JP2023512441A5 - - Google Patents

Info

Publication number
JP2023512441A5
JP2023512441A5 JP2022542475A JP2022542475A JP2023512441A5 JP 2023512441 A5 JP2023512441 A5 JP 2023512441A5 JP 2022542475 A JP2022542475 A JP 2022542475A JP 2022542475 A JP2022542475 A JP 2022542475A JP 2023512441 A5 JP2023512441 A5 JP 2023512441A5
Authority
JP
Japan
Prior art keywords
cdr
seq
optionally
antisense strand
nucleoside
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
JP2022542475A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023512441A (ja
JP7858532B2 (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/012650 external-priority patent/WO2021142227A1/en
Publication of JP2023512441A publication Critical patent/JP2023512441A/ja
Publication of JP2023512441A5 publication Critical patent/JP2023512441A5/ja
Priority to JP2025207980A priority Critical patent/JP2026062645A/ja
Application granted granted Critical
Publication of JP7858532B2 publication Critical patent/JP7858532B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2022542475A 2020-01-10 2021-01-08 筋肉標的複合体および筋肉の健康に関連する遺伝子のモジュレーションのためのその使用 Active JP7858532B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025207980A JP2026062645A (ja) 2020-01-10 2025-11-28 筋肉標的複合体および筋肉の健康に関連する遺伝子のモジュレーションのためのその使用

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202062959590P 2020-01-10 2020-01-10
US202062959469P 2020-01-10 2020-01-10
US202062959398P 2020-01-10 2020-01-10
US62/959,398 2020-01-10
US62/959,590 2020-01-10
US62/959,469 2020-01-10
PCT/US2021/012650 WO2021142227A1 (en) 2020-01-10 2021-01-08 Muscle targeting complexes and uses thereof for modulation of genes associated with muscle health

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025207980A Division JP2026062645A (ja) 2020-01-10 2025-11-28 筋肉標的複合体および筋肉の健康に関連する遺伝子のモジュレーションのためのその使用

Publications (3)

Publication Number Publication Date
JP2023512441A JP2023512441A (ja) 2023-03-27
JP2023512441A5 true JP2023512441A5 (enExample) 2024-01-17
JP7858532B2 JP7858532B2 (ja) 2026-05-14

Family

ID=76788282

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2022542475A Active JP7858532B2 (ja) 2020-01-10 2021-01-08 筋肉標的複合体および筋肉の健康に関連する遺伝子のモジュレーションのためのその使用
JP2025207980A Pending JP2026062645A (ja) 2020-01-10 2025-11-28 筋肉標的複合体および筋肉の健康に関連する遺伝子のモジュレーションのためのその使用

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025207980A Pending JP2026062645A (ja) 2020-01-10 2025-11-28 筋肉標的複合体および筋肉の健康に関連する遺伝子のモジュレーションのためのその使用

Country Status (6)

Country Link
US (1) US20230111147A1 (enExample)
EP (1) EP4087586A4 (enExample)
JP (2) JP7858532B2 (enExample)
CN (1) CN115335062A (enExample)
CA (1) CA3163283A1 (enExample)
WO (1) WO2021142227A1 (enExample)

