KR20150133752A - 근육 이영양증의 치료를 위한 엑손 스키핑 조성물 - Google Patents

근육 이영양증의 치료를 위한 엑손 스키핑 조성물 Download PDF

Info

Publication number
KR20150133752A
KR20150133752A KR1020157028188A KR20157028188A KR20150133752A KR 20150133752 A KR20150133752 A KR 20150133752A KR 1020157028188 A KR1020157028188 A KR 1020157028188A KR 20157028188 A KR20157028188 A KR 20157028188A KR 20150133752 A KR20150133752 A KR 20150133752A
Authority
KR
South Korea
Prior art keywords
antisense
antisense oligomer
oligomer
exon
dystrophin
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.)
Ceased
Application number
KR1020157028188A
Other languages
English (en)
Korean (ko)
Inventor
리차드 케이. 베스트윅
다이앤 엘리자베스 프랭크
Original Assignee
사렙타 쎄러퓨틱스 인코퍼레이티드
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 사렙타 쎄러퓨틱스 인코퍼레이티드 filed Critical 사렙타 쎄러퓨틱스 인코퍼레이티드
Publication of KR20150133752A publication Critical patent/KR20150133752A/ko
Ceased legal-status Critical Current

Links

Images

Classifications

    • 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
    • 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
    • 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
    • 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
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • 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
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • 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
    • C12N2310/351—Conjugate
    • 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
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide
    • 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
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3535—Nitrogen
    • 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)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (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)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Medicinal Preparation (AREA)
KR1020157028188A 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물 Ceased KR20150133752A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361782706P 2013-03-14 2013-03-14
US61/782,706 2013-03-14
PCT/US2014/029766 WO2014153240A2 (en) 2013-03-14 2014-03-14 Exon skipping compositions for treating muscular dystrophy

Related Child Applications (1)

Application Number Title Priority Date Filing Date
KR1020217000648A Division KR20210006516A (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물

Publications (1)

Publication Number Publication Date
KR20150133752A true KR20150133752A (ko) 2015-11-30

Family

ID=50694008

Family Applications (5)

Application Number Title Priority Date Filing Date
KR1020157028188A Ceased KR20150133752A (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
KR1020217000648A Ceased KR20210006516A (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
KR1020247018517A Ceased KR20240094032A (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
KR1020237016315A Ceased KR20230074606A (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
KR1020227014526A Ceased KR20220061280A (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물

Family Applications After (4)

Application Number Title Priority Date Filing Date
KR1020217000648A Ceased KR20210006516A (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
KR1020247018517A Ceased KR20240094032A (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
KR1020237016315A Ceased KR20230074606A (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
KR1020227014526A Ceased KR20220061280A (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물

Country Status (26)

Country Link
US (5) US20140315977A1 (OSRAM)
EP (3) EP3495485A3 (OSRAM)
JP (4) JP2016521119A (OSRAM)
KR (5) KR20150133752A (OSRAM)
CN (2) CN110066793A (OSRAM)
AU (3) AU2014236160A1 (OSRAM)
BR (1) BR112015023038A2 (OSRAM)
CA (1) CA2903872A1 (OSRAM)
CY (1) CY1121876T1 (OSRAM)
DK (1) DK2970964T3 (OSRAM)
EA (1) EA201591694A1 (OSRAM)
ES (1) ES2714290T3 (OSRAM)
HR (1) HRP20190382T1 (OSRAM)
HU (1) HUE042218T2 (OSRAM)
IL (2) IL241047B (OSRAM)
LT (1) LT2970964T (OSRAM)
MX (2) MX366274B (OSRAM)
NZ (1) NZ631132A (OSRAM)
PL (1) PL2970964T3 (OSRAM)
PT (1) PT2970964T (OSRAM)
RS (1) RS58529B1 (OSRAM)
SA (1) SA515361121B1 (OSRAM)
SI (1) SI2970964T1 (OSRAM)
SM (1) SMT201900139T1 (OSRAM)
TR (1) TR201903009T4 (OSRAM)
WO (1) WO2014153240A2 (OSRAM)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20190015311A (ko) * 2016-05-24 2019-02-13 사렙타 쎄러퓨틱스 인코퍼레이티드 올리고머의 제조 방법

