JPWO2020028864A5 - - Google Patents

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JPWO2020028864A5
JPWO2020028864A5 JP2021529261A JP2021529261A JPWO2020028864A5 JP WO2020028864 A5 JPWO2020028864 A5 JP WO2020028864A5 JP 2021529261 A JP2021529261 A JP 2021529261A JP 2021529261 A JP2021529261 A JP 2021529261A JP WO2020028864 A5 JPWO2020028864 A5 JP WO2020028864A5
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oligonucleotide
conjugate
transferrin receptor
complex
receptor antibody
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JP2021529261A
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Japanese (ja)
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JP2021533200A (ja
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Priority claimed from PCT/US2019/044990 external-priority patent/WO2020028864A1/en
Publication of JP2021533200A publication Critical patent/JP2021533200A/ja
Publication of JPWO2020028864A5 publication Critical patent/JPWO2020028864A5/ja
Priority to JP2024084487A priority Critical patent/JP2024122993A/ja
Pending legal-status Critical Current

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JP2021529261A 2018-08-02 2019-08-02 顔面・肩甲・上腕筋ジストロフィーを処置するための筋標的化複合体およびそれらの使用 Pending JP2021533200A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024084487A JP2024122993A (ja) 2018-08-02 2024-05-24 顔面・肩甲・上腕筋ジストロフィーを処置するための筋標的化複合体およびそれらの使用

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862713933P 2018-08-02 2018-08-02
US62/713,933 2018-08-02
PCT/US2019/044990 WO2020028864A1 (en) 2018-08-02 2019-08-02 Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy

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JP2024084487A Division JP2024122993A (ja) 2018-08-02 2024-05-24 顔面・肩甲・上腕筋ジストロフィーを処置するための筋標的化複合体およびそれらの使用

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JP2021533200A JP2021533200A (ja) 2021-12-02
JPWO2020028864A5 true JPWO2020028864A5 (enExample) 2022-08-09

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JP2021529261A Pending JP2021533200A (ja) 2018-08-02 2019-08-02 顔面・肩甲・上腕筋ジストロフィーを処置するための筋標的化複合体およびそれらの使用
JP2024084487A Pending JP2024122993A (ja) 2018-08-02 2024-05-24 顔面・肩甲・上腕筋ジストロフィーを処置するための筋標的化複合体およびそれらの使用

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US (12) US20220193250A1 (enExample)
EP (1) EP3830259A4 (enExample)
JP (2) JP2021533200A (enExample)
KR (1) KR20210081324A (enExample)
CN (1) CN112912499A (enExample)
AU (1) AU2019316103A1 (enExample)
CA (1) CA3108289A1 (enExample)
EA (1) EA202190416A1 (enExample)
IL (1) IL280426A (enExample)
MX (1) MX2021001284A (enExample)
SG (1) SG11202100928QA (enExample)
WO (1) WO2020028864A1 (enExample)

Families Citing this family (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
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
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
US20210308273A1 (en) 2018-08-02 2021-10-07 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
JP2021533200A (ja) 2018-08-02 2021-12-02 ダイン セラピューティクス, インコーポレーテッドDyne Therapeutics, Inc. 顔面・肩甲・上腕筋ジストロフィーを処置するための筋標的化複合体およびそれらの使用
CA3127243A1 (en) 2019-01-22 2020-07-30 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
EP3914259A4 (en) 2019-01-22 2023-05-17 Korro Bio, Inc. RNA-EDITING OLIGONUCLEOTIDES AND THEIR USE
WO2020154343A1 (en) * 2019-01-22 2020-07-30 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
US20230088865A1 (en) * 2020-01-10 2023-03-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
EP4121063A4 (en) * 2020-03-19 2024-07-03 Avidity Biosciences, Inc. COMPOSITIONS AND METHODS FOR TREATING FACIO-SCAPULO-HUMERAL MUSCULAR DYSTROPHY
EP4127175A4 (en) 2020-04-02 2025-01-22 Mirecule, Inc. Targeted inhibition using engineered oligonucleotides
EP4185314A4 (en) * 2020-07-23 2024-08-07 Dyne Therapeutics, Inc. MUSCLE-TARGETED COMPLEXES AND USES THEREOF FOR THE TREATMENT OF FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
WO2022056266A2 (en) 2020-09-11 2022-03-17 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of dux4, compositions thereof, and methods of use
KR20230117182A (ko) * 2020-12-04 2023-08-07 다인 세라퓨틱스, 인크. 항체-올리고뉴클레오티드 복합체 및 그의 용도
