BR112014026285A8 - oligonucleotídeos moduladores de rna com características melhoradas para o tratamento de distúrbios neuromusculares - Google Patents

oligonucleotídeos moduladores de rna com características melhoradas para o tratamento de distúrbios neuromusculares

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Publication number
BR112014026285A8
BR112014026285A8 BR112014026285A BR112014026285A BR112014026285A8 BR 112014026285 A8 BR112014026285 A8 BR 112014026285A8 BR 112014026285 A BR112014026285 A BR 112014026285A BR 112014026285 A BR112014026285 A BR 112014026285A BR 112014026285 A8 BR112014026285 A8 BR 112014026285A8
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BR
Brazil
Prior art keywords
rna
treatment
improved characteristics
neuromuscular disorders
modulating oligonucleotides
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BR112014026285A
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English (en)
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BR112014026285B1 (pt
BR112014026285A2 (pt
Inventor
Christian De Visser Peter
Allegonda Maria Mulders Susan
Original Assignee
Biomarin Tech Bv
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Publication date
Application filed by Biomarin Tech Bv filed Critical Biomarin Tech Bv
Publication of BR112014026285A2 publication Critical patent/BR112014026285A2/pt
Publication of BR112014026285A8 publication Critical patent/BR112014026285A8/pt
Publication of BR112014026285B1 publication Critical patent/BR112014026285B1/pt

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    • 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
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    • 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
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
BR112014026285-3A 2012-04-23 2013-04-23 Oligonucleotídeos moduladores de rna com características melhoradas para o tratamento de distúrbios neuromusculares BR112014026285B1 (pt)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201261636914P 2012-04-23 2012-04-23
EP12165139 2012-04-23
EP12165139.2 2012-04-23
US61/636,914 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 (3)

Publication Number Publication Date
BR112014026285A2 BR112014026285A2 (pt) 2017-06-27
BR112014026285A8 true BR112014026285A8 (pt) 2018-01-16
BR112014026285B1 BR112014026285B1 (pt) 2021-08-31

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BR112014026285-3A BR112014026285B1 (pt) 2012-04-23 2013-04-23 Oligonucleotídeos moduladores de rna com características melhoradas para o tratamento de distúrbios neuromusculares

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US (4) US9745576B2 (pt)
EP (4) EP2841578B1 (pt)
JP (4) JP6174678B2 (pt)
CN (2) CN104271741A (pt)
AU (1) AU2013253074C1 (pt)
BR (1) BR112014026285B1 (pt)
CA (2) CA3103101C (pt)
DK (1) DK2841578T3 (pt)
ES (2) ES2983421T3 (pt)
HK (1) HK1207118A1 (pt)
HR (1) HRP20171254T1 (pt)
HU (1) HUE033431T2 (pt)
LT (1) LT2841578T (pt)
NZ (1) NZ700561A (pt)
PL (1) PL2841578T3 (pt)
PT (1) PT2841578T (pt)
RS (1) RS56319B1 (pt)
SI (1) SI2841578T1 (pt)
WO (1) WO2013162363A1 (pt)

Families Citing this family (40)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2049664B1 (en) 2006-08-11 2011-09-14 Prosensa Technologies B.V. Single stranded oligonucleotides complementary to repetitive elements for treating DNA repeat instability associated genetic disorders
ES2914775T3 (es) 2007-10-26 2022-06-16 Academisch Ziekenhuis Leiden Medios y métodos para contrarresta trastornos del músculo
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
CA2744987C (en) 2008-12-02 2018-01-16 Chiralgen, Ltd. Method for the synthesis of phosphorus atom modified nucleic acids
CA2767253A1 (en) 2009-07-06 2011-01-13 Ontorii, Inc. Novel nucleic acid prodrugs and methods of use thereof
EP2620428B1 (en) 2010-09-24 2019-05-22 Wave Life Sciences Ltd. Asymmetric auxiliary group
US9476043B2 (en) * 2011-04-08 2016-10-25 Rula Zain-Luqman Diagnosis and treatment of friedreich's ataxia
EP3248982A1 (en) 2011-07-19 2017-11-29 Wave Life Sciences Ltd. Thiosulfonate reagents for the synthesis of functionalized nucleic acids
CA2862628C (en) 2012-01-27 2021-08-24 Prosensa Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
CA3103101C (en) * 2012-04-23 2023-01-17 Biomarin Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders
SG11201500239VA (en) 2012-07-13 2015-03-30 Wave Life Sciences Japan Asymmetric auxiliary group
RU2020127664A (ru) * 2012-10-15 2020-09-17 Ионис Фармасьютикалз, Инк. Композиции для модуляции экспрессии гена c9orf72
FI2906696T4 (fi) 2012-10-15 2023-03-18 Menetelmiä c9orf72:n ilmentymisen moduloimiseksi
US10577604B2 (en) * 2012-10-15 2020-03-03 Ionis Pharmaceuticals, Inc. Methods for monitoring C9ORF72 expression
KR20160062069A (ko) 2013-10-11 2016-06-01 아이오니스 파마수티컬즈, 인코포레이티드 Ｃ９ｏｒｆ７２ 발현을 조절하기 위한 조성물
RU2016133035A (ru) 2014-01-16 2018-02-21 Уэйв Лайф Сайенсес Лтд. Хиральный дизайн
CN116970607A (zh) 2014-03-19 2023-10-31 Ionis制药公司 用于调节共济失调蛋白2表达的组合物
US10006027B2 (en) 2014-03-19 2018-06-26 Ionis Pharmaceuticals, Inc. Methods for modulating Ataxin 2 expression
WO2016060919A1 (en) * 2014-10-14 2016-04-21 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
SG11201708468YA (en) 2015-04-16 2017-11-29 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
RU2022107926A (ru) * 2016-07-05 2022-04-27 Биомарин Текнолоджис Б.В. Олигонуклеотиды, производящие переключение или модулирование сплайсинга пре-рнк и содержащие бициклические каркасные фрагменты, с улучшенными характеристиками для лечения генетических заболеваний
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
AU2018354195A1 (en) * 2017-10-23 2020-06-04 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disease
PE20211398A1 (es) 2018-07-25 2021-07-27 Ionis Pharmaceuticals Inc Compuestos y metodos para reducir la expresion de la atxn2
KR20210081324A (ko) 2018-08-02 2021-07-01 다인 세라퓨틱스, 인크. 근육 표적화 복합체 및 안면견갑상완 근육 이영양증을 치료하기 위한 그의 용도
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
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
US12097263B2 (en) 2018-08-02 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
SG11202100934PA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating dystrophinopathies
AR120341A1 (es) * 2019-11-01 2022-02-09 Alnylam Pharmaceuticals Inc COMPOSICIONES DE AGENTES DE ARNi CONTRA LA HUNTINGTINA (HTT) Y SUS MÉTODOS DE USO
CN115038789A (zh) 2019-12-02 2022-09-09 塑造治疗公司 治疗性编辑
JP2024519204A (ja) * 2021-04-28 2024-05-09 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組成物
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
KR20240035825A (ko) 2021-07-09 2024-03-18 다인 세라퓨틱스, 인크. 디스트로핀병증을 치료하기 위한 근육 표적화 복합체 및 제제
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
EP4408999A1 (en) * 2021-09-29 2024-08-07 F. Hoffmann-La Roche AG Rna editing
WO2023164656A2 (en) * 2022-02-25 2023-08-31 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating atn1 expression
AU2023254846A1 (en) 2022-04-15 2024-10-10 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating myotonic dystrophy

