JP2022122928A5 - - Google Patents

Download PDF

Info

Publication number
JP2022122928A5
JP2022122928A5 JP2022085383A JP2022085383A JP2022122928A5 JP 2022122928 A5 JP2022122928 A5 JP 2022122928A5 JP 2022085383 A JP2022085383 A JP 2022085383A JP 2022085383 A JP2022085383 A JP 2022085383A JP 2022122928 A5 JP2022122928 A5 JP 2022122928A5
Authority
JP
Japan
Prior art keywords
pharmaceutical composition
antibody
binding fragment
conjugated
strand
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Granted
Application number
JP2022085383A
Other languages
English (en)
Japanese (ja)
Other versions
JP7420866B2 (ja
JP2022122928A (ja
Filing date
Publication date
Priority claimed from PCT/US2017/025608 external-priority patent/WO2017173408A1/en
Application filed filed Critical
Publication of JP2022122928A publication Critical patent/JP2022122928A/ja
Publication of JP2022122928A5 publication Critical patent/JP2022122928A5/ja
Priority to JP2024002769A priority Critical patent/JP2024050610A/ja
Application granted granted Critical
Publication of JP7420866B2 publication Critical patent/JP7420866B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2022085383A 2016-04-01 2022-05-25 核酸ポリペプチド組成物とその使用 Active JP7420866B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024002769A JP2024050610A (ja) 2016-04-01 2024-01-11 核酸ポリペプチド組成物とその使用

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201662316919P 2016-04-01 2016-04-01
US62/316,919 2016-04-01
PCT/US2017/025608 WO2017173408A1 (en) 2016-04-01 2017-03-31 Nucleic acid-polypeptide compositions and uses thereof
JP2018551978A JP2019513371A (ja) 2016-04-01 2017-03-31 核酸ポリペプチド組成物とその使用

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
JP2018551978A Division JP2019513371A (ja) 2016-04-01 2017-03-31 核酸ポリペプチド組成物とその使用

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024002769A Division JP2024050610A (ja) 2016-04-01 2024-01-11 核酸ポリペプチド組成物とその使用

Publications (3)

Publication Number Publication Date
JP2022122928A JP2022122928A (ja) 2022-08-23
JP2022122928A5 true JP2022122928A5 (enExample) 2022-12-21
JP7420866B2 JP7420866B2 (ja) 2024-01-23

Family

ID=59958453

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2018551978A Pending JP2019513371A (ja) 2016-04-01 2017-03-31 核酸ポリペプチド組成物とその使用
JP2022085383A Active JP7420866B2 (ja) 2016-04-01 2022-05-25 核酸ポリペプチド組成物とその使用
JP2024002769A Pending JP2024050610A (ja) 2016-04-01 2024-01-11 核酸ポリペプチド組成物とその使用

Family Applications Before (1)

Application Number Title Priority Date Filing Date
JP2018551978A Pending JP2019513371A (ja) 2016-04-01 2017-03-31 核酸ポリペプチド組成物とその使用

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024002769A Pending JP2024050610A (ja) 2016-04-01 2024-01-11 核酸ポリペプチド組成物とその使用

Country Status (11)

Country Link
US (10) US20170281795A1 (enExample)
EP (1) EP3436588A4 (enExample)
JP (3) JP2019513371A (enExample)
KR (3) KR102640587B1 (enExample)
CN (2) CN118662652A (enExample)
AU (3) AU2017240799B2 (enExample)
CA (1) CA3019568A1 (enExample)
MA (1) MA45328A (enExample)
MX (1) MX2023008041A (enExample)
SG (1) SG11201808619QA (enExample)
WO (1) WO2017173408A1 (enExample)

