PL3118311T3 - Antysensowny kwas nukleinowy - Google Patents

Antysensowny kwas nukleinowy

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Publication number
PL3118311T3
PL3118311T3 PL15761392T PL15761392T PL3118311T3 PL 3118311 T3 PL3118311 T3 PL 3118311T3 PL 15761392 T PL15761392 T PL 15761392T PL 15761392 T PL15761392 T PL 15761392T PL 3118311 T3 PL3118311 T3 PL 3118311T3
Authority
PL
Poland
Prior art keywords
antisensive
nucleic acid
nucleic
acid
antisensive nucleic
Prior art date
Application number
PL15761392T
Other languages
English (en)
Inventor
Tatsushi Wakayama
Haruna SEO
Youhei Satou
Shin'ichi Takeda
Tetsuya Nagata
Original Assignee
Nippon Shinyaku Co., Ltd.
National Center Of Neurology And Psychiatry
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 Nippon Shinyaku Co., Ltd., National Center Of Neurology And Psychiatry filed Critical Nippon Shinyaku Co., Ltd.
Publication of PL3118311T3 publication Critical patent/PL3118311T3/pl

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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/314Phosphoramidates
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    • C12N2310/315Phosphorothioates
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/3222'-R Modification
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3233Morpholino-type ring
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
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    • C12N2310/3521Methyl
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
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    • C12N2310/3525MOE, methoxyethoxy
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
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    • C12N2310/3533Halogen
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    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/353Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3535Nitrogen
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing

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  • Health & Medical Sciences (AREA)
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  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
PL15761392T 2014-03-12 2015-03-11 Antysensowny kwas nukleinowy PL3118311T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2014048897 2014-03-12
PCT/JP2015/057180 WO2015137409A1 (ja) 2014-03-12 2015-03-11 アンチセンス核酸
EP15761392.8A EP3118311B1 (en) 2014-03-12 2015-03-11 Antisense nucleic acid

Publications (1)

Publication Number Publication Date
PL3118311T3 true PL3118311T3 (pl) 2019-09-30

Family

ID=54071850

Family Applications (1)

Application Number Title Priority Date Filing Date
PL15761392T PL3118311T3 (pl) 2014-03-12 2015-03-11 Antysensowny kwas nukleinowy

Country Status (31)

Country Link
US (5) US9988629B2 (pl)
EP (2) EP3514234A1 (pl)
JP (1) JP6606488B2 (pl)
KR (1) KR102335801B1 (pl)
CN (2) CN110951732A (pl)
AU (2) AU2015227733B2 (pl)
BR (1) BR112016020618B1 (pl)
CA (1) CA2939948A1 (pl)
CY (1) CY1121322T1 (pl)
DK (1) DK3118311T3 (pl)
ES (1) ES2710802T3 (pl)
HR (1) HRP20190235T1 (pl)
HU (1) HUE042283T2 (pl)
IL (1) IL247663B (pl)
LT (1) LT3118311T (pl)
MX (1) MX2016011538A (pl)
MY (1) MY193708A (pl)
NZ (1) NZ724836A (pl)
PH (1) PH12016501761B1 (pl)
PL (1) PL3118311T3 (pl)
PT (1) PT3118311T (pl)
RS (1) RS58573B1 (pl)
RU (2) RU2702424C2 (pl)
SG (1) SG11201607095RA (pl)
SI (1) SI3118311T1 (pl)
SM (1) SMT201900169T1 (pl)
TR (1) TR201901939T4 (pl)
TW (2) TWI672314B (pl)
UA (1) UA120849C2 (pl)
WO (1) WO2015137409A1 (pl)
ZA (1) ZA201605864B (pl)

