AU2017240799B2 - Nucleic acid-polypeptide compositions and uses thereof - Google Patents

Nucleic acid-polypeptide compositions and uses thereof Download PDF

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Publication number
AU2017240799B2
AU2017240799B2 AU2017240799A AU2017240799A AU2017240799B2 AU 2017240799 B2 AU2017240799 B2 AU 2017240799B2 AU 2017240799 A AU2017240799 A AU 2017240799A AU 2017240799 A AU2017240799 A AU 2017240799A AU 2017240799 B2 AU2017240799 B2 AU 2017240799B2
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acid molecule
polynucleic acid
polynucleotide
molecule
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AU2017240799A1 (en
Inventor
Palani Balu
Rob BURKE
David Sai-Ho CHU
Michael Caramian COCHRAN
Beatrice Diana DARIMONT
Venkata Ramana Doppalapudi
Andrew John Geall
Rachel Elizabeth JOHNS
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Avidity Biosciences Inc
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Avidity Biosciences Inc
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Priority to AU2023203188A priority Critical patent/AU2023203188B2/en
Priority to AU2025263825A priority patent/AU2025263825A1/en
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Families Citing this family (80)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SI3215518T1 (sl) 2014-10-29 2021-08-31 Bicyclerd Limited Biciklični peptidni ligandi, značilni za MT1-MMP
JP6892433B2 (ja) 2015-04-03 2021-06-23 ユニバーシティ・オブ・マサチューセッツＵｎｉｖｅｒｓｉｔｙ Ｏｆ Ｍａｓｓａｃｈｕｓｅｔｔｓ 十分に安定化された非対称ｓｉｒｎａ
US10633653B2 (en) 2015-08-14 2020-04-28 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
JP7749201B6 (ja) 2016-01-31 2025-10-21 ユニバーシティー オブ マサチューセッツ 分岐オリゴヌクレオチド
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
JP6823269B2 (ja) * 2016-06-20 2021-02-03 株式会社ＧｅｎＡｈｅａｄ Ｂｉｏ 抗体−薬物コンジュゲート
WO2018031933A2 (en) 2016-08-12 2018-02-15 University Of Massachusetts Conjugated oligonucleotides
KR20190104381A (ko) 2017-01-06 2019-09-09 어비디티 바이오사이언시스 엘엘씨 핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법
US10844377B2 (en) 2017-06-23 2020-11-24 University Of Massachusetts Two-tailed self-delivering siRNA
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
CN111183147B (zh) 2017-08-04 2024-07-05 拜斯科技术开发有限公司 Cd137特异性的双环肽配体
WO2019055633A1 (en) * 2017-09-14 2019-03-21 Arrowhead Pharmaceuticals, Inc. RNAI AGENTS AND COMPOSITIONS FOR INHIBITING ANALOGUE EXPRESSION OF ANGIOPOIETIN 3 (ANGPTL3) AND METHODS OF USE
MX2020003596A (es) 2017-10-04 2020-07-22 Avidity Biosciences Inc Composiciones de acido nucleico-polipeptido y usos de las mismos.
JP7506601B2 (ja) * 2017-12-06 2024-06-26 アビディティー バイオサイエンシーズ，インク． 筋萎縮症と筋緊張性ジストロフィーを処置するための組成物および方法
TWI825046B (zh) 2017-12-19 2023-12-11 英商拜西可泰克斯有限公司 Epha2特用之雙環胜肽配位基
MA51583A (fr) * 2018-01-04 2020-11-11 Avidity Biosciences Inc Molécules d'acide nucléique hétéroduplex et leurs utilisations
WO2019162682A1 (en) 2018-02-23 2019-08-29 Bicycletx Limited Multimeric bicyclic peptide ligands
KR20200139236A (ko) * 2018-04-04 2020-12-11 바이사이클티엑스 리미티드 헤테로탠덤 비사이클릭 펩티드 복합체
US11180531B2 (en) 2018-06-22 2021-11-23 Bicycletx Limited Bicyclic peptide ligands specific for Nectin-4
GB201810316D0 (en) 2018-06-22 2018-08-08 Bicyclerd Ltd Peptide ligands for binding to EphA2
SG11202100928QA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
SG11202100934PA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
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
US11168141B2 (en) 2018-08-02 2021-11-09 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
KR20210093227A (ko) 2018-08-10 2021-07-27 유니버시티 오브 매사추세츠 Snp를 표적화하는 변형된 올리고뉴클레오티드
KR20210053930A (ko) 2018-08-31 2021-05-12 예일 유니버시티 공여자 올리고뉴클레오티드 기반의 유전자 편집을 향상시키기 위한 조성물 및 방법
US12331320B2 (en) 2018-10-10 2025-06-17 The Research Foundation For The State University Of New York Genome edited cancer cell vaccines
GB201820295D0 (en) 2018-12-13 2019-01-30 Bicyclerd Ltd Bicyclic peptide ligands specific for MT1-MMP
GB201820325D0 (en) 2018-12-13 2019-01-30 Bicyclerd Ltd Bicyclic peptide ligands specific for psma
GB201820288D0 (en) 2018-12-13 2019-01-30 Bicycle Tx Ltd Bicycle peptide ligaands specific for MT1-MMP
IL319265A (en) 2018-12-21 2025-04-01 Avidity Biosciences Inc Anti-transferrin receptor antibodies and their uses
WO2020128526A1 (en) 2018-12-21 2020-06-25 Bicycletx Limited Bicyclic peptide ligands specific for pd-l1
WO2020150636A1 (en) * 2019-01-18 2020-07-23 University Of Massachusetts Dynamic pharmacokinetic-modifying anchors
AU2020263487A1 (en) 2019-04-25 2021-12-16 Avidity Biosciences, Inc. Nucleic acid compositions and methods of multi-exon skipping
WO2020225577A1 (en) 2019-05-09 2020-11-12 Bicycletx Limited Bicyclic peptide ligands specific for ox40
WO2020247818A1 (en) 2019-06-06 2020-12-10 Avidity Biosciences, Inc. Una amidites and uses thereof
CA3142283A1 (en) 2019-06-06 2020-12-10 Avidity Biosciences, Inc. Nucleic acid-polypeptide compositions and uses thereof
TWI862640B (zh) 2019-07-30 2024-11-21 英商拜西可泰克斯有限公司 異質雙環肽複合物
MX2022001710A (es) 2019-08-09 2022-05-10 Univ Massachusetts Oligonucleótidos modificados químicamente dirigidos a los snp.