Families Citing this family (80)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2708459C2 (ru) 2014-10-29 2019-12-09 БайсиклРД Лимитед Бициклические пептидные лиганды, специфичные для мт1-ммр
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
WO2017030973A1 (en) 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
WO2017132669A1 (en) 2016-01-31 2017-08-03 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
WO2017221883A1 (ja) * 2016-06-20 2017-12-28 武田薬品工業株式会社 抗体-薬物コンジュゲート
US11753638B2 (en) 2016-08-12 2023-09-12 University Of Massachusetts Conjugated oligonucleotides
MX2019008199A (es) 2017-01-06 2019-11-25 Avidity Biosciences Llc Composiciones de acido nucleico polipeptido y metodos de induccion de la omision de exon.
JP7406793B2 (ja) 2017-06-23 2023-12-28 ユニバーシティー オブ マサチューセッツ 2テイル自己デリバリー型siRNAおよび関連方法
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
JP7670481B2 (ja) 2017-08-04 2025-04-30 バイスクルテクス・リミテッド Cd137に対して特異的な二環式ペプチドリガンド
CA3074320A1 (en) * 2017-09-14 2019-03-21 Arrowhead Pharmaceuticals, Inc. Rnai agents and compositions for inhibiting expression of angiopoietin-like 3 (angptl3), and methods of use
AU2018346507B2 (en) 2017-10-04 2025-03-13 Avidity Biosciences, Inc. Nucleic acid-polypeptide compositions and uses thereof
KR20250148679A (ko) 2017-12-06 2025-10-14 어비디티 바이오사이언시스 인크. 근위축증 및 근긴장성 이영양증을 치료하는 조성물 및 방법
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
JP2021514953A (ja) 2018-02-23 2021-06-17 バイスクルテクス・リミテッド 多量体二環式ペプチドリガンド
SG11202009773VA (en) * 2018-04-04 2020-10-29 Bicycletx Ltd Heterotandem bicyclic peptide complexes
GB201810316D0 (en) 2018-06-22 2018-08-08 Bicyclerd Ltd Peptide ligands for binding to EphA2
US11180531B2 (en) 2018-06-22 2021-11-23 Bicycletx Limited Bicyclic peptide ligands specific for Nectin-4
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
WO2020028832A1 (en) 2018-08-02 2020-02-06 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
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US20220193250A1 (en) 2018-08-02 2022-06-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
AU2019316640A1 (en) 2018-08-10 2021-03-18 University Of Massachusetts Modified oligonucleotides targeting SNPs
SG11202101975RA (en) 2018-08-31 2021-03-30 Univ Yale Compositions and methods for enhancing donor oligonucleotide-based gene editing
US12331320B2 (en) 2018-10-10 2025-06-17 The Research Foundation For The State University Of New York Genome edited cancer cell vaccines
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
GB201820295D0 (en) 2018-12-13 2019-01-30 Bicyclerd Ltd Bicyclic peptide ligands specific for MT1-MMP
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
CA3125441A1 (en) * 2019-01-18 2020-07-23 University Of Massachusetts Dynamic pharmacokinetic-modifying anchors
EP3959319A4 (en) 2019-04-25 2023-06-07 Avidity Biosciences, Inc. NUCLEIC ACID COMPOSITIONS AND METHODS FOR MULTI-EXON SKIPPING
CA3137095A1 (en) 2019-05-09 2020-11-12 Bicycletx Limited Bicyclic peptide ligands specific for ox40
US11578090B2 (en) 2019-06-06 2023-02-14 Avidity Biosciences, Inc. Nucleic acid-polypeptide compositions and uses thereof
CA3142337A1 (en) 2019-06-06 2020-12-10 Avidity Biosciences, Inc. Una amidites and uses thereof
TWI860386B (zh) 2019-07-30 2024-11-01 英商拜西可泰克斯有限公司 異質雙環肽複合物
CA3149835A1 (en) 2019-08-09 2021-02-18 University Of Massachusetts Chemically modified oligonucleotides targeting snps
KR20220101073A (ko) * 2019-08-30 2022-07-19 예일 유니버시티 핵산을 세포로 전달하기 위한 조성물 및 방법
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
EP4464721B1 (en) 2019-10-03 2025-06-11 BicycleTX Limited Heterotandem bicyclic peptide complexes
WO2021076546A1 (en) 2019-10-14 2021-04-22 Aro Biotherapeutics Company Cd71 binding fibronectin type iii domains
US12491255B2 (en) 2019-10-14 2025-12-09 Aro Biotherapeutics Company EPCAM binding fibronectin type III domains
WO2021076574A2 (en) * 2019-10-14 2021-04-22 Aro Biotherapeutics Company Fn3 domain-sirna conjugates and uses thereof
EP4069261A4 (en) * 2019-12-06 2023-05-03 Sirnaomics, Inc. PEPTIDE DOCKING VEHICLE FOR TARGETED NUCLEIC ACID DELIVERY
IL296387B2 (en) * 2020-03-19 2024-08-01 Avidity Biosciences Inc Preparations and methods for the treatment of facial, back and arm muscle atrophy
MX2022011880A (es) 2020-03-27 2022-10-20 Avidity Biosciences Inc Composiciones y metodos para tratar distrofia muscular.
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
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
CA3214552A1 (en) 2021-04-14 2022-10-20 Russell C. Addis Cd71 binding fibronectin type iii domains
US12239710B2 (en) 2021-04-14 2025-03-04 Aro Biotherapeutics Company FN3 domain-siRNA conjugates and uses thereof
CA3174095A1 (en) 2021-06-23 2022-12-29 Vignesh Narayan HARIHARAN Optimized anti-flt1 oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
EP4366784A1 (en) 2021-07-09 2024-05-15 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating dystrophinopathies
US11633498B2 (en) 2021-07-09 2023-04-25 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
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
CR20240139A (es) 2021-09-01 2024-06-14 Biogen Ma Inc Anticuerpos del receptor antitransferrina y usos de los mismos
KR20240055874A (ko) 2021-09-16 2024-04-29 어비디티 바이오사이언시스 인크. 안면견갑상완 근이영양증을 치료하는 조성물 및 방법
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
AU2023254846A1 (en) 2022-04-15 2024-10-10 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating myotonic dystrophy
EP4568701A2 (en) * 2022-08-08 2025-06-18 Eli Lilly And Co. 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
CN120882749A (zh) 2023-03-08 2025-10-31 渤健马萨诸塞州股份有限公司 抗转铁蛋白受体抗体及其用途
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
WO2025090898A1 (en) 2023-10-26 2025-05-01 Biogen Ma Inc. Anti-transferrin receptor antibodies and uses thereof cross reference to related applications
WO2025207959A1 (en) 2024-03-27 2025-10-02 Biogen Ma Inc. Anti-transferrin receptor antibodies and uses thereof

Family Cites Families (317)