Families Citing this family (53)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
USRE47769E1 (en) 2004-06-28 2019-12-17 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
HUE028662T2 (en) * 2007-10-26 2016-12-28 Academisch Ziekenhuis Leiden Preparations and methods for controlling muscle disorders
TR201902952T4 (tr) 2008-10-24 2019-03-21 Sarepta Therapeutics Inc Dmd için ekson atlama bileşimleri.
WO2011057350A1 (en) 2009-11-12 2011-05-19 The University Of 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
US9217148B2 (en) 2013-03-14 2015-12-22 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
MX373959B (es) 2013-03-15 2020-07-13 Sarepta Therapeutics Inc Composición para usarse en el tratamiento de la distrofia muscular de duchenne (dmd).
FR3044926B1 (fr) * 2015-12-09 2020-01-31 Genethon Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine
JOP20200228A1 (ar) 2015-12-21 2017-06-16 Novartis Ag تركيبات وطرق لخفض تعبير البروتين tau
US11472824B2 (en) 2016-05-24 2022-10-18 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
AU2017270598B2 (en) * 2016-05-24 2022-12-01 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
CN109311920B (zh) * 2016-05-24 2021-11-09 萨勒普塔医疗公司 制备磷酸二酰胺吗啉代寡聚物的方法
MA45183A (fr) * 2016-05-24 2019-04-10 Sarepta Therapeutics Inc Procédés de préparation d'oligomères morpholino de phosphorodiamidate
JP2019525742A (ja) 2016-06-30 2019-09-12 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーに対するエクソンスキッピングオリゴマー
CN109152775B (zh) * 2016-06-30 2022-04-26 萨勒普塔医疗公司 制备磷酸二酰胺吗啉代寡聚物的方法
US20180028554A1 (en) * 2016-07-05 2018-02-01 Biomarin Technologies B.V. Oligomers Having Bicyclic Scaffold Moeities
WO2018014042A1 (en) * 2016-07-15 2018-01-18 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of dystrophin transcript
EP3515445A4 (en) * 2016-09-20 2020-05-27 The Regents of The University of Colorado, A Body Corporate SYNTHESIS OF BACKBONE-MODIFIED MORPHOLINO-OLIGONUCLEOTIDES AND CHIMERES WITH THE AID OF PHOSPHORAMIDITE CHEMISTRY
EP3554553B1 (en) 2016-12-19 2022-07-20 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
KR20240006023A (ko) 2016-12-19 2024-01-12 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
CA3047010A1 (en) * 2016-12-19 2018-06-28 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
WO2019046755A1 (en) * 2017-08-31 2019-03-07 Sarepta Therapeutics, Inc. METHODS FOR TREATING MUSCLE DYSTROPHY
EA201991450A1 (ru) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
TWI812647B (zh) * 2017-09-25 2023-08-21 美商薩羅塔治療公司 經由速流合成以製備磷醯二胺嗎啉代寡聚物之製程
JP2020536057A (ja) 2017-09-28 2020-12-10 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーを処置するための併用療法
EP3765030A4 (en) 2018-03-16 2022-01-26 Sarepta Therapeutics, Inc. CHIMERIC PEPTIDES FOR ANTISENSE ADMINISTRATION
WO2019209764A2 (en) * 2018-04-26 2019-10-31 Sarepta Therapeutics, Inc. Exon skipping oligomers and oligomer conjugates for muscular dystrophy
US10765760B2 (en) 2018-05-29 2020-09-08 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
EP3810150A4 (en) * 2018-06-14 2023-01-25 Sarepta Therapeutics, Inc. EXON-SKIPPING OLIGOMERS AND OLIGOMER CONJUGATES FOR MUSCULAR DYSTROPHY
JP7345466B2 (ja) * 2018-06-26 2023-09-15 日本新薬株式会社 アンチセンスオリゴヌクレオチドを含有する組成物およびデュシェンヌ型筋ジストロフィーの治療へのその使用
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
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
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
EP3890783A1 (en) 2018-12-07 2021-10-13 Oxford University Innovation Limited Linkers
JP7687682B2 (ja) * 2019-04-08 2025-06-03 国立大学法人東京科学大学 筋疾患治療用医薬組成物
WO2021025899A1 (en) 2019-08-02 2021-02-11 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer pharmaceutical compositions
AU2020368539A1 (en) 2019-10-16 2022-04-28 Massachusetts Institute Of Technology Engineered muscle targeting compositions
EP4182315A4 (en) * 2020-07-10 2024-07-03 Changzhou Syntheall Pharmaceuticals Co., Ltd. Processes for preparing oligonucleotides
CA3211038A1 (en) * 2021-02-12 2022-08-18 Oxford University Innovation Limited Cell-penetrating peptide conjugates and methods of their use
JP2024519601A (ja) 2021-03-31 2024-05-20 エントラーダ セラピューティクス，インコーポレイティド 環状細胞透過性ペプチド
WO2022217366A1 (en) * 2021-04-14 2022-10-20 The Royal Institution For The Advancement Of Learning/Mcgill University Modified oligonucleotides for the treatment of duchenne muscular dystrophy
IL308353A (en) 2021-05-10 2024-01-01 Entrada Therapeutics Inc Compositions and methods for modulating tissue distribution of intracellular therapies
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
MX2024000489A (es) 2021-07-09 2024-04-09 Dyne Therapeutics Inc Complejos dirigidos al musculo y formulaciones para el tratamiento de las distrofinopatías.
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US20250066396A1 (en) 2021-12-27 2025-02-27 Nippon Shinyaku Co., Ltd. Method for producing oligonucleic acid compound
US20250195667A1 (en) * 2022-03-17 2025-06-19 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer conjugates
WO2024073588A1 (en) * 2022-09-28 2024-04-04 Board Of Regents, The University Of Texas System Respiratory dry powder delivery