Families Citing this family (39)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2702424C2 (ru) * 2014-03-12 2019-10-08 Ниппон Синяку Ко., Лтд. Антисмысловые нуклеиновые кислоты
WO2015194520A1 (ja) 2014-06-17 2015-12-23 日本新薬株式会社 アンチセンス核酸
CN113913426B (zh) 2015-09-15 2024-08-02 日本新药株式会社 反义核酸
SG10201912902TA (en) 2015-10-09 2020-02-27 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof
KR102775461B1 (ko) 2016-04-01 2025-02-28 어비디티 바이오사이언시스 인크. 핵산-폴리펩타이드 조성물 및 이의 용도
US20190262375A1 (en) 2016-06-30 2019-08-29 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystrophy
EP3485015A4 (en) * 2016-07-15 2020-07-29 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS OF MODULATION OF DYSTROPHINE TRANSCRIPT
US11118179B2 (en) * 2016-09-23 2021-09-14 Synthena Ag Mixed tricyclo-DNA, 2′-modified RNA oligonucleotide compositions and uses thereof
HRP20240705T1 (hr) 2016-12-19 2024-08-30 Sarepta Therapeutics, Inc. Oligomerni konjugati koji preskaču egzon za mišićnu distrofiju
KR102810425B1 (ko) * 2016-12-19 2025-05-21 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
KR102646318B1 (ko) 2016-12-19 2024-03-12 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
SG10202107429WA (en) 2017-01-06 2021-08-30 Avidity Biosciences Inc 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
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
JP2020536060A (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
BR112020020670A2 (pt) 2018-04-12 2021-03-02 Wave Life Sciences Ltd. composição de oligonucleotídeo, composição farmacêutica, método para alterar o splicing de uma transcrição alvo, método para tratar distrofia muscular, método para preparar um oligonucleotídeo ou uma composição de oligonucleotídeo do mesmo e oligonucleotídeo
TWI844541B (zh) 2018-05-11 2024-06-11 新加坡商波濤生命科學有限公司 寡核苷酸組成物及其使用方法
US10765760B2 (en) 2018-05-29 2020-09-08 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
US20210346290A1 (en) * 2018-06-13 2021-11-11 Kawasaki Institute Of Industrial Promotion POLYION COMPLEX MICELLE THAT INCLUDES ANTISENSE OLIGONUCLEOTIDE AND BLOCK COPOLYMER OF PEG BLOCK AND CATIONIC POLYMER, SAID BLOCK COPOLYMER HAVING NUMBER-AVERAGE MOLECULAR WEIGHT OF 3-10 kDa
WO2020028832A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
AU2019316103B2 (en) 2018-08-02 2026-02-19 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
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
EP3874044A1 (en) * 2018-11-02 2021-09-08 BioMarin Technologies B.V. Bispecific antisense oligonucleotides for dystrophin exon skipping
AU2019392928A1 (en) * 2018-12-06 2021-06-17 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
WO2020158792A1 (ja) * 2019-01-30 2020-08-06 国立研究開発法人国立精神・神経医療研究センター 核酸送達複合体
EP3959319A4 (en) 2019-04-25 2023-06-07 Avidity Biosciences, Inc. NUCLEIC ACID COMPOSITIONS AND METHODS FOR MULTI-EXON SKIPPING
MX2022007491A (es) * 2019-12-19 2022-08-15 Nippon Shinyaku Co Ltd Acido nucleico antisentido que permite la omision de exones.
PE20230237A1 (es) * 2020-02-28 2023-02-07 Nippon Shinyaku Co Ltd Acidos nucleicos antisentido que inducen la omision del exon 51
IL307937A (en) 2021-04-30 2023-12-01 Sarepta Therapeutics Inc Treatment methods for muscular dystrophy
JP2024525608A (ja) 2021-07-09 2024-07-12 ダイン セラピューティクス,インコーポレーテッド ジストロフィン異常症を処置するための筋標的化複合体および製剤
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
AU2022424485A1 (en) 2021-12-27 2024-07-11 Nippon Shinyaku Co., Ltd. Method for producing oligonucleic acid compound