* 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
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
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
JP2528107B2 (ja) 1985-03-15 1996-08-28 サマ−トン,ジエ−ムス ポリヌクレオチド測定試薬と方法
US5521063A (en) 1985-03-15 1996-05-28 Antivirals Inc. Polynucleotide reagent containing chiral subunits and methods of use
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library 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
DE69123979T2 (de) 1990-10-12 1997-04-30 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
CA2142711A1 (en) 1992-08-17 1994-03-03 Anna M. Richter 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
NZ288223A (en) 1994-06-17 1997-11-24 Hoffmann La Roche N,n'-bis(quinolin-4-yl)-diamine derivatives; antimalarial medicaments
US5716824A (en) 1995-04-20 1998-02-10 Ribozyme Pharmaceuticals, Inc. 2'-O-alkylthioalkyl and 2-C-alkylthioalkyl-containing enzymatic nucleic acids (ribozymes)
AU4188196A (en) 1994-12-22 1996-07-10 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
EP1108724B1 (en) 1996-01-16 2007-09-19 Sirna Therpeutics, Inc. Synthesis of methoxy nucleosides and enzymatic nucleic acid molecules
JP4046354B2 (ja) 1996-03-18 2008-02-13 ボード オブ リージェンツ,ザ ユニバーシティ オブ テキサス システム 増大した半減期を有する免疫グロブリン様ドメイン
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US9096636B2 (en) 1996-06-06 2015-08-04 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds and their use in gene modulation
US5849902A (en) 1996-09-26 1998-12-15 Oligos Etc. Inc. Three component chimeric antisense oligonucleotides
DE69735445T2 (de) 1996-12-10 2006-08-10 Sequenom, Inc., San Diego Abspaltbare, nicht-flüchtige moleküle zur massenmarkierung
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
PT1068241E (pt) 1998-04-02 2007-11-19 Genentech Inc Variantes de anticorpos e respectivos fragmentos
US6194551B1 (en) 1998-04-02 2001-02-27 Genentech, Inc. Polypeptide variants
US6528624B1 (en) 1998-04-02 2003-03-04 Genentech, Inc. Polypeptide variants
AU3751299A (en) 1998-04-20 1999-11-08 Ribozyme Pharmaceuticals, Inc. Nucleic acid molecules with novel chemical compositions capable of modulating gene expression
GB9816575D0 (en) 1998-07-31 1998-09-30 Zeneca Ltd Novel compounds
GB9818730D0 (en) 1998-08-27 1998-10-21 Univ Portsmouth Collections of compounds
DK1413582T3 (da) 1998-08-27 2006-07-17 Spirogen Ltd Dimere pyrrolobenzodiazepiner
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
PT1811036E (pt) 1999-02-22 2011-07-11 Univ Georgetown Imunolipossomas dirigidos por fragmento de anticorpo para distribuição sistémica de genes
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
US7829693B2 (en) 1999-11-24 2010-11-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a target gene
DE10160151A1 (de) 2001-01-09 2003-06-26 Ribopharma Ag Verfahren zur Hemmung der Expression eines vorgegebenen Zielgens
CA2328356A1 (en) 1999-12-22 2001-06-22 Itty Atcravi Recreational vehicles
CA2395660A1 (en) 1999-12-29 2001-07-12 Immunogen, Inc. Cytotoxic agents comprising modified doxorubicins and daunorubicins and their therapeutic use
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)
EP2361981B2 (en) 2000-03-30 2019-01-23 The Whitehead Institute for Biomedical Research RNA sequence-specific mediators of RNA interference
KR20080023768A (ko) 2000-03-30 2008-03-14 화이트헤드 인스티튜트 포 바이오메디칼 리서치 Rna 간섭의 rna 서열 특이적인 매개체
AU5752601A (en) 2000-05-04 2001-11-12 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
KR100909681B1 (ko) 2000-12-01 2009-07-29 막스-플랑크-게젤샤프트 츄어 푀르더룽 데어 비쎈샤프텐 에.파우. Rna 간섭을 매개하는 작은 rna 분자
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
US6881557B2 (en) 2001-07-12 2005-04-19 Arrowsmith Technologies Llp Super humanized antibodies
DE10133858A1 (de) 2001-07-12 2003-02-06 Aventis Pharma Gmbh Synthetische doppelsträngige Oligonucleotide zur gezielten Hemmung der Genexpression
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
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)
EP1432724A4 (en) 2002-02-20 2006-02-01 Sirna Therapeutics Inc INTERFERENCE MEDIATION INHIBITION OF GENE RNA FROM MAP KINASE
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
SI2357006T1 (sl) 2002-07-31 2016-01-29 Seattle Genetics, Inc. Konjugati zdravil in njihova uporaba za zdravljenje raka, avtoimunske bolezni ali infekcijske bolezni
US7452987B2 (en) 2002-08-05 2008-11-18 Silence Therapeutics Aktiengesellschaft (Ag) 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)
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
EP2213738B1 (en) 2002-11-14 2012-10-10 Dharmacon, Inc. siRNA molecules targeting Bcl-2
CA3001404C (en) 2002-11-25 2020-07-28 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
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
JP4605799B2 (ja) 2003-04-02 2011-01-05 ダーマコン, インコーポレイテッド Rna干渉において使用するための修飾ポリヌクレオチド
US20040224893A1 (en) 2003-05-06 2004-11-11 Li-Hsien Wang Methods of using IL-1 antagonists to treat neointimal hyperplasia
US7276497B2 (en) 2003-05-20 2007-10-02 Immunogen Inc. Cytotoxic agents comprising new maytansinoids
