JP2025126919A5 - - Google Patents

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Publication number
JP2025126919A5
JP2025126919A5 JP2025074246A JP2025074246A JP2025126919A5 JP 2025126919 A5 JP2025126919 A5 JP 2025126919A5 JP 2025074246 A JP2025074246 A JP 2025074246A JP 2025074246 A JP2025074246 A JP 2025074246A JP 2025126919 A5 JP2025126919 A5 JP 2025126919A5
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JP
Japan
Prior art keywords
antisense oligonucleotide
oligonucleotides
oligonucleotide analogue
carrier
analogue
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Pending
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JP2025074246A
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English (en)
Japanese (ja)
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JP2025126919A (ja
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Priority claimed from GBGB1711809.2A external-priority patent/GB201711809D0/en
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Publication of JP2025126919A publication Critical patent/JP2025126919A/ja
Publication of JP2025126919A5 publication Critical patent/JP2025126919A5/ja
Pending legal-status Critical Current

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JP2025074246A 2017-07-21 2025-04-28 ヒトジストロフィンプレmRNAのエクソン51に結合するアンチセンスオリゴヌクレオチド Pending JP2025126919A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
GBGB1711809.2A GB201711809D0 (en) 2017-07-21 2017-07-21 Antisense oligonucleotide
GB1711809.2 2017-07-21
JP2020524657A JP7365336B2 (ja) 2017-07-21 2018-07-20 ヒトジストロフィンプレmRNAのエクソン51に結合するアンチセンスオリゴヌクレオチド
PCT/CA2018/050881 WO2019014772A1 (en) 2017-07-21 2018-07-20 ANTISENSE OLIGONUCLEOTIDES THAT BIND EXON 51 OF HUMAN DYSTROPHIN PRE-MRNA
JP2023092396A JP7742205B2 (ja) 2017-07-21 2023-06-05 ヒトジストロフィンプレmRNAのエクソン51に結合するアンチセンスオリゴヌクレオチド

Related Parent Applications (1)

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JP2023092396A Division JP7742205B2 (ja) 2017-07-21 2023-06-05 ヒトジストロフィンプレmRNAのエクソン51に結合するアンチセンスオリゴヌクレオチド

Publications (2)

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JP2025126919A JP2025126919A (ja) 2025-08-29
JP2025126919A5 true JP2025126919A5 (enExample) 2025-10-06

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ID=59771647

Family Applications (3)

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JP2020524657A Active JP7365336B2 (ja) 2017-07-21 2018-07-20 ヒトジストロフィンプレmRNAのエクソン51に結合するアンチセンスオリゴヌクレオチド
JP2023092396A Active JP7742205B2 (ja) 2017-07-21 2023-06-05 ヒトジストロフィンプレmRNAのエクソン51に結合するアンチセンスオリゴヌクレオチド
JP2025074246A Pending JP2025126919A (ja) 2017-07-21 2025-04-28 ヒトジストロフィンプレmRNAのエクソン51に結合するアンチセンスオリゴヌクレオチド

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JP2020524657A Active JP7365336B2 (ja) 2017-07-21 2018-07-20 ヒトジストロフィンプレmRNAのエクソン51に結合するアンチセンスオリゴヌクレオチド
JP2023092396A Active JP7742205B2 (ja) 2017-07-21 2023-06-05 ヒトジストロフィンプレmRNAのエクソン51に結合するアンチセンスオリゴヌクレオチド

Country Status (8)

