CN115666589A - 治疗面肩肱型肌营养不良的组合物和方法 - Google Patents

治疗面肩肱型肌营养不良的组合物和方法 Download PDF

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CN115666589A
CN115666589A CN202180036457.6A CN202180036457A CN115666589A CN 115666589 A CN115666589 A CN 115666589A CN 202180036457 A CN202180036457 A CN 202180036457A CN 115666589 A CN115666589 A CN 115666589A
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polynucleic acid
acid molecule
cases
instances
sequence
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Chinese (zh)
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巴尔博拉·马勒科娃
罗伯·伯克
比亚特丽丝·戴安娜·达里蒙特
大卫·萨拉卡诺
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Avidity Biosciences Inc
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Avidity Biosciences Inc
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CN202180036457.6A 2020-03-19 2021-03-12 治疗面肩肱型肌营养不良的组合物和方法 Pending CN115666589A (zh)

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US202062992071P 2020-03-19 2020-03-19
US62/992,071 2020-03-19
US202063066655P 2020-08-17 2020-08-17
US63/066,655 2020-08-17
PCT/US2021/022214 WO2021188390A1 (en) 2020-03-19 2021-03-12 Compositions and methods of treating facioscapulohumeral muscular dystrophy

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JP2019513371A (ja) 2016-04-01 2019-05-30 アビディティー バイオサイエンシーズ エルエルシー 核酸ポリペプチド組成物とその使用
SG11202100928QA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular 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
WO2021188390A1 (en) 2020-03-19 2021-09-23 Avidity Biosciences, Inc. Compositions and methods of treating facioscapulohumeral muscular dystrophy
KR20230023612A (ko) 2020-04-02 2023-02-17 마이레큘, 인크. 조작된 올리고뉴클레오티드를 사용한 표적화된 억제
CA3189065A1 (en) 2020-09-11 2022-03-17 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of dux4, compositions thereof, and methods of use
CN118265544A (zh) * 2021-09-16 2024-06-28 艾维迪提生物科学公司 治疗面肩肱型肌营养不良的组合物和方法
AU2022422149A1 (en) * 2021-12-23 2024-08-01 Mirecule, Inc. Compositions for delivery of polynucleotides
EP4735602A2 (en) 2023-06-27 2026-05-06 Atrium Therapeutics, Inc. Compositions and methods of using prkag2-targeting antibody-oligonucleotide conjugates abstract
AU2024308906A1 (en) 2023-06-30 2026-02-05 Atrium Therapeutics, Inc. Compositions and methods of using pln-targeting antibody-oligonucleotide conjugates
AU2024299328A1 (en) 2023-07-21 2026-01-22 Marrow Therapeutics, Inc. Hematopoietic cell targeting conjugates and related methods

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018129384A1 (en) * 2017-01-06 2018-07-12 Avidity Biosciences Llc Nucleic acid-polypeptide compositions and methods of inducing exon skipping
US20190300903A1 (en) * 2011-07-25 2019-10-03 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of dux4
WO2020028864A1 (en) * 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
CN118265544A (zh) * 2021-09-16 2024-06-28 艾维迪提生物科学公司 治疗面肩肱型肌营养不良的组合物和方法

Family Cites Families (249)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPS5892644A (ja) 1981-11-26 1983-06-02 Taiho Yakuhin Kogyo Kk 新規なニトロ安息香酸アミド誘導体およびそれを含有する放射線増感剤
US4694778A (en) 1984-05-04 1987-09-22 Anicon, Inc. Chemical vapor deposition wafer boat
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
WO1986005519A1 (en) 1985-03-15 1986-09-25 James Summerton Polynucleotide assay reagent and method
US4866132A (en) 1986-04-17 1989-09-12 The Research Foundation Of State University Of New York Novel radiopaque barium polymer complexes, compositions of matter and articles prepared therefrom
US5616584A (en) 1986-09-25 1997-04-01 Sri International 1,2,4-benzotriazine oxides as radiosensitizers and selective cytotoxic agents
US4921963A (en) 1987-04-13 1990-05-01 British Columbia Cancer Foundation Platinum complexes with one radiosensitizing ligand
JPH0819111B2 (ja) 1987-10-22 1996-02-28 ポーラ化成工業株式会社 ２―ニトロイミダゾール誘導体及びこれを有効成分とする放射線増感剤
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
US5256334A (en) 1988-09-08 1993-10-26 The Research Foundation Of The State University Of New York Homogeneous radiopaque polymer-organobismuth composites
WO1991004753A1 (en) 1989-10-02 1991-04-18 Cetus Corporation Conjugates of antisense oligonucleotides and therapeutic uses thereof
US5208020A (en) 1989-10-25 1993-05-04 Immunogen Inc. Cytotoxic agents comprising maytansinoids and their therapeutic use
ES2061416T3 (es) 1990-10-12 1997-03-01 Max Planck Gesellschaft Ribozimas modificadas.