Families Citing this family (25)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4169576A1 (en) 2018-03-23 2023-04-26 University of Massachusetts Gene therapeutics for treating bone disorders
WO2020028832A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US12097263B2 (en) 2018-08-02 2024-09-24 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
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
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
EP3830259A4 (en) 2018-08-02 2022-05-04 Dyne Therapeutics, Inc. MUSCLE TARGETED COMPLEXES AND THEIR USES IN THE TREATMENT OF FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
US20230111212A1 (en) * 2020-01-10 2023-04-13 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
EP4087653A4 (en) * 2020-01-10 2024-06-19 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
WO2021154477A1 (en) 2020-01-31 2021-08-05 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
US11672872B2 (en) 2021-07-09 2023-06-13 Dyne Therapeutics, Inc. Anti-transferrin receptor antibody and uses thereof
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
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
WO2023283623A1 (en) 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating dystrophinopathies
US20250082759A1 (en) 2021-12-22 2025-03-13 Sapreme Technologies B.V. Compositions comprising therapeutic nucleic acid and targeted saponin for the treatment of muscle-wasting disorders
EP4452281A1 (en) 2021-12-22 2024-10-30 Sapreme Technologies B.V. Compositions comprising therapeutic nucleic acid and saponin for the treatment of muscle-wasting disorders
CA3240612A1 (en) 2021-12-22 2023-06-29 Miriam Verena Bujny Targeted saponin-nucleic acid conjugates for treatment of muscle wasting disorders
IL316143A (en) 2022-04-15 2024-12-01 Dyne Therapeutics Inc Muscle targeting complexes and formulations for the treatment of myotonic dystrophy
WO2023230562A2 (en) * 2022-05-26 2023-11-30 Board Of Regents Of The University Ofnebraska Rna compositions and therapeutic methods thereof
WO2024220758A1 (en) * 2023-04-20 2024-10-24 University Of Massachusetts Rnas targeting activin a subunits
EP4698656A1 (en) * 2023-04-20 2026-02-25 University of Massachusetts Rna therapeutics for fibrodysplasia ossificans progressiva and uses thereof
US12527877B2 (en) 2024-01-24 2026-01-20 Arrowhead Pharmaceuticals, Inc. Anti-transferrin receptor antibodies, conjugates thereof, and uses thereof