Country Link
US (3) US11142767B2 (enExample)
EP (1) EP3655535A4 (enExample)
JP (3) JP7365336B2 (enExample)
KR (2) KR20240049639A (enExample)
CN (2) CN117701566A (enExample)
CA (1) CA3105864A1 (enExample)
GB (1) GB201711809D0 (enExample)
WO (1) WO2019014772A1 (enExample)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
MX2019008199A (es) 2017-01-06 2019-11-25 Avidity Biosciences Llc Composiciones de acido nucleico polipeptido y metodos de induccion de la omision de exon.
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
WO2019241385A2 (en) * 2018-06-13 2019-12-19 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystropy
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
KR20210081324A (ko) 2018-08-02 2021-07-01 다인 세라퓨틱스, 인크. 근육 표적화 복합체 및 안면견갑상완 근육 이영양증을 치료하기 위한 그의 용도
SG11202100934PA (en) 2018-08-02 2021-02-25 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
JP7687682B2 (ja) * 2019-04-08 2025-06-03 国立大学法人東京科学大学 筋疾患治療用医薬組成物
US20230121577A1 (en) * 2020-04-15 2023-04-20 The Regents Of The University Of California Compositions and Methods for Treatment
IL301781A (en) * 2020-09-30 2023-05-01 Biomarin Tech Bv Antisense oligonucleotides targeting the exon 51 of dystrophin gene
EP3978608A1 (en) * 2020-10-05 2022-04-06 SQY Therapeutics Oligomeric compound for dystrophin rescue in dmd patients throughout skipping of exon-51
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
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
AU2022309028A1 (en) 2021-07-09 2024-01-25 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating dystrophinopathies
CA3229661A1 (en) 2021-09-01 2023-03-09 Xiang Li Compounds and methods for skipping exon 44 in duchenne muscular dystrophy
WO2023168427A1 (en) 2022-03-03 2023-09-07 Yale University Compositions and methods for delivering therapeutic polynucleotides for exon skipping
US12071621B2 (en) 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping
CN115806984B (zh) * 2022-10-18 2023-10-10 昆明理工大学 环状rna及载体和载体的应用
WO2025072246A1 (en) 2023-09-26 2025-04-03 Entrada Therapeutics, Inc. Compounds and methods for skipping exon 50 in duchenne muscular dystrophy