Family Cites Families (52)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CH445129A (fr) 1964-04-29 1967-10-15 Nestle Sa Procédé pour la préparation de composés d'inclusion à poids moléculaire élevé
US3459731A (en) 1966-12-16 1969-08-05 Corn Products Co Cyclodextrin polyethers and their production
US3453257A (en) 1967-02-13 1969-07-01 Corn Products Co Cyclodextrin with cationic properties
US3426011A (en) 1967-02-13 1969-02-04 Corn Products Co Cyclodextrins with anionic properties
US3453259A (en) 1967-03-22 1969-07-01 Corn Products Co Cyclodextrin polyol ethers and their oxidation products
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
EP0215942B1 (en) 1985-03-15 1995-07-12 Antivirals Inc. Polynucleotide assay reagent and method
US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group Polynucleotide assay reagent and method
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5521063A (en) 1985-03-15 1996-05-28 Antivirals Inc. Polynucleotide reagent containing chiral subunits and methods of use
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
KR0166088B1 (ko) 1990-01-23 1999-01-15 . 수용해도가 증가된 시클로덱스트린 유도체 및 이의 용도
US5149797A (en) 1990-02-15 1992-09-22 The Worcester Foundation For Experimental Biology Method of site-specific alteration of rna and production of encoded polypeptides
CA2140343A1 (en) 1992-07-17 1994-02-03 Sean M. Sullivan Method and reagent for treatment of animal diseases
AU6786594A (en) 1993-05-11 1994-12-12 University Of North Carolina At Chapel Hill, The Antisense oligonucleotides which combat aberrant splicing and methods of using the same
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
IL115849A0 (en) 1994-11-03 1996-01-31 Merz & Co Gmbh & Co Tangential filtration preparation of liposomal drugs and liposome product thereof
WO1999042091A2 (en) 1998-02-19 1999-08-26 Massachusetts Institute Of Technology Use of polycations as endosomolytic agents
US6683173B2 (en) 1998-04-03 2004-01-27 Epoch Biosciences, Inc. Tm leveling methods
US6210892B1 (en) 1998-10-07 2001-04-03 Isis Pharmaceuticals, Inc. Alteration of cellular behavior by antisense modulation of mRNA processing
EP1242052A4 (en) 1999-12-29 2003-07-02 A James Mixson HISTIDIN COPOLYMER AND METHODS OF USE THEREOF
US7070807B2 (en) 1999-12-29 2006-07-04 Mixson A James Branched histidine copolymers and methods for using same
CA2448120A1 (en) 2001-05-24 2002-11-28 Genzyme Corporation Muscle-specific expression vectors
CA2524255C (en) 2003-03-21 2014-02-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
USRE47769E1 (en) * 2004-06-28 2019-12-17 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
US8067571B2 (en) * 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
JP2009515523A (ja) 2005-11-10 2009-04-16 サンタリス ファーマ アー／エス Ｔｎｆスーパーファミリー受容体に対するスプライス切替オリゴマーならびに疾病治療における該オリゴマーの使用
US7943762B2 (en) 2006-05-10 2011-05-17 Avi Biopharma, Inc. Oligonucleotide analogs having cationic intersubunit linkages
US20070265215A1 (en) * 2006-05-11 2007-11-15 Iversen Patrick L Antisense restenosis composition and method
US20100016215A1 (en) * 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
HUE028662T2 (en) * 2007-10-26 2016-12-28 Academisch Ziekenhuis Leiden Preparations and methods for controlling muscle disorders
US8299206B2 (en) 2007-11-15 2012-10-30 Avi Biopharma, Inc. Method of synthesis of morpholino oligomers
US8076476B2 (en) 2007-11-15 2011-12-13 Avi Biopharma, Inc. Synthesis of morpholino oligomers using doubly protected guanine morpholino subunits
WO2009127230A1 (en) 2008-04-16 2009-10-22 Curevac Gmbh MODIFIED (m)RNA FOR SUPPRESSING OR AVOIDING AN IMMUNOSTIMULATORY RESPONSE AND IMMUNOSUPPRESSIVE COMPOSITION
US8084601B2 (en) * 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
TR201902952T4 (tr) * 2008-10-24 2019-03-21 Sarepta Therapeutics Inc Dmd için ekson atlama bileşimleri.
WO2011057350A1 (en) * 2009-11-12 2011-05-19 The University Of Western Australia Antisense molecules and methods for treating pathologies
CA2799501C (en) 2010-05-28 2022-02-15 Sarepta Therapeutics, Inc. Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
TWI541024B (zh) * 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
US9161948B2 (en) * 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
KR102229650B1 (ko) 2011-05-05 2021-03-19 사렙타 쎄러퓨틱스, 인코퍼레이티드 펩타이드 올리고뉴클레오타이드 접합체
EP2750715B1 (en) * 2011-08-30 2018-10-31 The Regents of The University of California Identification of small molecules that enhance therapeutic exon skipping
CN111893117B (zh) * 2012-01-27 2024-06-04 比奥马林技术公司 治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸
EP2828395B1 (en) * 2012-03-20 2018-10-24 Sarepta Therapeutics, Inc. Boronic acid conjugates of oligonucleotide analogues
BR112015014987A2 (pt) * 2012-12-20 2017-08-15 Sarepta Therapeutics Inc Aprimoradas composições que pulam éxon para o tratamento da distrofia muscular

