KR102775461B1 - 핵산-폴리펩타이드 조성물 및 이의 용도 - Google Patents

핵산-폴리펩타이드 조성물 및 이의 용도 Download PDF

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KR102775461B1
KR102775461B1 KR1020247005800A KR20247005800A KR102775461B1 KR 102775461 B1 KR102775461 B1 KR 102775461B1 KR 1020247005800 A KR1020247005800 A KR 1020247005800A KR 20247005800 A KR20247005800 A KR 20247005800A KR 102775461 B1 KR102775461 B1 KR 102775461B1
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polynucleic acid
acid molecule
molecule
polynucleotide
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KR20240026257A (ko
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앤드루 존 길
벤카타 라마나 도팔라푸디
데이빗 사이호 추
마이클 캐러미언 코크런
레이첼 엘리자베스 존스
팔라니 발루
롭 버크
비어트리스 다이애나 다리몬트
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어비디티 바이오사이언시스 인크.
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    • C07—ORGANIC CHEMISTRY
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    • A61K47/6889—Conjugates wherein the antibody being the modifying agent and wherein the linker, binder or spacer confers particular properties to the conjugates, e.g. peptidic enzyme-labile linkers or acid-labile linkers, providing for an acid-labile immuno conjugate wherein the drug may be released from its antibody conjugated part in an acidic, e.g. tumoural or environment
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Families Citing this family (80)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HUE054526T2 (hu) 2014-10-29 2021-09-28 Bicyclerd Ltd MT1-MMP-ra specifikus biciklusos peptid ligandumok
EP3277811B1 (en) 2015-04-03 2020-12-23 University of Massachusetts Fully stabilized asymmetric sirna
EP3334499A4 (en) 2015-08-14 2019-04-17 University of Massachusetts BIOACTIVE CONJUGATES FOR THE RELEASE OF OLIGONUCLEOTIDES
US10478503B2 (en) 2016-01-31 2019-11-19 University Of Massachusetts Branched oligonucleotides
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
EP3473270B1 (en) * 2016-06-20 2022-11-30 Genahead Bio, Inc. Antibody-drug conjugate
US11753638B2 (en) 2016-08-12 2023-09-12 University Of Massachusetts Conjugated oligonucleotides
JP7573966B2 (ja) * 2017-01-06 2024-10-28 アビディティー バイオサイエンシーズ，インク． エクソンスキッピングを誘導する核酸ポリペプチド組成物および方法
EP3642341A4 (en) 2017-06-23 2021-06-16 University Of Massachusetts TWO-DAY SELF-RELEASING SIRNA AND RELATED PROCEDURES
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
US11261214B2 (en) 2017-08-04 2022-03-01 Bicycletx Limited Bicyclic peptide ligand specific for CD137
SG11201912188RA (en) * 2017-09-14 2020-01-30 Arrowhead Pharmaceuticals Inc Rnai agents and compositions for inhibiting expression of angiopoietin-like 3 (angptl3), and methods of use
CN118267396A (zh) * 2017-10-04 2024-07-02 艾维迪提生物科学公司 核酸-多肽组合物及其用途
IL319835A (en) 2017-12-06 2025-05-01 Avidity Biosciences Inc Compositions and methods for treating muscular dystrophy and myotonic dystrophy
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
WO2019136180A2 (en) * 2018-01-04 2019-07-11 Avidity Biosciences Llc Heteroduplex nucleic acid molecules and uses thereof
US10875894B2 (en) 2018-02-23 2020-12-29 Bicycletx Limited Multimeric bicyclic peptide ligands
MX2020010444A (es) * 2018-04-04 2021-01-08 Bicycletx Ltd Complejos de péptidos bicíclicos en heterotándem.
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
MX2021001281A (es) 2018-08-02 2021-07-15 Dyne Therapeutics Inc Complejos dirigidos a músculos y sus usos para el tratamiento de distrofinopatías.
