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DK3554553T3 - Exon-overspringnings-oligomerkonjugat til muskeldystrofi - Google Patents

Exon-overspringnings-oligomerkonjugat til muskeldystrofi Download PDF

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Publication number
DK3554553T3
DK3554553T3 DK17832401.8T DK17832401T DK3554553T3 DK 3554553 T3 DK3554553 T3 DK 3554553T3 DK 17832401 T DK17832401 T DK 17832401T DK 3554553 T3 DK3554553 T3 DK 3554553T3
Authority
DK
Denmark
Prior art keywords
exon
muscular dystrophy
oligomer conjugate
skipping oligomer
skipping
Prior art date
Application number
DK17832401.8T
Other languages
English (en)
Inventor
Marco A Passini
Gunnar J Hanson
Original Assignee
Sarepta Therapeutics Inc
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 Sarepta Therapeutics Inc filed Critical Sarepta Therapeutics Inc
Application granted granted Critical
Publication of DK3554553T3 publication Critical patent/DK3554553T3/da

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Classifications

    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • 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
    • 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
    • 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
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
DK17832401.8T 2016-12-19 2017-12-13 Exon-overspringnings-oligomerkonjugat til muskeldystrofi DK3554553T3 (da)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662436182P 2016-12-19 2016-12-19
US201762443476P 2017-01-06 2017-01-06
US201762479173P 2017-03-30 2017-03-30
US201762562080P 2017-09-22 2017-09-22
PCT/US2017/066222 WO2018118599A1 (en) 2016-12-19 2017-12-13 Exon skipping oligomer conjugates for muscular dystrophy

Publications (1)

Publication Number Publication Date
DK3554553T3 true DK3554553T3 (da) 2022-09-19

Family

ID=61006315

Family Applications (1)

Application Number Title Priority Date Filing Date
DK17832401.8T DK3554553T3 (da) 2016-12-19 2017-12-13 Exon-overspringnings-oligomerkonjugat til muskeldystrofi

Country Status (27)

Country Link
US (4) US10888578B2 (da)
EP (1) EP3554553B1 (da)
JP (3) JP7118063B2 (da)
KR (2) KR102552428B1 (da)
CN (1) CN110290812B (da)
AU (1) AU2017382723B2 (da)
CA (1) CA3046793A1 (da)
CO (1) CO2019007397A2 (da)
DK (1) DK3554553T3 (da)
ES (1) ES2927079T3 (da)
HR (1) HRP20220943T1 (da)
HU (1) HUE059843T2 (da)
IL (2) IL267244B (da)
LT (1) LT3554553T (da)
MD (1) MD3554553T2 (da)
MX (1) MX2019006882A (da)
MY (1) MY195801A (da)
NZ (1) NZ755416A (da)
PL (1) PL3554553T3 (da)
PT (1) PT3554553T (da)
RS (1) RS63610B1 (da)
SA (1) SA519402154B1 (da)
SG (1) SG10202012839TA (da)
SM (1) SMT202200366T1 (da)
TW (1) TWI760402B (da)
WO (1) WO2018118599A1 (da)
ZA (1) ZA201904092B (da)

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EP3464305B1 (en) 2016-05-24 2024-08-21 Sarepta Therapeutics, Inc. Processes for preparing oligomers
HRP20240583T1 (hr) 2016-05-24 2024-07-19 Sarepta Therapeutics, Inc. Postupci za pripremu fosforodiamidat morfolino oligomera
KR20240006057A (ko) * 2016-12-19 2024-01-12 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
WO2018118627A1 (en) 2016-12-19 2018-06-28 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
MD3554553T2 (ro) * 2016-12-19 2022-10-31 Sarepta Therapeutics Inc Conjugați de oligomeri de omitere a exonului, pentru distrofie musculară
WO2018129384A1 (en) 2017-01-06 2018-07-12 Avidity Biosciences Llc Nucleic acid-polypeptide compositions and methods of inducing exon skipping
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US20220193246A1 (en) * 2019-04-18 2022-06-23 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
EP3987029A1 (en) * 2019-06-19 2022-04-27 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy
CN118109468A (zh) 2019-12-26 2024-05-31 日本新药株式会社 诱导外显子50的跳读的反义核酸
KR20220145865A (ko) 2020-02-28 2022-10-31 니뽄 신야쿠 가부시키가이샤 엑손 51의 스키핑을 유도하는 안티센스 핵산
CA3211038A1 (en) * 2021-02-12 2022-08-18 Oxford University Innovation Limited Cell-penetrating peptide conjugates and methods of their use
AU2022267324A1 (en) 2021-04-30 2023-12-14 Sarepta Therapeutics, Inc. Treatment methods for muscular dystrophy
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IL310001A (en) 2021-07-08 2024-03-01 Nippon Shinyaku Co Ltd Precipitation suppressing agent
EP4368186A4 (en) 2021-07-08 2025-06-25 Nippon Shinyaku Co., Ltd. NEPHROTOXICITY REDUCING AGENT
JPWO2023282344A1 (da) 2021-07-08 2023-01-12
CN120129745A (zh) 2022-03-10 2025-06-10 日本新药株式会社 抗病毒反义低聚物
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