DE4216134A1 (de) 1991-06-20 1992-12-24 Europ Lab Molekularbiolog Synthetische katalytische oligonukleotidstrukturen
JPH07509444A (ja) 1991-11-26 1995-10-19 アルカーメス・インコーポレーテツド トランスフェリンセレプター特異的抗体−神経薬又は診断薬複合体の製造法
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
FI950730L (fi) 1992-08-17 1995-04-10 Quadra Logic Tech Inc Menetelmä ei-toivottujen solujen tai kudosten kasvun tuhoamiseksi tai ehkäisemiseksi
US5346981A (en) 1993-01-13 1994-09-13 Miles Inc. Radiopaque polyurethanes
KR100361933B1 (ko) 1993-09-08 2003-02-14 라 졸라 파마슈티칼 컴파니 화학적으로정의된비중합성결합가플랫폼분자및그것의콘주게이트
US5599796A (en) 1993-12-02 1997-02-04 Emory University Treatment of urogenital cancer with boron neutron capture therapy
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
PT765313E (pt) 1994-06-17 2003-11-28 Hoffmann La Roche Derivados de n,n'-bis(quinolin-4-il)-diamina a sua preparacao e a sua utilizacao como agentes anti-malaria
US5716824A (en) 1995-04-20 1998-02-10 Ribozyme Pharmaceuticals, Inc. 2'-O-alkylthioalkyl and 2-C-alkylthioalkyl-containing enzymatic nucleic acids (ribozymes)
WO1996019487A1 (en) 1994-12-22 1996-06-27 Nissan Chemical Industries, Ltd. Organobismuth derivatives and process for producing the same
US5700825A (en) 1995-03-31 1997-12-23 Florida State University Radiosensitizing diamines and their pharmaceutical preparations
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
JP4046354B2 (ja) 1996-03-18 2008-02-13 ボード オブ リージェンツ，ザ ユニバーシティ オブ テキサス システム 増大した半減期を有する免疫グロブリン様ドメイン
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US9096636B2 (en) 1996-06-06 2015-08-04 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds and their use in gene modulation
US5849902A (en) 1996-09-26 1998-12-15 Oligos Etc. Inc. Three component chimeric antisense oligonucleotides
EP0963443B1 (en) 1996-12-10 2006-03-08 Sequenom, Inc. Releasable nonvolatile mass-label molecules
US20030073207A1 (en) 1997-01-31 2003-04-17 Saghir Akhtar Enzymatic nucleic acid treatment of diseases or conditions related to levels of epidermal growth factor receptors
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
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
ATE375365T1 (de) 1998-04-02 2007-10-15 Genentech Inc Antikörper varianten und fragmente davon
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
GB9818730D0 (en) 1998-08-27 1998-10-21 Univ Portsmouth Collections of compounds
DE69925133T2 (de) 1998-08-27 2006-01-19 Spirogen Ltd., Ryde Pyrrolobenzodiazepine
GB9818731D0 (en) 1998-08-27 1998-10-21 Univ Portsmouth Compounds
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
US6849272B1 (en) 1999-04-21 2005-02-01 Massachusetts Institute Of Technology Endosomolytic agents and cell delivery systems
DE10160151A1 (de) 2001-01-09 2003-06-26 Ribopharma Ag Verfahren zur Hemmung der Expression eines vorgegebenen Zielgens
DE10100586C1 (de) 2001-01-09 2002-04-11 Ribopharma Ag Verfahren zur Hemmung der Expression eines Ziegens
US7829693B2 (en) 1999-11-24 2010-11-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a target gene
CA2328356A1 (en) 1999-12-22 2001-06-22 Itty Atcravi Recreational vehicles
AU767394C (en) 1999-12-29 2005-04-21 Immunogen, Inc. Cytotoxic agents comprising modified doxorubicins and daunorubicins and their therapeutic use
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
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
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)
ES2336887T5 (es) 2000-03-30 2019-03-06 Whitehead Inst Biomedical Res Mediadores de interferencia por ARN específicos de secuencias de ARN
EP2796553B1 (en) 2000-03-30 2019-06-19 Whitehead Institute for Biomedical Research RNA sequence-specific mediators of RNA interference
US6784291B2 (en) 2000-05-04 2004-08-31 Avi Biopharma, Inc. Splice-region antisense composition and method
CA2429814C (en) 2000-12-01 2014-02-18 Thomas Tuschl Rna interference mediating small rna molecules
US8546143B2 (en) 2001-01-09 2013-10-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a target gene
US6884869B2 (en) 2001-04-30 2005-04-26 Seattle Genetics, Inc. Pentapeptide compounds and uses related thereto
DE10133858A1 (de) 2001-07-12 2003-02-06 Aventis Pharma Gmbh Synthetische doppelsträngige Oligonucleotide zur gezielten Hemmung der Genexpression
MXPA05000511A (es) 2001-07-12 2005-09-30 Jefferson Foote Anticuepros super humanizados.
