JP7118063B2 - 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コンジュゲート - Google Patents

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コンジュゲート Download PDF

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JP7118063B2
JP7118063B2 JP2019531378A JP2019531378A JP7118063B2 JP 7118063 B2 JP7118063 B2 JP 7118063B2 JP 2019531378 A JP2019531378 A JP 2019531378A JP 2019531378 A JP2019531378 A JP 2019531378A JP 7118063 B2 JP7118063 B2 JP 7118063B2
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dystrophin
exon
antisense oligomer
antisense
skipping
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JP2020503009A5 (enExample
JP2020503009A (ja
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マルコ エー． パッシーニ，
ガンナー ジェイ． ハンソン，
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サレプタ セラピューティクス， インコーポレイテッド
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    • 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)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Microbiology (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)
JP2019531378A 2016-12-19 2017-12-13 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コンジュゲート Active JP7118063B2 (ja)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201662436182P 2016-12-19 2016-12-19
US62/436,182 2016-12-19
US201762443476P 2017-01-06 2017-01-06
US62/443,476 2017-01-06
US201762479173P 2017-03-30 2017-03-30
US62/479,173 2017-03-30
US201762562080P 2017-09-22 2017-09-22
US62/562,080 2017-09-22
PCT/US2017/066222 WO2018118599A1 (en) 2016-12-19 2017-12-13 Exon skipping oligomer conjugates for muscular dystrophy

Related Child Applications (1)

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JP2020202518A Division JP2021045160A (ja) 2016-12-19 2020-12-07 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コンジュゲート

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JP2020503009A JP2020503009A (ja) 2020-01-30
JP2020503009A5 JP2020503009A5 (enExample) 2021-01-21
JP7118063B2 true JP7118063B2 (ja) 2022-08-15

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JP2019531378A Active JP7118063B2 (ja) 2016-12-19 2017-12-13 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コンジュゲート
JP2020202518A Withdrawn JP2021045160A (ja) 2016-12-19 2020-12-07 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コンジュゲート
JP2023136250A Active JP7691462B2 (ja) 2016-12-19 2023-08-24 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コンジュゲート

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JP2023136250A Active JP7691462B2 (ja) 2016-12-19 2023-08-24 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コンジュゲート

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US (4) US10888578B2 (enExample)
EP (1) EP3554553B1 (enExample)
JP (3) JP7118063B2 (enExample)
KR (2) KR102810425B1 (enExample)
CN (1) CN110290812B (enExample)
AU (1) AU2017382723B2 (enExample)
CA (1) CA3046793A1 (enExample)
CO (1) CO2019007397A2 (enExample)
DK (1) DK3554553T3 (enExample)
ES (1) ES2927079T3 (enExample)
HR (1) HRP20220943T1 (enExample)
HU (1) HUE059843T2 (enExample)
IL (2) IL267244B (enExample)
LT (1) LT3554553T (enExample)
MD (1) MD3554553T2 (enExample)
MX (1) MX2019006882A (enExample)
MY (1) MY195801A (enExample)
NZ (1) NZ755416A (enExample)
PL (1) PL3554553T3 (enExample)
PT (1) PT3554553T (enExample)
RS (1) RS63610B1 (enExample)
SA (1) SA519402154B1 (enExample)
SG (1) SG10202012839TA (enExample)
SM (1) SMT202200366T1 (enExample)
TW (1) TWI760402B (enExample)
WO (1) WO2018118599A1 (enExample)
ZA (1) ZA201904092B (enExample)

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KR102504759B1 (ko) 2016-05-24 2023-02-28 사렙타 쎄러퓨틱스 인코퍼레이티드 포스포로디아미데이트 모르폴리노 올리고머의 제조 방법
MD3554553T2 (ro) * 2016-12-19 2022-10-31 Sarepta Therapeutics Inc Conjugați de oligomeri de omitere a exonului, pentru distrofie musculară
MA65895B1 (fr) 2016-12-19 2024-06-28 Sarepta Therapeutics, Inc. Conjugués d'oligomères induisant le saut d'exons pour la dystrophie musculaire
DK3554552T3 (da) * 2016-12-19 2022-10-24 Sarepta Therapeutics Inc Exon-skipping-oligomerkonjugater mod muskeldystrofi
WO2018129384A1 (en) 2017-01-06 2018-07-12 Avidity Biosciences Llc Nucleic acid-polypeptide compositions and methods of inducing exon skipping
GB201711809D0 (en) * 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
GB201821269D0 (en) 2018-12-28 2019-02-13 Nippon Shinyaku Co Ltd Myostatin signal inhibitor
JP2022528725A (ja) * 2019-04-18 2022-06-15 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治療するための組成物
WO2020257489A1 (en) * 2019-06-19 2020-12-24 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy
TWI874546B (zh) 2019-12-26 2025-03-01 日商日本新藥股份有限公司 誘導外顯子５０的跳讀的反義核酸
AU2021226089A1 (en) 2020-02-28 2022-09-15 National Center Of Neurology And Psychiatry Antisense nucleic acid inducing skipping of exon 51
WO2022171972A1 (en) * 2021-02-12 2022-08-18 Oxford University Innovation Limited Cell-penetrating peptide conjugates and methods of their use
WO2022232478A1 (en) 2021-04-30 2022-11-03 Sarepta Therapeutics, Inc. Treatment methods for muscular dystrophy
KR20240024176A (ko) 2021-06-23 2024-02-23 니뽄 신야쿠 가부시키가이샤 안티센스 올리고머의 조합
CN118201606A (zh) 2021-07-08 2024-06-14 日本新药株式会社 肾毒性减轻剂
AU2022306542A1 (en) 2021-07-08 2024-01-18 Nippon Shinyaku Co., Ltd. Precipitation suppressing agent
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JPWO2023171804A1 (enExample) 2022-03-10 2023-09-14
IL315280A (en) 2022-03-17 2024-10-01 Sarepta Therapeutics Inc Phosphorodiamidate morpholino oligomer conjugates

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