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
EA202190416A1 (ru) 2018-08-02 2021-06-23 Дайн Терапьютикс, Инк. Мышечно-специфические комлексы и их применение для лечения плече-лопаточно-лицевой мышечной дистрофии
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
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
BR112021002440A2 (pt) 2018-08-10 2021-05-04 University Of Massachusetts oligonucleotídeos modificados visando snps
JP7570107B2 (ja) 2018-08-31 2024-10-21 イェール ユニバーシティー ドナーオリゴヌクレオチドベースの遺伝子編集を亢進するための組成物および方法
US12331320B2 (en) 2018-10-10 2025-06-17 The Research Foundation For The State University Of New York Genome edited cancer cell vaccines
GB201820288D0 (en) 2018-12-13 2019-01-30 Bicycle Tx Ltd Bicycle peptide ligaands specific for MT1-MMP
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
IL319265A (en) 2018-12-21 2025-04-01 Avidity Biosciences Inc Anti-transferrin receptor antibodies and their uses
EP3897849A1 (en) 2018-12-21 2021-10-27 BicycleTx Limited Bicyclic peptide ligands specific for pd-l1
JP2022523467A (ja) * 2019-01-18 2022-04-25 ユニバーシティ・オブ・マサチューセッツ 動的な薬物動態を修飾するアンカー
CA3137715A1 (en) 2019-04-25 2020-10-29 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
AU2020289464B2 (en) 2019-06-06 2025-12-04 Avidity Biosciences, Inc. Nucleic acid-polypeptide compositions and uses thereof
CN114375297A (zh) 2019-06-06 2022-04-19 艾维迪提生物科学公司 Una亚酰胺及其用途
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
WO2021030213A1 (en) 2019-08-09 2021-02-18 University Of Massachusetts Chemically modified oligonucleotides targeting snps
US12485180B2 (en) 2019-08-30 2025-12-02 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
EP4592309A3 (en) 2019-10-03 2025-10-22 BicycleTx Limited Heterotandem bicyclic peptide complexes
US12491255B2 (en) 2019-10-14 2025-12-09 Aro Biotherapeutics Company EPCAM binding fibronectin type III domains
US11781138B2 (en) * 2019-10-14 2023-10-10 Aro Biotherapeutics Company FN3 domain-siRNA conjugates and uses thereof
CN119119240A (zh) 2019-10-14 2024-12-13 Aro生物疗法公司 结合cd71的纤维粘连蛋白iii型结构域
WO2021113851A2 (en) * 2019-12-06 2021-06-10 Sirnaomics, Inc. Peptide docking vehicle for targeted nucleic acid delivery
KR20220156867A (ko) 2020-03-19 2022-11-28 어비디티 바이오사이언시스 인크. 안면견갑상완 근이영양증의 치료용 조성물 및 방법
EP4126066B1 (en) 2020-03-27 2025-11-19 Avidity Biosciences, Inc. Compositions and methods of treating muscle dystrophy
EP4631524A3 (en) * 2020-06-24 2025-12-17 Sapreme Technologies B.V. Combination comprising an adc or an aoc comprising a vhh, and a saponin or a ligand-saponin conjugate
EP4192506A1 (en) * 2020-08-07 2023-06-14 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
WO2022221505A2 (en) 2021-04-14 2022-10-20 Aro Biotherapeutics Company Cd71 binding fibronectin type iii domains
IL307595A (en) 2021-04-14 2023-12-01 Aro Biotherapeutics Company FN3 RNA-silencing region conjugates and their uses
IL309334A (en) 2021-06-23 2024-02-01 Univ Massachusetts Anti-FLT1 optimized oligonucleotide compounds for the treatment of preeclampsia and other angiogenic disorders
US11648318B2 (en) 2021-07-09 2023-05-16 Dyne Therapeutics, Inc. Anti-transferrin receptor (TFR) antibody and uses thereof
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
JP2024525608A (ja) 2021-07-09 2024-07-12 ダイン セラピューティクス，インコーポレーテッド ジストロフィン異常症を処置するための筋標的化複合体および製剤
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11633498B2 (en) 2021-07-09 2023-04-25 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
MX2024002753A (es) 2021-09-01 2024-05-20 Biogen Ma Inc Anticuerpos anti receptor de transferrina y usos de los mismos.
EP4401792A4 (en) 2021-09-16 2025-12-03 Avidity Biosciences Inc COMPOSITIONS AND METHODS OF TREATMENT OF FACIO-SCAPULO-HUMERAL MUSCULAR DYSTROPHY
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
AU2022422149A1 (en) 2021-12-23 2024-08-01 Mirecule, Inc. Compositions for delivery of polynucleotides
US12071621B2 (en) 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping
EP4508090A1 (en) 2022-04-15 2025-02-19 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating myotonic dystrophy
CA3263853A1 (en) * 2022-08-08 2024-02-15 Eli Lilly And Company TRANSFERRIN RECEPTOR-BINDING PROTEINS AND CONJUGATES
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
WO2024187057A2 (en) 2023-03-08 2024-09-12 Biogen Ma Inc. Anti-transferrin receptor antibodies and uses thereof
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
WO2025006639A2 (en) 2023-06-27 2025-01-02 Avidity Biosciences, Inc. Compositions and methods of using prkag2-targeting antibody-oligonucleotide conjugates abstract
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

Family Cites Families (318)

* 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
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
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
ATE124999T1 (de) 1985-03-15 1995-07-15 Antivirals Inc Immunotestmittel für polynukleotid und verfahren.