US6942972B2 (en) 2001-10-24 2005-09-13 Beckman Coulter, Inc. Efficient synthesis of protein-oligonucleotide conjugates
US6716821B2 (en) 2001-12-21 2004-04-06 Immunogen Inc. Cytotoxic agents bearing a reactive polyethylene glycol moiety, cytotoxic conjugates comprising polyethylene glycol linking groups, and methods of making and using the same
US20060009409A1 (en) 2002-02-01 2006-01-12 Woolf Tod M Double-stranded oligonucleotides
EP1432724A4 (en) 2002-02-20 2006-02-01 Sirna Therapeutics Inc RNA inhibition mediated inhibition of MAP KINASE GENES
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)
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)
US20040023220A1 (en) 2002-07-23 2004-02-05 Lawrence Greenfield Integrated method for PCR cleanup and oligonucleotide removal
DK1545613T3 (da) 2002-07-31 2011-11-14 Seattle Genetics Inc Auristatinkonjugater og deres anvendelse til behandling af cancer, en autoimmun sygdom eller en infektiøs sygdom
DE60310944T3 (de) 2002-08-05 2017-08-03 Silence Therapeutics Gmbh Weitere neue formen von interferierende rns moleküle
US7923547B2 (en) 2002-09-05 2011-04-12 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
AU2003295600A1 (en) 2002-11-14 2004-06-15 Dharmacon, Inc. Functional and hyperfunctional sirna
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
US8084582B2 (en) 2003-03-03 2011-12-27 Xencor, Inc. Optimized anti-CD20 monoclonal antibodies having Fc variants
JP4605799B2 (ja) 2003-04-02 2011-01-05 ダーマコン， インコーポレイテッド Ｒｎａ干渉において使用するための修飾ポリヌクレオチド
US20040224893A1 (en) 2003-05-06 2004-11-11 Li-Hsien Wang Methods of using IL-1 antagonists to treat neointimal hyperplasia
US7276497B2 (en) 2003-05-20 2007-10-02 Immunogen Inc. Cytotoxic agents comprising new maytansinoids
PT1633767T (pt) 2003-06-02 2019-02-27 Univ Massachusetts Métodos e composições para controlar a eficácia do silenciamento de arn
EP1633890B2 (en) 2003-06-02 2020-11-18 University of Massachusetts METHODS AND COMPOSITIONS FOR ENHANCING THE EFFICACY AND SPECIFICITY OF FNAi
US7750144B2 (en) 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
AU2004263830B2 (en) 2003-06-13 2008-12-18 Alnylam Pharmaceuticals, Inc. Double-stranded ribonucleic acid with increased effectiveness in an organism
GB0416511D0 (en) 2003-10-22 2004-08-25 Spirogen Ltd Pyrrolobenzodiazepines
US8133733B2 (en) 2003-10-24 2012-03-13 Gencia Corporation Nonviral vectors for delivering polynucleotides to target tissues
EP2478912B1 (en) 2003-11-06 2016-08-31 Seattle Genetics, Inc. Auristatin conjugates with anti-HER2 or anti-CD22 antibodies and their use in therapy
US8389710B2 (en) 2004-02-27 2013-03-05 Operational Technologies Corporation Therapeutic nucleic acid-3′-conjugates
WO2005085260A1 (en) 2004-03-09 2005-09-15 Spirogen Limited Pyrrolobenzodiazepines
CA2559955C (en) 2004-03-15 2016-02-16 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded rna
KR101147147B1 (ko) 2004-04-01 2012-05-25 머크 샤프 앤드 돔 코포레이션 Ｒｎａ 간섭의 오프 타겟 효과 감소를 위한 변형된폴리뉴클레오타이드
AU2005247509C1 (en) 2004-05-27 2012-09-20 Alnylam Pharmaceuticals, Inc. Nuclease resistant double-stranded ribonucleic acid
EP2933332A1 (en) 2004-06-28 2015-10-21 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
US8288352B2 (en) 2004-11-12 2012-10-16 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