ATE485394T1 (de) 2003-06-02 2010-11-15 Univ Massachusetts Verfahren und zusammensetzungen zur verbesserung der wirksamkeit und spezifität von fnai
US7750144B2 (en) 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
AU2004248136B2 (en) 2003-06-02 2011-09-15 University Of Massachusetts Methods and compositions for controlling efficacy of RNA silencing
US7786290B2 (en) 2003-06-13 2010-08-31 Alnylam Pharmaceuticals, Inc. Double-stranded ribonucleic acid with increased effectiveness in an organism
GB0416511D0 (en) 2003-10-22 2004-08-25 Spirogen Ltd Pyrrolobenzodiazepines
US8133733B2 (en) 2003-10-24 2012-03-13 Gencia Corporation Nonviral vectors for delivering polynucleotides to target tissues
BR122018071808B8 (pt) 2003-11-06 2020-06-30 Seattle Genetics Inc conjugado
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
WO2005089287A2 (en) 2004-03-15 2005-09-29 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
EP1765847A4 (en) 2004-05-27 2010-10-20 Alnylam Pharmaceuticals Inc NUCLEASERESISTENT DOUBLE-STRANDED RIBONUCLEIC ACID
PL1766010T3 (pl) 2004-06-28 2011-07-29 Univ Western Australia Antysensowne oligonukleotydy do indukcji pominięcia egzonu i sposoby ich zastosowania
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
SI1879901T1 (sl) 2005-04-21 2010-04-30 Spirogen Ltd Pirolobenzodiazepini
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
AU2006249340A1 (en) * 2005-05-26 2006-11-30 Ensemble Discovery Corportion Biodetection by nucleic acid-templated chemistry
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
CN101287835A (zh) 2005-08-17 2008-10-15 株式会社百奥尼 用于siRNA细胞内输送的siRNA-亲水性聚合物结合物及其方法
DK1931780T3 (en) 2005-08-29 2016-01-25 Regulus Therapeutics Inc Antisense-forbindelser med forøget anti-microrna-aktivitet
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
DK1813614T3 (da) 2006-01-25 2012-01-23 Sanofi Sa Cytotoksiske midler, der omfatter nye tomaymycinderivater
US7750116B1 (en) 2006-02-18 2010-07-06 Seattle Genetics, Inc. Antibody drug conjugate metabolites
SI2735568T1 (en) 2006-05-10 2018-01-31 Sarepta Therapeutics, Inc. Analogues of the oligonucleotide, with cationic links between subunits
EP1857548A1 (en) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
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
US8598333B2 (en) 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
US7521232B2 (en) 2006-05-31 2009-04-21 Icx Nomadics, Inc. Emissive species for clinical imaging
CN101563105B (zh) 2006-07-10 2013-01-23 拜奥根Idec马萨诸塞公司 用于抑制smad4-缺陷癌症的组合物和方法
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
WO2008022309A2 (en) 2006-08-18 2008-02-21 F. Hoffmann-La Roche Ag Polyconjugates for in vivo delivery of polynucleotides
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
EP2148886B1 (en) 2007-05-10 2014-02-19 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
WO2009005793A2 (en) 2007-06-29 2009-01-08 Avi Biopharma, Inc. Tissue specific peptide conjugates and methods
SI2019104T1 (sl) 2007-07-19 2013-12-31 Sanofi Citotoksična sredstva, ki obsegajo nove tomaimicinske derivate, in njihova terapevtska uporaba
CN105018492B (zh) 2007-08-27 2018-08-24 北京强新生物科技有限公司 不对称干扰rna的组合物及其用途
WO2009108217A2 (en) 2007-09-18 2009-09-03 Intradigm Corporation Compositions comprising k-ras sirna and methods of use
JP5723154B2 (ja) 2007-09-19 2015-05-27 アプライド バイオシステムズ リミテッド ライアビリティー カンパニー RNAiにおけるオフターゲット表現型の影響を減少させるためのSiRNA配列非依存性修飾フォーマットおよびその安定化型
SI2211904T1 (sl) 2007-10-19 2016-12-30 Seattle Genetics, Inc. CD19 vezavna sredstva in njihove uporabe
CN101896186A (zh) 2007-10-26 2010-11-24 莱顿教学医院 对抗肌肉病症的方式和方法
WO2009061941A2 (en) 2007-11-06 2009-05-14 Osmetech Molecular Diagnostics Baseless nucleotide analogues and uses thereof
CN102083460A (zh) 2008-01-18 2011-06-01 米迪缪尼有限公司 用于位点特异性偶联的半胱氨酸工程化抗体
WO2009099942A2 (en) 2008-01-31 2009-08-13 Alnylam Pharmaceuticals, Inc. Chemically modified oligonucleotides and uses thereof
WO2009099991A2 (en) 2008-01-31 2009-08-13 The Brigham And Women's Hospital, Inc. Treatment of cancer
RU2010136023A (ru) 2008-02-01 2012-03-10 Асцендис Фарма Ас (Dk) Пролекарство, содержащее саморасщепляемый линкер
CN104975020B (zh) 2008-02-11 2020-01-17 菲奥医药公司 经修饰的RNAi多核苷酸及其用途
CA2718942A1 (en) 2008-03-18 2009-09-24 Seattle Genetics, Inc. Auristatin drug linker conjugates
US8575123B2 (en) 2008-04-11 2013-11-05 Tekmira Pharmaceuticals Corporation Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
TWI465247B (zh) 2008-04-11 2014-12-21 Catalyst Biosciences Inc 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
US20110130346A1 (en) 2008-05-30 2011-06-02 Isis Innovation Limited Peptide conjugates for delvery of biologically active compounds
EP2283029A1 (en) 2008-06-04 2011-02-16 Medical Research Council Peptides
DK2285350T3 (en) * 2008-06-16 2017-12-11 Pfizer PROCEDURES FOR THE PREPARATION OF DIBLOCK COPOLYMERS FUNCTIONALIZED WITH TARGETING AGENT FOR USE IN THE MANUFACTURE OF THERAPEUTIC NANOPARTICLES
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
EP2320953B1 (en) 2008-08-08 2015-07-15 Genisphere, LLC Long-acting dna dendrimers and methods thereof