US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group Polynucleotide assay reagent and method
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
DE552178T1 (de) 1990-10-12 1994-02-03 Max Planck Gesellschaft Abgeänderte ribozyme.
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
FI950730A7 (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
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
CN1120156C (zh) 1994-06-17 2003-09-03 弗·哈夫曼-拉罗切有限公司 N,n′－二(喹啉－4－基)－二胺衍生物、其制备及作为抗疟药的应用
US5716824A (en) 1995-04-20 1998-02-10 Ribozyme Pharmaceuticals, Inc. 2'-O-alkylthioalkyl and 2-C-alkylthioalkyl-containing enzymatic nucleic acids (ribozymes)
US5939045A (en) 1994-12-22 1999-08-17 Nissan Chemical Industries, Ltd. Organic bismuth derivatives for X-ray imaging
US5889136A (en) 1995-06-09 1999-03-30 The Regents Of The University Of Colorado Orthoester protecting groups in RNA synthesis
EP0886641A2 (en) 1996-01-16 1998-12-30 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
US9096636B2 (en) 1996-06-06 2015-08-04 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds and their use in gene modulation
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
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
US6194551B1 (en) 1998-04-02 2001-02-27 Genentech, Inc. Polypeptide variants
US6528624B1 (en) 1998-04-02 2003-03-04 Genentech, Inc. Polypeptide variants
IL138608A0 (en) 1998-04-02 2001-10-31 Genentech Inc Antibody variants and fragments thereof
CA2326823A1 (en) 1998-04-20 1999-10-28 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
ES2260570T3 (es) 1998-08-27 2006-11-01 Spirogen Limited Pirrolobenziodiazepinas dimericas.
GB9818730D0 (en) 1998-08-27 1998-10-21 Univ Portsmouth Collections of compounds
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
US7479276B1 (en) 1999-02-22 2009-01-20 Synergene Therapeutics, Inc. 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
DE10100586C1 (de) 2001-01-09 2002-04-11 Ribopharma Ag Verfahren zur Hemmung der Expression eines Ziegens
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
CA2328356A1 (en) 1999-12-22 2001-06-22 Itty Atcravi Recreational vehicles
NZ518764A (en) 1999-12-29 2004-02-27 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
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)
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
DK2028278T3 (da) 2000-03-30 2014-06-23 Univ Massachusetts RNA-sekvensspecifikke formidlere af RNA-interferens
DE60140676D1 (de) 2000-03-30 2010-01-14 Massachusetts Inst Technology Mediatoren von rns-interferenz, die rns-sequenzspezifisch sind
WO2001083740A2 (en) 2000-05-04 2001-11-08 Avi Biopharma, Inc. Splice-region antisense composition and method
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
CZ308053B6 (cs) 2000-12-01 2019-11-27 Max Planck Gesellschaft Izolovaná molekula dvouřetězcové RNA, způsob 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
CN1671416B (zh) 2001-07-12 2013-01-02 杰斐逊·富特 超人源化抗体
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
AU2003207708A1 (en) 2002-02-20 2003-09-09 Sirna Therapeutics, Inc. Rna interference 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)
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
SI1545613T1 (sl) 2002-07-31 2011-11-30 Seattle Genetics Inc Avristatinski konjugati in njihova uporaba za zdravljenje raka avtoimunske bolezni ali infekcijskebolezni
EP1857547B2 (en) 2002-08-05 2020-12-02 Silence Therapeutics GmbH Further novel forms of interfering 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)
US8090542B2 (en) 2002-11-14 2012-01-03 Dharmacon Inc. Functional and hyperfunctional siRNA
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
EP2392660A3 (en) 2002-11-25 2012-03-28 Masafumi Matsuo ENA Nucleic Acid Drugs Modifying Splicing in mRNA Precursor
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
EP1608733B1 (en) 2003-04-02 2011-12-07 Dharmacon, Inc. Modified polynucleotides for use in rna interference
