NZ700561A - Rna modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders - Google Patents

Rna modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders

Info

Publication number
NZ700561A
NZ700561A NZ700561A NZ70056113A NZ700561A NZ 700561 A NZ700561 A NZ 700561A NZ 700561 A NZ700561 A NZ 700561A NZ 70056113 A NZ70056113 A NZ 70056113A NZ 700561 A NZ700561 A NZ 700561A
Authority
NZ
New Zealand
Prior art keywords
treatment
improved characteristics
neuromuscular disorders
treating
modulating oligonucleotides
Prior art date
Application number
NZ700561A
Other languages
English (en)
Inventor
Visser Peter Christian De
Susan Allegonda Maria Mulders
Original Assignee
Biomarin Technologies B V
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 Biomarin Technologies B V filed Critical Biomarin Technologies B V
Publication of NZ700561A publication Critical patent/NZ700561A/en

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
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/333—Modified A
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/335—Modified T or U
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/336—Modified G
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Pain & Pain Management (AREA)
  • Psychology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
NZ700561A 2012-04-23 2013-04-23 Rna modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders NZ700561A (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261636914P 2012-04-23 2012-04-23
EP12165139 2012-04-23
PCT/NL2013/050306 WO2013162363A1 (en) 2012-04-23 2013-04-23 RNA modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders

Publications (1)

Publication Number Publication Date
NZ700561A true NZ700561A (en) 2016-07-29

Family

ID=49483552

Family Applications (1)

Application Number Title Priority Date Filing Date
NZ700561A NZ700561A (en) 2012-04-23 2013-04-23 Rna modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders

Country Status (19)

Country Link
US (5) US9745576B2 (enExample)
EP (4) EP3228712A1 (enExample)
JP (5) JP6174678B2 (enExample)
CN (2) CN110025628B (enExample)
AU (1) AU2013253074C1 (enExample)
BR (1) BR112014026285B1 (enExample)
CA (2) CA2870697C (enExample)
DK (1) DK2841578T3 (enExample)
ES (2) ES2639066T3 (enExample)
HR (1) HRP20171254T1 (enExample)
HU (1) HUE033431T2 (enExample)
LT (1) LT2841578T (enExample)
NZ (1) NZ700561A (enExample)
PL (2) PL2841578T3 (enExample)
PT (1) PT2841578T (enExample)
RS (1) RS56319B1 (enExample)
SI (1) SI2841578T1 (enExample)
SM (1) SMT201700339T1 (enExample)
WO (1) WO2013162363A1 (enExample)