Family Cites Families (147)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
US4364934A (en) 1979-12-04 1982-12-21 Ortho Pharmaceutical Corporation Monoclonal antibody to a human early thymocyte antigen and methods for preparing same
US4469863A (en) 1980-11-12 1984-09-04 Ts O Paul O P Nonionic nucleic acid alkyl and aryl phosphonates and processes for manufacture and use thereof
US5023243A (en) 1981-10-23 1991-06-11 Molecular Biosystems, Inc. Oligonucleotide therapeutic agent and method of making same
US4476301A (en) 1982-04-29 1984-10-09 Centre National De La Recherche Scientifique Oligonucleotides, a process for preparing the same and their application as mediators of the action of interferon
US5550111A (en) 1984-07-11 1996-08-27 Temple University-Of The Commonwealth System Of Higher Education Dual action 2',5'-oligoadenylate antiviral derivatives and uses thereof
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US6548640B1 (en) 1986-03-27 2003-04-15 Btg International Limited Altered antibodies
EP0260032B1 (en) 1986-09-08 1994-01-26 Ajinomoto Co., Inc. Compounds for the cleavage at a specific position of RNA, oligomers employed for the formation of said compounds, and starting materials for the synthesis of said oligomers
JP3101690B2 (ja) 1987-03-18 2000-10-23 エス・ビィ・2・インコーポレイテッド 変性抗体の、または変性抗体に関する改良
US5276019A (en) 1987-03-25 1994-01-04 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
US5264423A (en) 1987-03-25 1993-11-23 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
US5677425A (en) 1987-09-04 1997-10-14 Celltech Therapeutics Limited Recombinant antibody
US4924624A (en) 1987-10-22 1990-05-15 Temple University-Of The Commonwealth System Of Higher Education 2,',5'-phosphorothioate oligoadenylates and plant antiviral uses thereof
US5188897A (en) 1987-10-22 1993-02-23 Temple University Of The Commonwealth System Of Higher Education Encapsulated 2',5'-phosphorothioate oligoadenylates
JP3019994B2 (ja) 1987-11-30 2000-03-15 ユニバーシティ オブ アイオワ リサーチ ファウンデーション 新規なオリゴデオキシヌクレオチド、それを使用した標的遺伝子の発現をブロックする方法、及び新規なオリゴデオキシヌクレオチド並びにそれを使用した標的遺伝子の発現を阻止する方法
US5403711A (en) 1987-11-30 1995-04-04 University Of Iowa Research Foundation Nucleic acid hybridization and amplification method for detection of specific sequences in which a complementary labeled nucleic acid probe is cleaved
EP0406309A4 (en) 1988-03-25 1992-08-19 The University Of Virginia Alumni Patents Foundation Oligonucleotide n-alkylphosphoramidates
US5278302A (en) 1988-05-26 1994-01-11 University Patents, Inc. Polynucleotide phosphorodithioates
US5223409A (en) 1988-09-02 1993-06-29 Protein Engineering Corp. Directed evolution of novel binding proteins
US5256775A (en) 1989-06-05 1993-10-26 Gilead Sciences, Inc. Exonuclease-resistant oligonucleotides
US5399676A (en) 1989-10-23 1995-03-21 Gilead Sciences Oligonucleotides with inverted polarity
US5177198A (en) 1989-11-30 1993-01-05 University Of N.C. At Chapel Hill Process for preparing oligoribonucleoside and oligodeoxyribonucleoside boranophosphates
US5587361A (en) 1991-10-15 1996-12-24 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
US5623065A (en) 1990-08-13 1997-04-22 Isis Pharmaceuticals, Inc. Gapped 2' modified oligonucleotides
US7101993B1 (en) 1990-01-11 2006-09-05 Isis Pharmaceuticals, Inc. Oligonucleotides containing 2′-O-modified purines
US5220007A (en) 1990-02-15 1993-06-15 The Worcester Foundation For Experimental Biology Method of site-specific alteration of RNA and production of encoded polypeptides
US5149797A (en) 1990-02-15 1992-09-22 The Worcester Foundation For Experimental Biology Method of site-specific alteration of rna and production of encoded polypeptides