Family Cites Families (35)

* 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
WO1995030774A1 (en) 1994-05-05 1995-11-16 Beckman Instruments, Inc. Oligonucleotide repeat arrays
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.
US20030109476A1 (en) 2001-08-07 2003-06-12 Kmiec Eric B. 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
US7589189B2 (en) 2003-05-14 2009-09-15 Japan Science And Technology Agency Inhibition of the expression of huntingtin gene
AU2006210973A1 (en) 2005-01-31 2006-08-10 University Of Iowa Research Foundation Nucleic acid silencing of Huntington's Disease gene
PL2161038T3 (pl) 2006-01-26 2014-06-30 Ionis Pharmaceuticals Inc Kompozycje i ich zastosowanie ukierunkowane na huntingtynę
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
EP2049664B1 (en) * 2006-08-11 2011-09-14 Prosensa Technologies B.V. Single stranded oligonucleotides complementary to repetitive elements for treating DNA repeat instability associated genetic disorders
AU2008273096B2 (en) 2007-07-12 2013-05-02 Academisch Ziekenhuis Leiden Molecules for targeting compounds to various selected organs or tissues
NZ587178A (en) * 2008-02-08 2011-11-25 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
WO2010006237A2 (en) 2008-07-11 2010-01-14 Alnylam Pharmaceuticals, Inc. Phosphorothioate oligonucleotides non-nucleosidic phosphorothiotes as delivery agents for irna agents
AU2009276763B2 (en) 2008-07-29 2015-07-16 The Board Of Regents Of The University Of Texas Sytem 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
EP2536738A4 (en) 2010-02-08 2014-09-17 Isis Pharmaceuticals Inc METHODS AND COMPOSITIONS SUITABLE FOR THE TREATMENT OF CONDITIONS OR CONDITIONS IN RELATION TO THE EXPANSION OF REPETITIONS
CN107353317A (zh) 2010-05-28 2017-11-17 萨勒普塔医疗公司 具有修饰的亚基间键和/或端基的寡核苷酸类似物
KR20180105730A (ko) 2010-07-19 2018-09-28 아이오니스 파마수티컬즈, 인코포레이티드 근육긴장성 이영양증-단백질 키나제(dmpk) 발현의 조절 방법
CN103052405B (zh) 2010-08-20 2015-11-25 里普利科股份有限公司 寡核苷酸螯合物
WO2012109395A1 (en) 2011-02-08 2012-08-16 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
CN103619356B (zh) 2011-05-05 2017-09-12 萨勒普塔医疗公司 肽寡核苷酸缀合物
ES2832531T3 (es) 2011-11-30 2021-06-10 Sarepta Therapeutics Inc Oligonucleótidos para el tratamiento de enfermedades por expansión de repeticiones
WO2013120003A1 (en) 2012-02-08 2013-08-15 Isis Pharmaceuticals, Inc. Modulation of rna by repeat targeting
CA3103101C (en) * 2012-04-23 2023-01-17 Biomarin Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders

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CN110025628B (zh) 2023-03-31
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US20180066256A1 (en) 2018-03-08
US20220389421A1 (en) 2022-12-08
US20200149043A1 (en) 2020-05-14
EP3604536A1 (en) 2020-02-05
DK2841578T3 (en) 2017-07-31
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AU2013253074B2 (en) 2018-02-22
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