WO2022147207A1 (en) * 2020-12-31 2022-07-07 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
EP4359534A1 (en) * 2021-06-21 2024-05-01 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating pompe disease
WO2023283629A1 (en) * 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations thereof for treating facioscapulohumeral muscular dystrophy
US11633498B2 (en) 2021-07-09 2023-04-25 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
EP4366784A1 (en) 2021-07-09 2024-05-15 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating dystrophinopathies
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11648318B2 (en) 2021-07-09 2023-05-16 Dyne Therapeutics, Inc. Anti-transferrin receptor (TFR) antibody and uses thereof
EP4124345A1 (en) * 2021-07-30 2023-02-01 4basebio UK Ltd Nanoparticles for muscle delivery
WO2023006985A1 (en) * 2021-07-30 2023-02-02 4Basebio Uk Ltd Nanoparticles and peptides for the delivery of cargos to muscle cells
US11827702B2 (en) 2021-09-01 2023-11-28 Biogen Ma Inc. Anti-transferrin receptor antibodies and uses thereof
KR20240055874A (ko) 2021-09-16 2024-04-29 어비디티 바이오사이언시스 인크. 안면견갑상완 근이영양증을 치료하는 조성물 및 방법
EP4429711A1 (en) * 2021-11-12 2024-09-18 Dyne Therapeutics, Inc. Muscle targeting complexes for treating facioscapulohumeral muscular dystrophy
KR20250004770A (ko) 2022-04-15 2025-01-08 다인 세라퓨틱스, 인크. 근긴장성 이영양증을 치료하기 위한 근육 표적화 복합체 및 제제
EP4615418A1 (en) * 2022-11-10 2025-09-17 Ohio State Innovation Foundation Designer extracellular vesicles for targeted delivery to muscle cells
WO2025024334A1 (en) 2023-07-21 2025-01-30 Marrow Therapeutics, Inc. Hematopoietic cell targeting conjugates and related methods

Family Cites Families (324)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US2631173A (en) 1949-01-04 1953-03-10 Phillips Petroleum Co Production of cyclooctadiene
IL54795A (en) 1978-05-26 1980-10-26 Yeda Res & Dev Analgesic compositions comprising substituted bicyclic and spiroaliphatic amines and certain novel compounds of these types
DE68916959T2 (de) 1988-03-02 1995-01-19 Uab Research Foundation Reversibel mechanisch-chemisch arbeitende maschinen, die aus bioelastomeren bestehen, die zu regelbaren inversen temperaturübergängen fähig sind, zur umwandlung von chemischer in mechanische arbeit.
WO1991004753A1 (en) 1989-10-02 1991-04-18 Cetus Corporation Conjugates of antisense oligonucleotides and therapeutic uses thereof
US7015315B1 (en) 1991-12-24 2006-03-21 Isis Pharmaceuticals, Inc. Gapped oligonucleotides
US6100099A (en) 1994-09-06 2000-08-08 Abbott Laboratories Test strip having a diagonal array of capture spots
US6214345B1 (en) 1993-05-14 2001-04-10 Bristol-Myers Squibb Co. Lysosomal enzyme-cleavable antitumor drug conjugates
US6255455B1 (en) 1996-10-11 2001-07-03 The Trustees Of The University Of Pennsylvania Rh(D)-binding proteins and magnetically activated cell sorting method for production thereof
US6136603A (en) 1999-03-26 2000-10-24 Isis Pharmaceuticals Inc. Antisense modulation of interleukin-5 signal transduction
AU3412801A (en) 2000-02-22 2001-09-03 Banyu Pharma Co Ltd Novel imidazoline compounds
JP2003533986A (ja) 2000-05-04 2003-11-18 エイブイアイ バイオファーマ, インコーポレイテッド スプライス領域アンチセンス組成物および方法
EP1191097A1 (en) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
US20040063654A1 (en) 2001-11-02 2004-04-01 Davis Mark E. Methods and compositions for therapeutic use of RNA interference
US7241444B2 (en) 2002-01-18 2007-07-10 Pierre Fabre Medicament Anti-IGF-IR antibodies and uses thereof
WO2003078960A2 (en) 2002-03-11 2003-09-25 Momenta Pharmaceuticals, Inc. Analysis of sulfated polysaccharides
DK2284266T3 (da) 2002-11-14 2014-01-13 Thermo Fisher Scient Biosciences Inc sIRNA-MOLEKYLE MOD TP53
US7250496B2 (en) 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
DK2752488T3 (da) 2002-11-18 2020-04-20 Roche Innovation Ct Copenhagen As Antisense-design
EP2135948B1 (en) 2002-11-25 2014-09-17 Masafumi Matsuo ENA nucleic acid drugs modifying splicing in mRNA precursor
ES2361924T3 (es) 2002-12-20 2011-06-24 X-Ceptor Therapeutics, Inc. Derivados de isoquinolinona y su uso como agentes terapéuticos.