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
CA2527958C (en) 2003-06-02 2014-04-08 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of rnai
EP1633767B1 (en) 2003-06-02 2018-11-21 University of Massachusetts Methods and compositions for controlling efficacy of rna silencing
US7750144B2 (en) 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
EP3604537B1 (en) 2003-06-13 2021-12-08 Alnylam Europe AG 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
KR101438983B1 (ko) 2003-11-06 2014-09-05 시애틀 지네틱스, 인크. 리간드에 접합될 수 있는 모노메틸발린 화합물
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
JP5166861B2 (ja) 2004-03-09 2013-03-21 スピロゲン リミティッド ピロロベンゾジアゼピン
US20050277610A1 (en) 2004-03-15 2005-12-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
US20060029597A1 (en) * 2004-03-31 2006-02-09 Chimeric Technologies 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
EP2399924B1 (en) 2004-05-27 2015-01-28 Alnylam Pharmaceuticals, Inc. Nuclease resistant double-stranded ribonucleic acid
EP2206781B1 (en) 2004-06-28 2015-12-02 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
CA2602663A1 (en) * 2005-03-31 2006-10-05 Xencor, Inc. Fc variants with optimized properties
WO2006111759A1 (en) 2005-04-21 2006-10-26 Spirogen Limited Pyrrolobenzodiazepines
JP2008538500A (ja) 2005-04-22 2008-10-30 アカデミス ツィーケンホイス ライデン ＳＲタンパク質の結合に対する干渉とＲＮＡ二次構造に対する干渉による、ｍＲＮＡ前駆体におけるエクソン認識の調節
KR20080028886A (ko) * 2005-05-26 2008-04-02 앙상블 디스커버리 코포레이션 핵산-템플레이팅된 화학 반응에 의한 생검출
US7736647B2 (en) * 2005-06-15 2010-06-15 Monoclonal Antibodies Therapeutics Anti-CD71 monoclonal antibodies and uses thereof for treating malignant tumor cells
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
DK1931780T3 (en) 2005-08-29 2016-01-25 Regulus Therapeutics Inc Antisense-forbindelser med forøget anti-microrna-aktivitet
EP1937312B1 (en) 2005-08-30 2016-06-29 Ionis 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
SI1813614T1 (sl) 2006-01-25 2012-01-31 Sanofi 174 Citotoksična sredstva, ki obsegajo nove tomajmicinske derivate
US7750116B1 (en) 2006-02-18 2010-07-06 Seattle Genetics, Inc. Antibody drug conjugate metabolites
CA2651881A1 (en) 2006-05-10 2008-03-27 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
AU2007272995B2 (en) 2006-07-10 2013-02-07 Biogen Idec Ma Inc. Compositions and methods for inhibiting growth of smad4-deficient cancers
DK2049664T3 (da) 2006-08-11 2012-01-02 Prosensa Technologies Bv Enkeltstrengede oligonukleotider, som er komplementære til repetitive elementer, til behandling af med DNA-repetitiv-ustabilitet associerede lidelser
KR101133799B1 (ko) 2006-08-18 2012-04-24 에프. 호프만-라 로슈 아게 폴리뉴클레오타이드의 생체내 전달을 위한 다가접합체
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
WO2008138561A1 (en) 2007-05-10 2008-11-20 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
WO2009005793A2 (en) 2007-06-29 2009-01-08 Avi Biopharma, Inc. Tissue specific peptide conjugates and methods
ES2435779T3 (es) 2007-07-19 2013-12-23 Sanofi Agentes citotóxicos que comprenden nuevos derivados de tomaimicina y su uso terapéutico
DK2193140T3 (en) 2007-08-27 2017-01-23 Iglobe Health Inst Llc COMPOSITIONS OF ASYMMETRIC INTERFERRING RNA AND ITS APPLICATIONS
EP2190995A2 (en) 2007-09-18 2010-06-02 Intradigm Corporation Compositions comprising k-ras sirna and methods of use
WO2009039173A2 (en) 2007-09-19 2009-03-26 Applied Biosystems Inc. SiRNA SEQUENCE-INDEPENDENT MODIFICATION FORMATS FOR REDUCING OFF-TARGET PHENOTYPIC EFFECTS IN RNAi, AND STABILIZED FORMS THEREOF
LT2211904T (lt) 2007-10-19 2016-11-25 Seattle Genetics, Inc. Cd19 surišantys agentai ir jų panaudojimas
EP2607484B1 (en) 2008-10-27 2016-01-06 BioMarin Technologies B.V. Methods and means for efficient skipping of exon 45 in Duchenne Muscular Dystrophy pre-mRNA
ES2564563T3 (es) 2007-10-26 2016-03-23 Academisch Ziekenhuis Leiden Medios y métodos para contrarrestar trastornos del músculo
WO2009061941A2 (en) 2007-11-06 2009-05-14 Osmetech Molecular Diagnostics Baseless nucleotide analogues and uses thereof
