JP6317675B2 - 延長リピート病を処置するためのオリゴヌクレオチド - Google Patents

延長リピート病を処置するためのオリゴヌクレオチド Download PDF

Info

Publication number
JP6317675B2
JP6317675B2 JP2014544959A JP2014544959A JP6317675B2 JP 6317675 B2 JP6317675 B2 JP 6317675B2 JP 2014544959 A JP2014544959 A JP 2014544959A JP 2014544959 A JP2014544959 A JP 2014544959A JP 6317675 B2 JP6317675 B2 JP 6317675B2
Authority
JP
Japan
Prior art keywords
group
antisense oligonucleotide
apn
oligonucleotides
oligonucleotide
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
JP2014544959A
Other languages
English (en)
Japanese (ja)
Other versions
JP2015506669A5 (enExample
JP2015506669A (ja
Inventor
ピーター リンズリー，
ピーター リンズリー，
ブライアン ジェイムス レパート，
ブライアン ジェイムス レパート，
グンナール ジェイ． ハンソン，
グンナール ジェイ． ハンソン，
Original Assignee
サレプタ セラピューティクス， インコーポレイテッド
サレプタ セラピューティクス， インコーポレイテッド
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 サレプタ セラピューティクス， インコーポレイテッド, サレプタ セラピューティクス， インコーポレイテッド filed Critical サレプタ セラピューティクス， インコーポレイテッド
Publication of JP2015506669A publication Critical patent/JP2015506669A/ja
Publication of JP2015506669A5 publication Critical patent/JP2015506669A5/ja
Application granted granted Critical
Publication of JP6317675B2 publication Critical patent/JP6317675B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • 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
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02—Phosphorus compounds
    • C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/6558—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing at least two different or differently substituted hetero rings neither condensed among themselves nor condensed with a common carbocyclic ring or ring system
    • C07F9/65583—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing at least two different or differently substituted hetero rings neither condensed among themselves nor condensed with a common carbocyclic ring or ring system each of the hetero rings containing nitrogen as ring hetero atom
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02—Phosphorus compounds
    • C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/6561—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing systems of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring or ring system, with or without other non-condensed hetero rings
    • C07F9/65616—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing systems of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring or ring system, with or without other non-condensed hetero rings containing the ring system having three or more than three double bonds between ring members or between ring members and non-ring members, e.g. purine or analogs
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/156—Polymorphic or mutational markers

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Neurology (AREA)
  • Analytical Chemistry (AREA)
  • Plant Pathology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurosurgery (AREA)
  • Pathology (AREA)
  • Immunology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Pain & Pain Management (AREA)
  • Psychology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2014544959A 2011-11-30 2012-11-30 延長リピート病を処置するためのオリゴヌクレオチド Active JP6317675B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201161565475P 2011-11-30 2011-11-30
US61/565,475 2011-11-30
PCT/US2012/067470 WO2013082548A1 (en) 2011-11-30 2012-11-30 Oligonucleotides for treating expanded repeat diseases

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2018008762A Division JP6718474B2 (ja) 2011-11-30 2018-01-23 延長リピート病を処置するためのオリゴヌクレオチド

Publications (3)

Publication Number Publication Date
JP2015506669A JP2015506669A (ja) 2015-03-05
JP2015506669A5 JP2015506669A5 (enExample) 2016-01-28
JP6317675B2 true JP6317675B2 (ja) 2018-04-25

Family

ID=47470150

Family Applications (4)

Application Number Title Priority Date Filing Date
JP2014544959A Active JP6317675B2 (ja) 2011-11-30 2012-11-30 延長リピート病を処置するためのオリゴヌクレオチド
JP2018008762A Active JP6718474B2 (ja) 2011-11-30 2018-01-23 延長リピート病を処置するためのオリゴヌクレオチド
JP2020033113A Active JP7057385B2 (ja) 2011-11-30 2020-02-28 延長リピート病を処置するためのオリゴヌクレオチド
JP2022010846A Pending JP2022048248A (ja) 2011-11-30 2022-01-27 延長リピート病を処置するためのオリゴヌクレオチド

Family Applications After (3)

Application Number Title Priority Date Filing Date
JP2018008762A Active JP6718474B2 (ja) 2011-11-30 2018-01-23 延長リピート病を処置するためのオリゴヌクレオチド
JP2020033113A Active JP7057385B2 (ja) 2011-11-30 2020-02-28 延長リピート病を処置するためのオリゴヌクレオチド
JP2022010846A Pending JP2022048248A (ja) 2011-11-30 2022-01-27 延長リピート病を処置するためのオリゴヌクレオチド

Country Status (5)