WO2021042060A1 (en) 2019-08-30 2021-03-04 Yale University Compositions and methods for delivery of nucleic acids to cells
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
CN119192401A (zh) 2019-10-03 2024-12-27 拜斯科技术开发有限公司 异串联双环肽复合物
WO2021076574A2 (en) * 2019-10-14 2021-04-22 Aro Biotherapeutics Company Fn3 domain-sirna conjugates and uses thereof
US12491255B2 (en) 2019-10-14 2025-12-09 Aro Biotherapeutics Company EPCAM binding fibronectin type III domains
EP4045061A4 (en) 2019-10-14 2024-04-17 ARO Biotherapeutics Company FIBRONECTIN TYPE III DOMAINS BINDING TO CD137
CA3160576A1 (en) * 2019-12-06 2021-06-10 Sirnaomics, Inc. Peptide docking vehicle for targeted nucleic acid delivery
CA3172111A1 (en) 2020-03-19 2021-09-23 Barbora MALECOVA Compositions and methods of treating facioscapulohumeral muscular dystrophy
EP4656242A2 (en) 2020-03-27 2025-12-03 Avidity Biosciences, Inc. Compositions and methods of treating muscle dystrophy
WO2021259506A1 (en) * 2020-06-24 2021-12-30 Sapreme Technologies B.V. Combination comprising an adc or an aoc comprising a vhh, and a saponin or a ligand-saponin conjugate
EP4295917A3 (en) * 2020-08-07 2024-02-28 Tambo, Inc. Trans-cyclooctene bioorthogonal agents and uses in cancer and immunotherapy
CN111996193B (zh) * 2020-09-11 2024-02-20 北京键凯科技股份有限公司 一种有效抑制表皮生长因子受体表达的siRNA序列
US20250302979A1 (en) * 2021-04-02 2025-10-02 Avidity Biosciences, Inc. Antibody-oligonucleotide conjugate and antibody-peptide-oligonucleotide conjugate compositions and methods of inducing exon skipping
CA3214552A1 (en) 2021-04-14 2022-10-20 Russell C. Addis Cd71 binding fibronectin type iii domains
BR112023021318A2 (pt) 2021-04-14 2023-12-19 Aro Biotherapeutics Company Conjugados de domínio fn3-sirna e usos dos mesmos
IL309334A (en) 2021-06-23 2024-02-01 Univ Massachusetts Anti-FLT1 optimized oligonucleotide compounds for the treatment of preeclampsia and other angiogenic disorders
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11672872B2 (en) 2021-07-09 2023-06-13 Dyne Therapeutics, Inc. Anti-transferrin receptor antibody and uses thereof
MX2024000489A (es) 2021-07-09 2024-04-09 Dyne Therapeutics Inc Complejos dirigidos al musculo y formulaciones para el tratamiento de las distrofinopatías.
US11633498B2 (en) 2021-07-09 2023-04-25 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
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
KR20240107202A (ko) 2021-09-01 2024-07-08 바이오젠 엠에이 인코포레이티드 항-트랜스페린 수용체 항체 및 이의 용도
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
CN113943805A (zh) * 2021-11-02 2022-01-18 上海市生物医药技术研究院 基因突变检测引物组及其用途
CN114099692B (zh) * 2021-11-30 2023-06-09 西南大学 一种抗菌肽-细胞膜复合物、制备方法和应用
KR20240125036A (ko) 2021-12-23 2024-08-19 마이레큘, 인크. 폴리뉴클레오타이드의 전달을 위한 조성물
US12071621B2 (en) 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping
CA3255934A1 (en) 2022-04-15 2023-10-19 Dyne Therapeutics, Inc. MUSCLE TARGETTING COMPLEXES AND FORMULATIONS FOR THE TREATMENT OF MYOTONIC DYSTROPHY
MA71741A (fr) * 2022-08-08 2025-05-30 Eli Lilly And Co. Protéines de liaison au récepteur de la transferrine et conjugués
CN115607563B (zh) * 2022-08-15 2023-11-07 江苏蓝果临床营养科技有限公司 一种烟酰胺单核苷酸复合物及其制备方法
KR20250173499A (ko) 2023-03-08 2025-12-10 바이오젠 엠에이 인코포레이티드 항-트랜스페린 수용체 항체 및 이의 용도
CN116381125A (zh) * 2023-06-05 2023-07-04 迦进生物医药(上海)有限公司 评估与蛋白偶联的核酸稳定性的方法及试剂盒
US12491257B2 (en) 2023-06-27 2025-12-09 Avidity Biosciences, Inc. Compositions and methods of using PRKAG2-targeting antibody-oligonucleotide conjugates
WO2025007063A1 (en) 2023-06-30 2025-01-02 Avidity Biosciences, Inc. Compositions and methods of using pln-targeting antibody-oligonucleotide conjugates
WO2025024334A1 (en) 2023-07-21 2025-01-30 Marrow Therapeutics, Inc. Hematopoietic cell targeting conjugates and related methods
TW202517685A (zh) 2023-10-26 2025-05-01 美商百健Ｍａ公司 抗運鐵蛋白受體抗體及其用途
WO2025207959A1 (en) 2024-03-27 2025-10-02 Biogen Ma Inc. Anti-transferrin receptor antibodies and uses thereof

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006128138A2 (en) * 2005-05-26 2006-11-30 Ensemble Discovery Corportion Biodetection by nucleic acid-templated chemistry
US20130172238A1 (en) * 2010-06-11 2013-07-04 Andreas Mitsch Method for selective oligonucleotide modification

Family Cites Families (315)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US1000075A (en) 1910-07-27 1911-08-08 William R Collins Tunneling-machine.