US20110033378A1 (en) 2008-01-18 2011-02-10 Medlmmune, Llc. Cysteine Engineered Antibodies For Site-Specific Conjugation
US20110065774A1 (en) 2008-01-31 2011-03-17 Alnylam Pharmaceuticals Chemically modified oligonucleotides and uses thereof
US20110081362A1 (en) 2008-01-31 2011-04-07 The Brigham And Women's Hospital, Inc. Treatment of cancer
RS58948B1 (sr) 2008-02-01 2019-08-30 Ascendis Pharma As Prolek koji sadrži linker koji se može sam odcepiti
US10131904B2 (en) 2008-02-11 2018-11-20 Rxi Pharmaceuticals Corporation Modified RNAi polynucleotides and uses thereof
EP2265283B1 (en) 2008-03-18 2014-09-03 Seattle Genetics, Inc. Auristatin drug linker conjugates
CA3044980A1 (en) 2008-04-11 2009-10-15 Alnylam Pharmaceuticals, Inc. Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
TWI538916B (zh) 2008-04-11 2016-06-21 介控生化科技公司 經修飾的因子vii多肽和其用途
AU2009236270B2 (en) 2008-04-15 2014-06-26 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
WO2009144481A2 (en) 2008-05-30 2009-12-03 Isis Innovation Limited Conjugates for delivery 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
JP5583667B2 (ja) 2008-08-08 2014-09-03 ジェニスフィア・エルエルシー 長時間作用型ｄｎａデンドリマーおよびその方法
ES2915692T3 (es) 2008-08-13 2022-06-24 California Inst Of Techn Nanopartículas portadoras y composiciones, métodos y sistemas relacionados
US9433684B2 (en) 2008-08-19 2016-09-06 Nektar Therapeutics Conjugates of small-interfering nucleic acids
EP2331690B1 (en) 2008-09-02 2016-01-13 Alnylam Pharmaceuticals Inc. 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
US8664189B2 (en) 2008-09-22 2014-03-04 Rxi Pharmaceuticals Corporation RNA interference in skin indications
AU2013204838B2 (en) 2008-10-24 2016-12-22 Sarepta Therapeutics, Inc. Multiple exon skipping compositions for DMD
CA2740328A1 (en) 2008-10-24 2010-04-29 Avi Biopharma, Inc. Multiple exon skipping compositions for dmd
KR101602431B1 (ko) 2008-11-05 2016-03-10 삼성전자 주식회사 냉장고
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
CN105175434A (zh) 2009-02-05 2015-12-23 伊缪诺金公司 新型苯并二氮杂*衍生物
US8481698B2 (en) 2009-03-19 2013-07-09 The President And Fellows Of Harvard College Parallel proximity ligation event analysis
NZ594339A (en) * 2009-03-25 2013-06-28 Genentech Inc Anti-fgfr3 antibodies and methods using same
CA2757354A1 (en) 2009-04-02 2010-10-07 Laura P.W. Ranum Nucleotide repeat expansion-associated polypeptides and uses thereof
HUE028036T2 (en) 2009-04-10 2016-11-28 Ass Inst De Myologie Tricyclic DNA anti-sense oligonucleotides, compositions and methods for treating a disease
US20120046342A1 (en) 2009-04-24 2012-02-23 Prosensa 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
RU2569186C2 (ru) 2009-06-04 2015-11-20 Новартис Аг СПОСОБЫ ИДЕНТИФИКАЦИИ САЙТОВ ДЛЯ КОНЪЮГАЦИИ IgG
JP5918129B2 (ja) 2009-06-22 2016-05-18 メディミューン，エルエルシー 部位特異的共役のための操作されたＦｃ領域
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
EP2456470A1 (en) 2009-07-22 2012-05-30 Cenix Bioscience GmbH Delivery system and conjugates for compound delivery via naturally occurring intracellular transport routes
IT1394860B1 (it) 2009-07-22 2012-07-20 Kemotech S R L Composti farmaceutici
US20120172415A1 (en) 2009-08-31 2012-07-05 Thomas Voit Exon Skipping Therapy for Functional Amelioration of Semifunctional Dystrophin in Becker and Duchenne Muscular Dystrophy
CN102666874B (zh) 2009-10-07 2016-06-01 宏观基因有限公司 由于岩藻糖基化程度的改变而表现出改善的效应子功能的含Fc区的多肽及其使用方法
LT2499249T (lt) 2009-11-12 2018-12-27 The University Of Western Australia Priešprasmės molekulės ir patologijų gydymo būdai
WO2011078797A2 (en) 2009-12-22 2011-06-30 Singapore Health Services Pte. Ltd Antisense oligonucleotides and uses threreof
KR20130028055A (ko) 2010-01-28 2013-03-18 글락소 그룹 리미티드 Ｃｄ １２７ 결합 단백질
WO2011130371A1 (en) 2010-04-13 2011-10-20 Life Technologies Corporation Compositions and methods for inhibition of nucleic acids function
MX368648B (es) 2010-04-15 2019-10-09 Seattle Genetics Inc Pirrolobenzodiazepinas usadas para tratar enfermedades proliferativas.