US5321131A (en) 1990-03-08 1994-06-14 Hybridon, Inc. Site-specific functionalization of oligodeoxynucleotides for non-radioactive labelling
US5427908A (en) 1990-05-01 1995-06-27 Affymax Technologies N.V. Recombinant library screening methods
WO1991018913A1 (en) 1990-06-07 1991-12-12 City Of Hope Ribozyme mediated reversal of transformation by cleavage of the hras oncogene rna
US5270163A (en) 1990-06-11 1993-12-14 University Research Corporation Methods for identifying nucleic acid ligands
US5650275A (en) 1990-06-11 1997-07-22 Nexstar Pharmacueticals Inc Target detection method using spectroscopically detectable nucleic acid ligands
US5789157A (en) 1990-06-11 1998-08-04 Nexstar Pharmaceuticals, Inc. Systematic evolution of ligands by exponential enrichment: tissue selex
US5683867A (en) 1990-06-11 1997-11-04 Nexstar Pharmaceuticals, Inc. Systematic evolution of ligands by exponential enrichment: blended SELEX
US5567588A (en) 1990-06-11 1996-10-22 University Research Corporation Systematic evolution of ligands by exponential enrichment: Solution SELEX
ATE318832T1 (de) 1990-06-11 2006-03-15 Gilead Sciences Inc Verfahren zur vervendung von nukleinsäureliganden
US5864026A (en) 1990-06-11 1999-01-26 Nexstar Pharmaceuticals, Inc. Systematic evolution of ligands by exponential enrichment: tissue selex
US6365730B1 (en) 1990-06-19 2002-04-02 Gene Shears Pty. Limited DNA-Armed ribozymes and minizymes
ES2139598T3 (es) 1990-07-10 2000-02-16 Medical Res Council Procedimientos para la produccion de miembros de parejas de union especifica.
AU651380B2 (en) 1990-07-16 1994-07-21 University Of Medicine And Dentistry Of New Jersey Selection of ribozymes that efficiently cleave target RNA
US5177196A (en) 1990-08-16 1993-01-05 Microprobe Corporation Oligo (α-arabinofuranosyl nucleotides) and α-arabinofuranosyl precursors thereof
AU649074B2 (en) 1990-10-12 1994-05-12 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Modified ribozymes
EP0563251A1 (en) 1990-12-20 1993-10-06 Exxon Chemical Patents Inc. Uv/eb curable butyl copolymers for lithographic and corrosion-resistant coating applications
DE69233697T2 (de) 1991-03-01 2008-01-24 Dyax Corp., Cambridge Verfahren zur Entwicklung von bindenden Mikroproteinen
PT100379B (pt) 1991-04-10 1999-01-29 Scripps Research Inst Bibliotecas de receptores heterodimericos usando fagomideos
US7015315B1 (en) 1991-12-24 2006-03-21 Isis Pharmaceuticals, Inc. Gapped oligonucleotides
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
EP0590058B1 (en) 1991-06-14 2003-11-26 Genentech, Inc. HUMANIZED Heregulin ANTIBODy
IE922437A1 (en) 1991-07-25 1993-01-27 Idec Pharma Corp Recombinant antibodies for human therapy
US5571799A (en) 1991-08-12 1996-11-05 Basco, Ltd. (2'-5') oligoadenylate analogues useful as inhibitors of host-v5.-graft response
US5700922A (en) 1991-12-24 1997-12-23 Isis Pharmaceuticals, Inc. PNA-DNA-PNA chimeric macromolecules
GB9206422D0 (en) 1992-03-24 1992-05-06 Bolt Sarah L Antibody preparation
AU4116793A (en) 1992-04-24 1993-11-29 Board Of Regents, The University Of Texas System Recombinant production of immunoglobulin-like domains in prokaryotic cells
US5652355A (en) 1992-07-23 1997-07-29 Worcester Foundation For Experimental Biology Hybrid oligonucleotide phosphorothioates
US5436143A (en) 1992-12-23 1995-07-25 Hyman; Edward D. Method for enzymatic synthesis of oligonucleotides
US5476925A (en) 1993-02-01 1995-12-19 Northwestern University Oligodeoxyribonucleotides including 3'-aminonucleoside-phosphoramidate linkages and terminal 3'-amino groups
GB9304618D0 (en) 1993-03-06 1993-04-21 Ciba Geigy Ag Chemical compounds