WO2004069991A2 (en) 2003-02-10 2004-08-19 Santaris Pharma A/S Oligomeric compounds for the modulation of survivin expression
CA2524255C (en) 2003-03-21 2014-02-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
EP1620564A4 (en) 2003-04-18 2008-03-12 Cytovia Inc METHODS FOR TREATING DISEASES INDUCING APOPTOSIS INDUCTION AND SCREENING ASSAYS
US7442372B2 (en) 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
US20050053981A1 (en) 2003-09-09 2005-03-10 Swayze Eric E. Gapped oligomeric compounds having linked bicyclic sugar moieties at the termini
ES2344302T3 (es) 2004-02-10 2010-08-24 Zystor Therapeutics , Inc. Alfa glucosidasa acida y fragmentos de la misma.
US8404747B2 (en) 2004-03-05 2013-03-26 The General Hospital Corporation Compositions and methods for modulating interaction between polypeptides
WO2005113571A2 (en) 2004-05-13 2005-12-01 The University Of North Carolina At Chapel Hill Methods for the delivery of oligomeric compounds
JP5697297B2 (ja) 2004-05-14 2015-04-08 ロゼッタ ジノミクス リミテッド マイクロnasおよびその使用
ATE498685T1 (de) 2004-06-28 2011-03-15 Univ Western Australia Antisense-oligonukleotide zur induktion von exon- skipping sowie verfahren zur verwendung davon
USRE48960E1 (en) 2004-06-28 2022-03-08 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
WO2006022688A1 (en) 2004-08-06 2006-03-02 Applera Corporation Diagnosis of pancreatic cancer by using pancreatic targets
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
PT1812031E (pt) 2004-11-01 2015-10-01 Univ California Composições e métodos para modificação de biomoléculas
US8431558B2 (en) 2004-11-01 2013-04-30 The Regents Of The University Of California Compositions and methods for modification of biomolecules
US20120122801A1 (en) 2005-01-05 2012-05-17 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
WO2006091796A2 (en) 2005-02-22 2006-08-31 Acucela, Inc. Compositions and methods for diagnosing and treating retinal diseases
JP2009511023A (ja) 2005-10-06 2009-03-19 ユニバーシティー、オブ、デラウェア ハンチントン病の治療のためのgに富むポリヌクレオチド
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
US20090214531A1 (en) 2006-01-30 2009-08-27 John Mekalanos Methods and Compositions for Treating and Preventing Bacterial Infections
CA2641359C (en) 2006-02-09 2022-10-04 Genzyme Corporation Slow intraventricular delivery
US20070249014A1 (en) 2006-02-10 2007-10-25 Invitrogen Corporation Labeling and detection of post translationally modified proteins
US8859752B2 (en) 2006-04-18 2014-10-14 The Trustees Of The University Of Pennsylvania SIRNA-based therapy of Fibrodyplasia Ossificans Progressiva (FOP)
WO2007136989A2 (en) 2006-05-05 2007-11-29 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of dgat2
EP1857548A1 (en) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
WO2008018795A1 (en) 2006-08-11 2008-02-14 Prosensa Technologies B.V. Methods and means for treating dna repeat instability associated genetic disorders
JP5665317B2 (ja) 2006-10-18 2015-02-04 アイシス ファーマシューティカルズ, インコーポレーテッド アンチセンス化合物
ES2569365T3 (es) 2006-11-29 2016-05-10 Nationwide Children's Hospital Inhibición de miostatina para la potenciación de músculo y/o la mejora de la función muscular
PT2457919T (pt) 2007-01-18 2019-09-20 Genzyme Corp Oligossacáridos compreendendo um grupo amino-oxi e os seus conjugados
DK2149605T3 (da) 2007-03-22 2013-09-30 Santaris Pharma As Korte RNA antagonist forbindelser til modulering af det ønskede mRNA
EP2149605B1 (en) 2007-03-22 2013-07-03 Santaris Pharma A/S Short RNA antagonist compounds for the modulation of target mRNA
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
PT2203173E (pt) 2007-10-26 2016-03-15 Academisch Ziekenhuis Leiden Resumo
WO2009075815A1 (en) 2007-12-07 2009-06-18 Duke University Immunomodulating gene therapy
WO2009124295A2 (en) 2008-04-04 2009-10-08 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleosides and having reduced toxicity
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
HUE025212T2 (en) 2008-07-08 2016-03-29 Univ Duke A method for treating glycogen storage disease
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
TR201902952T4 (tr) 2008-10-24 2019-03-21 Sarepta Therapeutics Inc Dmd için ekson atlama bileşimleri.