Families Citing this family (47)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK2049664T3 (da) 2006-08-11 2012-01-02 Prosensa Technologies Bv Enkeltstrengede oligonukleotider, som er komplementære til repetitive elementer, til behandling af med DNA-repetitiv-ustabilitet associerede lidelser
EP2614827B1 (en) 2007-10-26 2017-06-28 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
EP2119783A1 (en) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
CN102282155B (zh) 2008-12-02 2017-06-09 日本波涛生命科学公司 磷原子修饰的核酸的合成方法
WO2011005761A1 (en) 2009-07-06 2011-01-13 Ontorii, Inc Novel nucleic acid prodrugs and methods use thereof
WO2012039448A1 (ja) 2010-09-24 2012-03-29 株式会社キラルジェン 不斉補助基
WO2012138289A1 (en) * 2011-04-08 2012-10-11 Zain-Luqman Rula Diagnosis and treatment of friedreich's ataxia
EP2734208B1 (en) 2011-07-19 2017-03-01 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
EP4043039A1 (en) 2012-01-27 2022-08-17 BioMarin Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
EP3228712A1 (en) * 2012-04-23 2017-10-11 BioMarin Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders
AU2013288048A1 (en) 2012-07-13 2015-01-22 Wave Life Sciences Ltd. Asymmetric auxiliary group
WO2014062686A1 (en) 2012-10-15 2014-04-24 Isis Pharmaceuticals, Inc. Methods for modulating c9orf72 expression
BR112015008399A8 (pt) * 2012-10-15 2017-10-03 Ionis Pharmaceuticals Inc Composto para modular a expressão de c90rf72, seu uso, oligonucleotídeo modificado, composto de fita dupla e composição
WO2014062736A1 (en) * 2012-10-15 2014-04-24 Isis Pharmaceuticals, Inc. Methods for monitoring c9orf72 expression
EP3055414A4 (en) 2013-10-11 2017-07-19 Ionis Pharmaceuticals, Inc. Compositions for modulating c9orf72 expression
CN113278617A (zh) 2014-01-16 2021-08-20 波涛生命科学有限公司 手性设计
US10006027B2 (en) 2014-03-19 2018-06-26 Ionis Pharmaceuticals, Inc. Methods for modulating Ataxin 2 expression
CA2942340A1 (en) 2014-03-19 2015-09-24 Ionis Pharmaceuticals, Inc. Compositions for modulating ataxin 2 expression
US10538762B2 (en) 2014-10-14 2020-01-21 The Board Of Regents Of The University Of Texas System Allele selective inhibition of mutant C9orf72 foci expression by duplex RNAS targeting the expanded hexanucleotide repeat
WO2016168592A2 (en) * 2015-04-16 2016-10-20 Ionis Pharmaceuticals, Inc. Compositions for modulating c9orf72 expression
WO2017079291A1 (en) 2015-11-02 2017-05-11 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating c90rf72
EP3397288A4 (en) 2015-12-31 2019-09-11 Ionis Pharmaceuticals, Inc. PROCESS FOR REDUCING ATAXIN-2 EXPRESSION
US20180028554A1 (en) * 2016-07-05 2018-02-01 Biomarin Technologies B.V. Oligomers Having Bicyclic Scaffold Moeities
CN118416088A (zh) 2017-03-03 2024-08-02 加利福尼亚大学董事会 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向
CA3083582A1 (en) * 2017-10-23 2019-05-02 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disease
EA202190299A1 (ru) 2018-07-25 2021-06-11 Ионис Фармасьютикалз, Инк. Соединения и способы для снижения экспрессии atxn2
US12370264B1 (en) 2018-08-02 2025-07-29 Dyne Therapeutics, Inc. Complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of delivering oligonucleotide to a subject
US12097263B2 (en) 2018-08-02 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
JP7611825B2 (ja) 2018-08-02 2025-01-10 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それらの使用
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
SG11202100928QA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
MX2022004388A (es) * 2019-11-01 2022-05-06 Alnylam Pharmaceuticals Inc Composiciones de agentes de arni de huntingtina (htt) y metodos de uso de las mismas.
CN115038789A (zh) 2019-12-02 2022-09-09 塑造治疗公司 治疗性编辑
BR112022021333A2 (pt) * 2020-05-01 2022-12-13 Ionis Pharmaceuticals Inc Compostos e métodos para modular atxn1
JP2023542889A (ja) 2020-09-17 2023-10-12 キュー－ステート バイオサイエンシーズ， インコーポレイテッド 複数の標的を介して疼痛を処置するための治療用組成物
US20240209377A1 (en) * 2021-04-28 2024-06-27 Q-State Biosciences, Inc. Therapeutic compositions for treating pain via multiple targets
AU2022309028A1 (en) 2021-07-09 2024-01-25 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating dystrophinopathies
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US11969475B2 (en) 2021-07-09 2024-04-30 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
AR127158A1 (es) * 2021-09-29 2023-12-27 Hoffmann La Roche Oligonucléotido antisentido para la edición de arn asociado a enfermedades de poliglutamina
WO2023164656A2 (en) * 2022-02-25 2023-08-31 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating atn1 expression
KR20250004770A (ko) 2022-04-15 2025-01-08 다인 세라퓨틱스, 인크. 근긴장성 이영양증을 치료하기 위한 근육 표적화 복합체 및 제제
WO2025068368A1 (en) * 2023-09-26 2025-04-03 Katholieke Universiteit Leuven Inhibitors for treating cancer
WO2025255388A1 (en) * 2024-06-05 2025-12-11 Camp4 Therapeutics Corporation Modulation of syngap1 gene transcription using antisense oligonucleotides targeting regulatory rnas