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
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group Polynucleotide assay reagent and method
EP0215942B1 (en) 1985-03-15 1995-07-12 Antivirals Inc. Polynucleotide assay reagent and method
US5521063A (en) 1985-03-15 1996-05-28 Antivirals Inc. Polynucleotide reagent containing chiral subunits and methods of use
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
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
PL169576B1 (pl) 1990-10-12 1996-08-30 Max Planck Gesellschaft Sposób wytwarzania czasteczki RNA o aktywnosci katalitycznej PL PL
DE4216134A1 (de) 1991-06-20 1992-12-24 Europ Lab Molekularbiolog Synthetische katalytische oligonukleotidstrukturen
EP0531597A1 (en) 1991-09-12 1993-03-17 Merrell Dow Pharmaceuticals Inc. Novel unsaturated acyclic phosphonate derivatives of purine and pyrimidine
GB9123947D0 (en) * 1991-11-12 1992-01-02 Imp Cancer Res Tech Therapeutic compounds
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
AU4695493A (en) 1992-08-17 1994-03-15 Quadra Logic Technologies Inc. Method for destroying or inhibiting growth of unwanted cells or tissues
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
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
US5889136A (en) 1995-06-09 1999-03-30 The Regents Of The University Of Colorado Orthoester protecting groups in RNA synthesis
AU1430097A (en) 1996-01-16 1997-08-11 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
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
JP2001524808A (ja) 1996-12-10 2001-12-04 ジーントレイス・システムズ・インコーポレイテッド 放出可能な不揮発性の質量標識分子
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
DE69937291T2 (de) 1998-04-02 2008-07-10 Genentech, Inc., South San Francisco Antikörpervarianten 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
GB9818731D0 (en) 1998-08-27 1998-10-21 Univ Portsmouth Compounds
GB9818730D0 (en) 1998-08-27 1998-10-21 Univ Portsmouth Collections of compounds
ES2244210T3 (es) 1998-08-27 2005-12-01 Spirogen Limited Pirrolobenzodiazepinas.
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
AU3703100A (en) 1999-02-22 2000-09-14 Georgetown University Antibody fragment-targeted immunoliposomes for systemic gene delivery
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
US7829693B2 (en) 1999-11-24 2010-11-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a target gene
DE10100586C1 (de) 2001-01-09 2002-04-11 Ribopharma Ag Verfahren zur Hemmung der Expression eines Ziegens
CA2328356A1 (en) 1999-12-22 2001-06-22 Itty Atcravi Recreational vehicles
PT1242438E (pt) 1999-12-29 2007-02-28 Immunogen Inc Agentes citotóxicos compreendendo dixorrubicinas e daunorrubicinas modificadas e seu uso terapêutico
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)
DK2796553T3 (da) 2000-03-30 2019-09-30 Whitehead Inst Biomedical Res Rna-sekvensspecifikke formidlere af rna-interferens
ES2461765T3 (es) 2000-03-30 2014-05-21 The Whitehead Institute For Biomedical Research Procedimientos de producción de células silenciadas u organismos silenciados por medio de mediadores específicos de secuencia de ARN de interferencia de ARN y usos de los mismos.
KR20020097241A (ko) 2000-05-04 2002-12-31 에이브이아이 바이오파마 인코포레이티드 스플라이스-영역 안티센스 조성물 및 방법
JP2002095476A (ja) 2000-09-20 2002-04-02 Eisai Co Ltd トランスフェリン受容体を介した遺伝子導入法
EP1191097A1 (en) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
CZ302719B6 (cs) 2000-12-01 2011-09-21 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Izolovaná molekula dvouretezcové RNA, zpusob její výroby a její použití
US8546143B2 (en) 2001-01-09 2013-10-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a target gene
WO2003071872A1 (en) 2002-02-22 2003-09-04 University Of Maryland, Baltimore Novel treatment of neurodegenerative diseases by altering levels of trkb isoforms and/or trkc isoforms
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
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)
EP1432724A4 (en) 2002-02-20 2006-02-01 Sirna Therapeutics Inc INTERFERENCE MEDIATION INHIBITION OF GENE RNA FROM MAP KINASE
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)
US20040012626A1 (en) 2002-07-22 2004-01-22 Brookins Timothy J. Method for creating configurable and customizable web user interfaces
US20040023220A1 (en) 2002-07-23 2004-02-05 Lawrence Greenfield Integrated method for PCR cleanup and oligonucleotide removal
PT2357006E (pt) 2002-07-31 2016-01-22 Seattle Genetics Inc Conjugados de fármacos e sua utilização para tratamento do cancro, de uma doença autoimune ou de uma doença infeciosa
DK1527176T4 (en) 2002-08-05 2017-07-03 Silence Therapeutics Gmbh ADDITIONAL NEW FORMS OF INTERFERRING RNA MOLECULES
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
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
EP1560931B1 (en) 2002-11-14 2011-07-27 Dharmacon, Inc. Functional and hyperfunctional sirna
CA2942791C (en) 2002-11-25 2019-08-20 Masafumi Matsuo 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
CA2524255C (en) 2003-03-21 2014-02-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
EP1608733B1 (en) 2003-04-02 2011-12-07 Dharmacon, Inc. Modified polynucleotides for use in rna interference