BR112012026801B8 (pt) 2010-04-15 2021-05-25 Medimmune Ltd conjugados de pirrolobenzodiazepina direcionados, composição farmacêutica, uso dos mesmos para tratamento de uma doença proliferativa ou autoimune e ligante de medicamento
US8993738B2 (en) 2010-04-28 2015-03-31 Isis Pharmaceuticals, Inc. Modified nucleosides, analogs thereof and oligomeric compounds prepared therefrom
EP2625186B1 (en) 2010-04-28 2016-07-27 Ionis Pharmaceuticals, Inc. 5' modified nucleosides and oligomeric compounds prepared therefrom
KR102182663B1 (ko) 2010-05-28 2020-11-25 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
CA2805765A1 (en) 2010-07-19 2012-01-26 C. Frank Bennett Modulation of dystrophia myotonica-protein kinase (dmpk) expression
EP2409983A1 (en) 2010-07-19 2012-01-25 Leibniz-Institut für Pflanzenbiochemie (IPB) Tubulysin analogues
EP3192880B1 (en) 2010-08-18 2019-10-09 Fred Hutchinson Cancer Research Center Nucleic acid agents for use in treating facioscapulohumeral dystrophy (fshd)
US20130195799A1 (en) 2010-08-19 2013-08-01 Peg Biosciences, Inc. Synergistic biomolecule-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
EP2632472B1 (en) 2010-10-29 2017-12-13 Sirna Therapeutics, Inc. Rna interference mediated inhibition of gene expression using short interfering nucleic acids (sina)
MX2013005046A (es) 2010-11-03 2013-12-12 Immunogen Inc Agentes citotoxicos que comprenden nuevos derivados de ansamitocina.
US8501930B2 (en) 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
MX347298B (es) * 2010-12-29 2017-04-21 Arrowhead Res Corp Conjugados de suministro de polinucleotidos in vivo que tienen acoplamientos sensibles a enzima.
MX2013009191A (es) 2011-02-08 2013-11-04 Charlotte Mecklenburg Hospital Oligonucleotidos antisentido.
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
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
JP5980202B2 (ja) * 2011-05-09 2016-08-31 株式会社ペルセウスプロテオミクス トランスフェリン受容体を特異的に認識できる抗体
WO2012177837A2 (en) 2011-06-21 2012-12-27 Immunogen, Inc. Novel maytansinoid derivatives with peptide linker and conjugates thereof
WO2013012752A2 (en) 2011-07-15 2013-01-24 Sarepta Therapeutics, Inc. Methods and compositions for manipulating translation of protein isoforms from alternative initiation start sites
ES2626488T3 (es) 2011-07-19 2017-07-25 Wave Life Sciences Pte. Ltd. Procedimientos para la síntesis de ácidos 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
EP3640332A1 (en) 2011-08-29 2020-04-22 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
US9302014B2 (en) 2011-08-30 2016-04-05 Medical Research Council Cell-penetrating peptides having a central hydrophobic domain
JP6393617B2 (ja) 2011-10-14 2018-09-19 シアトル ジェネティクス，インコーポレーテッド ピロロベンゾジアゼピンおよび標的コンジュゲート
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
CN105861503A (zh) 2011-11-18 2016-08-17 阿尔尼拉姆医药品有限公司 修饰的RNAi试剂
US20130224228A1 (en) 2011-12-05 2013-08-29 Igenica, 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
CA3111357A1 (en) 2011-12-23 2013-06-27 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
WO2013110005A1 (en) * 2012-01-18 2013-07-25 Wisconsin Alumni Research Foundation Boronate-mediated delivery of molecules into cells
CN118581086A (zh) 2012-01-27 2024-09-03 比奥马林技术公司 用于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
US10010623B2 (en) 2012-02-16 2018-07-03 Ucl Business Plc Lysosome-cleavable linker
BR112014020697B1 (pt) 2012-02-24 2024-01-09 Alteogen Inc Anticorpo modificado compreendendo um motivo contendo cisteína na terminação do anticorpo, conjugado anticorpo-droga modificado compreendendo uma droga ligada ao anticorpo modificado e método de produção do mesmo
WO2013138662A1 (en) 2012-03-16 2013-09-19 4S3 Bioscience, Inc. Antisense conjugates for decreasing expression of dmpk
US20150238515A1 (en) 2012-05-02 2015-08-27 Arrowhead Research Corporation Organic compositions to treat kras-related diseases
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