DK2344637T4 (en) 2008-10-27 2018-04-23 Biomarin Tech Bv Methods and means for efficiently skipping exon 45 in Duchenne muscular dystrophy pre-mRNA
BR122020010601B8 (pt) 2008-12-16 2021-07-27 Genzyme Corp conjugados de proteína-oligossacarídeo, seus usos, e composições farmacêuticas
US8466340B2 (en) 2009-02-18 2013-06-18 Amicus Therapeutics, Inc. Mouse model for pompe disease and methods of use thereof
NZ595955A (en) 2009-04-24 2012-10-26 Prosensa Technologies Bv Oligonucleotide comprising an inosine for treating dmd
CN102459597B (zh) * 2009-05-08 2020-06-30 库尔纳公司 通过针对dmd家族的天然反义转录物的抑制治疗肌营养蛋白家族相关疾病
EP3679942A1 (en) 2009-06-17 2020-07-15 BioMarin Pharmaceutical Inc. Formulations for lysosomal enzymes
FR2947269B1 (fr) 2009-06-29 2013-01-18 Sanofi Aventis Nouveaux composes anticancereux
WO2011036640A2 (en) 2009-09-24 2011-03-31 Cellectis Meganuclease reagents of uses thereof for treating genetic diseases caused by frame shift/non sense mutations
CA2780563C (en) 2009-11-12 2025-05-06 Univ Western Australia Antisense Molecules and Methods for Treating Pathologies
WO2011078797A2 (en) 2009-12-22 2011-06-30 Singapore Health Services Pte. Ltd Antisense oligonucleotides and uses threreof
WO2011130164A2 (en) 2010-04-13 2011-10-20 The Regents Of The University Of California Unconjugated anti-tfr antibodies and compositions thereof for the treatment of cancer
US20110256157A1 (en) 2010-04-15 2011-10-20 Spirogen Limited Pyrrolobenzodiazepines and conjugates thereof
ES2527536T7 (es) 2010-04-27 2016-03-08 Synaffix B.V. Compuestos de ciclooctina fusionada y su uso en reacciones clic sin metales
EP2576574A2 (en) 2010-05-28 2013-04-10 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.
EP2593142B8 (en) 2010-07-12 2018-12-26 Pfizer Healthcare Ireland Multifunctional antibody conjugates
KR20180105730A (ko) 2010-07-19 2018-09-28 아이오니스 파마수티컬즈, 인코포레이티드 근육긴장성 이영양증-단백질 키나제(dmpk) 발현의 조절 방법
US10865445B2 (en) 2010-08-18 2020-12-15 Fred Hutchinson Cancer Research Center Methods for alleviating facioscapulohumeral dystrophy (FSHD) by N siRNA molecule inhibiting the expression of DUX4-FL
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
US20120225034A1 (en) 2010-09-02 2012-09-06 Universite De Mons Agents useful in treating facioscapulohumeral muscular dystrophy
EA034333B1 (ru) 2010-11-30 2020-01-29 Дженентек, Инк. Варианты антитела для переноса соединения через гематоэнцефалический барьер
WO2012109296A1 (en) 2011-02-08 2012-08-16 The Charlotte-Mecklenburg Hospital Authority D/B/A Carolinas Medical Center Antisense oligonucleotides
EP2673361B1 (en) 2011-02-08 2016-04-13 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
WO2012144906A1 (en) 2011-04-22 2012-10-26 Prosensa Technologies B.V. New compounds for treating, delaying and/or preventing a human genetic disorder such as myotonic dystrophy type 1 (dm1)
CN103732259A (zh) 2011-06-24 2014-04-16 森彻斯有限公司 细胞外靶向药物缀合物
ES2990023T3 (es) * 2011-07-25 2024-11-28 Nationwide Childrens Hospital Inc Productos de virus recombinantes y métodos para inhibición de la expresión de DUX4
WO2013019623A2 (en) 2011-07-29 2013-02-07 Fred Hutchinson Cancer Research Center Methods and compositions for modulating the innate immune response and/or myogenesis in a mammalian subject
EP2753607B1 (en) 2011-09-09 2016-04-13 Berry And Associates, Inc. Bicyclo[6.1.0]non-4-yne reagents for chemical modification of oligonucleotides
WO2013085550A2 (en) 2011-12-05 2013-06-13 Duke University V1v2 immunogens
EP2788087A4 (en) 2011-12-06 2015-08-26 Ohio State Innovation Foundation Non-ionic, low-osmolar contrast agents for the administration of antisense oligonucleotides and the treatment of diseases
CN107881175B (zh) 2011-12-28 2022-05-13 日本新药株式会社 反义核酸
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
NZ627896A (en) 2012-01-27 2016-11-25 Biomarin Technologies B V Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