US7612062B2 (en) 2005-04-21 2009-11-03 Spirogen Limited Pyrrolobenzodiazepines
WO2006128138A2 (en) 2005-05-26 2006-11-30 Ensemble Discovery Corportion Biodetection by nucleic acid-templated chemistry
WO2007008848A2 (en) 2005-07-07 2007-01-18 Seattle Genetics, Inc. Monomethylvaline compounds having phenylalanine carboxy modifications at the c-terminus
CA2619533C (en) 2005-08-17 2014-02-04 Bioneer Corporation Sirna-hydrophilic polymer conjugates for intracellular delivery of sirna and method thereof
US8501703B2 (en) 2005-08-30 2013-08-06 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds for modulation of splicing
RS52060B (sr) 2006-01-25 2012-04-30 Sanofi Citotoksični agensi koji sadrže nove derivate tomaimicina
US7750116B1 (en) 2006-02-18 2010-07-06 Seattle Genetics, Inc. Antibody drug conjugate metabolites
PL2735568T3 (pl) 2006-05-10 2018-02-28 Sarepta Therapeutics, Inc. Analogi oligonukleotydowe mające kationowe połączenia pomiędzy podjednostkami
EP1857548A1 (en) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
US8598333B2 (en) 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
US7521232B2 (en) 2006-05-31 2009-04-21 Icx Nomadics, Inc. Emissive species for clinical imaging
CN101563105B (zh) 2006-07-10 2013-01-23 拜奥根Idec马萨诸塞公司 用于抑制smad4-缺陷癌症的组合物和方法
SI2049664T1 (sl) 2006-08-11 2012-04-30 Prosensa Technologies Bv Enojna veriga oligonukleotidov, komplementarna repetitivnim elementom, za zdravljenje genetskih bolezni, povezanih z nestabilnostjo dnk ponovitev
CN101500548A (zh) 2006-08-18 2009-08-05 弗·哈夫曼-拉罗切有限公司 用于体内递送多核苷酸的多缀合物
WO2008138561A1 (en) 2007-05-10 2008-11-20 R & D Biopharmaceuticals Gmbh Tubulysine derivatives
SI2019104T1 (sl) 2007-07-19 2013-12-31 Sanofi Citotoksična sredstva, ki obsegajo nove tomaimicinske derivate, in njihova terapevtska uporaba
EP3889261A1 (en) 2007-08-27 2021-10-06 1Globe Health Institute LLC Compositions of asymmetric interfering rna and uses thereof
JP2010538678A (ja) 2007-09-18 2010-12-16 イントラドイグム コーポレーション Ｋ−ｒａｓｓｉＲＮＡ含有組成物及びそれらの使用法
EP2195428B1 (en) 2007-09-19 2013-12-11 Applied Biosystems, LLC SiRNA SEQUENCE-INDEPENDENT MODIFICATION FORMATS FOR REDUCING OFF-TARGET PHENOTYPIC EFFECTS IN RNAi, AND STABILIZED FORMS THEREOF
ES2639852T3 (es) 2007-10-26 2017-10-30 Academisch Ziekenhuis Leiden Medios y métodos para contrarrestar los trastornos musculares
WO2009099991A2 (en) 2008-01-31 2009-08-13 The Brigham And Women's Hospital, Inc. Treatment of cancer
WO2009099942A2 (en) 2008-01-31 2009-08-13 Alnylam Pharmaceuticals, Inc. Chemically modified oligonucleotides and uses thereof
AU2009209565B2 (en) 2008-02-01 2013-09-19 Ascendis Pharma As Prodrug comprising a self-cleavable linker
CA2715289C (en) 2008-02-11 2019-12-24 Rxi Pharmaceuticals Corporation Modified rnai polynucleotides and uses thereof
DK2265283T3 (da) 2008-03-18 2014-10-20 Seattle Genetics Inc Auristatin-lægemiddel-linker-konjugater
CA2721183C (en) 2008-04-11 2019-07-16 Alnylam Pharmaceuticals, Inc. Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
JP5667043B2 (ja) 2008-04-15 2015-02-12 アメリカ合衆国ＧＯＶＥＲＮＭＥＮＴ ＯＦ ＴＨＥ ＵＮＩＴＥＤ ＳＴＡＴＥＳ ＯＦ ＡＭＥＲＩＣＡ， ａｓ ｒｅｐｒｅｓｅｎｔｅｄ ｂｙ ＴＨＥ ＳＥＣＲＥＴＡＲＹ， ＤＥＰＡＲＴＭＥＮＴ ＯＦ ＨＥＡＬＴＨＡＮＤ ＨＵＭＡＮ ＳＥＲＶＩＣＥＳ 阻害性オリゴヌクレオチドを送達するための組成物および方法
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