WO2010019718A2 (en) 2008-08-13 2010-02-18 California Institute Of Technology Carrier nanoparticles and related compositions, methods and systems
EP2323695B1 (en) 2008-08-19 2018-12-05 Nektar Therapeutics Complexes of small-interfering nucleic acids
US8318693B2 (en) 2008-09-02 2012-11-27 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
US8796443B2 (en) 2008-09-22 2014-08-05 Rxi Pharmaceuticals Corporation Reduced size self-delivering RNAi compounds
AU2013204438B2 (en) 2008-10-24 2016-12-22 Sarepta Therapeutics, Inc. Multiple exon skipping compositions for DMD
SMT201900159T1 (it) 2008-10-24 2019-05-10 Sarepta Therapeutics Inc Composizioni di salto di esone per dmd
DK2607484T3 (en) 2008-10-27 2016-03-07 Biomarin Technologies B V Methods and means for efficient skipping of exon 45 in Duchenne muscular dystrophy pre-MRNA
KR101602431B1 (ko) 2008-11-05 2016-03-10 삼성전자 주식회사 냉장고
KR101881596B1 (ko) 2008-12-02 2018-07-24 웨이브 라이프 사이언시스 재팬 인코포레이티드 인 원자 변형된 핵산의 합성 방법
EP3100745B1 (en) 2009-02-05 2018-04-18 Immunogen, Inc. Novel benzodiazepine derivatives
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
PE20120553A1 (es) * 2009-03-25 2012-05-18 Genentech Inc Anticuerpos anti-fgfr3
US20130115603A9 (en) 2009-04-02 2013-05-09 Regents Of The University Of Minnesota Nucleotide repeat expansion-associated polypeptides and uses thereof
PL2417257T3 (pl) 2009-04-10 2016-11-30 Antysensowne oligonukleotydy tricyklo-dna, kompozycje i sposoby leczenia choroby
CA2759899A1 (en) 2009-04-24 2010-10-28 Prosensa Technologies B.V. Oligonucleotide comprising an inosine for treating dmd
US8604184B2 (en) 2009-05-05 2013-12-10 The United States Of America As Represented By The Secretary Of The Air Force Chemically programmable immunity
BRPI1013016A2 (pt) 2009-06-04 2016-03-29 Massachusetts Inst Technology "métodos para identificação de locais para conjugação de igg"
CN102802661B (zh) 2009-06-22 2016-01-13 米迪缪尼有限公司 用于位点特异性偶联的工程改造的Fc区
US20110269665A1 (en) 2009-06-26 2011-11-03 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
US9050375B2 (en) * 2009-07-06 2015-06-09 Hoffmann-La Roche, Inc. 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
JP2012533587A (ja) * 2009-07-22 2012-12-27 セニックス バイオサイエンス ゲーエムベーハー 自然に存在する細胞内輸送経路を介して化合物を送達するための送達システム及びコンジュゲート
WO2011024077A2 (en) 2009-08-31 2011-03-03 INSERM (Institut National de la Santé et de la Recherche Médicale) Exon skipping therapy for functional amelioration of semi functional dystrophin in becker and duchenne muscular dystrophy
JP5898082B2 (ja) 2009-10-07 2016-04-06 マクロジェニクス,インコーポレーテッド フコシル化程度の変更により改良されたエフェクター機能を示すFc領域含有ポリペプチドおよびその使用法
KR20230137491A (ko) 2009-11-12 2023-10-04 더 유니버시티 오브 웨스턴 오스트레일리아 안티센스 분자 및 이를 이용한 질환 치료방법
WO2011078797A2 (en) 2009-12-22 2011-06-30 Singapore Health Services Pte. Ltd Antisense oligonucleotides and uses threreof
UY33202A (es) 2010-01-28 2011-08-31 Glaxo Group Ltd Proteínas de unión a cd127
DK2558578T3 (en) 2010-04-13 2016-01-25 Life Technologies Corp CONFIGURATIONS AND METHODS FOR INHIBITION OF nucleic acid function
NZ602932A (en) 2010-04-15 2014-08-29 Seattle Genetics Inc Targeted pyrrolobenzodiazapine conjugates
MX368648B (es) 2010-04-15 2019-10-09 Seattle Genetics Inc Pirrolobenzodiazepinas usadas para tratar enfermedades proliferativas.
WO2011139699A2 (en) 2010-04-28 2011-11-10 Isis Pharmaceuticals, Inc. 5' modified nucleosides and oligomeric compounds prepared therefrom
JP6005628B2 (ja) 2010-04-28 2016-10-12 アイオーニス ファーマシューティカルズ, インコーポレーテッドIonis Pharmaceuticals,Inc. 修飾ヌクレオシド、その類似体、およびこれらから調製されるオリゴマー化合物
NZ603606A (en) 2010-05-28 2015-06-26 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.
JP2013531981A (ja) * 2010-06-11 2013-08-15 アンチセンス・ファーマ・ゲーエムベーハー 選択的オリゴヌクレオチド修飾の方法
AU2011282217B2 (en) 2010-07-19 2015-12-03 Ionis Pharmaceuticals, Inc. 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
WO2012031243A2 (en) 2010-09-03 2012-03-08 Avi Biopharma, 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)
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
CA2816041C (en) * 2010-12-29 2019-01-08 Arrowhead Research Corporation In vivo polynucleotide delivery conjugates having enzyme sensitive linkages
CN103501793A (zh) 2011-02-08 2014-01-08 夏洛特-梅克伦堡医院(商业用名:卡罗来纳保健系统) 反义寡核苷酸
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
HUE041335T2 (hu) 2011-03-29 2019-05-28 Roche Glycart Ag Antitest FC-variánsok
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
US9598496B2 (en) * 2011-05-09 2017-03-21 Perseus Proteomics Inc. Antibody capable of specifically recognizing transferrin receptor
EA029797B1 (ru) 2011-06-21 2018-05-31 Иммуноджен, Инк. Новые производные майтанзиноида с пептидным линкером и их конъюгаты
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
BR112014001244A2 (pt) 2011-07-19 2017-02-21 Wave Life Sciences Pte Ltd métodos para a síntese de ácidos nucléicos funcionalizados
US9418216B2 (en) 2011-07-21 2016-08-16 Microsoft Technology Licensing, Llc Cloud service authentication
CA3106285A1 (en) 2011-07-25 2013-01-31 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of dux4
EP2751270B1 (en) 2011-08-29 2018-08-22 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