WO2013120038A2 (en) 2012-02-10 2013-08-15 Regents Of The University Of Minnesota Morpholino targeting dux4 for treating fshd
ES2812849T3 (es) 2012-02-24 2021-03-18 Abbvie Stemcentrx Llc Anticuerpos anti-DLL3 y procedimientos de utilización de los mismos
CN104379162B (zh) 2012-03-15 2017-03-15 奥克西雷恩英国有限公司 用于治疗蓬佩氏病的方法和材料
US9610362B2 (en) 2012-03-16 2017-04-04 Valerion Therapeutics, Llc Antisense conjugates for decreasing expression of DMPK
BR112014026285B1 (pt) 2012-04-23 2021-08-31 Biomarin Technologies B.V Oligonucleotídeos moduladores de rna com características melhoradas para o tratamento de distúrbios neuromusculares
EP3608407A1 (en) 2012-07-03 2020-02-12 BioMarin Technologies B.V. Oligonucleotide for the treatment of muscular dystrophy patients
EP2684892A1 (en) 2012-07-13 2014-01-15 Association Française contre les Myopathies Compositions and methods for duchenne muscular dystrophy gene therapy
ES2791778T3 (es) 2012-08-01 2020-11-05 Ikaika Therapeutics Llc Mitigación del daño tisular y la fibrosis a través de anticuerpos anti-LTBP4
EP2900821B1 (en) 2012-09-25 2020-04-01 Genzyme Corporation Peptide-linked morpholino antisense oligonucleotides for treatment of myotonic dystrophy
EP2906699A4 (en) 2012-10-11 2016-06-08 Ionis Pharmaceuticals Inc OLIGOMER COMPOUNDS WITH BICYCLIC NUCLEOSIDES AND USES THEREOF
JP6855661B2 (ja) 2012-10-23 2021-04-07 シンアフィックス ビー.ブイ. 修飾抗体、抗体コンジュゲート及びそれらを調製する方法
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
HUE042218T2 (hu) 2013-03-14 2019-06-28 Sarepta Therapeutics Inc Exonátugrást elõsegítõ készítmények izomdisztrófia kezelésére
NZ775701A (en) 2013-03-14 2022-08-26 Sarepta Therapeutics Inc Exon skipping compositions for treating muscular dystrophy
NZ732507A (en) 2013-03-15 2018-08-31 Sarepta Therapeutics Inc Improved compositions for treating muscular dystrophy
EP4324480A3 (en) 2013-05-20 2024-05-08 F. Hoffmann-La Roche AG Anti-transferrin receptor antibodies and methods of use
WO2014189370A1 (en) 2013-05-24 2014-11-27 Stichting Katholieke Universiteit Substituted azadibenzocyclooctyne compounds and their use in metal-free click reactions
JP7085716B2 (ja) 2013-06-05 2022-06-17 デューク ユニバーシティ Rnaガイド遺伝子編集及び遺伝子調節
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
CN105682687A (zh) 2013-08-16 2016-06-15 Rana医疗有限公司 异染色质形成性非编码rna
EP3033425A4 (en) 2013-08-16 2017-07-26 Rana Therapeutics, Inc. Compositions and methods for modulating expression of frataxin
AU2014312210A1 (en) 2013-08-28 2016-04-07 Abbvie Stemcentrx Llc Engineered anti-DLL3 conjugates and methods of use
WO2015042581A1 (en) 2013-09-23 2015-03-26 President And Fellows Of Harvard College Allele-specific rna silencing for the treatment of hypertrophic cardiomyopathy
EP3058083B1 (en) 2013-10-14 2018-04-11 SynAffix B.V. Modified glycoprotein, protein-conjugate and process for the preparation thereof
EP3057618B1 (en) 2013-10-14 2022-12-14 SynAffix B.V. Glycoengineered antibody, antibody-conjugate and methods for their preparation
EP2862928A1 (en) 2013-10-18 2015-04-22 Université de Strasbourg Dynamin 2 inhibitor for the treatment of centronuclear myopathies
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
WO2015073848A1 (en) 2013-11-14 2015-05-21 Shehadeh Lina A Methods and compositions employing an osteopontin aptamer to deliver nucleic acids into smooth muscle, endothelial, cardiac and progenitor/stem cells
EP3097079B1 (en) 2014-01-24 2021-05-12 SynAffix B.V. Process for the cycloaddition of a hetero(aryl) 1,3-dipole compound with a (hetero)cycloalkyne
US10266502B2 (en) 2014-01-24 2019-04-23 Synaffix B.V. Process for the cycloaddition of a halogenated 1,3-dipole compound with a (hetero)cycloalkyne
WO2015134365A2 (en) 2014-03-03 2015-09-11 Acharjee Sujata Chimeric dystrophin-vsv-g-protein to treat dystrophinopathies
TR201901939T4 (tr) 2014-03-12 2019-03-21 Nat Center Neurology & Psychiatry Antisens nükleik asit.