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20190015311A (ko) * 2016-05-24 2019-02-13 사렙타 쎄러퓨틱스 인코퍼레이티드 올리고머의 제조 방법

Also Published As

Publication number Publication date
WO2014153240A3 (en) 2014-12-31
PT2970964T (pt) 2019-03-14
KR20240094032A (ko) 2024-06-24
MX366274B (es) 2019-07-04
CN110066793A (zh) 2019-07-30
CY1121876T1 (el) 2020-10-14
MX2015012149A (es) 2016-06-02
AU2014236160A1 (en) 2015-10-01
US20140315977A1 (en) 2014-10-23
KR20230074606A (ko) 2023-05-30
TR201903009T4 (tr) 2019-03-21
IL272415A (en) 2020-03-31
IL241047B (en) 2020-02-27
ES2714290T3 (es) 2019-05-28
WO2014153240A2 (en) 2014-09-25
EP3495485A2 (en) 2019-06-12
JP2016521119A (ja) 2016-07-21
SMT201900139T1 (it) 2019-05-10
EP3760720A1 (en) 2021-01-06
PL2970964T3 (pl) 2019-06-28
EA201591694A1 (ru) 2016-06-30
HUE042218T2 (hu) 2019-06-28
NZ631132A (en) 2017-06-30
US20220364093A1 (en) 2022-11-17
EP2970964B8 (en) 2019-03-06
US20180002689A1 (en) 2018-01-04
KR20220061280A (ko) 2022-05-12
SI2970964T1 (sl) 2019-05-31
AU2022252769A1 (en) 2022-11-10
US20160040162A1 (en) 2016-02-11
JP2024112929A (ja) 2024-08-21
CA2903872A1 (en) 2014-09-25
RS58529B1 (sr) 2019-04-30
CN105392884B (zh) 2018-11-30
US20200040336A1 (en) 2020-02-06
EP3495485A3 (en) 2019-08-21
BR112015023038A2 (pt) 2017-11-14
AU2020203473A1 (en) 2020-06-18
HRP20190382T1 (hr) 2019-04-19
HK1218138A1 (en) 2017-02-03
DK2970964T3 (en) 2019-04-01
CN105392884A (zh) 2016-03-09
EP2970964B1 (en) 2018-12-26
JP2022031948A (ja) 2022-02-22
EP2970964A2 (en) 2016-01-20
KR20210006516A (ko) 2021-01-18
IL241047A0 (en) 2015-11-30
MX2019000224A (es) 2021-06-15
JP2020039362A (ja) 2020-03-19
LT2970964T (lt) 2019-04-25
SA515361121B1 (ar) 2019-05-07

Similar Documents

Publication Publication Date Title
AU2022202393B2 (en) Exon skipping compositions for treating muscular dystrophy
KR20150133752A (ko) 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
JP2019050834A (ja) 筋ジストロフィを処置するための改善されたエキソンスキッピング組成物
HK1218138B (en) Exon skipping compositions for treating muscular dystrophy

Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20151008

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
A201 Request for examination
PA0201 Request for examination

Patent event code: PA02012R01D

Patent event date: 20190313

Comment text: Request for Examination of Application

E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

Comment text: Notification of reason for refusal

Patent event date: 20200305

Patent event code: PE09021S01D

E601 Decision to refuse application
PE0601 Decision on rejection of patent

Patent event date: 20201011

Comment text: Decision to Refuse Application

Patent event code: PE06012S01D

Patent event date: 20200305

Comment text: Notification of reason for refusal

Patent event code: PE06011S01I

PA0104 Divisional application for international application

Comment text: Divisional Application for International Patent

Patent event code: PA01041R01D

Patent event date: 20210108