WO2004100987A2 (en) 2003-05-06 2004-11-25 Regeneron Pharmaceuticals, Inc. 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
AU2004252442B2 (en) 2003-06-02 2010-04-08 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
PT1633767T (pt) 2003-06-02 2019-02-27 Univ Massachusetts Métodos e composições para controlar a eficácia do silenciamento de arn
ES2761262T3 (es) 2003-06-13 2020-05-19 Alnylam Europe Ag Acido ribonucleico bicatenario con elevada eficacia en un organismo
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
SG149815A1 (en) 2003-11-06 2009-02-27 Seattle Genetics Inc Monomethylvaline compounds capable of conjugation to ligands
US7494925B2 (en) 2004-02-23 2009-02-24 Micron Technology, Inc. Method for making through-hole conductors for semiconductor substrates
US8389710B2 (en) 2004-02-27 2013-03-05 Operational Technologies Corporation Therapeutic nucleic acid-3′-conjugates
US7528126B2 (en) 2004-03-09 2009-05-05 Spirogen Limited Pyrrolobenzodiazepines
US8084599B2 (en) 2004-03-15 2011-12-27 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
WO2005094364A2 (en) * 2004-03-31 2005-10-13 Chimeric Technologies, Inc. Iga antibody protein as a cytotoxic drug
KR101147147B1 (ko) 2004-04-01 2012-05-25 머크 샤프 앤드 돔 코포레이션 Ｒｎａ 간섭의 오프 타겟 효과 감소를 위한 변형된폴리뉴클레오타이드
CA2568013C (en) 2004-05-27 2015-11-24 Alnylam Pharmaceuticals, Inc. Nuclease resistant double-stranded ribonucleic acid
USRE47769E1 (en) 2004-06-28 2019-12-17 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
EP1817336B1 (en) 2004-11-12 2019-01-09 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
AU2006230413B8 (en) * 2005-03-31 2011-01-20 Xencor, Inc Fc variants with optimized properties
WO2006111759A1 (en) 2005-04-21 2006-10-26 Spirogen Limited Pyrrolobenzodiazepines
KR20080031164A (ko) 2005-04-22 2008-04-08 아카데미슈 지켄후이스 라이덴 ＳＲ 단백질의 결합의 방해에 의해 그리고 이차 ＲＮＡ구조의 방해에 의한 ｐｒｅ－ｍＲＮＡ에서 엑손 인식의조절
US7736647B2 (en) * 2005-06-15 2010-06-15 Monoclonal Antibodies Therapeutics Anti-CD71 monoclonal antibodies and uses thereof for treating malignant tumor cells
US8871720B2 (en) 2005-07-07 2014-10-28 Seattle Genetics, Inc. Monomethylvaline compounds having phenylalanine carboxy modifications at the C-terminus
AU2006280600B2 (en) 2005-08-17 2012-01-19 Bioneer Corporation Sirna-hydrophilic polymer conjugates for intracellular delivery of siRNA and method thereof
EP1931780B1 (en) 2005-08-29 2016-01-06 Regulus Therapeutics Inc. Antisense compounds having enhanced anti-microrna activity
US8501703B2 (en) 2005-08-30 2013-08-06 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
PL1813614T3 (pl) 2006-01-25 2012-03-30 Sanofi Sa Środki cytotoksyczne zawierające nowe pochodne tomaymycyny
US7750116B1 (en) 2006-02-18 2010-07-06 Seattle Genetics, Inc. Antibody drug conjugate metabolites
US7943762B2 (en) 2006-05-10 2011-05-17 Avi Biopharma, Inc. Oligonucleotide analogs having cationic intersubunit linkages
US8198252B2 (en) * 2006-05-19 2012-06-12 Board Of Regents, The University Of Texas System SIRNA inhibition of PI3K P85, P110, and AKT2 and methods of use
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
SG173364A1 (en) 2006-07-10 2011-08-29 Biogen Idec Inc Compositions and methods for inhibiting growth of smad4-deficient cancers
ES2373246T3 (es) 2006-08-11 2012-02-01 Prosensa Technologies B.V. Oligonucleótidos monocatenarios complementarios de elementos repetitivos para el tratamiento de trastornos genéticos asociados a la inestabilidad de repeticiones de adn.
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
US9371348B2 (en) 2006-11-27 2016-06-21 The Trustees Of The University Of Pennsylvania Photocleavable oligonucleotide and uses thereof
WO2011100493A1 (en) 2010-02-12 2011-08-18 Solulink, Inc. Preparation and/or purification of oligonucleotide conjugates
CA2723671C (en) 2007-05-10 2018-06-19 R & D Biopharmaceuticals Gmbh Tubulysine derivatives
US9063129B2 (en) 2007-06-15 2015-06-23 Idexx Laboratories, Inc. Device, kit and method for hookworm antigen capture and detection
CA2691673A1 (en) 2007-06-29 2009-01-08 Avi Biopharma, Inc. Tissue specific peptide conjugates and methods
PT2019104E (pt) 2007-07-19 2013-12-03 Sanofi Sa Agentes citotóxicos compreendendo novos derivados de tomaimicina e sua utilização terapêutica
ES2617877T3 (es) 2007-08-27 2017-06-20 1Globe Health Institute Llc Composiciones de ARN interferente asimétrico y uso de las mismas
CA2704737A1 (en) 2007-09-18 2009-09-03 Intradigm Corporation Compositions comprising k-ras sirna and methods of use
DK2195428T3 (en) 2007-09-19 2014-03-03 Applied Biosystems Llc SIRNA SEQUENCE-INDEPENDENT MODIFICATION FORMS TO REDUCE TARGET-FAILING PHENOTYPIC EFFECTS OF RNAI, AND STABILIZED FORMS THEREOF
SI2211904T1 (sl) 2007-10-19 2016-12-30 Seattle Genetics, Inc. CD19 vezavna sredstva in njihove uporabe
HUE028662T2 (en) 2007-10-26 2016-12-28 Academisch Ziekenhuis Leiden Preparations and methods for controlling muscle disorders
US7820391B2 (en) 2007-11-06 2010-10-26 Osmetech Molecular Diagnostics Baseless nucleotide analogues and uses thereof
MX2010007767A (es) 2008-01-18 2010-08-09 Medimmune Llc Anticuerpos manipulados con cisteina para conjugacion especifica de sitio.