CN120129745A (zh) 2022-03-10 2025-06-10 日本新药株式会社 抗病毒反义低聚物
JP2025509438A (ja) 2022-03-17 2025-04-11 サレプタ セラピューティクス, インコーポレイテッド ホスホロジアミデートモルホリノオリゴマーコンジュゲート
US12071621B2 (en) 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
DE69033986T2 (de) 1989-12-20 2003-03-13 Antivirals Inc., Portland Ungeladene, auf Morpholin basierende Polymere mit Chiralen, Phosphor enthaltenden Brücken zwischen den Untereinheiten
JP2924179B2 (ja) 1993-02-19 1999-07-26 日本新薬株式会社 グリセロール誘導体,デバイス及び医薬組成物
IL115849A0 (en) 1994-11-03 1996-01-31 Merz & Co Gmbh & Co Tangential filtration preparation of liposomal drugs and liposome product thereof
EP1191097A1 (en) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
EP2530155B1 (en) 2002-11-25 2016-03-16 Masafumi Matsuo ENA nucleic acid drugs modifying splicing in mRNA precursor
SI2206781T1 (sl) * 2004-06-28 2016-05-31 The University Of Western Australia Protismiselni oligonukleotidi za induciranje preskakovanja eksona in postopki njihove uporabe
WO2006038608A1 (ja) 2004-10-05 2006-04-13 Nippon Shinyaku Co., Ltd. オリゴ二本鎖rna及び医薬組成物
CA2605512A1 (en) * 2005-04-22 2006-10-26 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the binding of sr proteins and by interfering with secondary rna structure.
US20090069260A1 (en) 2005-05-30 2009-03-12 Nippon Shinyaku Co., Ltd Method for producing a nucleic-acid-containing complex preparation
US8067571B2 (en) * 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
RU2418068C2 (ru) * 2005-08-17 2011-05-10 Сирна Терапьютикс, Инк. Молекулы химически модифицированной короткой интерферирующей нуклеиновой кислоты, которые опосредуют интерференцию рнк
WO2007145661A2 (en) * 2005-11-30 2007-12-21 Guild Associates, Inc. Differentially fluorescent yeast biosensors for the detection and biodegradation of chemical agents
EP2119738B1 (en) 2007-02-05 2014-04-16 Nippon Shinyaku Co., Ltd. Polyethylene glycol derivative
DK2203173T3 (en) * 2007-10-26 2016-02-29 Academisch Ziekenhuis Leiden Materials and methods for prevention of muscle diseases
CA2884340C (en) 2007-11-15 2017-07-25 Sarepta Therapeutics, Inc. Method of synthesis of morpholino oligomers
US8084601B2 (en) * 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
EP2762567B1 (en) * 2008-10-24 2016-07-13 Sarepta Therapeutics, Inc. Multiple exon skipping compositions for DMD
CA2741629C (en) 2008-10-27 2022-07-05 Academisch Ziekenhuis Leiden Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mrna
US20110269665A1 (en) 2009-06-26 2011-11-03 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
SMT201800579T1 (it) * 2009-11-12 2019-01-11 Univ Western Australia Molecole antisenso e metodi per trattare patologie
JP2013530154A (ja) * 2010-05-28 2013-07-25 サレプタ セラピューティクス, インコーポレイテッド 修飾されたサブユニット間結合および/または末端基を有するオリゴヌクレオチドアナログ
IT1400425B1 (it) * 2010-06-08 2013-05-31 Amsterdam Molecular Therapeutics Bv Modified snrnas for use in therapy.
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
EP2672977A1 (en) * 2011-02-08 2013-12-18 The Charlotte-Mecklenburg Hospital Authority d/b/a Carolina Healthcare System Antisense oligonucleotides
WO2012150960A1 (en) * 2011-05-05 2012-11-08 Avi Biopharma, Inc. Peptide oligonucleotide conjugates
CN108611349A (zh) * 2011-12-28 2018-10-02 日本新药株式会社 反义核酸
RU2702424C2 (ru) * 2014-03-12 2019-10-08 Ниппон Синяку Ко., Лтд. Антисмысловые нуклеиновые кислоты

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