WO2009144481A2 (en) 2008-05-30 2009-12-03 Isis Innovation Limited Conjugates for delivery of biologically active compounds
GB0813432D0 (en) 2008-07-22 2008-08-27 Spirogen Ltd Pyrrolobenzodiazepines
US20120122800A1 (en) 2008-08-08 2012-05-17 Genisphere, LLC Hatfield, Pennsylvania Long-Acting DNA Dendrimers and Methods Thereof
US8557292B2 (en) 2008-08-13 2013-10-15 California Institute Of Technology Carrier nanoparticles and related compositions, methods and systems
WO2010028054A1 (en) 2008-09-02 2010-03-11 Alnylam Europe Ag. Compositions and methods for inhibiting expression of mutant egfr gene
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
EP2342340A1 (en) 2008-09-22 2011-07-13 Rxi Pharmaceuticals Corporation Rna interference in skin indications
BR122020021379B1 (pt) 2008-10-24 2021-05-11 Sarepta Therapeutics, Inc. oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular
AU2009310557B2 (en) 2008-10-27 2014-09-11 Academisch Ziekenhuis Leiden Methods and means for efficient skipping of exon 45 in Duchenne Muscular Dystrophy pre-mRNA
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
US20100249214A1 (en) 2009-02-11 2010-09-30 Dicerna Pharmaceuticals Multiplex dicer substrate rna interference molecules having joining sequences
RU2683325C2 (ru) 2009-02-05 2019-03-28 Иммьюноджен, Инк. Новые производные бензодиазепина
US8481698B2 (en) 2009-03-19 2013-07-09 The President And Fellows Of Harvard College Parallel proximity ligation event analysis
CA2757354A1 (en) 2009-04-02 2010-10-07 Laura P.W. Ranum Nucleotide repeat expansion-associated polypeptides and uses thereof
EP2421971B1 (en) 2009-04-24 2016-07-06 BioMarin Technologies B.V. Oligonucleotide comprising an inosine for treating dmd
CN102459298A (zh) 2009-05-05 2012-05-16 阿尔特姆恩科技责任有限公司 化学可编程免疫
MX2011012665A (es) 2009-06-04 2012-03-07 Novartis Ag Metodos para identificacion de sitios para conjugacion de igg.
US20110269665A1 (en) 2009-06-26 2011-11-03 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
WO2011003780A1 (en) 2009-07-06 2011-01-13 F. Hoffmann-La Roche Ag Bi-specific digoxigenin binding antibodies
IT1394860B1 (it) 2009-07-22 2012-07-20 Kemotech S R L Composti farmaceutici
JP2012533587A (ja) 2009-07-22 2012-12-27 セニックス バイオサイエンス ゲーエムベーハー 自然に存在する細胞内輸送経路を介して化合物を送達するための送達システム及びコンジュゲート
EP2473607A2 (en) 2009-08-31 2012-07-11 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
JP5898082B2 (ja) 2009-10-07 2016-04-06 マクロジェニクス，インコーポレーテッド フコシル化程度の変更により改良されたエフェクター機能を示すＦｃ領域含有ポリペプチドおよびその使用法
TR201816523T4 (tr) 2009-11-12 2018-11-21 Univ Western Australia Patolojilerin tedavisine yönelik antisens moleküller ve yöntemler.
CA2789560A1 (en) 2010-02-12 2011-08-18 Solulink, Inc. Preparation and/or purification of oligonucleotide conjugates
CA2795349C (en) 2010-04-15 2016-11-29 Seattle Genetics, Inc. Targeted pyrrolobenzodiazepine conjugates
JP5875083B2 (ja) 2010-04-15 2016-03-02 メディミューン リミテッド 増殖性疾患治療用ピロロベンゾジアゼピン
EP2601204B1 (en) 2010-04-28 2016-09-07 Ionis Pharmaceuticals, Inc. Modified nucleosides and oligomeric compounds prepared therefrom
KR20200133284A (ko) 2010-05-28 2020-11-26 사렙타 쎄러퓨틱스, 인코퍼레이티드 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체
IT1400425B1 (it) 2010-06-08 2013-05-31 Amsterdam Molecular Therapeutics Bv Modified snrnas for use in therapy.