EP3119909B1 (en) 2014-03-18 2019-11-13 Fred Hutchinson Cancer Research Center Dux4-induced gene expression in facioscapulohumeral muscular dystrophy (fshd)
CA2946983A1 (en) 2014-04-25 2015-10-29 Yamaha Motor Power Products Kabushiki Kaisha Engine
JP6750148B2 (ja) 2014-04-25 2020-09-02 公益財団法人野口研究所 糖鎖切断抗体の製造方法及び均一糖鎖抗体
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
EP3159409B1 (en) 2014-06-17 2019-12-04 Nippon Shinyaku Co., Ltd. Antisense nucleic acid for use in the treatment of duchenne's muscular dystrophy
AU2015301221B2 (en) 2014-08-04 2020-07-02 MiRagen Therapeutics, Inc. Inhibitors of MYH7B and uses thereof
US11046988B2 (en) 2014-08-04 2021-06-29 Synaffix B.V. Process for the modification of a glycoprotein using a β-(1,4)-N-acetylgalactosaminyl transferase or a mutant thereof
AU2015301809A1 (en) 2014-08-11 2017-02-02 Shire Human Genetic Therapies, Inc. Mannose-6-phosphate bearing peptides fused to lysosomal enzymes
US20160075772A1 (en) 2014-09-12 2016-03-17 Regeneron Pharmaceuticals, Inc. Treatment of Fibrodysplasia Ossificans Progressiva
DK3201320T5 (da) 2014-09-30 2024-10-14 Amicus Therapeutics Inc Højpotent sur alfa-glucosidase med forbedrede carbohydrater
ES2740907T3 (es) 2014-10-03 2020-02-07 Synaffix Bv Enlazador de sulfamida, conjugados de los mismos y métodos de preparación
CA2967830A1 (en) 2014-11-14 2016-05-19 Ossianix, Inc. Tfr selective binding compounds and related methods
EP3221362B1 (en) 2014-11-19 2019-07-24 F.Hoffmann-La Roche Ag Anti-transferrin receptor antibodies and methods of use
AU2015349890B2 (en) 2014-11-21 2018-08-30 Merck Sharp & Dohme Llc Insulin receptor partial agonists
US20190002876A1 (en) 2014-12-09 2019-01-03 The Board Of Regents Of The University Of Texas System Compositions and methods for treatment of friedreich's ataxia
WO2016115490A1 (en) 2015-01-16 2016-07-21 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of dux4
US20180216111A1 (en) 2015-02-27 2018-08-02 Sarepta Therapeutics, Inc. Antisense-induced exon2 inclusion in acid alpha-glucosidase
EP3134520B1 (en) 2015-04-23 2017-12-20 Synaffix B.V. Process for the modification of a glycoprotein using a glycosyltransferase that is or is derived from a (1,4)-n-acetylgalactosaminyltransferase
TWI787796B (zh) 2015-05-04 2022-12-21 美商Cytomx生物製藥公司 抗cd71抗體類、可活化之抗cd71抗體類及使用彼等之方法
NZ737757A (en) 2015-05-19 2023-07-28 Sarepta Therapeutics Inc Peptide oligonucleotide conjugates
EA039366B1 (ru) 2015-06-24 2022-01-19 Джей Си Ар ФАРМАСЬЮТИКАЛЗ КО., ЛТД. Антитело к рецептору трансферрина человека, проникающее через гематоэнцефалический барьер
RU2737637C2 (ru) 2015-07-22 2020-12-01 Инатерис Антитела против tfr и их применение при лечении пролиферативных и воспалительных расстройств
SMT202000379T1 (it) 2015-09-15 2020-09-10 Nippon Shinyaku Co Ltd Acido nucleico antisenso
WO2017049157A1 (en) 2015-09-18 2017-03-23 Duke University Methods and compositions for the treatment of steatosis-associated disorders
US20180237775A1 (en) 2015-09-21 2018-08-23 Association Institut De Myologie Antisense oligonucleotides and uses thereof
WO2017049407A1 (en) 2015-09-23 2017-03-30 UNIVERSITé LAVAL Modification of the dystrophin gene and uses thereof
MA45819A (fr) 2015-10-09 2018-08-15 Sarepta Therapeutics Inc Compositions et méthodes pour traiter la dystrophie musculaire de duchenne et troubles associés
SG10201912902TA (en) 2015-10-09 2020-02-27 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof
CN108348621A (zh) 2015-11-05 2018-07-31 竹治疗股份有限公司 用于基因治疗的经修饰的弗里德赖希共济失调基因及载体
MX2018007061A (es) 2015-12-08 2018-08-15 Regeneron Pharma Composiciones y metodos para enzimas de internalizacion.