Family Cites Families (160)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
US4694778A (en) 1984-05-04 1987-09-22 Anicon, Inc. Chemical vapor deposition wafer boat
US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group Polynucleotide assay reagent and method
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
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5521063A (en) 1985-03-15 1996-05-28 Antivirals Inc. Polynucleotide reagent containing chiral subunits and methods of use
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
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US4828971A (en) 1988-03-24 1989-05-09 Eastman Kodak Company Thermally processable element comprising a backing layer
CA1251835A (en) 1988-04-05 1989-03-28 Wai-Cheung Tang Dielectric image-resonator multiplexer
WO1991004753A1 (en) 1989-10-02 1991-04-18 Cetus Corporation Conjugates of antisense oligonucleotides and therapeutic uses thereof
AU649074B2 (en) 1990-10-12 1994-05-12 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Modified ribozymes
DE4216134A1 (de) 1991-06-20 1992-12-24 Europ Lab Molekularbiolog Synthetische katalytische oligonukleotidstrukturen
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
US5736557A (en) 1994-06-17 1998-04-07 Hoffman-La Roche, Inc. N,N-bis (quinolin-4-yl)-diamine derivatives, their preparation and their use as antimalarials
US5716824A (en) 1995-04-20 1998-02-10 Ribozyme Pharmaceuticals, Inc. 2'-O-alkylthioalkyl and 2-C-alkylthioalkyl-containing enzymatic nucleic acids (ribozymes)
US5889136A (en) 1995-06-09 1999-03-30 The Regents Of The University Of Colorado Orthoester protecting groups in RNA synthesis
WO1997026270A2 (en) 1996-01-16 1997-07-24 Ribozyme Pharmaceuticals, Inc. Synthesis of methoxy nucleosides and enzymatic nucleic acid molecules
AU728657B2 (en) 1996-03-18 2001-01-18 Board Of Regents, The University Of Texas System Immunoglobulin-like domains with increased half-lives
US5849902A (en) 1996-09-26 1998-12-15 Oligos Etc. Inc. Three component chimeric antisense oligonucleotides
US6111086A (en) 1998-02-27 2000-08-29 Scaringe; Stephen A. Orthoester protecting groups
US6528624B1 (en) 1998-04-02 2003-03-04 Genentech, Inc. Polypeptide variants
DK1068241T3 (da) 1998-04-02 2008-02-04 Genentech Inc Antistofvarianter og fragmenter deraf
US6194551B1 (en) 1998-04-02 2001-02-27 Genentech, Inc. Polypeptide variants
EP1071753A2 (en) 1998-04-20 2001-01-31 Ribozyme Pharmaceuticals, Inc. Nucleic acid molecules with novel chemical compositions capable of modulating gene expression
GB9816575D0 (en) 1998-07-31 1998-09-30 Zeneca Ltd Novel compounds
CA2328356A1 (en) 1999-12-22 2001-06-22 Itty Atcravi Recreational vehicles
DK1242438T3 (da) 1999-12-29 2007-02-12 Immunogen Inc Cytotoksiske midler omfattende modificerede doxorubiciner og daunorubiciner og deres terapeutiske anvendelse
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
US8202979B2 (en) 2002-02-20 2012-06-19 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid
US8273866B2 (en) 2002-02-20 2012-09-25 Merck Sharp & Dohme Corp. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SINA)
US6784291B2 (en) 2000-05-04 2004-08-31 Avi Biopharma, Inc. Splice-region antisense composition and method
EP1191097A1 (en) * 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
US6884869B2 (en) 2001-04-30 2005-04-26 Seattle Genetics, Inc. Pentapeptide compounds and uses related thereto
US9181551B2 (en) 2002-02-20 2015-11-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
AU2003207708A1 (en) 2002-02-20 2003-09-09 Sirna Therapeutics, Inc. Rna interference mediated inhibition of map kinase genes