US6214345B1 (en) 1993-05-14 2001-04-10 Bristol-Myers Squibb Co. Lysosomal enzyme-cleavable antitumor drug conjugates
AU691811B2 (en) 1993-06-16 1998-05-28 Celltech Therapeutics Limited Antibodies
DK0733059T3 (da) 1993-12-09 2000-10-16 Univ Jefferson Forbindelser og fremgangsmåder til site-rettede mutationer i eukaryote celler
US5625050A (en) 1994-03-31 1997-04-29 Amgen Inc. Modified oligonucleotides and intermediates useful in nucleic acid therapeutics
US5650502A (en) 1994-11-09 1997-07-22 Hybridon, Inc. Ribozyme analogs having rigid non-nucleotidic linkers
US5869046A (en) 1995-04-14 1999-02-09 Genentech, Inc. Altered polypeptides with increased half-life
US6121022A (en) 1995-04-14 2000-09-19 Genentech, Inc. Altered polypeptides with increased half-life
US5693773A (en) 1995-06-07 1997-12-02 Hybridon Incorporated Triplex-forming antisense oligonucleotides having abasic linkers targeting nucleic acids comprising mixed sequences of purines and pyrimidines
US5652356A (en) 1995-08-17 1997-07-29 Hybridon, Inc. Inverted chimeric and hybrid oligonucleotides
CA2249195A1 (en) 1996-03-18 1997-09-25 Board Of Regents, The University Of Texas System Immunoglobin-like domains with increased half lives
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
WO1998023289A1 (en) 1996-11-27 1998-06-04 The General Hospital Corporation MODULATION OF IgG BINDING TO FcRn
US6277375B1 (en) 1997-03-03 2001-08-21 Board Of Regents, The University Of Texas System Immunoglobulin-like domains with increased half-lives
WO1999029888A1 (en) 1997-12-05 1999-06-17 The Scripps Research Institute Humanization of murine antibody
US6242246B1 (en) 1997-12-15 2001-06-05 Somalogic, Inc. Nucleic acid ligand diagnostic Biochip
US5989823A (en) 1998-09-18 1999-11-23 Nexstar Pharmaceuticals, Inc. Homogeneous detection of a target through nucleic acid ligand-ligand beacon interaction
US6194551B1 (en) 1998-04-02 2001-02-27 Genentech, Inc. Polypeptide variants
GB9809951D0 (en) 1998-05-08 1998-07-08 Univ Cambridge Tech Binding molecules
US6737056B1 (en) 1999-01-15 2004-05-18 Genentech, Inc. Polypeptide variants with altered effector function
HUP0104865A3 (en) 1999-01-15 2004-07-28 Genentech Inc Polypeptide variants with altered effector function
BRPI0008131B8 (pt) 1999-02-12 2021-05-25 Daiichi Sankyo Co Ltd composto ou um sal deste, análogo de oligonucleotídeo, composição farmacêutica, sonda para um gene,iniciador para começar a amplificação, uso de um análogo de oligonucleotídeo ou de um sal deste farmacologicamente aceitável, agente antisentido, e, agente antígeno
US6569630B1 (en) 1999-07-02 2003-05-27 Conceptual Mindworks, Inc. Methods and compositions for aptamers against anthrax
US7658921B2 (en) 2000-12-12 2010-02-09 Medimmune, Llc Molecules with extended half-lives, compositions and uses thereof
EP1355919B1 (en) 2000-12-12 2010-11-24 MedImmune, LLC Molecules with extended half-lives, compositions and uses thereof
US20050124566A1 (en) 2001-05-18 2005-06-09 Sirna Therapeutics, Inc. RNA interference mediated inhibition of myostatin gene expression using short interfering nucleic acid (siNA)
US20040121353A1 (en) 2002-05-23 2004-06-24 Ceptyr, Inc. Modulation of TCPTP signal transduction by RNA interference
EP2284269B1 (en) 2002-11-18 2017-08-09 Roche Innovation Center Copenhagen A/S Antisense design
CA2515623A1 (en) 2003-02-10 2004-08-19 Santaris Pharma A/S Oligomeric compounds for the modulation of survivin expression
GB0407315D0 (en) 2003-07-15 2004-05-05 Cambridge Antibody Tech Human antibody molecules
MXPA06000619A (es) 2003-07-15 2006-04-11 Hybridon Inc Estimulacion sinergistica del sistema inmune usando oligonucleotidos inmunoestimuladores y/o compuestos inmunomeros en conjunto con citocinas y/o agentes quimioterapeuticos o terapia de radiacion.