ES2887208T3 (es) 2012-05-15 2021-12-22 Concortis Biosystems Corp Conjugados de fármacos y usos de los mismos
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
ES2940887T3 (es) 2012-07-13 2023-05-12 Wave Life Sciences Ltd Método de preparación de oligonucleótidos quirales
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
AU2013328580B2 (en) 2012-10-12 2016-01-21 Medimmune Limited Pyrrolobenzodiazepines and conjugates thereof
JP6386461B2 (ja) 2012-10-26 2018-09-05 エヌライフ、セラピューティックス、ソシエダッド、リミターダＮｌｉｆｅ Ｔｈｅｒａｐｅｕｔｉｃｓ， Ｓ．Ｌ． 特定のニューロン種へのオリゴヌクレオチド分子の選択的送達によるパーキンソン病の治療のための組成物および方法
WO2014069520A1 (ja) 2012-10-31 2014-05-08 武田薬品工業株式会社 新規修飾核酸
WO2014076196A1 (en) 2012-11-15 2014-05-22 Santaris Pharma A/S Anti apob antisense conjugate compounds
EP2922818B1 (en) 2012-11-24 2018-09-05 Hangzhou Dac Biotech Co., Ltd Hydrophilic linkers and their uses for conjugation of drugs to cell binding molecules
AU2013364158A1 (en) 2012-12-20 2015-07-09 Sarepta Therapeutics, Inc. Improved exon skipping compositions for treating muscular dystrophy
RU2015129800A (ru) 2012-12-21 2017-01-30 Биоэллаенс К. В. Гидрофильные саморазрушающиеся линкеры и их конъюгаты
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
AR096287A1 (es) 2013-03-13 2015-12-23 Spirogen Sàrl Pirrolobenzodiacepinas y conjugados
BR112015023001B8 (pt) 2013-03-14 2022-08-09 Sarepta Therapeutics Inc Oligonucleotídeo antisenso, composição farmacêutica compreendendo o mesmo e uso da dita composição para o tratamento de 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
AU2014233456B2 (en) 2013-03-15 2019-02-21 Sarepta Therapeutics, Inc. Improved 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
MY183572A (en) 2013-03-15 2021-02-26 Regeneron Pharma Biologically active molecules, conjugates thereof, and therapeutic uses
CA2908096C (en) * 2013-03-27 2022-05-03 Isarna Therapeutics Gmbh Modified tgf-beta oligonucleotides
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
US20180104349A9 (en) 2013-04-28 2018-04-19 Gang Qin Novel linker, preparation method, and application thereof
AU2014268726B2 (en) 2013-05-20 2019-10-31 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
ES2987399T3 (es) 2013-06-05 2024-11-14 Univ Duke Edición génica guiada por ARN y regulación génica
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
EP3052654A4 (en) 2013-09-30 2017-05-03 The Regents of the University of California Identification of structurally similar small molecules that enhance therapeutic exon skipping
CA3187392A1 (en) 2013-10-15 2015-04-23 Seagen Inc. Pegylated drug-linkers for improved ligand-drug conjugate pharmacokinetics
RU2016134258A (ru) 2013-10-15 2018-02-28 Сорренто Терапьютикс Инк. Конъюгат лекарственного средства с направляющей молекулой и двумя различными лекарственными средствами
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
US10378017B2 (en) 2013-12-02 2019-08-13 Brandeis University High temperature selection of nucleotide-supported carbohydrate vaccines and resulting glycosylated oligonucleotides
CN113278617A (zh) 2014-01-16 2021-08-20 波涛生命科学有限公司 手性设计
US10358643B2 (en) 2014-01-30 2019-07-23 Hoffmann-La Roche, Inc. Poly oligomer compound with biocleavable conjugates
NZ724836A (en) 2014-03-12 2022-07-01 Nippon Shinyaku Co Ltd Antisense nucleic acids
IL295906A (en) * 2014-03-21 2022-10-01 Abbvie Inc Anti-egfr antibodies and antibody drug conjugates
US20170051286A1 (en) 2014-05-01 2017-02-23 Larry J. Smith METHODS AND MODIFICATIONS THAT PRODUCE ssRNAi COMPOUNDS WITH ENHANCED ACTIVITY, POTENCY AND DURATION OF EFFECT
TWI787678B (zh) 2014-05-23 2022-12-21 美商健臻公司 藉由使用反義寡核苷酸創造提前終止密碼子抑制或下調肝醣合成酶
ES2765463T3 (es) 2014-06-17 2020-06-09 Nippon Shinyaku Co Ltd Acido nucleico antisentido para usar en el tratamiento de la distrofia muscular de Duchenne
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
AU2015280252A1 (en) 2014-06-23 2017-01-12 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
EP3221362B1 (en) 2014-11-19 2019-07-24 F.Hoffmann-La Roche Ag Anti-transferrin receptor antibodies and methods of use