US20110081362A1 (en) 2008-01-31 2011-04-07 The Brigham And Women's Hospital, Inc. Treatment of cancer
US20110065774A1 (en) 2008-01-31 2011-03-17 Alnylam Pharmaceuticals Chemically modified oligonucleotides and uses thereof
MX2010008024A (es) 2008-02-01 2010-12-21 Ascendis Pharma As Profarmaco que comprende un enlazador que se puede separar por si mismo.
JP5697993B2 (ja) 2008-02-11 2015-04-08 アールエックスアイ ファーマシューティカルズ コーポレーション 修飾ＲＮＡｉポリヌクレオチドおよびその使用
EP2265283B1 (en) 2008-03-18 2014-09-03 Seattle Genetics, Inc. Auristatin drug linker conjugates
TWI465247B (zh) 2008-04-11 2014-12-21 Catalyst Biosciences Inc 經修飾的因子ｖｉｉ多肽和其用途
EP2274425A2 (en) 2008-04-11 2011-01-19 Alnylam Pharmaceuticals Inc. Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
WO2009129281A2 (en) 2008-04-15 2009-10-22 Government Of The United States Of America, As Represented By The Secretary, Department Of Health & Human Services Compositions and methods for delivering inhibitory oligonucleotides
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
JP2011523557A (ja) 2008-06-04 2011-08-18 メディカル リサーチ カウンシル ペプチド
PT2285350T (pt) * 2008-06-16 2018-01-04 Pfizer Métodos para a preparação de copolímeros em dibloco funcionalizados com agente de direcionamento para utilização no fabrico de nanopartículas terapêuticas
GB0813432D0 (en) 2008-07-22 2008-08-27 Spirogen Ltd Pyrrolobenzodiazepines
JP5028355B2 (ja) * 2008-08-01 2012-09-19 株式会社オティックス 可変動弁機構
WO2010017544A2 (en) 2008-08-08 2010-02-11 Genisphere, Inc. Long-acting dna dendrimers and methods thereof
KR101661746B1 (ko) 2008-08-13 2016-09-30 캘리포니아 인스티튜트 오브 테크놀로지 캐리어 나노입자, 그리고 관련된 조성물, 방법 및 시스템
WO2010021720A1 (en) 2008-08-19 2010-02-25 Nektar Therapeutics Conjugates of small-interfering nucleic acids
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
CA2753338A1 (en) 2008-09-22 2010-03-25 Rxi Pharmaceuticals Corporation Neutral nanotransporters
TR201902952T4 (tr) 2008-10-24 2019-03-21 Sarepta Therapeutics Inc Dmd için ekson atlama bileşimleri.
AU2013204438B2 (en) 2008-10-24 2016-12-22 Sarepta Therapeutics, Inc. Multiple exon skipping compositions for DMD
PT2344637E (pt) 2008-10-27 2015-03-23 Academisch Ziekenhuis Leiden Métodos e meios para o salto eficiente do exão 45 no pré-mrna de distrofia muscular de duchenne
KR101602431B1 (ko) 2008-11-05 2016-03-10 삼성전자 주식회사 냉장고
SG171914A1 (en) 2008-12-02 2011-07-28 Chiralgen Ltd Method for the synthesis of phosphorus atom modified nucleic acids
CN108727407B (zh) 2009-02-05 2022-01-28 伊缪诺金公司 新型苯并二氮杂䓬衍生物
WO2010093788A2 (en) 2009-02-11 2010-08-19 Dicerna Pharmaceuticals, Inc. Multiplex dicer substrate rna interference molecules having joining sequences
US8481698B2 (en) 2009-03-19 2013-07-09 The President And Fellows Of Harvard College Parallel proximity ligation event analysis
RU2568066C2 (ru) * 2009-03-25 2015-11-10 Дженентек, Инк. Антитела против fgfr3 и способы их применения
CA2757354A1 (en) 2009-04-02 2010-10-07 Laura P.W. Ranum 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
JP2012524540A (ja) 2009-04-24 2012-10-18 プロセンサ テクノロジーズ ビー．ブイ． Ｄｍｄを処置するためのイノシンを含むオリゴヌクレオチド
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
KR101861295B1 (ko) 2009-06-04 2018-05-25 노파르티스 아게 ＩｇＧ 콘쥬게이션을 위한 자리의 확인 방법
AU2010270979B2 (en) 2009-06-22 2015-04-23 Medimmune, Llc Engineered Fc regions for site-specific conjugation
US20110269665A1 (en) 2009-06-26 2011-11-03 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
SG177560A1 (en) * 2009-07-06 2012-03-29 Hoffmann La Roche Bi-specific digoxigenin binding antibodies
WO2011005860A2 (en) 2009-07-07 2011-01-13 Alnylam Pharmaceuticals, Inc. 5' phosphate mimics
IT1394860B1 (it) 2009-07-22 2012-07-20 Kemotech S R L Composti farmaceutici
EP2456470A1 (en) 2009-07-22 2012-05-30 Cenix Bioscience GmbH Delivery system and conjugates for compound delivery via naturally occurring intracellular transport routes
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
CA2776385C (en) 2009-10-07 2019-04-09 Macrogenics, Inc. Fc region-containing polypeptides that exhibit improved effector function due to alterations of the extent of fucosylation, and methods for their use
WO2011057350A1 (en) 2009-11-12 2011-05-19 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
AR080027A1 (es) 2010-01-28 2012-03-07 Glaxo Group Ltd Proteinas de union a cd127
WO2011130371A1 (en) 2010-04-13 2011-10-20 Life Technologies Corporation Compositions and methods for inhibition of nucleic acids function
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
KR101671360B1 (ko) 2010-04-15 2016-11-01 시애틀 지네틱스, 인크. 표적화된 피롤로벤조디아제핀 접합체
WO2011139702A2 (en) 2010-04-28 2011-11-10 Isis Pharmaceuticals, Inc. Modified nucleosides and oligomeric compounds prepared therefrom
EP3091027B1 (en) 2010-04-28 2018-01-17 Ionis Pharmaceuticals, Inc. 5' modified nucleosides and oligomeric compounds prepared therefrom
CA2799501C (en) 2010-05-28 2022-02-15 Sarepta Therapeutics, Inc. Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
IT1400425B1 (it) 2010-06-08 2013-05-31 Amsterdam Molecular Therapeutics Bv Modified snrnas for use in therapy.