IN2014MN00357A (enExample) 2011-08-30 2015-06-19 Medical Res Council
CA2850373C (en) 2011-10-14 2019-07-16 Seattle Genetics, Inc. Pyrrolobenzodiazepines and targeted conjugates
WO2013071079A1 (en) 2011-11-09 2013-05-16 President And Fellows Of Harvard College Lin28/let-7 crystal structures, purification protocols, and molecular probes suitable for screening assays and therapeutics
FI3366775T4 (fi) 2011-11-18 2025-11-14 Alnylam Pharmaceuticals Inc Modifioituja rnai-agensseja
WO2013085925A1 (en) 2011-12-05 2013-06-13 Igenica, Inc. Antibody-drug conjugates and related compounds, compositions, and methods
ES2707232T3 (es) * 2011-12-16 2019-04-03 Univ Nat Corp Tokyo Medical & Dental Acido nucleico bicatenario quimérico
CA2859755C (en) 2011-12-23 2021-04-20 Pfizer Inc. Engineered antibody constant regions for site-specific conjugation and methods and uses therefor
TW201333040A (zh) * 2012-01-06 2013-08-16 Bioalliance Cv 抗-輸鐵蛋白受器之抗體及其使用方法
WO2013110005A1 (en) * 2012-01-18 2013-07-25 Wisconsin Alumni Research Foundation Boronate-mediated delivery of molecules into cells
BR112014018427B1 (pt) 2012-01-27 2021-11-03 Biomarin Technologies B.V. Oligonucleotídeos moduladores de rna com características melhoradas para o tratamento da distrofia muscular de duchenne e becker
WO2013121175A1 (en) 2012-02-16 2013-08-22 Ucl Business Plc Lysosome-cleavable linker
CN104220458B (zh) 2012-02-24 2018-08-14 阿特根公司 结合有含半胱氨酸残基的基序的修饰抗体,含该修饰抗体的修饰抗体-药物缀合物以及其制造方法
CA2907072A1 (en) 2012-03-16 2013-09-19 Valerion Therapeutics, Llc 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
WO2013166004A2 (en) 2012-05-02 2013-11-07 Novartis Ag Organic compositions to treat kras-related diseases
US9504756B2 (en) 2012-05-15 2016-11-29 Seattle Genetics, Inc. Self-stabilizing linker conjugates
CN104662000B (zh) 2012-05-15 2018-08-17 索伦托医疗有限公司 药物偶联物及其偶联方法和用途
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
CN112007045A (zh) 2012-07-13 2020-12-01 波涛生命科学有限公司 手性控制
JP2015529073A (ja) 2012-08-20 2015-10-05 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 生物可逆性基を有するポリヌクレオチド
WO2014057074A1 (en) 2012-10-12 2014-04-17 Spirogen Sàrl Pyrrolobenzodiazepines and conjugates thereof
ES2967530T3 (es) 2012-10-26 2024-04-30 Palomo Ltd 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
CA2889044A1 (en) 2012-11-15 2014-05-22 Roche Innovation Center Copenhagen A/S Anti apob antisense conjugate compounds
WO2014080251A1 (en) 2012-11-24 2014-05-30 Hangzhou Dac Biotech Co., Ltd. Hydrophilic linkers and their uses for conjugation of drugs to cell binding molecules
EP2935584A1 (en) 2012-12-20 2015-10-28 Sarepta Therapeutics, Inc. Improved exon skipping compositions for treating muscular dystrophy
TWI716339B (zh) 2012-12-21 2021-01-21 荷蘭商台醫(有限合夥)公司 親水性自消耗連接子及彼等之共軛物
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
EP2968596B1 (en) 2013-03-13 2019-03-06 Medimmune Limited Pyrrolobenzodiazepines and conjugates thereof
NZ631245A (en) 2013-03-14 2017-09-29 Sarepta Therapeutics Inc Exon skipping compositions for treating muscular dystrophy
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
EA038512B1 (ru) 2013-03-15 2021-09-08 Регенерон Фармасьютикалз, Инк. Конъюгаты майтанзиноидов и их терапевтическое применение
US9577459B2 (en) 2013-03-15 2017-02-21 Sequana Medical Ag Systems and methods for regulating inductive energy transfer to an implantable system
HK1220154A1 (zh) 2013-03-15 2017-04-28 Sarepta Therapeutics, Inc. 改进的用於治疗肌营养不良的组合物
CN105378083B (zh) * 2013-03-27 2018-09-21 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
WO2014169049A1 (en) 2013-04-09 2014-10-16 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
JP2016520574A (ja) 2013-04-28 2016-07-14 秦剛 新規リンカー、その製造方法およびその応用
PH12021550015A1 (en) 2013-05-20 2022-05-11 Genentech Inc Anti-transferrin receptor antibodies and methods of use
RU2655439C2 (ru) 2013-05-31 2018-05-28 Займворкс Инк. Гетеромультимеры с уменьшенной или подавленной эффекторной функцией
KR20230136697A (ko) 2013-06-05 2023-09-26 듀크 유니버시티 Rna-가이드 유전자 편집 및 유전자 조절
WO2014197854A1 (en) 2013-06-06 2014-12-11 Igenica Biotherapeutics, Inc. Novel linkers for antibody-drug conjugates and related compounds, compositions, and methods of use
US20140363454A1 (en) 2013-06-06 2014-12-11 Igenica Biotherapeutics, Inc. Antibody-Drug Conjugates, Compositions and Methods of Use
TW201536329A (zh) 2013-08-09 2015-10-01 Isis Pharmaceuticals Inc 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法
EP2845607A1 (en) 2013-09-09 2015-03-11 University of Vienna Antisense oligonucleotides with improved pharmacokinetic properties
WO2015038426A1 (en) 2013-09-13 2015-03-19 Asana Biosciences, Llc Self-immolative linkers containing mandelic acid derivatives, drug-ligand conjugates for targeted therapies and uses thereof
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
WO2015057876A1 (en) 2013-10-15 2015-04-23 Sorrento Therapeutics Inc. Drug-conjugates with a targeting molecule and two different drugs
NZ758050A (en) 2013-10-15 2024-03-22 Seagen Inc Pegylated drug-linkers for improved ligand-drug conjugate pharmacokinetics