US9657294B2 (en) 2002-02-20 2017-05-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
AU2002257333A1 (en) * 2002-02-22 2003-09-09 Linda L. Bambrick Novel treatment of neurodegenerative diseases by altering levels of trkb isoforms and/or trkc isoforms
US20040023220A1 (en) 2002-07-23 2004-02-05 Lawrence Greenfield Integrated method for PCR cleanup and oligonucleotide removal
PT1527176E (pt) 2002-08-05 2007-04-30 Atugen Ag Novas formas de muléculas de arn de interferência
US7956176B2 (en) 2002-09-05 2011-06-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US7250496B2 (en) 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
DK2284266T3 (da) 2002-11-14 2014-01-13 Thermo Fisher Scient Biosciences Inc sIRNA-MOLEKYLE MOD TP53
CA2796924C (en) * 2002-11-25 2016-12-13 Nonprofit Organization Translational Research Organization Of Duchenne Muscular Dystrophy Ena nucleic acid pharmaceuticals capable of modifying splicing of mrna precursors
US8084582B2 (en) 2003-03-03 2011-12-27 Xencor, Inc. Optimized anti-CD20 monoclonal antibodies having Fc variants
AU2003225410A1 (en) 2003-03-21 2004-10-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
CN104998273A (zh) 2003-11-06 2015-10-28 西雅图基因公司 能够与配体偶联的单甲基缬氨酸化合物
US7494925B2 (en) 2004-02-23 2009-02-24 Micron Technology, Inc. Method for making through-hole conductors for semiconductor substrates
EP1766010B1 (en) 2004-06-28 2011-02-16 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
FR2874384B1 (fr) * 2004-08-17 2010-07-30 Genethon Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables
CA2586909A1 (en) 2004-11-12 2006-12-14 Seattle Genetics, Inc. Auristatins having an aminobenzoic acid unit at the n terminus
US7429482B2 (en) 2005-01-13 2008-09-30 United States Of America As Represented By The Department Of Veterans Affairs Screening tools for discovery of novel anabolic agents
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.
US7736647B2 (en) 2005-06-15 2010-06-15 Monoclonal Antibodies Therapeutics Anti-CD71 monoclonal antibodies and uses thereof for treating malignant tumor cells
WO2007028065A2 (en) 2005-08-30 2007-03-08 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds for modulation of splicing
KR100699874B1 (ko) 2005-11-08 2007-03-28 삼성전자주식회사 삽입형 연결부를 갖는 비. 지. 에이 패키지 그 제조방법 및이를 포함하는 보드 구조
HUE035799T2 (en) 2006-05-10 2018-05-28 Sarepta Therapeutics Inc Cationic oligonucleotide analogues containing subunits
EP1857548A1 (en) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
PT2049664E (pt) 2006-08-11 2012-01-03 Prosensa Technologies Bv Oligonucleotídeos de cadeia simples complementares dos elementos repetitivos para tratar perturbações genéticas associadas à instabilidade das repetições do adn
US9063129B2 (en) 2007-06-15 2015-06-23 Idexx Laboratories, Inc. Device, kit and method for hookworm antigen capture and detection
WO2009005793A2 (en) * 2007-06-29 2009-01-08 Avi Biopharma, Inc. Tissue specific peptide conjugates and methods
DK2185816T3 (en) 2007-08-29 2018-03-12 Mhi Vestas Offshore Wind As MONOPILE FOUNDATION FOR OFFSHORE WINDMILL
PL2203173T3 (pl) 2007-10-26 2016-06-30 Academisch Ziekenhuis Leiden Środki i sposoby przeciwdziałania zaburzeniom mięśni
WO2009099942A2 (en) 2008-01-31 2009-08-13 Alnylam Pharmaceuticals, Inc. Chemically modified oligonucleotides and uses thereof
US20110081362A1 (en) 2008-01-31 2011-04-07 The Brigham And Women's Hospital, Inc. Treatment of cancer
EP2842575B1 (en) 2008-03-18 2017-09-27 Seattle Genetics, Inc. Auristatin drug linker conjugates
EP3604533A1 (en) 2008-04-11 2020-02-05 Arbutus Biopharma Corporation Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