Country Link
US (3) US10066228B2 (enExample)
EP (2) EP3858847A1 (enExample)
JP (4) JP6317675B2 (enExample)
ES (1) ES2832531T3 (enExample)
WO (1) WO2013082548A1 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018082717A (ja) * 2011-11-30 2018-05-31 サレプタ セラピューティクス， インコーポレイテッド 延長リピート病を処置するためのオリゴヌクレオチド

Families Citing this family (51)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6047270B2 (ja) 2006-08-11 2016-12-21 バイオマリン テクノロジーズ ベー．フェー． Ｄｎａリピートの不安定性に関連した遺伝的障害を治療するための方法及び手段
NZ584793A (en) 2007-10-26 2012-05-25 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
BRPI0923225A2 (pt) 2008-12-02 2016-10-04 Chiralgen Ltd metodo para sintese de acidos nucleicos modificados no atomo de fosforo
EP2451461A4 (en) 2009-07-06 2013-05-29 Ontorii Inc NOVEL NUCLEIC ACID PRODRUGS AND METHOD OF USE THEREOF
DK2620428T3 (da) 2010-09-24 2019-07-01 Wave Life Sciences Ltd Asymmetrisk hjælpegruppe
US9476043B2 (en) * 2011-04-08 2016-10-25 Rula Zain-Luqman Diagnosis and treatment of friedreich's ataxia
CN107365339A (zh) 2011-07-19 2017-11-21 波涛生命科学有限公司 合成官能化核酸的方法
ES2727481T3 (es) 2011-11-30 2019-10-16 Sarepta Therapeutics Inc Inclusión inducida de exón en atrofia muscular espinal
PT2841578T (pt) 2012-04-23 2017-08-10 Biomarin Tech Bv Resumo
WO2014010250A1 (en) 2012-07-13 2014-01-16 Chiralgen, Ltd. Asymmetric auxiliary group
US10111962B2 (en) 2012-09-25 2018-10-30 Genzyme Corporation Peptide-linked morpholino antisense oligonucleotides for treatment of myotonic dystrophy
FI2906696T4 (fi) 2012-10-15 2023-03-18 Menetelmiä c9orf72:n ilmentymisen moduloimiseksi
EP2906697A4 (en) 2012-10-15 2016-06-22 Ionis Pharmaceuticals Inc METHOD FOR MONITORING THE C9ORF72 EXPRESSION
US10443052B2 (en) 2012-10-15 2019-10-15 Ionis Pharmaceuticals, Inc. Compositions for modulating C9ORF72 expression
MY192689A (en) 2013-10-11 2022-09-01 Ionis Pharmaceuticals Inc Compositions for modulating c9orf72 expression
US11162096B2 (en) 2013-10-14 2021-11-02 Ionis Pharmaceuticals, Inc Methods for modulating expression of C9ORF72 antisense transcript
EP3094728B1 (en) 2014-01-16 2022-03-09 Wave Life Sciences Ltd. Chiral design
SI3119888T1 (sl) 2014-03-19 2021-11-30 Ionis Pharmaceuticals, Inc. Sestavki za moduliranje izražanja ataksina 2
WO2015143245A1 (en) 2014-03-19 2015-09-24 Isis Pharmaceuticals, Inc. Methods for modulating ataxin 2 expression
US20160017327A1 (en) * 2014-07-11 2016-01-21 The Johns Hopkins University Phosphorodiamidate morpholino oligomers (pmos) and their use in suppression of mutant huntingtin expression and attenuation of neurotoxicity
US20190002876A1 (en) * 2014-12-09 2019-01-03 The Board Of Regents Of The University Of Texas System Compositions and methods for treatment of friedreich's ataxia
US10793855B2 (en) 2015-01-06 2020-10-06 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of C9ORF72 antisense transcript
WO2016167780A1 (en) 2015-04-16 2016-10-20 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
WO2016168592A2 (en) * 2015-04-16 2016-10-20 Ionis Pharmaceuticals, Inc. Compositions for modulating c9orf72 expression
KR102644053B1 (ko) 2015-05-19 2024-03-06 사렙타 쎄러퓨틱스 인코퍼레이티드 펩티드 올리고뉴클레오티드 콘주게이트
CN117965536A (zh) 2015-07-22 2024-05-03 波涛生命科学有限公司 寡核苷酸组合物及其方法
RS62930B1 (sr) 2015-08-05 2022-03-31 Eisai R&D Man Co Ltd Postupak za pripremu suštinski dijastereomerno čistog fosforodiamidatnog oligomera, fosforodiamidatni oligomer proizveden takvim postupkom i farmaceutska kompozicija koja sadrži fosforodiamidatni oligomer
WO2017040271A1 (en) 2015-08-28 2017-03-09 Sarepta Therapeutics, Inc. Modified antisense oligomers for exon inclusion in spinal muscular atrophy
WO2017079291A1 (en) * 2015-11-02 2017-05-11 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating c90rf72
AU2016359629B2 (en) * 2015-11-23 2023-03-09 Ranjan BATRA Tracking and manipulating cellular RNA via nuclear delivery of CRISPR/Cas9
WO2017117496A1 (en) 2015-12-31 2017-07-06 Ionis Pharmaceuticals, Inc. Methods for reducing ataxin-2 expression
EP3452596A4 (en) 2016-05-04 2020-03-18 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS
EA039716B1 (ru) * 2016-05-17 2022-03-03 Сарепта Терапьютикс, Инк. Пептид-олигонуклеотидные конъюгаты
MX390906B (es) 2016-05-24 2025-03-21 Sarepta Therapeutics Inc Procesos para preparar oligómeros de morfolino fosforodiamidato.
CN109152775B (zh) * 2016-06-30 2022-04-26 萨勒普塔医疗公司 制备磷酸二酰胺吗啉代寡聚物的方法
WO2018208998A1 (en) 2017-05-10 2018-11-15 The Regents Of The University Of California Directed editing of cellular rna via nuclear delivery of crispr/cas9
WO2018212271A1 (ja) * 2017-05-18 2018-11-22 国立大学法人京都大学 脊髄小脳変性症３６型の予防又は治療用組成物
US10668247B2 (en) * 2017-11-15 2020-06-02 Alcyone Lifesciences, Inc. Therapy specific, pre-programmed auto injection device
US11560401B2 (en) 2018-05-10 2023-01-24 Nippon Shinyaku Co., Ltd. Method for preparing oligonucleic acid compound
AU2019310097A1 (en) 2018-07-25 2021-02-04 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing ATXN2 expression
CA3110661A1 (en) 2018-08-29 2020-03-05 University Of Massachusetts Inhibition of protein kinases to treat friedreich ataxia
US12637682B2 (en) 2019-04-16 2026-05-26 The Regents Of The University Of California Protein translational control
EP3966328A4 (en) * 2019-05-06 2023-10-18 University Of Massachusetts Anti-c9orf72 oligonucleotides and related methods
JP2022551986A (ja) 2019-10-16 2022-12-14 ザ・ブロード・インスティテュート・インコーポレイテッド 修飾筋肉標的化組成物
EP4059944A4 (en) 2019-11-13 2024-04-10 Nippon Shinyaku Co., Ltd. METHOD FOR PREPARING AN OLIGONUCLICE ACID COMPOUND
EP4059943A4 (en) 2019-11-13 2024-01-31 Nippon Shinyaku Co., Ltd. METHOD FOR PRODUCING OLIGONUCLEIC ACID COMPOUND
US12668814B2 (en) 2020-07-22 2026-06-30 The Broad Institute, Inc. Engineered muscle targeting compositions
US12378267B2 (en) 2021-06-17 2025-08-05 Entrada Therapeutics, Inc. Synthesis of FMOC-protected morpholino monomers and oligomers
WO2023225506A1 (en) * 2022-05-16 2023-11-23 The University Of North Carolina At Chapel Hill Compositions and methods comprising synthetic rna molecules for treatment of intragenic nucleotide repeat disorders
WO2025137623A1 (en) * 2023-12-22 2025-06-26 Iris Medicine, Inc. Methods and compositions for treating ctg repeat expansion diseases
WO2025151707A1 (en) * 2024-01-11 2025-07-17 Entrada Therapeutics, Inc. Method for preparing multimers of phosphorodiamidate morpholino oligomers