WO2005013901A2 (en) 2003-07-31 2005-02-17 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for use in modulation of small non-coding rnas
US20050053981A1 (en) 2003-09-09 2005-03-10 Swayze Eric E. Gapped oligomeric compounds having linked bicyclic sugar moieties at the termini
US20050074801A1 (en) 2003-09-09 2005-04-07 Monia Brett P. Chimeric oligomeric compounds comprising alternating regions of northern and southern conformational geometry
GB0324854D0 (en) 2003-10-24 2003-11-26 Expresson Biosystems Ltd App/ena antisense
CN1961003B (zh) 2004-03-31 2013-03-27 健泰科生物技术公司 人源化抗TGF-β抗体
KR101295139B1 (ko) 2004-06-07 2013-08-12 레이븐 바이오테크놀로지스, 인코퍼레이티드 트랜스페린 리셉터 항체
AR049390A1 (es) 2004-06-09 2006-07-26 Wyeth Corp Anticuerpos contra la interleuquina-13 humana y usos de los mismos
WO2006086667A2 (en) 2005-02-09 2006-08-17 Avi Bio Pharma, Inc. Antisense composition and method for treating muscle atrophy
CA2538208A1 (en) 2005-05-04 2006-11-04 Universite Laval Modulation of myostatin and use thereof in cell transplantation-based treatment of muscle disease
US7736647B2 (en) 2005-06-15 2010-06-15 Monoclonal Antibodies Therapeutics Anti-CD71 monoclonal antibodies and uses thereof for treating malignant tumor cells
JP2007104971A (ja) * 2005-10-14 2007-04-26 New Industry Research Organization ミオスタチンのスプライシング制御アンチセンスオリゴヌクレオチド
DE602007009487D1 (de) 2006-01-27 2010-11-11 Isis Pharmaceutical Inc 6-modifizierte bicyclische nukleinsäureanaloga
EP2388328A1 (en) 2006-01-27 2011-11-23 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for the use in modulation of micrornas
US7569686B1 (en) 2006-01-27 2009-08-04 Isis Pharmaceuticals, Inc. Compounds and methods for synthesis of bicyclic nucleic acid analogs
CA3042781C (en) 2006-04-03 2021-10-19 Roche Innovation Center Copenhagen A/S Pharmaceutical composition comprising anti-mirna antisense oligonucleotides
EP2505646A1 (en) 2006-05-05 2012-10-03 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of CRP
CA2651453C (en) 2006-05-11 2014-10-14 Isis Pharmaceuticals, Inc. 5'-modified bicyclic nucleic acid analogs
US20080081791A1 (en) 2006-07-06 2008-04-03 Weida Huang Methods of using combinations of siRNAs for treating a disease or a disorder, and for enhancing siRNA efficacy in RNAi
EP2076597A2 (en) 2006-10-09 2009-07-08 Santaris Pharma A/S Rna antagonist compounds for the modulation of pcsk9
EP2410053B2 (en) 2006-10-18 2020-07-15 Ionis Pharmaceuticals, Inc. Antisense compounds
US8318438B2 (en) 2007-03-01 2012-11-27 The United States Of America As Represented By The Secretary Of The Air Force Aptamer-based assays
EP2149605B1 (en) 2007-03-22 2013-07-03 Santaris Pharma A/S Short RNA antagonist compounds for the modulation of target mRNA
US8580756B2 (en) 2007-03-22 2013-11-12 Santaris Pharma A/S Short oligomer antagonist compounds for the modulation of target mRNA
PT2175884T (pt) 2007-07-12 2016-09-21 Gitr Inc Terapias de combinação empregando moléculas de ligação a gitr
WO2009090182A1 (en) 2008-01-14 2009-07-23 Santaris Pharma A/S C4'-substituted - dna nucleotide gapmer oligonucleotides
EP2268811A1 (en) 2008-03-07 2011-01-05 Santaris Pharma A/S Pharmaceutical compositions for treatment of microrna related diseases
US8846639B2 (en) 2008-04-04 2014-09-30 Isis Pharmaceutical, Inc. Oligomeric compounds comprising bicyclic nucleosides and having reduced toxicity
WO2009126913A1 (en) * 2008-04-11 2009-10-15 Cedars-Sinai Medical Center Poly(beta malic acid) with pendant leu-leu-leu tripeptide for effective cytoplasmic drug delivery
US20100077498A1 (en) 2008-09-11 2010-03-25 Pardridge William M Compositions and methods for blood-brain barrier delivery in the mouse