FR3044926B1 (fr) 2015-12-09 2020-01-31 Genethon Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine
NZ742916A (en) 2015-12-18 2023-03-31 Eisai R&D Man Co Ltd C-terminal lysine conjugated immunoglobulins
US10874746B2 (en) 2016-02-08 2020-12-29 Synaffix B.V. Sulfamide linkers for use in bioconjugates
JP7366541B2 (ja) 2016-02-19 2023-10-23 コード バイオセラピューティクス インコーポレイテッド 核酸キャリア及び治療上の使用方法
CA3019128A1 (en) 2016-03-30 2017-10-05 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
KR102343162B1 (ko) 2016-03-30 2021-12-23 아미쿠스 세라퓨틱스, 인코포레이티드 고 m6p 재조합 단백질의 선택 방법
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
JP7253379B2 (ja) 2016-04-02 2023-04-06 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 組織特異的発現のための改変u6プロモーターシステム
FR3049951A1 (fr) 2016-04-12 2017-10-13 Univ Rabelais Francois Nouvelles constructions peptidiques et leur utilisation dans le traitement de la toxoplasmose
EP3445405A4 (en) 2016-04-18 2019-12-18 Sarepta Therapeutics, Inc. ANTISENSE OLIGOMERS AND METHOD FOR USE THEREOF TO TREAT DISEASES RELATING TO THE ACID ALPHA GLUCOSIDASE GENE
MA45290A (fr) 2016-05-04 2019-03-13 Wave Life Sciences Ltd Procédés et compositions d'agents biologiquement actifs
US10953076B2 (en) 2016-05-24 2021-03-23 Merck Sharp & Dohme Corp. Insulin receptor partial agonists and GLP-1 analogues
US11013757B2 (en) 2016-06-03 2021-05-25 Wave Life Sciences Ltd. Oligonucleotides, compositions and methods thereof
CN109310765A (zh) * 2016-06-20 2019-02-05 领先基因生物技术股份有限公司 抗体-药物偶联物
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
KR20230175312A (ko) 2016-07-05 2023-12-29 바이오마린 테크놀로지스 비.브이. 유전적 장애 치료를 위한, 특징이 개선된, 비사이클릭 스캐폴드 모이어티를 포함하는 프리-mrna 스플라이스 스위칭 또는 조정 올리고뉴클레오티드
WO2018014042A1 (en) 2016-07-15 2018-01-18 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of dystrophin transcript
EP3487523B1 (en) 2016-07-19 2023-09-06 Duke University Therapeutic applications of cpf1-based genome editing
EP3293259A1 (en) 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
MX2019002842A (es) 2016-09-12 2019-08-29 Genethon Variantes de alfa-glucosidasa acida y usos de las mismas.
US10933081B2 (en) 2016-09-21 2021-03-02 Alnylam Pharmaceuticals, Inc. Myostatin iRNA compositions and methods of use thereof
ES3038159T3 (en) 2016-11-16 2025-10-09 Academisch Ziekenhuis Leiden Substances for targeting various selected organs or tissues
US10947540B2 (en) 2016-11-29 2021-03-16 Association Institut De Myologie Allele-specific silencing therapy for Dynamin 2-related diseases
CA3044531A1 (en) 2016-11-28 2018-05-31 The Board Of Regents Of The University Of Texas System Prevention of muscular dystrophy by crispr/cpf1-mediated gene editing
AU2017370730A1 (en) 2016-12-08 2019-06-27 The Board Of Regents Of The University Of Texas System DMD reporter models containing humanized duschene muscular dystrophy mutations
WO2018118627A1 (en) 2016-12-19 2018-06-28 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
JP6956741B2 (ja) 2016-12-26 2021-11-02 Jcrファーマ株式会社 血液脳関門を通過する新規な抗ヒトトランスフェリン受容体抗体
KR102471458B1 (ko) 2016-12-28 2022-11-25 제이씨알 파마 가부시키가이샤 동결건조 제제
JOP20190166A1 (ar) 2017-01-05 2019-07-02 Univ Texas استراتيجية مثلى من أجل تعديلات تخطي إكسون باستخدام crispr/cas9 مع متواليات توجيه ثلاثي
WO2018129384A1 (en) 2017-01-06 2018-07-12 Avidity Biosciences Llc Nucleic acid-polypeptide compositions and methods of inducing exon skipping
WO2018140831A2 (en) 2017-01-27 2018-08-02 Silverback Therapeutics, Inc. Tumor targeting conjugates and methods of use thereof
CN110809583A (zh) 2017-06-07 2020-02-18 瑞泽恩制药公司 用于内化酶的组合物和方法
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
CA3099522C (en) 2017-09-19 2024-10-08 Univ Alberta GAPMERES AND METHODS OF USING THESE FOR THE TREATMENT OF MUSCULAR DYSTROPHY