AU2011263642B2 (en) 2010-06-11 2014-09-04 Isarna Therapeutics Gmbh Method for selective oligonucleotide modification
EP2409983A1 (en) 2010-07-19 2012-01-25 Leibniz-Institut für Pflanzenbiochemie (IPB) Tubulysin analogues
EP2595663A4 (en) 2010-07-19 2014-03-05 Isis Pharmaceuticals Inc MODULATION OF DYSTROPHIA MYOTONICA PROTEIN KINASE (DMPK) EXPRESSION
EP3192880B1 (en) 2010-08-18 2019-10-09 Fred Hutchinson Cancer Research Center Nucleic acid agents for use in treating facioscapulohumeral dystrophy (fshd)
EP2606072A4 (en) 2010-08-19 2016-04-20 Peg Biosciences Inc SYNERGISTIC BIOMOLECULAR POLYMER CONJUGATES
ES2596977T3 (es) 2010-09-02 2017-01-13 Université de Mons Agentes útiles en el tratamiento de la distrofia muscular facioescapulohumeral
WO2012058210A1 (en) 2010-10-29 2012-05-03 Merck Sharp & Dohme Corp. RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACIDS (siNA)
AU2011323316B2 (en) 2010-11-03 2016-02-25 Immunogen, Inc. Cytotoxic agents comprising new ansamitocin derivatives
US8501930B2 (en) 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
CA2816041C (en) 2010-12-29 2019-01-08 Arrowhead Research Corporation In vivo polynucleotide delivery conjugates having enzyme sensitive linkages
EP2672977A1 (en) 2011-02-08 2013-12-18 The Charlotte-Mecklenburg Hospital Authority d/b/a Carolina Healthcare System Antisense oligonucleotides
EP2686345B1 (en) 2011-03-16 2018-04-25 Amgen Inc. Fc variants
US9045750B2 (en) 2011-03-18 2015-06-02 Yuelong Ma Humanized lewis-Y specific antibody-based delivery of dicer substrate siRNA (D-siRNA) against STAT3
ES2692268T5 (en) 2011-03-29 2025-02-26 Roche Glycart Ag Antibody fc variants
JP6209159B2 (ja) 2011-06-21 2017-10-04 イミュノジェン・インコーポレーテッド ペプチドリンカーを有する新規メイタンシノイド誘導体およびその結合体
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
CN103796657B (zh) 2011-07-19 2017-07-11 波涛生命科学有限公司 合成官能化核酸的方法
WO2013033230A1 (en) 2011-08-29 2013-03-07 Isis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
CA2850373C (en) 2011-10-14 2019-07-16 Seattle Genetics, Inc. Pyrrolobenzodiazepines and targeted conjugates
WO2013071079A1 (en) 2011-11-09 2013-05-16 President And Fellows Of Harvard College Lin28/let-7 crystal structures, purification protocols, and molecular probes suitable for screening assays and therapeutics
SI3366775T2 (sl) 2011-11-18 2025-12-31 Alnylam Pharmaceuticals, Inc. Modificirana sredstva RNAI
RU2014124984A (ru) 2011-12-05 2016-01-27 Идженика Биотерапьютикс, Инк. Конъюгаты антитело-лекарственное средство и родственные соединения, композиции и способы
AU2013203422A1 (en) 2012-01-06 2013-07-18 Bioalliance C.V. Anti-transferrin receptor antibodies and methods using same
WO2013121175A1 (en) 2012-02-16 2013-08-22 Ucl Business Plc Lysosome-cleavable linker
AU2013222935B2 (en) 2012-02-24 2016-03-10 Alteogen Inc. Modified antibody in which motif comprising cysteine residue is bound, modified antibody-drug conjugate comprising the modified antibody, and production method for same
EA201492004A1 (ru) 2012-05-02 2015-08-31 Новартис Аг Органические композиции для лечения kras-ассоциированных заболеваний
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
US9504756B2 (en) 2012-05-15 2016-11-29 Seattle Genetics, Inc. Self-stabilizing linker conjugates
CN104640572B (zh) 2012-05-15 2018-04-27 索伦托医疗有限公司 药物偶联物，偶联方法，及其用途
KR102213609B1 (ko) 2012-07-13 2021-02-08 웨이브 라이프 사이언시스 리미티드 키랄 제어
CN104781271B (zh) 2012-08-20 2018-07-06 加利福尼亚大学董事会 具有生物可逆的基团的多核苷酸
KR102058381B1 (ko) 2012-08-24 2019-12-24 강원대학교산학협력단 인간 ｌ１ｃａｍ에 대한 인간화 항체 및 그의 제조 방법
MX338711B (es) 2012-10-12 2016-04-28 Medimmune Ltd Pirrolobenzodiazepinas y conjugados de las mismas.