Family Cites Families (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group Polynucleotide assay reagent and method
JP2528107B2 (ja) 1985-03-15 1996-08-28 サマ−トン，ジエ−ムス ポリヌクレオチド測定試薬と方法
US5521063A (en) 1985-03-15 1996-05-28 Antivirals Inc. Polynucleotide reagent containing chiral subunits and methods of use
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
AU659482B2 (en) 1991-06-28 1995-05-18 Massachusetts Institute Of Technology Localized oligonucleotide therapy
WO1997022371A1 (en) 1995-12-18 1997-06-26 Collagen Corporation Crosslinked polymer compositions and methods for their use
US6245747B1 (en) 1996-03-12 2001-06-12 The Board Of Regents Of The University Of Nebraska Targeted site specific antisense oligodeoxynucleotide delivery method
FR2802206B1 (fr) 1999-12-14 2005-04-22 Sod Conseils Rech Applic Derives de 4-aminopiperidine et leur utilisation en tant que medicament
AU5752601A (en) 2000-05-04 2001-11-12 Avi Biopharma Inc Splice-region antisense composition and method
US6965025B2 (en) 2001-12-10 2005-11-15 Isis Pharmaceuticals, Inc. Antisense modulation of connective tissue growth factor expression
PT2548560E (pt) 2005-06-23 2015-09-22 Isis Pharmaceuticals Inc Composições e métodos de modulação da excisão-união de smn2
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
LT2735568T (lt) * 2006-05-10 2017-11-27 Sarepta Therapeutics, Inc. Oligonukleotido analogai, turintys katijonines jungtis tarp subvienetų
JP6047270B2 (ja) * 2006-08-11 2016-12-21 バイオマリン テクノロジーズ ベー．フェー． Ｄｎａリピートの不安定性に関連した遺伝的障害を治療するための方法及び手段
US20100016215A1 (en) * 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
MX2010004955A (es) 2007-11-15 2010-06-30 Avi Biopharma Inc Metodo de sintesis de oligomeros de morfolina.
DE102008000410A1 (de) 2008-02-26 2009-08-27 Wacker Chemie Ag Verfahren zur Herstellung von Alkylchlorsilanen aus den Rückständen der Direktsynthese von Alkylchlorsilanen
AU2009223125A1 (en) 2008-03-12 2009-09-17 Amicus Therapeutics, Inc. Assays for diagnosing and evaluating treatment options for Pompe disease
US20120149757A1 (en) 2009-04-13 2012-06-14 Krainer Adrian R Compositions and methods for modulation of smn2 splicing
US20110269665A1 (en) * 2009-06-26 2011-11-03 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
US8802642B2 (en) 2010-04-28 2014-08-12 Iowa State University Research Foundation, Inc. Spinal muscular atrophy treatment via targeting SMN2 catalytic core
JP5585822B2 (ja) 2010-05-11 2014-09-10 東レ・ファインケミカル株式会社 光学活性ニペコチン酸誘導体の製造方法
KR101981705B1 (ko) 2010-05-28 2019-05-24 사렙타 쎄러퓨틱스, 인코퍼레이티드 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체
AU2011367230B2 (en) 2011-05-05 2017-08-10 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
US9161948B2 (en) * 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
WO2013082548A1 (en) * 2011-11-30 2013-06-06 Sarepta Therapeutics, Inc. Oligonucleotides for treating expanded repeat diseases
ES2727481T3 (es) 2011-11-30 2019-10-16 Sarepta Therapeutics Inc Inclusión inducida de exón en atrofia muscular espinal

