CN110290812B - 用于肌肉萎缩症的外显子跳跃寡聚体缀合物 - Google Patents

用于肌肉萎缩症的外显子跳跃寡聚体缀合物 Download PDF

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CN110290812B
CN110290812B CN201780086301.2A CN201780086301A CN110290812B CN 110290812 B CN110290812 B CN 110290812B CN 201780086301 A CN201780086301 A CN 201780086301A CN 110290812 B CN110290812 B CN 110290812B
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exon
antisense oligomer
dystrophin
present disclosure
antisense
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CN110290812A (zh
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M.A.帕西尼
G.J.汉森
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Sarepta Therapeutics Inc
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Sarepta Therapeutics Inc
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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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  • Life Sciences & Earth Sciences (AREA)
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  • 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)
CN201780086301.2A 2016-12-19 2017-12-13 用于肌肉萎缩症的外显子跳跃寡聚体缀合物 Active CN110290812B (zh)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201662436182P 2016-12-19 2016-12-19
US62/436182 2016-12-19
US201762443476P 2017-01-06 2017-01-06
US62/443476 2017-01-06
US201762479173P 2017-03-30 2017-03-30
US62/479173 2017-03-30
US201762562080P 2017-09-22 2017-09-22
US62/562080 2017-09-22
PCT/US2017/066222 WO2018118599A1 (en) 2016-12-19 2017-12-13 Exon skipping oligomer conjugates for muscular dystrophy

Publications (2)

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CN110290812A CN110290812A (zh) 2019-09-27
CN110290812B true CN110290812B (zh) 2023-06-02

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CA (1) CA3046793A1 (enExample)
CO (1) CO2019007397A2 (enExample)
DK (1) DK3554553T3 (enExample)
ES (1) ES2927079T3 (enExample)
HR (1) HRP20220943T1 (enExample)
HU (1) HUE059843T2 (enExample)
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LT (1) LT3554553T (enExample)
MD (1) MD3554553T2 (enExample)
MX (1) MX2019006882A (enExample)
MY (1) MY195801A (enExample)
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SA (1) SA519402154B1 (enExample)
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SM (1) SMT202200366T1 (enExample)
TW (1) TWI760402B (enExample)
WO (1) WO2018118599A1 (enExample)
ZA (1) ZA201904092B (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110636866B (zh) * 2016-12-19 2024-10-18 萨勒普塔医疗公司 用于肌肉萎缩症的外显子跳跃寡聚体缀合物

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TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
MD3464305T2 (ro) 2016-05-24 2025-01-31 Sarepta Therapeutics Inc Procedee de preparare de oligomeri
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
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
WO2023282345A1 (ja) 2021-07-08 2023-01-12 日本新薬株式会社 腎毒性軽減剤
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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WO2012029986A1 (ja) * 2010-09-01 2012-03-08 日本新薬株式会社 アンチセンス核酸
CN104603271A (zh) * 2012-07-03 2015-05-06 普罗森萨科技有限公司 用于治疗肌肉萎缩症患者的寡核苷酸
CN105307723A (zh) * 2013-03-15 2016-02-03 萨勒普塔医疗公司 改进的用于治疗肌营养不良的组合物

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