US8248352B2 (en) 2008-04-25 2012-08-21 Lg Display Co., Ltd. Driving circuit of liquid crystal display
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
US20110130346A1 (en) 2008-05-30 2011-06-02 Isis Innovation Limited Peptide conjugates for delvery of biologically active compounds
US8575305B2 (en) 2008-06-04 2013-11-05 Medical Research Council Cell penetrating peptides
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
AU2013204438B2 (en) 2008-10-24 2016-12-22 Sarepta Therapeutics, Inc. Multiple exon skipping compositions for DMD
KR20180118828A (ko) * 2008-10-24 2018-10-31 사렙타 쎄러퓨틱스 인코퍼레이티드 Dmd를 위한 다중 엑손 스키핑 조성물
DK2607484T3 (en) 2008-10-27 2016-03-07 Biomarin Technologies B V Methods and means for efficient skipping of exon 45 in Duchenne muscular dystrophy pre-MRNA
KR101602431B1 (ko) 2008-11-05 2016-03-10 삼성전자 주식회사 냉장고
CA2744987C (en) 2008-12-02 2018-01-16 Chiralgen, Ltd. Method for the synthesis of phosphorus atom modified nucleic acids
US20130115603A9 (en) 2009-04-02 2013-05-09 Regents Of The University Of Minnesota Nucleotide repeat expansion-associated polypeptides and uses thereof
CN102625840A (zh) * 2009-04-10 2012-08-01 肌肉学研究协会 用于治疗疾病的三环dna反义寡核苷酸、组合物和方法
WO2010123369A1 (en) 2009-04-24 2010-10-28 Prosensa Technologies B.V. Oligonucleotide comprising an inosine for treating dmd
WO2010129666A1 (en) 2009-05-05 2010-11-11 Altermune Technologies, Llc Chemically programmable immunity
US20110269665A1 (en) 2009-06-26 2011-11-03 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
WO2011024077A2 (en) 2009-08-31 2011-03-03 INSERM (Institut National de la Santé et de la Recherche Médicale) Exon skipping therapy for functional amelioration of semi functional dystrophin in becker and duchenne muscular dystrophy
TR201804897T4 (tr) 2009-10-07 2018-06-21 Macrogenics Inc Fukosi̇lasyon ölçüsünün deği̇şi̇mleri̇nden dolayi geli̇şmi̇ş efektör i̇şlevi̇ sergi̇leyen fc bölgesi̇ni̇ i̇çeren poli̇pepti̇tler ve bunlarin kullanimlarina yöneli̇k yöntemler
EP3431603A1 (en) * 2009-11-12 2019-01-23 The University Of Western Australia Antisense molecules and methods for treating pathologies
WO2011078797A2 (en) * 2009-12-22 2011-06-30 Singapore Health Services Pte. Ltd Antisense oligonucleotides and uses threreof
US8697688B2 (en) 2010-04-15 2014-04-15 Seattle Genetics Inc. Pyrrolobenzodiazepines used to treat proliferative diseases
MX2012011900A (es) 2010-04-15 2013-03-21 Seattle Genetics Inc Conjugados de pirrolobenzodiazepina diana.
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
IT1400425B1 (it) 2010-06-08 2013-05-31 Amsterdam Molecular Therapeutics Bv Modified snrnas for use in therapy.
US8936910B2 (en) 2010-06-11 2015-01-20 Antisense Pharma Gmbh Method for selective oligonucleotide modification
CN103167883B (zh) 2010-07-19 2016-08-03 F·C·贝内特 肌强直性营养障碍蛋白激酶(dmpk)表达的调节
ES2663009T3 (es) 2010-10-29 2018-04-10 Sirna Therapeutics, Inc. Inhibición de la expresión génica mediada por interferencia por ARN utilizando ácidos nucleicos de interferencia cortos (ANic)
US8501930B2 (en) * 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
EP2672977A1 (en) 2011-02-08 2013-12-18 The Charlotte-Mecklenburg Hospital Authority d/b/a Carolina Healthcare System Antisense oligonucleotides
SI2691417T2 (sl) 2011-03-29 2025-05-30 Roche Glycart Ag FC variante protitelesa
US20140128449A1 (en) 2011-04-07 2014-05-08 The Board Of Regents Of The University Of Texas System Oligonucleotide modulation of splicing
US9161948B2 (en) 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
EP2708560A4 (en) 2011-05-09 2015-01-14 Perseus Proteomics Inc ANTIBODIES FOR THE SPECIFIC DETECTION OF TRANSFERRIN RECEPTORS