Family Cites Families (37)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5695933A (en) 1993-05-28 1997-12-09 Massachusetts Institute Of Technology Direct detection of expanded nucleotide repeats in the human genome
DE69503126T2 (de) 1994-05-05 1998-11-12 Beckman Instruments Inc Repetitive oligonukleotide matrix
US6683173B2 (en) 1998-04-03 2004-01-27 Epoch Biosciences, Inc. Tm leveling methods
US5945290A (en) * 1998-09-18 1999-08-31 Isis Pharmaceuticals, Inc. Antisense modulation of RhoA expression
US6900187B2 (en) * 1999-02-26 2005-05-31 The University Of British Columbia TRPM-2 antisense therapy using an oligonucleotide having 2′-O-(2-methoxy)ethyl modifications
US20050277133A1 (en) 2001-05-18 2005-12-15 Sirna Therapeutics, Inc. RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA)
ES2301659T3 (es) * 2001-07-06 2008-07-01 Topigen Pharmaceuticals Inc. Metodos para aumentar la eficacia in vivo de oligonucleotidos e inhibir la inflamacion en mamiferos.
WO2003013437A2 (en) 2001-08-07 2003-02-20 University Of Delaware Compositions and methods for the prevention and treatment of huntington's disease
US20060074034A1 (en) 2001-09-17 2006-04-06 Collins Douglas A Cobalamin mediated delivery of nucleic acids, analogs and derivatives thereof
US20050255086A1 (en) 2002-08-05 2005-11-17 Davidson Beverly L Nucleic acid silencing of Huntington's Disease gene
US7892793B2 (en) 2002-11-04 2011-02-22 University Of Massachusetts Allele-specific RNA interference
US7655785B1 (en) 2002-11-14 2010-02-02 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory oligonucleotides and uses thereof
US7514551B2 (en) 2003-04-03 2009-04-07 Enzo Life Sciences, Inc. Multisignal labeling reagents, and processes and uses therefor
AU2004239114B2 (en) 2003-05-14 2008-03-13 Japan Science And Technology Agency Inhibition of the expression of huntingtin gene
WO2006083800A2 (en) 2005-01-31 2006-08-10 University Of Iowa Research Foundation Nucleic acid silencing of huntington's disease gene
DK2161038T3 (da) 2006-01-26 2014-03-03 Isis Pharmaceuticals Inc Sammensætninger og deres anvendelser rettet mod Huntington
DK2049664T3 (da) * 2006-08-11 2012-01-02 Prosensa Technologies Bv Enkeltstrengede oligonukleotider, som er komplementære til repetitive elementer, til behandling af med DNA-repetitiv-ustabilitet associerede lidelser
CN101501193B (zh) * 2006-08-11 2013-07-03 普罗森那技术公司 用于治疗与dna重复不稳定性相关的遗传病的方法和手段
CA2693048C (en) 2007-07-12 2016-10-18 Prosensa Technologies B.V. Molecules for targeting compounds to various selected organs or tissues
CA2714120A1 (en) * 2008-02-08 2009-08-13 Prosensa Holding Bv Methods and means for treating dna repeat instability associated genetic disorders
AU2009270020B2 (en) 2008-07-10 2014-09-25 Regenesance B.V. Complement antagonists and uses thereof
US20110218334A1 (en) 2008-07-11 2011-09-08 Alnylam Pharmaceuticals, Inc. PHOSPHOROTHIOATE OLIGONUCLEOTIDES AND NON-NUCLEOSIDIC PHOSPHOROTHIOATES AS DELIVERY AGENTS FOR iRNA AGENTS
EP2317847B1 (en) 2008-07-29 2019-04-17 The Board of Regents of The University of Texas System Selective inhibition of polyglutamine protein expression
EP2370582B1 (en) * 2008-12-04 2017-05-10 CuRNA, Inc. Treatment of tumor suppressor gene related diseases by inhibition of natural antisense transcript to the gene
MA33488B1 (fr) 2009-06-08 2012-08-01 Miragen Therapeutics Motifs de modification chimique pour des inhibiteurs et mimétiques de miarn
WO2011097614A1 (en) 2010-02-08 2011-08-11 Isis Pharmaceuticals, Inc. Mehods and compositions useful in diseases or conditions related to repeat expansion
WO2011097641A1 (en) * 2010-02-08 2011-08-11 Isis Pharmaceuticals, Inc. Methods and compositions useful in treatment of diseases or conditions related to repeat expansion
KR102095478B1 (ko) 2010-05-28 2020-04-01 사렙타 쎄러퓨틱스, 인코퍼레이티드 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체
AU2011282243B2 (en) 2010-07-19 2016-09-22 Ionis Pharmaceuticals, Inc. Modulation of nuclear-retained RNA
NZ606364A (en) 2010-08-20 2014-12-24 Replicor Inc Oligonucleotide chelate complexes
EP3467109A1 (en) 2011-02-08 2019-04-10 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
EP2699269A1 (en) * 2011-04-22 2014-02-26 Prosensa Technologies B.V. New compounds for treating, delaying and/or preventing a human genetic disorder such as myotonic dystrophy type 1 (dm1)
CN103619356B (zh) 2011-05-05 2017-09-12 萨勒普塔医疗公司 肽寡核苷酸缀合物
JP6317675B2 (ja) 2011-11-30 2018-04-25 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
EP4043039A1 (en) * 2012-01-27 2022-08-17 BioMarin Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
EP2812342B1 (en) 2012-02-08 2017-11-15 Ionis Pharmaceuticals, Inc. Modulation of rna by repeat targeting
EP3228712A1 (en) * 2012-04-23 2017-10-11 BioMarin Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders

