211A1 (en) 2016-05-11
JP2021072820A (ja) 2021-05-13
HRP20160336T1 (hr) 2016-04-22
JP6867621B1 (ja) 2021-04-28
US10662217B2 (en) 2020-05-26
JP2021072821A (ja) 2021-05-13
SMT201900559T1 (it) 2019-11-13
EP2612917A1 (en) 2013-07-10
PL3018211T3 (pl) 2020-02-28
LT3018211T (lt) 2019-10-25
US9708361B2 (en) 2017-07-18
CN103154245B (zh) 2016-06-01
RS59361B1 (sr) 2019-11-29
EP4403632A3 (en) 2024-10-23
CA2809637A1 (en) 2012-03-08
ES2567411T3 (es) 2016-04-22
US9079934B2 (en) 2015-07-14
US11028122B1 (en) 2021-06-08
US20200102343A1 (en) 2020-04-02
AU2011296882A1 (en) 2013-04-18
WO2012029986A1 (ja) 2012-03-08
RU2013114396A (ru) 2014-10-10
EP3018211B1 (en) 2019-08-07
US20210284680A1 (en) 2021-09-16
JP2021104037A (ja) 2021-07-26
US20190211049A1 (en) 2019-07-11
PT3018211T (pt) 2019-10-28
JP2019062913A (ja) 2019-04-25
SI3018211T1 (sl) 2019-11-29
KR101310569B1 (ko) 2013-09-23
US10329319B2 (en) 2019-06-25
TW201215408A (en) 2012-04-16
CY1117367T1 (el) 2017-04-26
PL2612917T3 (pl) 2016-07-29
JP6867620B1 (ja) 2021-04-28
JPWO2012029986A1 (ja) 2013-10-31
CA2809637C (en) 2018-06-05
US10870676B2 (en) 2020-12-22
US20190211050A1 (en) 2019-07-11
US10407461B2 (en) 2019-09-10
US20210198306A1 (en) 2021-07-01
DK3018211T3 (da) 2019-10-14
JP2020072724A (ja) 2020-05-14
EP3581655A1 (en) 2019-12-18
EP4403632A2 (en) 2024-07-24
US20190315796A1 (en) 2019-10-17
JP6867619B2 (ja) 2021-04-28
SMT201600111B (it) 2016-07-01
US10487106B2 (en) 2019-11-26
US20210179659A1 (en) 2021-06-17
KR20130069762A (ko) 2013-06-26
US20200115411A1 (en) 2020-04-16
DK2612917T3 (en) 2016-04-18
JP2016104021A (ja) 2016-06-09
JP6465932B2 (ja) 2019-02-06
US10385092B2 (en) 2019-08-20
JP2023036865A (ja) 2023-03-14

Similar Documents

Publication Publication Date Title
US20250042933A1 (en) Antisense nucleic acids
AU2020200679A1 (en) Antisense nucleic acids
EP4596047A2 (en) Antisense nucleic acid for use in treating duchenne muscular dystrophy
HK40112203A (en) Antisense nucleic acids
HK40110953A (en) Antisense nucleic acids
HK40017657A (en) Antisense nucleic acids
AU2015203659A1 (en) Antisense nucleic acid

Legal Events

Date Code Title Description
DA3 Amendments made section 104

Free format text: THE NATURE OF THE AMENDMENT IS: AMEND THE INVENTION TITLE TO READ ANTISENSE NUCLEIC ACIDS

FGA Letters patent sealed or granted (standard patent)