US10100305B2 (en) 2011-07-15 2018-10-16 Sarepta Therapeutics, Inc. Methods and compositions for manipulating translation of protein isoforms from alternative initiation of start sites
WO2013012758A1 (en) 2011-07-19 2013-01-24 Ontorii, Inc. Methods for the synthesis of functionalized nucleic acids
US9418216B2 (en) 2011-07-21 2016-08-16 Microsoft Technology Licensing, Llc Cloud service authentication
ES2876939T3 (es) 2011-08-30 2021-11-15 Res & Innovation Uk Péptidos penetradores de células que tienen un dominio hidrófobo central
CA2850373C (en) 2011-10-14 2019-07-16 Seattle Genetics, Inc. Pyrrolobenzodiazepines and targeted conjugates
KR102853694B1 (ko) 2011-11-18 2025-09-03 알닐람 파마슈티칼스 인코포레이티드 변형된 RNAi 제제
JP2015509922A (ja) * 2012-01-27 2015-04-02 プロセンサ テクノロジーズ ビー.ブイ.Prosensa Technologies B.V. デュシェンヌ型及びベッカー型筋ジストロフィーの治療のための改善された特徴を有するrna調節オリゴヌクレオチド
EP2814512A1 (en) 2012-02-16 2014-12-24 UCL Business Plc. Lysosome-cleavable linker
AR090905A1 (es) 2012-05-02 2014-12-17 Merck Sharp & Dohme Conjugados que contienen tetragalnac y peptidos y procedimientos para la administracion de oligonucleotidos, composicion farmaceutica
WO2013173391A1 (en) 2012-05-15 2013-11-21 Concortis Biosystems, Corp Drug-conjugates, conjugation methods, and uses thereof
US9504756B2 (en) 2012-05-15 2016-11-29 Seattle Genetics, Inc. Self-stabilizing linker conjugates
CN110257379B (zh) * 2012-07-03 2023-08-11 马林生物科技有限公司 用于治疗肌肉萎缩症患者的寡核苷酸
KR102712879B1 (ko) 2012-07-13 2024-10-04 웨이브 라이프 사이언시스 리미티드 키랄 제어
DK2839860T3 (da) 2012-10-12 2019-06-17 Medimmune Ltd Pyrrolobenzodiazepiner og konjugater deraf
JP6133431B2 (ja) 2012-11-24 2017-05-24 ハンジョウ ディーエーシー バイオテック シーオー.,エルティディ.Hangzhou Dac Biotech Co.,Ltd. 親水性連結体及び薬物分子と細胞結合分子との共役反応における親水性連結体の使用
EA201591178A1 (ru) 2012-12-20 2015-11-30 Сарепта Терапьютикс, Инк. Улучшенные композиции для пропускания экзона для лечения мышечной дистрофии
MX379355B (es) 2012-12-21 2025-03-11 Altrubio Inc Enlazadores autodestructivos hidrofilos y conjugados de los mismos.
US9481905B2 (en) 2013-02-19 2016-11-01 Orizhan Bioscience Limited Method of using neutrilized DNA (N-DNA) as surface probe for high throughput detection platform
US9319764B2 (en) 2013-03-08 2016-04-19 Merry Electronics Co., Ltd. MEMS microphone packaging structure
KR20230116945A (ko) 2013-03-14 2023-08-04 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
CA2902872A1 (en) 2013-03-15 2014-09-18 Regeneron Pharmaceuticals, Inc. Biologically active molecules, conjugates thereof, and therapeutic uses
BR112015022998A2 (pt) 2013-03-15 2017-11-14 Sarepta Therapeutics Inc composições melhoradas para o tratamento de distrofia muscular
EP2777714A1 (en) 2013-03-15 2014-09-17 NBE-Therapeutics LLC Method of producing an immunoligand/payload conjugate by means of a sequence-specific transpeptidase enzyme
US9577459B2 (en) 2013-03-15 2017-02-21 Sequana Medical Ag Systems and methods for regulating inductive energy transfer to an implantable system
US9295731B2 (en) 2013-04-01 2016-03-29 Mark Quang Nguyen Cleavable drug conjugates, compositions thereof and methods of use
US9849066B2 (en) 2013-04-24 2017-12-26 Corning Incorporated Delamination resistant pharmaceutical glass containers containing active pharmaceutical ingredients
CN105722851A (zh) 2013-04-28 2016-06-29 秦刚 一种新型的连接子及其制备方法和用途
IL242088B2 (en) 2013-05-20 2023-12-01 Genentech Inc Anti-transferrin receptor antibodies and methods of use
BR112015029788B1 (pt) 2013-05-31 2024-01-02 Zymeworks Inc HETERO-MULTÍMERO, USO E MÉTODO PARA PREPARAR DO MESMO, COMPOSIÇÃO FARMACÊUTICA E MÉTODO PARA REDUZIR FUNÇÃO EFETORA DE UM CONSTRUTO IgG FC