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018082717A (ja) * 2011-11-30 2018-05-31 サレプタ セラピューティクス， インコーポレイテッド 延長リピート病を処置するためのオリゴヌクレオチド

Also Published As

Publication number Publication date
JP2022048248A (ja) 2022-03-25
WO2013082548A1 (en) 2013-06-06
ES2832531T3 (es) 2021-06-10
JP2018082717A (ja) 2018-05-31
US10066228B2 (en) 2018-09-04
JP2020078347A (ja) 2020-05-28
JP2015506669A (ja) 2015-03-05
US20140303238A1 (en) 2014-10-09
EP2785729A1 (en) 2014-10-08
EP2785729B1 (en) 2020-11-04
JP7057385B2 (ja) 2022-04-19
US20190100755A1 (en) 2019-04-04
HK1202875A1 (en) 2015-10-09
US11674139B2 (en) 2023-06-13
JP6718474B2 (ja) 2020-07-08
US20240209363A1 (en) 2024-06-27
EP3858847A1 (en) 2021-08-04

Similar Documents

Publication Publication Date Title
JP7057385B2 (ja) 延長リピート病を処置するためのオリゴヌクレオチド
JP6496549B2 (ja) 脊髄性筋萎縮症における誘発されたエクソン包含
JP7416852B2 (ja) Ｈｔｒａ１の発現を調節するためのアンチセンスオリゴヌクレオチド
KR20160132056A (ko) 안티센스 핵산
KR20190040098A (ko) 안티센스 핵산
JP6987041B2 (ja) 脊髄性筋萎縮症におけるエクソン包含のための修飾アンチセンスオリゴマー
JP2016185991A (ja) ペプチドオリゴヌクレオチドコンジュゲート
US20220042022A1 (en) Antisense oligonucleotides for modulating htra1 expression
HK1202875B (en) Oligonucleotides for treating expanded repeat diseases
HK1202891B (en) Induced exon inclusion in spinal muscle atrophy
HK40021168A (en) Antisense oligonucleotides for modulating htra1 expression
HK1262526A1 (en) Antisense oligonucleotides for modulating htra1 expression

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20151130

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20151130

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20160915

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20161213

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20170214

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20170315

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20170802

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20171016

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20171219

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20180123

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20180319

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20180330

R150 Certificate of patent or registration of utility model

Ref document number: 6317675

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250