Also Published As

Publication number Publication date
AU2013253074A1 (en) 2014-10-16
US20250011784A1 (en) 2025-01-09
PL2841578T3 (pl) 2017-12-29
ES2983421T3 (es) 2024-10-23
US20180066256A1 (en) 2018-03-08
JP2020182472A (ja) 2020-11-12
EP3604536A1 (en) 2020-02-05
CA3103101A1 (en) 2013-10-31
PT2841578T (pt) 2017-08-10
US11345915B2 (en) 2022-05-31
US20200149043A1 (en) 2020-05-14
HUE033431T2 (en) 2017-11-28
BR112014026285B1 (pt) 2021-08-31
EP4400169A3 (en) 2024-12-25
JP2018027080A (ja) 2018-02-22
AU2013253074C1 (en) 2018-06-14
RS56319B1 (sr) 2017-12-29
JP6174678B2 (ja) 2017-08-02
EP3604536B1 (en) 2024-05-29
PL3604536T3 (pl) 2024-12-16
EP2841578B1 (en) 2017-06-07
JP2025111478A (ja) 2025-07-30
BR112014026285A2 (pt) 2017-06-27
BR112014026285A8 (pt) 2018-01-16
CA2870697C (en) 2021-11-23
SMT201700339T1 (it) 2017-09-07
CN104271741A (zh) 2015-01-07
CA2870697A1 (en) 2013-10-31
EP3604536C0 (en) 2024-05-29
US20220389421A1 (en) 2022-12-08
DK2841578T3 (en) 2017-07-31
ES2639066T3 (es) 2017-10-25
CN110025628A (zh) 2019-07-19
LT2841578T (lt) 2017-09-25
JP2015514418A (ja) 2015-05-21
CA3103101C (en) 2023-01-17
JP2022126711A (ja) 2022-08-30
US20150148404A1 (en) 2015-05-28
EP2841578A1 (en) 2015-03-04
CN110025628B (zh) 2023-03-31
EP4400169A2 (en) 2024-07-17
JP7459691B2 (ja) 2024-04-02
WO2013162363A1 (en) 2013-10-31
AU2013253074B2 (en) 2018-02-22
HK1207118A1 (en) 2016-01-22
EP3228712A1 (en) 2017-10-11
HRP20171254T1 (hr) 2018-01-12
US9745576B2 (en) 2017-08-29
SI2841578T1 (sl) 2017-12-29