AU2011282243B2 (en) 2010-07-19 2016-09-22 Ionis Pharmaceuticals, Inc. Modulation of nuclear-retained RNA
EP2409983A1 (en) 2010-07-19 2012-01-25 Leibniz-Institut für Pflanzenbiochemie (IPB) Tubulysin analogues
EP4050109A1 (en) 2010-08-18 2022-08-31 Fred Hutchinson Cancer Center Agents for use in treating facioscapulohumeral dystrophy (fshd)
EP2606072A4 (en) 2010-08-19 2016-04-20 Peg Biosciences Inc SYNERGISTIC BIOMOLECULAR POLYMER CONJUGATES
US10017763B2 (en) 2010-09-03 2018-07-10 Sarepta Therapeutics, Inc. dsRNA molecules comprising oligonucleotide analogs having modified intersubunit linkages and/or terminal groups
EP3327125B1 (en) 2010-10-29 2020-08-05 Sirna Therapeutics, Inc. Rna interference mediated inhibition of gene expression using short interfering nucleic acids (sina)
CN103269712B (zh) 2010-11-03 2016-09-21 伊缪诺金公司 包含新型安丝菌素衍生物的细胞毒性剂
US8501930B2 (en) 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
KR20130136494A (ko) * 2010-12-29 2013-12-12 애로우헤드 리서치 코오포레이션 효소 민감성 결합을 갖는 Ｉｎ Ｖｉｖｏ 폴리뉴클레오티드 전달 컨쥬게이트
JP2014507143A (ja) 2011-02-08 2014-03-27 ザ シャーロット−メクレンバーグ ホスピタル オーソリティ ドゥーイング／ビジネス／アズ キャロライナズ ヘルスケア システム アンチセンスオリゴヌクレオチド
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
KR20160044598A (ko) 2011-03-29 2016-04-25 로슈 글리카트 아게 항체 Fc 변이체
WO2012138487A2 (en) 2011-04-07 2012-10-11 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
CN103534272B (zh) * 2011-05-09 2016-05-11 株式会社英仙蛋白质科学 能够特异性识别转铁蛋白受体的抗体
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
AU2012284259A1 (en) 2011-07-15 2014-03-06 Sarepta Therapeutics, Inc. Methods and compositions for manipulating translation of protein isoforms from alternative initiation start sites
MX347361B (es) 2011-07-19 2017-04-12 Wave Life Sciences Ltd Metodos para la sintesis de acidos nucleicos funcionalizados.
US9418216B2 (en) 2011-07-21 2016-08-16 Microsoft Technology Licensing, Llc Cloud service authentication
EP4488370A2 (en) 2011-07-25 2025-01-08 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expresssion of dux4
WO2013033230A1 (en) 2011-08-29 2013-03-07 Isis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
CN103998458B (zh) 2011-08-30 2018-10-09 医学研究理事会 具有中央疏水域的细胞穿透肽
WO2013055990A1 (en) 2011-10-14 2013-04-18 Seattle Genetics, Inc. Pyrrolobenzodiazepines and targeted conjugates
US9593141B2 (en) 2011-11-09 2017-03-14 President And Fellows Of Harvard College Lin28/let-7 crystal structures, purification protocols, and molecular probes suitable for screening assays and therapeutics
DK3366775T4 (da) 2011-11-18 2025-10-27 Alnylam Pharmaceuticals Inc Modificerede rnai-midler
CA2857398A1 (en) 2011-12-05 2013-06-13 Igenica Biotherapeutics, Inc. Antibody-drug conjugates and related compounds, compositions, and methods
CA3106312C (en) * 2011-12-16 2025-09-23 National University Corporation Tokyo Medical And Dental University CHIMERIC DOUBLE-STRANDED NUCLEIC ACID
EP2794653B1 (en) 2011-12-23 2019-03-13 Pfizer Inc Engineered antibody constant regions for site-specific conjugation and methods and uses therefor
WO2013103800A1 (en) * 2012-01-06 2013-07-11 Bioalliance C.V. Anti-transferrin receptor antibodies and methods using same
US9234048B2 (en) * 2012-01-18 2016-01-12 Wisconsin Alumni Research Foundation Boronate-mediated delivery of molecules into cells
CN111893117B (zh) 2012-01-27 2024-06-04 比奥马林技术公司 治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸
EP2814512A1 (en) 2012-02-16 2014-12-24 UCL Business Plc. Lysosome-cleavable linker
EP2818480B1 (en) 2012-02-24 2020-08-26 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
WO2013138662A1 (en) 2012-03-16 2013-09-19 4S3 Bioscience, Inc. Antisense conjugates for decreasing expression of dmpk
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
MX2014013367A (es) 2012-05-02 2014-12-08 Novartis Ag Composiciones organicas para tratar enfermedades relacionadas con kras.