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
CN113278617A (zh) 2014-01-16 2021-08-20 波涛生命科学有限公司 手性设计
CA2935426C (en) 2014-01-30 2023-07-25 F. Hoffmann-La Roche Ag Polyoligomer compound with biocleavable conjugates for reducing or inhibiting expression of a nucleic acid target
SG11201607095RA (en) 2014-03-12 2016-10-28 Nippon Shinyaku Co Ltd Antisense nucleic acids
WO2015168661A1 (en) 2014-05-01 2015-11-05 Smith Larry J METHODS AND MODIFICATIONS THAT PRODUCE ssRNAi COMPOUNDS WITH ENHANCED ACTIVITY, POTENCY AND DURATION OF EFFECT
EP3146050B1 (en) 2014-05-23 2020-08-12 Genzyme Corporation Inhibiting or downregulating glycogen synthase by creating premature stop codons using antisense oligonucleotides
CN111575282B (zh) 2014-06-17 2024-05-14 日本新药株式会社 反义核酸
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
CN106795515B (zh) 2014-06-23 2021-06-08 孟山都技术公司 用于经由rna干扰调控基因表达的组合物和方法
IL316808A (en) 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
CN107001473B (zh) 2014-11-19 2021-07-09 豪夫迈·罗氏有限公司 抗-运铁蛋白受体抗体及使用方法
BR112017015203A2 (pt) 2015-01-16 2018-03-13 Hope City anticorpos de penetração celular
PL3277814T3 (pl) 2015-04-03 2020-11-30 University Of Massachusetts Związki oligonukleotydowe ukierunkowane na mrna huntingtyny
WO2016170348A2 (en) 2015-04-22 2016-10-27 Mina Therapeutics Limited Sarna compositions and methods of use
ES2905181T3 (es) 2015-05-01 2022-04-07 Prec Biosciences Inc Deleción precisa de secuencias cromosómicas in vivo
CA2986228A1 (en) 2015-05-19 2016-11-24 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
IL302486A (en) 2015-06-24 2023-06-01 Hoffmann La Roche Antibodies against the transnephrine receptor with adapted affinity
AU2016306275A1 (en) 2015-08-07 2018-02-08 Arrowhead Pharmaceuticals, Inc. RNAi therapy for Hepatitis B virus infection
JP2018529715A (ja) 2015-09-30 2018-10-11 サレプタ セラピューティクス, インコーポレイテッド 筋ジストロフィーを処置するための方法
GB2545898B (en) 2015-12-21 2019-10-09 Sutura Therapeutics Ltd Improved drug delivery by conjugating oligonucleotides to stitched/stapled peptides
CN108699155B (zh) 2016-03-01 2023-03-21 豪夫迈·罗氏有限公司 具有改变的细胞死亡诱导的奥滨尤妥珠单抗变体
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
WO2017221883A1 (ja) * 2016-06-20 2017-12-28 武田薬品工業株式会社 抗体-薬物コンジュゲート
US11427838B2 (en) 2016-06-29 2022-08-30 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of myotonic dystrophy type 1 (DM1) and other related disorders
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
US20190256868A1 (en) 2016-10-28 2019-08-22 Genethon Compositions and methods for the treatment of myotonic dystrophy
KR102645243B1 (ko) 2016-11-23 2024-03-11 알닐람 파마슈티칼스 인코포레이티드 표적외 효과가 감소된 변형 rna 작용제
SG10202012839TA (en) 2016-12-19 2021-01-28 Sarepta Therapeutics Inc Exon skipping oligomer conjugates for muscular dystrophy
MX2019008199A (es) 2017-01-06 2019-11-25 Avidity Biosciences Llc Composiciones de acido nucleico polipeptido y metodos de induccion de la omision de exon.
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
DK3444010T3 (da) 2017-08-14 2021-03-15 Scandiedge Therapeutics Ab Pklr-inhibering i behandlingen af nafld og hcc
US20200282074A1 (en) 2017-09-22 2020-09-10 Avidity Biosciences, Inc. Nucleic acid-polypeptide compositions and methods of inducing exon skipping
AU2018346507B2 (en) 2017-10-04 2025-03-13 Avidity Biosciences, Inc. Nucleic acid-polypeptide compositions and uses thereof
KR20250148679A (ko) 2017-12-06 2025-10-14 어비디티 바이오사이언시스 인크. 근위축증 및 근긴장성 이영양증을 치료하는 조성물 및 방법
MA51583A (fr) 2018-01-04 2020-11-11 Avidity Biosciences Inc Molécules d'acide nucléique hétéroduplex et leurs utilisations
US10910559B2 (en) 2018-06-01 2021-02-02 Massachusetts Institute Of Technology Optoelectronic memristor devices including one or more solid electrolytes with electrically controllable optical properties
WO2020028832A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US20220193250A1 (en) 2018-08-02 2022-06-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
IL320597A (en) 2018-08-02 2025-07-01 Dyne Therapeutics Inc Transferrin receptor-targeting conjugates and their uses
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
US11578090B2 (en) 2019-06-06 2023-02-14 Avidity Biosciences, Inc. Nucleic acid-polypeptide compositions and uses thereof
CA3142337A1 (en) 2019-06-06 2020-12-10 Avidity Biosciences, Inc. Una amidites 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
IL296387B2 (en) 2020-03-19 2024-08-01 Avidity Biosciences Inc Preparations and methods for the treatment of facial, back and arm muscle atrophy
MX2022011880A (es) 2020-03-27 2022-10-20 Avidity Biosciences Inc Composiciones y metodos para tratar distrofia muscular.
WO2021226107A1 (en) 2020-05-05 2021-11-11 Avidity Biosciences, Inc. Compositions and methods of treating pompe disease
KR20240035826A (ko) 2021-07-09 2024-03-18 다인 세라퓨틱스, 인크. 근육 표적화 복합체 및 디스트로핀병증을 치료하기 위한 그의 용도
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
KR20240055874A (ko) 2021-09-16 2024-04-29 어비디티 바이오사이언시스 인크. 안면견갑상완 근이영양증을 치료하는 조성물 및 방법
US12071621B2 (en) 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping

Similar Documents

Publication Publication Date Title
JP2022122928A5 (enExample)
JPWO2018129384A5 (enExample)
JP2020505330A5 (enExample)
JP2019513371A5 (enExample)
IL274906B1 (en) Compositions and methods of treating muscle atrophy and myotonic dystrophy
JPWO2020132584A5 (enExample)
JP2021500862A5 (enExample)
JP2023015095A5 (enExample)
US20210121580A1 (en) Functionalized DNA Dendrimers For Gene Delivery To Cells
JP5952423B2 (ja) 新規オリゴヌクレオチド接合体およびその用途
JP6422961B2 (ja) 改善された高効率ナノ粒子型オリゴヌクレオチド構造体およびその製造方法
IL317871A (en) Muscle targeting complexes and their uses for the treatment of FACIOSCAPULOHUMERAL muscular dystrophy
JP2013531981A (ja) 選択的オリゴヌクレオチド修飾の方法
JPWO2020028861A5 (enExample)
JPWO2020028864A5 (enExample)
JPWO2021142227A5 (enExample)
JPWO2020247782A5 (enExample)
JP2025026839A5 (enExample)
JPWO2021142275A5 (enExample)
JP7657756B2 (ja) mRNAの機能状態を変化させてその選択的かつ特異的な認識を可能にする方法
WO2024137545A1 (en) Arnatar compounds and methods for enhanced cellular uptake
JPWO2023196400A5 (enExample)
JPWO2019158942A5 (enExample)
JP2020510622A5 (enExample)
CN115244064A (zh) 治疗性分子的靶向递送