US10704060B2 (en) * 2013-06-05 2020-07-07 Duke University RNA-guided gene editing and gene regulation
US11229711B2 (en) 2013-06-06 2022-01-25 Magenta Therapeutics, Inc. Linkers for antibody-drug conjugates and related compounds, compositions, and methods of use
TW201536329A (zh) 2013-08-09 2015-10-01 Isis Pharmaceuticals Inc 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法
WO2015038426A1 (en) 2013-09-13 2015-03-19 Asana Biosciences, Llc Self-immolative linkers containing mandelic acid derivatives, drug-ligand conjugates for targeted therapies and uses thereof
US9982260B2 (en) 2013-09-30 2018-05-29 The Regents Of The University Of California Identification of structurally similar small molecules that enhance therapeutic exon skipping
AU2014337555C1 (en) 2013-10-15 2021-01-28 Seagen Inc. PEGylated drug-linkers for improved Ligand-Drug Conjugate pharmacokinetics
CN106132431A (zh) 2013-10-15 2016-11-16 索伦托治疗有限公司 具有靶向分子和两种不同药物的药物偶联物
US10239957B2 (en) 2013-11-06 2019-03-26 Merck Sharp & Dohme Corp. Peptide containing conjugates for dual molecular delivery of oligonucleotides
WO2015075747A1 (en) 2013-11-19 2015-05-28 Council Of Scientific And Industrial Research Cell-penetrating peptide for biomolecule delivery
EP4137572A1 (en) 2014-01-16 2023-02-22 Wave Life Sciences Ltd. Chiral design
LT3118311T (lt) 2014-03-12 2019-04-10 Nippon Shinyaku Co., Ltd. Priešprasminė nukleorūgštis
MY194170A (en) 2014-06-17 2022-11-16 Nippon Shinyaku Co Ltd Antisense nucleic acids
US20180305689A1 (en) 2015-04-22 2018-10-25 Mina Therapeutics Limited Sarna compositions and methods of use
EP3297649B1 (en) 2015-05-19 2023-10-11 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
UA123053C2 (uk) 2015-06-24 2021-02-10 Ф. Хоффманн-Ля Рош Аг Антитіло до рецептора трансферину зі спеціально підібраною афінністю
EP3653216A1 (en) * 2015-09-30 2020-05-20 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy
GB2545898B (en) 2015-12-21 2019-10-09 Sutura Therapeutics Ltd Improved drug delivery by conjugating oligonucleotides to stitched/stapled peptides
EP3423491A1 (en) 2016-03-01 2019-01-09 H. Hoffnabb-La Roche Ag Obinutuzumab variants having altered cell death induction
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
MA45290A (fr) 2016-05-04 2019-03-13 Wave Life Sciences Ltd Procédés et compositions d'agents biologiquement actifs
US20190240346A1 (en) 2016-06-20 2019-08-08 Genahead Bio, Inc. Antibody-drug conjugate
EP3478829A1 (en) 2016-06-29 2019-05-08 Crispr Therapeutics AG Materials and methods for treatment of myotonic dystrophy type 1 (dm1) and other related disorders
MX2019006882A (es) * 2016-12-19 2019-08-16 Sarepta Therapeutics Inc Conjugados de oligomeros de omision de exon para distrofia muscular.
MX2019008199A (es) 2017-01-06 2019-11-25 Avidity Biosciences Llc Composiciones de acido nucleico polipeptido y metodos de induccion de la omision de exon.
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
CN111770757A (zh) 2017-09-22 2020-10-13 艾维迪提生物科学公司 诱导外显子跳读的核酸-多肽组合物和方法
WO2019113393A1 (en) 2017-12-06 2019-06-13 Avidity Biosciences Llc Compositions and methods of treating muscle atrophy and myotonic dystrophy
IL319265A (en) 2018-12-21 2025-04-01 Avidity Biosciences Inc Anti-transferrin receptor antibodies and their uses
AU2020263487A1 (en) 2019-04-25 2021-12-16 Avidity Biosciences, Inc. Nucleic acid compositions and methods of multi-exon skipping
US20220288218A1 (en) * 2019-07-09 2022-09-15 The Governors Of The University Of Alberta Exons 45-55 skipping using mutation-tailored cocktails of antisense morpholinos in the dmd gene

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