US9504756B2 (en) 2012-05-15 2016-11-29 Seattle Genetics, Inc. Self-stabilizing linker conjugates
US9981046B2 (en) 2012-05-15 2018-05-29 Concortis Biosystems, Corp., a wholly owned Subsidiary of Sorrento Therapeutics, Inc. Drug-conjugates, conjugation methods, and uses thereof
RU2674600C2 (ru) 2012-07-03 2018-12-11 Просенса Текнолоджиз Б.В. Олигонуклеотид для лечения пациентов с мышечной дистрофией
EP2872147B1 (en) 2012-07-13 2022-12-21 Wave Life Sciences Ltd. Method for making chiral oligonucleotides
CA2880869A1 (en) 2012-08-20 2014-02-27 The Regents Of The University Of California Polynucleotides having bioreversible groups
AU2013328580B2 (en) 2012-10-12 2016-01-21 Medimmune Limited Pyrrolobenzodiazepines and conjugates thereof
MX2015005352A (es) 2012-10-26 2015-10-29 Nlife Therapeutics S L Composiciones y métodos para el tratamiento de la enfermedad de parkinson mediante la administración selectiva de moléculas de oligonucleótidos a tipos específicos de neuronas.
EP2915815B1 (en) 2012-10-31 2018-01-17 Takeda Pharmaceutical Company Limited New modified nucleic acid
RU2015119411A (ru) * 2012-11-15 2017-01-10 Рош Инновейшен Сентер Копенгаген А/С Конъюгаты антисмысловых соединений, направленные на аполипопротеин в
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
BR112015014987A2 (pt) 2012-12-20 2017-08-15 Sarepta Therapeutics Inc Aprimoradas composições que pulam éxon para o tratamento da distrofia muscular
AU2013364065B2 (en) 2012-12-21 2018-10-04 Altrubio Inc. 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
US9319764B2 (en) 2013-03-08 2016-04-19 Merry Electronics Co., Ltd. MEMS microphone packaging structure
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
US9217148B2 (en) 2013-03-14 2015-12-22 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
US9577459B2 (en) 2013-03-15 2017-02-21 Sequana Medical Ag Systems and methods for regulating inductive energy transfer to an implantable system
BR112015022585B1 (pt) 2013-03-15 2023-04-04 Regeneron Pharmaceuticals, Inc Composto, composição farmacêutica, conjugado droga-anticorpo, e, uso dos mesmos
MX373959B (es) 2013-03-15 2020-07-13 Sarepta Therapeutics Inc Composición para usarse en el tratamiento de la distrofia muscular de duchenne (dmd).
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
CN109234274B (zh) * 2013-03-27 2021-09-21 伊萨纳治疗有限公司 修饰的TGF-β寡核苷酸
US9295731B2 (en) 2013-04-01 2016-03-29 Mark Quang Nguyen Cleavable drug conjugates, compositions thereof and methods of use
US9861574B2 (en) 2013-04-09 2018-01-09 Duke University 2-fluoro-modified RNAs as immunostimulators
US9849066B2 (en) 2013-04-24 2017-12-26 Corning Incorporated Delamination resistant pharmaceutical glass containers containing active pharmaceutical ingredients
GB2529356B (en) 2013-04-28 2020-12-23 Genequantum Healthcare Co Ltd Novel linkers, coupling intermediates, conjugates, preparation method and application thereof
HRP20240107T1 (hr) 2013-05-20 2024-04-12 F. Hoffmann - La Roche Ag Protutijela na transferinski receptor i postupci uporabe
DK3004174T3 (da) 2013-05-31 2019-07-22 Zymeworks Inc Heteromultimerer med reduceret eller nedreguleret effektorfunktion
KR102733409B1 (ko) 2013-06-05 2024-11-21 듀크 유니버시티 Rna-가이드 유전자 편집 및 유전자 조절
US11229711B2 (en) 2013-06-06 2022-01-25 Magenta Therapeutics, Inc. 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
WO2015048792A1 (en) 2013-09-30 2015-04-02 The Regents Of The University Of California Identification of structurally similar small molecules that enhance therapeutic exon skipping
KR102538993B1 (ko) 2013-10-15 2023-06-02 씨젠 인크. 개선된 리간드-약물 컨쥬게이트 약물동력학을 위한 peg화된 약물-링커
CA2934030A1 (en) 2013-10-15 2015-04-23 Sorrento Therapeutics Inc. Drug-conjugates with a targeting molecule and two different drugs
EP3065758B1 (en) 2013-11-06 2019-09-25 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
US10378017B2 (en) 2013-12-02 2019-08-13 Brandeis University High temperature selection of nucleotide-supported carbohydrate vaccines and resulting glycosylated oligonucleotides
CN106068325B (zh) 2014-01-16 2021-07-09 波涛生命科学有限公司 手性设计
WO2015113922A1 (en) 2014-01-30 2015-08-06 Roche Innovation Center Copenhagen A/S Poly oligomer compound with biocleavable conjugates
EP3514234A1 (en) 2014-03-12 2019-07-24 Nippon Shinyaku Co., Ltd. Antisense nucleic acid
MA39941A (fr) 2014-05-01 2017-03-08 Larry J Smith Procédés et modifications permettant de produire des composés arni simple brin à activité, potentiel et durée d'effet améliorés
US20170182189A1 (en) 2014-05-23 2017-06-29 Genzyme Corporation Inhibiting or downregulating glycogen synthase by creating premature stop codons using antisense oligonucleotides
UA121117C2 (uk) 2014-06-17 2020-04-10 Ніппон Шин'Яку Ко., Лтд. Антисенсова нуклеїнова кислота
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
CA2953347A1 (en) 2014-06-23 2015-12-30 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
JP6779876B2 (ja) 2014-11-19 2020-11-04 ジェネンテック， インコーポレイテッド 抗トランスフェリン受容体抗体及びその使用方法
JP7260248B2 (ja) 2015-01-16 2023-04-18 シティ・オブ・ホープ 細胞透過性抗体
DK3277814T3 (da) 2015-04-03 2020-08-24 Univ Massachusetts Oligonukleotidforbindelser til målretning mod huntingtin-mrna
WO2016170348A2 (en) 2015-04-22 2016-10-27 Mina Therapeutics Limited Sarna compositions and methods of use
US20180344817A1 (en) 2015-05-01 2018-12-06 Precision Biosciences, Inc. Precise deletion of chromosomal sequences in vivo and treatment of nucleotide repeat expansion disorders using engineered nucleases
MX388743B (es) 2015-05-19 2025-03-20 Sarepta Therapeutics Inc Conjugados oligonucleótido-péptido.