CN104837996A (zh) 2012-11-15 2015-08-12 罗氏创新中心哥本哈根有限公司 抗apob反义缀合物化合物
CA2891280C (en) 2012-11-24 2018-03-20 Hangzhou Dac Biotech Co., Ltd. Hydrophilic linkers and their uses for conjugation of drugs to cell binding molecules
US9089614B2 (en) 2012-12-21 2015-07-28 Bioalliance C.V. Hydrophilic self-immolative linkers and conjugates thereof
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
TWI680766B (zh) 2013-03-13 2020-01-01 英商梅迪繆思有限公司 吡咯并苯并二氮呯及其共軛物
CN110218727A (zh) 2013-03-14 2019-09-10 萨勒普塔医疗公司 用于治疗肌营养不良的外显子跳跃组合物
JP6847388B2 (ja) 2013-03-15 2021-03-31 レゲネロン ファーマシューティカルス，インコーポレーテッド 生物活性分子、そのコンジュゲート、及び治療用途
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
US20140329762A1 (en) 2013-03-15 2014-11-06 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
DK2978845T3 (da) 2013-03-27 2020-08-10 Isarna Therapeutics Gmbh Modificerede tgf-beta-oligonukleotider
US9295731B2 (en) 2013-04-01 2016-03-29 Mark Quang Nguyen Cleavable drug conjugates, compositions thereof and methods of use
WO2014169049A1 (en) 2013-04-09 2014-10-16 Duke University 2' fluoro-modified rnas as immunostimulators
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
EP3594240B1 (en) 2013-05-20 2023-12-06 F. Hoffmann-La Roche AG Anti-transferrin receptor antibodies and methods of use
KR102332303B1 (ko) 2013-05-31 2021-11-26 자임워크스 인코포레이티드 감소되거나 침묵화된 효과기 기능을 갖는 이종다량체
WO2014197854A1 (en) 2013-06-06 2014-12-11 Igenica Biotherapeutics, Inc. Novel linkers for antibody-drug conjugates and related compounds, compositions, and methods of use
US20140363454A1 (en) 2013-06-06 2014-12-11 Igenica Biotherapeutics, Inc. Antibody-Drug Conjugates, 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
EP2845607A1 (en) 2013-09-09 2015-03-11 University of Vienna Antisense oligonucleotides with improved pharmacokinetic properties
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
NZ758049A (en) 2013-10-15 2024-03-22 Seagen Inc Pegylated drug-linkers for improved ligand-drug conjugate pharmacokinetics
AU2014337317A1 (en) 2013-10-15 2016-09-15 Sorrento Therapeutics Inc. Drug-conjugates with a targeting molecule and two different drugs
EP3610884B1 (en) 2013-11-06 2021-08-18 Merck Sharp & Dohme Corp. Peptide containing conjugates for dual molecular delivery of oligonucleotides
US10378017B2 (en) 2013-12-02 2019-08-13 Brandeis University High temperature selection of nucleotide-supported carbohydrate vaccines and resulting glycosylated oligonucleotides
BR112016016400A2 (pt) 2014-01-16 2017-10-03 Wave Life Sciences Ltd Composições de oligonucleotídeos quiralmente controlados, seu uso, sua composição farmacêutica, e métodos
CA2935426C (en) 2014-01-30 2023-07-25 F. Hoffmann-La Roche Ag Polyoligomer compound with biocleavable conjugates for reducing or inhibiting expression of a nucleic acid target
MX373052B (es) 2014-06-17 2020-05-11 Nippon Shinyaku Co Ltd Acidos nucleicos antisentido.