WO2016115500A1 (en) 2015-01-16 2016-07-21 City Of Hope Cell penetrating antibodies
EP3766973A1 (en) 2015-04-03 2021-01-20 University Of Massachusetts Oligonucleotide compounds for targeting huntingtin mrna
WO2016170348A2 (en) 2015-04-22 2016-10-27 Mina Therapeutics Limited Sarna compositions and methods of use
EP3289076B1 (en) 2015-05-01 2021-11-17 Precision Biosciences, Inc. Precise deletion of chromoscomal sequences in vivo
CN107921092B (zh) 2015-05-19 2022-04-08 萨勒普塔医疗公司 肽寡核苷酸缀合物
WO2016207240A1 (en) 2015-06-24 2016-12-29 F. Hoffmann-La Roche Ag Anti-transferrin receptor antibodies with tailored affinity
JP7336191B2 (ja) 2015-08-07 2023-08-31 アローヘッド ファーマシューティカルズ インコーポレイテッド Ｂ型肝炎ウイルス感染に対するＲＮＡｉ療法
CN108738310A (zh) 2015-09-30 2018-11-02 萨勒普塔医疗公司 用于治疗肌营养不良的方法
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
EP3473270B1 (en) 2016-06-20 2022-11-30 Genahead Bio, Inc. Antibody-drug conjugate
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
EP3532615A1 (en) 2016-10-28 2019-09-04 Genethon Compositions and methods for the treatment of myotonic dystrophy
CN110337493B (zh) 2016-10-28 2024-03-12 吉尼松公司 用于治疗肌强直性营养不良的组合物和方法
EP3544617A4 (en) 2016-11-23 2020-08-05 Alnylam Pharmaceuticals, Inc. REDUCED OFF-TARGET MODIFIED RNA AGENTS
LT3554553T (lt) 2016-12-19 2022-08-25 Sarepta Therapeutics, Inc. Egzoną šalinantys oligomero konjugatai nuo raumenų distrofojos
JP7573966B2 (ja) 2017-01-06 2024-10-28 アビディティー バイオサイエンシーズ，インク． エクソンスキッピングを誘導する核酸ポリペプチド組成物および方法
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
EP3444010B1 (en) 2017-08-14 2020-12-09 ScandiEdge Therapeutics AB Pklr inhibition in the treatment of nafld and hcc
AU2018335880B2 (en) 2017-09-22 2025-04-10 Avidity Biosciences, Inc. Nucleic acid-polypeptide compositions and methods of inducing exon skipping
CN118267396A (zh) 2017-10-04 2024-07-02 艾维迪提生物科学公司 核酸-多肽组合物及其用途
IL319835A (en) 2017-12-06 2025-05-01 Avidity Biosciences Inc Compositions and methods for treating muscular dystrophy and myotonic dystrophy
WO2019136180A2 (en) 2018-01-04 2019-07-11 Avidity Biosciences Llc Heteroduplex nucleic acid molecules and uses thereof
WO2019232302A1 (en) 2018-06-01 2019-12-05 Massachusetts Institute Of Technology Optoelectronic memristor devices
CA3108335A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle-targeting complexes and uses thereof
MX2021001281A (es) 2018-08-02 2021-07-15 Dyne Therapeutics Inc Complejos dirigidos a músculos y sus usos para el tratamiento de distrofinopatías.
EA202190416A1 (ru) 2018-08-02 2021-06-23 Дайн Терапьютикс, Инк. Мышечно-специфические комлексы и их применение для лечения плече-лопаточно-лицевой мышечной дистрофии
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
AU2020289464B2 (en) 2019-06-06 2025-12-04 Avidity Biosciences, Inc. Nucleic acid-polypeptide compositions and uses thereof
CN114375297A (zh) 2019-06-06 2022-04-19 艾维迪提生物科学公司 Una亚酰胺及其用途
EP3997228A4 (en) 2019-07-09 2024-05-15 The Governors of the University of Alberta SKIP OF EXONS 45 TO 55 USING MUTATION-ADAPTED COCKTAILS, OF ANTISENS MORPHOLINOS, IN THE DMD GENE
KR20220156867A (ko) 2020-03-19 2022-11-28 어비디티 바이오사이언시스 인크. 안면견갑상완 근이영양증의 치료용 조성물 및 방법
EP4126066B1 (en) 2020-03-27 2025-11-19 Avidity Biosciences, Inc. Compositions and methods of treating muscle dystrophy
US20230330246A1 (en) 2020-05-05 2023-10-19 Avidity Biosciences, Inc. Compositions and methods of treating pompe disease
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
WO2023283615A1 (en) 2021-07-09 2023-01-12 Dynetherapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
EP4401792A4 (en) 2021-09-16 2025-12-03 Avidity Biosciences Inc COMPOSITIONS AND METHODS OF TREATMENT OF FACIO-SCAPULO-HUMERAL MUSCULAR DYSTROPHY
US12071621B2 (en) 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Clinical Cancer Research,21권,6호,1383-1394면(2015.03.15.)
Therapeutic Delivery,4권,7호,791-809면(2013)

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