AU2016282334B2 (en) 2015-06-24 2022-07-21 F. Hoffmann-La Roche Ag Anti-transferrin receptor antibodies with tailored affinity
CN115957337A (zh) 2015-08-07 2023-04-14 箭头药业股份有限公司 乙型肝炎病毒感染的RNAi疗法
WO2017059131A1 (en) 2015-09-30 2017-04-06 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
JP2019515874A (ja) 2016-03-01 2019-06-13 エフ・ホフマン−ラ・ロシュ・アクチェンゲゼルシャフト 改変された細胞死誘導を有するオビヌツズマブ変異体
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
MA45470A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques kras et leurs utilisations
MA45290A (fr) 2016-05-04 2019-03-13 Wave Life Sciences Ltd Procédés et compositions d'agents biologiquement actifs
JP6823269B2 (ja) 2016-06-20 2021-02-03 株式会社ＧｅｎＡｈｅａｄ Ｂｉｏ 抗体−薬物コンジュゲート
WO2018002812A1 (en) 2016-06-29 2018-01-04 Crispr Therapeutics Ag Materials and methods for treatment of myotonic dystrophy type 1 (dm1) and other related disorders
JP7157052B2 (ja) 2016-10-28 2022-10-19 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
EP3532614A1 (en) 2016-10-28 2019-09-04 Genethon Compositions and methods for the treatment of myotonic dystrophy
AU2017363892B2 (en) 2016-11-23 2023-06-15 Alnylam Pharmaceuticals, Inc. Modified RNA agents with reduced off-target effect
EP3554553B1 (en) 2016-12-19 2022-07-20 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
KR20190104381A (ko) 2017-01-06 2019-09-09 어비디티 바이오사이언시스 엘엘씨 핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
DK3444010T3 (da) 2017-08-14 2021-03-15 Scandiedge Therapeutics Ab Pklr-inhibering i behandlingen af nafld og hcc
KR20250065737A (ko) 2017-09-22 2025-05-13 어비디티 바이오사이언시스 인크. 핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법
MX2020003596A (es) 2017-10-04 2020-07-22 Avidity Biosciences Inc Composiciones de acido nucleico-polipeptido y usos de las mismos.
JP7506601B2 (ja) 2017-12-06 2024-06-26 アビディティー バイオサイエンシーズ，インク． 筋萎縮症と筋緊張性ジストロフィーを処置するための組成物および方法
MA51583A (fr) 2018-01-04 2020-11-11 Avidity Biosciences Inc Molécules d'acide nucléique hétéroduplex et leurs utilisations
WO2019232302A1 (en) 2018-06-01 2019-12-05 Massachusetts Institute Of Technology Optoelectronic memristor devices
SG11202100934PA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating dystrophinopathies
EP3829649A4 (en) 2018-08-02 2022-05-04 Dyne Therapeutics, Inc. ANTI-MUSCLE COMPLEXES AND THEIR USES
SG11202100928QA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
CN109020990A (zh) 2018-09-20 2018-12-18 广西壮族自治区药用植物园 苯基苯并呋喃化合物、及其制备方法、组合物和医药用途
IL319265A (en) 2018-12-21 2025-04-01 Avidity Biosciences Inc Anti-transferrin receptor antibodies and their uses
WO2020247818A1 (en) 2019-06-06 2020-12-10 Avidity Biosciences, Inc. Una amidites and uses thereof
CA3142283A1 (en) 2019-06-06 2020-12-10 Avidity Biosciences, Inc. Nucleic acid-polypeptide compositions and uses thereof
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
CA3172111A1 (en) 2020-03-19 2021-09-23 Barbora MALECOVA Compositions and methods of treating facioscapulohumeral muscular dystrophy
EP4656242A2 (en) 2020-03-27 2025-12-03 Avidity Biosciences, Inc. Compositions and methods of treating muscle dystrophy
WO2021226107A1 (en) 2020-05-05 2021-11-11 Avidity Biosciences, Inc. Compositions and methods of treating pompe disease
CN118591391A (zh) 2021-07-09 2024-09-03 达因疗法公司 肌肉靶向复合物及其用于治疗肌养蛋白病的用途
US11638761B2 (en) 2021-07-09 2023-05-02 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
US12071621B2 (en) 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006128138A2 (en) * 2005-05-26 2006-11-30 Ensemble Discovery Corportion Biodetection by nucleic acid-templated chemistry
US20130172238A1 (en) * 2010-06-11 2013-07-04 Andreas Mitsch Method for selective oligonucleotide modification

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