Similar Documents

Publication Publication Date Title
NZ700561A (en) Rna modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders
NZ631245A (en) Exon skipping compositions for treating muscular dystrophy
NZ627896A (en) Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
EP3800255A3 (en) Rna-guided transcriptional regulation and methods of using the same for the treatment of back pain
EP4252845A3 (en) Pre-mrna splice switching or modulating oligonucleotides comprising bicyclic scaffold moieties, with improved characteristics for the treatment of genetic disorders
CY1124197T1 (el) Antinohmatika πολυνουκλεοτιδια για να διεγειρεται παραλειψη εξονιου και μεθοδοι θεραπειας δυστροφιων
MX2013009191A (es) Oligonucleotidos antisentido.
MX2021005357A (es) Polimeros oligonucleotidos que inhiben el transporte de antigeno s y metodos.
NZ733882A (en) Compositions for modulating c9orf72 expression
WO2016106400A3 (en) Rna interference agents for gst-pi gene modulation
NZ631132A (en) Exon skipping compositions for treating muscular dystrophy
MY194058A (en) Cyclic dinucleotides for treating conditions associated with sting activity such as cancer
MX358603B (es) Tratamiento de enfermedades relacionadas con un gen supresor de tumor mediante inhibicion del transcrito antisentido natural para el gen.
WO2011038210A3 (en) Treatment of filaggrin (flg) related diseases by modulation of flg expression and activity
SG10201705285SA (en) Antisense oligonucleotides
WO2010138806A3 (en) Treatment of antiviral gene related diseases by inhibition of natural antisense transcript to an antiviral gene
MX363846B (es) Moduladores de oligonucleotido de linfoma linfocitico cronico/linfoma de celulas b 11a (bcl11a) y sus usos.
WO2011103528A3 (en) Treatment of pyrroline-5-carboxylate reductase 1 (pycr1) related diseases by inhibition of natural antisense transcript to pycr1
PH12014502569A1 (en) Method for treating non-small cell lung cancer
PH12021551025A1 (en) 5-halouracil-modified micrornas and their use in the treatment of cancer
CY1114844T1 (el) Ολιγο-νουκλεοτιδιο (oligonucleotide) και συνθεσεις που το περιλαμβανουν για την θεραπεια νεοπλασματος β κυτταρων
MX359337B (es) Oligonucleotidos antisentido para el tratamiento de celulas madre del cancer.
WO2015069906A3 (en) Modified dna quadruplex-forming oligonucleotides and methods of use
MX2025000726A (es) Composiciones de oligonucleótidos y métodos para éstas
MX2018012545A (es) Reactivos para el tratamiento de distrofia muscular oculofaríngea (opmd) y su uso.

Legal Events

Date Code Title Description
PSEA Patent sealed
RENW Renewal (renewal fees accepted)

Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 23 APR 2018 BY COMPUTER PACKAGES INC

Effective date: 20170331

RENW Renewal (renewal fees accepted)

Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 23 APR 2019 BY COMPUTER PACKAGES INC

Effective date: 20180331

RENW Renewal (renewal fees accepted)

Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 23 APR 2020 BY COMPUTER PACKAGES INC

Effective date: 20190402

RENW Renewal (renewal fees accepted)

Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 23 APR 2021 BY IPAN GMBH

Effective date: 20200406

RENW Renewal (renewal fees accepted)

Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 23 APR 2022 BY IPAN GMBH

Effective date: 20210406

ASS Change of ownership

Owner name: VICO THERAPEUTICS B.V., NL

Effective date: 20220308

RENW Renewal (renewal fees accepted)

Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 23 APR 2023 BY IPAN GMBH

Effective date: 20220406

RENW Renewal (renewal fees accepted)

Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 23 APR 2024 BY IPAN GMBH

Effective date: 20230405

RENW Renewal (renewal fees accepted)

Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 23 APR 2025 BY IPAN GMBH

Effective date: 20240404

RENW Renewal (renewal fees accepted)

Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 23 APR 2026 BY IPAN GMBH

Effective date: 20250404