TWI695011B (zh) 2014-06-18 2020-06-01 美商梅爾莎納醫療公司 抗ｈｅｒ２表位之單株抗體及其使用之方法
US10988764B2 (en) 2014-06-23 2021-04-27 Monsanto Technology Llc Compositions and methods for regulating gene expression via RNA interference
IL316808A (en) 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
US10508151B2 (en) 2014-11-19 2019-12-17 Genentech, Inc. Anti-transferrin receptor antibodies and methods of use
US10538763B2 (en) 2015-01-16 2020-01-21 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of DUX4
US20180305689A1 (en) 2015-04-22 2018-10-25 Mina Therapeutics Limited Sarna compositions and methods of use
ES2905181T3 (es) 2015-05-01 2022-04-07 Prec Biosciences Inc Deleción precisa de secuencias cromosómicas in vivo
AU2016264471B2 (en) 2015-05-19 2022-01-27 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
HUE057952T2 (hu) 2015-06-24 2022-06-28 Hoffmann La Roche Anti-transzferrin receptor antitestek testreszabott affinitással
WO2017007886A2 (en) 2015-07-08 2017-01-12 Idera Pharmaceuticals, Inc. Compositions for inhibiting dux4 gene expression and uses thereof
JP6749589B2 (ja) * 2015-07-31 2020-09-02 国立大学法人北海道大学 抗Ｅｖａ１タンパク質抗体
EP3423491A1 (en) 2016-03-01 2019-01-09 H. Hoffnabb-La Roche Ag Obinutuzumab variants having altered cell death induction
JP2019513371A (ja) 2016-04-01 2019-05-30 アビディティー バイオサイエンシーズ エルエルシー 核酸ポリペプチド組成物とその使用
CN109462995A (zh) 2016-05-04 2019-03-12 波涛生命科学有限公司 生物活性剂的方法和组合物
DK3473270T5 (da) 2016-06-20 2024-09-09 Genahead Bio Inc Konjugat af antistof-lægemiddel
US11427838B2 (en) 2016-06-29 2022-08-30 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of myotonic dystrophy type 1 (DM1) and other related disorders
JP7211940B2 (ja) 2016-10-28 2023-01-24 ジェネトン 筋緊張性ジストロフィーの治療のための組成物および方法
CN110234762A (zh) 2016-10-28 2019-09-13 吉尼松公司 用于治疗肌强直性营养不良的组合物和方法
US11504391B1 (en) 2016-11-23 2022-11-22 Alnylam Pharmaceuticals, Inc. Modified RNA agents with reduced off-target effect
JOP20190187A1 (ar) 2017-02-03 2019-08-01 Novartis Ag مترافقات عقار جسم مضاد لـ ccr7
EP3444010B1 (en) * 2017-08-14 2020-12-09 ScandiEdge Therapeutics AB Pklr inhibition in the treatment of nafld and hcc
MX2020003130A (es) 2017-09-22 2020-09-25 Avidity Biosciences Inc Composiciones y metodos de induccion de salto de exon en acidos nucleicos-polipeptidos.
AU2018345772B2 (en) 2017-10-02 2025-04-17 Research Institute At Nationwide Children's Hospital MiRNA detargeting system for tissue specific interference
EP3691657A4 (en) 2017-10-04 2021-07-21 Avidity Biosciences, Inc. NUCLEIC ACID POLYPEPTIDE COMPOSITIONS AND USES THEREOF
BR112020006677A2 (pt) 2017-10-05 2020-10-06 Fulcrum Therapeutics, Inc. uso de inibidores p38 para reduzir a expressão de dux4
AU2018378812B2 (en) 2017-12-06 2025-05-22 Avidity Biosciences, Inc. Compositions and methods of treating muscle atrophy and myotonic dystrophy
WO2020028836A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle-targeting complexes and uses thereof in treating muscle atrophy
CA3108282A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
IL312067B2 (en) 2018-12-21 2025-08-01 Avidity Biosciences Inc Anti-transferrin receptor antibodies and uses thereof
EP3906308A1 (en) 2018-12-31 2021-11-10 Research Institute at Nationwide Children's Hospital Dux4 rna silencing using rna targeting crispr-cas13b
WO2020203880A1 (ja) 2019-03-29 2020-10-08 田辺三菱製薬株式会社 Ｄｕｘ４の発現を調節するための化合物、方法及び医薬組成物
US12006499B2 (en) 2019-06-06 2024-06-11 Avidity Biosciences, Inc. Una amidites and uses thereof
KR20220125801A (ko) 2020-01-10 2022-09-14 다인 세라퓨틱스, 인크. 근육 표적화 복합체 및 안면견갑상완 근육 이영양증을 치료하기 위한 그의 용도
WO2021188390A1 (en) 2020-03-19 2021-09-23 Avidity Biosciences, Inc. Compositions and methods of treating facioscapulohumeral muscular dystrophy
CA3189861A1 (en) 2020-09-01 2022-03-10 Sean Christopher DAUGHERTY Dux4 inhibitors and methods of use thereof
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
EP4370678A2 (en) 2021-07-14 2024-05-22 Mirecule, Inc. Oligonucleotides and compositions thereof for neuromuscular disorders

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20190300903A1 (en) * 2011-07-25 2019-10-03 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of dux4
WO2018129384A1 (en) * 2017-01-06 2018-07-12 Avidity Biosciences Llc Nucleic acid-polypeptide compositions and methods of inducing exon skipping
WO2020028864A1 (en) * 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
CN118265544A (zh) * 2021-09-16 2024-06-28 艾维迪提生物科学公司 治疗面肩肱型肌营养不良的组合物和方法

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