GB0904214D0 (en) * 2009-03-11 2009-04-22 Ucb Pharma Sa Biological products
US9364495B2 (en) 2009-10-20 2016-06-14 Roche Innovation Center Copenhagen A/S Oral delivery of therapeutically effective LNA oligonucleotides
US8859629B2 (en) 2010-04-27 2014-10-14 Synaffix B.V. Fused cyclooctyne compounds and their use in metal-free click reactions
CA2807552A1 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
CN103443125B (zh) 2010-11-30 2019-05-03 霍夫曼-拉罗奇有限公司 低亲和力血脑屏障受体抗体及其用途
US10017764B2 (en) 2011-02-08 2018-07-10 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
US20140088174A1 (en) 2011-04-05 2014-03-27 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Compounds and methods for altering activin receptor-like kinase signaling
HK1200484A1 (zh) 2011-09-14 2015-08-07 Translate Bio Ma, Inc. 多聚寡核苷酸化合物
US20150247141A1 (en) 2012-09-14 2015-09-03 Rana Therapeutics, Inc. Multimeric oligonucleotide compounds
US9695418B2 (en) 2012-10-11 2017-07-04 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleosides and uses thereof
HRP20180268T4 (hr) 2012-10-23 2026-03-27 Synaffix B.V. Modificirano antitijelo, konjugat antitijela i postupak za njihovu pripremu
EP2951304B1 (en) 2013-02-04 2020-07-08 Ionis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
SG11201509566RA (en) 2013-05-20 2015-12-30 Genentech Inc Anti-transferrin receptor antibodies and methods of use
US10004814B2 (en) 2013-11-11 2018-06-26 Sirna Therapeutics, Inc. Systemic delivery of myostatin short interfering nucleic acids (siNA) conjugated to a lipophilic moiety
JP6616189B2 (ja) 2013-12-25 2019-12-04 Jcrファーマ株式会社 血液脳関門を通過する新規抗トランスフェリン受容体抗体
EP3094728B1 (en) 2014-01-16 2022-03-09 Wave Life Sciences Ltd. Chiral design
WO2015152183A1 (ja) 2014-03-31 2015-10-08 大日本住友製薬株式会社 進行性骨化性線維異形成症の予防剤及び治療剤
CN103882024A (zh) * 2014-04-21 2014-06-25 石河子大学 一种RNAi干扰片段、RNAi干扰载体及其应用
JP6779876B2 (ja) * 2014-11-19 2020-11-04 ジェネンテック, インコーポレイテッド 抗トランスフェリン受容体抗体及びその使用方法
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
CA2980611A1 (en) 2015-04-07 2016-10-13 Paranta Biosciences Limited A method of treating neoplasias
CN107810273B (zh) 2015-04-23 2022-09-27 西纳福克斯股份有限公司 用为或衍生自β-(1,4)-N-乙酰半乳糖胺转移酶的糖基转移酶修饰糖蛋白的方法
DK3473270T5 (da) * 2016-06-20 2024-09-09 Genahead Bio Inc Konjugat af antistof-lægemiddel
EP3516062A1 (en) * 2016-09-21 2019-07-31 Alnylam Pharmaceuticals, Inc. Myostatin irna compositions and methods of use thereof
TWI833178B (zh) * 2016-12-26 2024-02-21 日商Jcr製藥股份有限公司 通過血腦障壁之新穎抗人類運鐵蛋白受體抗體
CN118638787A (zh) 2017-12-06 2024-09-13 艾维迪提生物科学公司 治疗肌萎缩和强直性肌营养不良的组合物和方法
CN111683972A (zh) * 2018-02-05 2020-09-18 Jcr制药股份有限公司 用于将药剂递送至肌肉的方法
CN112930193A (zh) * 2018-08-02 2021-06-08 达因疗法公司 肌肉靶向复合物及其在治疗肌肉萎缩中的用途

Similar Documents

Publication Publication Date Title
JP2023510351A5 (enExample)
JP2023512442A5 (enExample)
JP2023509793A5 (enExample)
JP2024501872A5 (enExample)
JP2021533199A5 (enExample)
JP2024525614A5 (enExample)
JP2023510350A5 (enExample)
JP2023535078A5 (enExample)
JP2023535073A5 (enExample)
JP2021533200A5 (enExample)
JP2021532832A5 (enExample)
JP7803926B2 (ja) 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それらの使用
JP2021533197A5 (enExample)
JP2024524222A5 (enExample)
IL317871A (en) Muscle targeting complexes and their uses for the treatment of FACIOSCAPULOHUMERAL muscular dystrophy
JP2022122928A5 (enExample)
IL274906B2 (en) Compositions and methods for treating muscular dystrophy and myotonic dystrophy
JP2023535444A5 (enExample)
JP2025067909A5 (enExample)
JP2020505330A5 (enExample)
JP2024527362A5 (enExample)
JP2024535797A5 (enExample)
JP2021533198A5 (enExample)
JP2024525612A5 (enExample)
JP2024503609A5 (enExample)