KR20230116945A - 근육 이영양증의 치료를 위한 엑손 스키핑 조성물 - Google Patents

근육 이영양증의 치료를 위한 엑손 스키핑 조성물 Download PDF

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KR20230116945A
KR20230116945A KR1020237024770A KR20237024770A KR20230116945A KR 20230116945 A KR20230116945 A KR 20230116945A KR 1020237024770 A KR1020237024770 A KR 1020237024770A KR 20237024770 A KR20237024770 A KR 20237024770A KR 20230116945 A KR20230116945 A KR 20230116945A
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South Korea
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antisense
acid
dystrophin
oligomers
exon
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KR1020237024770A
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Korean (ko)
Inventor
리차드 케이. 베스트윅
다이앤 엘리자베스 프랭크
프레드 조셉 슈넬
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사렙타 쎄러퓨틱스 인코퍼레이티드
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Priority to KR1020247007336A priority Critical patent/KR20240034881A/ko
Publication of KR20230116945A publication Critical patent/KR20230116945A/ko
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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
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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
    • 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
    • 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
    • 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
    • A61K47/56—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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59—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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
    • A61K47/60—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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
    • 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
    • A61K47/645—Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
    • A61K47/6455—Polycationic oligopeptides, polypeptides or polyamino acids, e.g. for complexing nucleic acids
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/09—Recombinant DNA-technology
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/33—Chemical structure of the base
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/3513—Protein; Peptide
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    • 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
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    • C12N2320/33—Alteration of splicing
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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  • Polymers With Sulfur, Phosphorus Or Metals In The Main Chain (AREA)
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KR1020237024770A 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물 Ceased KR20230116945A (ko)

Priority Applications (1)

Application Number Priority Date Filing Date Title
KR1020247007336A KR20240034881A (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201361784547P 2013-03-14 2013-03-14
US61/784,547 2013-03-14
KR1020237012235A KR102558958B1 (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
PCT/US2014/029689 WO2014153220A2 (en) 2013-03-14 2014-03-14 Exon skipping compositions for treating muscular dystrophy

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KR1020237012235A Division KR102558958B1 (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물

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KR1020247007336A Division KR20240034881A (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물

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KR20230116945A true KR20230116945A (ko) 2023-08-04

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Family Applications (7)

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KR1020237024770A Ceased KR20230116945A (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
KR1020247007336A Ceased KR20240034881A (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
KR1020257009068A Pending KR20250047401A (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
KR1020157028186A Active KR102258326B1 (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
KR1020237012235A Active KR102558958B1 (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
KR1020217015536A Active KR102390965B1 (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
KR1020227013492A Active KR102521608B1 (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물

Family Applications After (6)

Application Number Title Priority Date Filing Date
KR1020247007336A Ceased KR20240034881A (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
KR1020257009068A Pending KR20250047401A (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
KR1020157028186A Active KR102258326B1 (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
KR1020237012235A Active KR102558958B1 (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
KR1020217015536A Active KR102390965B1 (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
KR1020227013492A Active KR102521608B1 (ko) 2013-03-14 2014-03-14 근육 이영양증의 치료를 위한 엑손 스키핑 조성물

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US (11) US9217148B2 (enExample)
EP (3) EP2970963B1 (enExample)
JP (5) JP6449231B2 (enExample)
KR (7) KR20230116945A (enExample)
CN (3) CN117844807A (enExample)
AU (4) AU2014236140B2 (enExample)
BR (2) BR122020016865B1 (enExample)
CA (1) CA2906209A1 (enExample)
EA (2) EA202090946A3 (enExample)
ES (1) ES2762881T3 (enExample)
IL (3) IL293975B1 (enExample)
MX (2) MX366485B (enExample)
NZ (4) NZ631245A (enExample)
SA (1) SA515361125B1 (enExample)
WO (1) WO2014153220A2 (enExample)

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EP1766010B1 (en) 2004-06-28 2011-02-16 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
ES2852549T3 (es) 2005-02-09 2021-09-13 Sarepta Therapeutics Inc Composición antisentido para tratamiento de la atrofia muscular
KR20180118828A (ko) 2008-10-24 2018-10-31 사렙타 쎄러퓨틱스 인코퍼레이티드 Dmd를 위한 다중 엑손 스키핑 조성물
EP3431603A1 (en) 2009-11-12 2019-01-23 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
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
KR20230116945A (ko) 2013-03-14 2023-08-04 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
BR112015022998A2 (pt) 2013-03-15 2017-11-14 Sarepta Therapeutics Inc composições melhoradas para o tratamento de distrofia muscular
CA2931829A1 (en) * 2013-12-02 2015-06-11 Ionis Pharmaceuticals, Inc. Antisense compounds and uses thereof
MX2017011004A (es) * 2015-02-27 2018-02-09 Sarepta Therapeutics Inc Inclusion del exon2 inducida por antisentido en alfa-glucosidasa.acida.
EP3271460A4 (en) * 2015-03-17 2019-03-13 The General Hospital Corporation RNA INTERACTOM OF POLYCOMB REPRESSIVE COMPLEX 1 (PRC1)
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
WO2017062835A2 (en) 2015-10-09 2017-04-13 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders
SG10201912902TA (en) 2015-10-09 2020-02-27 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof
JOP20200228A1 (ar) 2015-12-21 2017-06-16 Novartis Ag تركيبات وطرق لخفض تعبير البروتين tau
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