KR20250065737A (ko) 2017-09-22 2025-05-13 어비디티 바이오사이언시스 인크. 핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법
EA201991450A1 (ru) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
US20210145852A1 (en) 2017-09-28 2021-05-20 Sarepta Therapeutics, Inc. Combination Therapies for Treating Muscular Dystrophy
US20200248178A1 (en) 2017-09-28 2020-08-06 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
EP3668997A4 (en) 2017-10-02 2021-05-19 Research Institute at Nationwide Children's Hospital SYSTEM FOR MIRNA DETARGETING FOR TISSUE-SPECIFIC INTERFERENCE
SG11202003126VA (en) 2017-10-04 2020-05-28 Avidity Biosciences Inc Nucleic acid-polypeptide compositions and uses thereof
US11555189B2 (en) 2017-10-18 2023-01-17 Sarepta Therapeutics, Inc. Antisense oligomer compounds
WO2019092507A2 (en) 2017-11-09 2019-05-16 Crispr Therapeutics Ag Crispr/cas systems for treatment of dmd
WO2019110725A1 (en) 2017-12-06 2019-06-13 Synaffix B.V. Enediyne conjugates
CN118667812A (zh) 2017-12-06 2024-09-20 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
WO2019126641A2 (en) 2017-12-21 2019-06-27 Ionis Pharmaceuticals, Inc. Modulation of frataxin expression
US20210095283A1 (en) 2018-01-04 2021-04-01 Avidity Biosciences, Inc. Heteroduplex nucleic acid molecules and uses thereof
EP3735462A1 (en) 2018-01-05 2020-11-11 The Board of Regents of The University of Texas System Therapeutic crispr/cas9 compositions and methods of use
BR112020015617A2 (pt) 2018-01-31 2021-01-05 The Board Of Regents Of The University Of Texas System Composições e métodos para correção de mutações da distrofina em cardiomiócitos humanos
WO2019152820A1 (en) 2018-02-02 2019-08-08 Fred Hutchinson Cancer Research Center Methods for treating facioscapulohumeral muscular dystrophy
KR20200116952A (ko) 2018-02-05 2020-10-13 제이씨알 파마 가부시키가이샤 약제를 근육에 송달하기 위한 방법
KR20200118151A (ko) 2018-02-07 2020-10-14 리제너론 파마슈티칼스 인코포레이티드 치료 단백질 전달을 위한 방법 및 조성물
AU2019252680B2 (en) 2018-04-12 2025-11-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
WO2019209764A2 (en) 2018-04-26 2019-10-31 Sarepta Therapeutics, Inc. Exon skipping oligomers and oligomer conjugates for muscular dystrophy
WO2019215175A1 (en) 2018-05-08 2019-11-14 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating myh7 expression
SG11202010841QA (en) 2018-05-11 2020-11-27 Alpha Anomeric Sas Oligonucleotides conjugates comprising 7'-5'-alpha-anomeric-bicyclic sugar nucleosides
CA3098624A1 (en) 2018-05-11 2019-11-14 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
TWI869346B (zh) 2018-05-30 2025-01-11 瑞士商諾華公司 Entpd2抗體、組合療法、及使用該等抗體和組合療法之方法
US20220251551A1 (en) 2018-06-13 2022-08-11 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystrophy
WO2019246480A1 (en) 2018-06-21 2019-12-26 The Board Of Regents Of The University Of Texas System Correction of dystrophin exon 43, exon 45, or exon 52 deletions in duchenne muscular dystrophy
EP3829594A4 (en) 2018-08-02 2022-05-11 Dyne Therapeutics, Inc. MUSCLE TARGETING COMPLEXES AND THEIR USES FOR THE TREATMENT OF HYPERTROPHIC CARDIOMYOPATHY
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
WO2020028844A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating centronuclear myopathy
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
MX2021001283A (es) 2018-08-02 2021-07-15 Dyne Therapeutics Inc Complejos dirigidos al músculo y sus usos para tratar la distrofia miotónica.
WO2020028836A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle-targeting complexes and uses thereof in treating muscle atrophy
US12097263B2 (en) 2018-08-02 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
EP3829645A4 (en) 2018-08-02 2022-05-18 Dyne Therapeutics, Inc. MUSCLE TARGETING COMPLEXES AND THEIR USES FOR THE TREATMENT OF PROGRESSIVE FIBRODYSPLASIA OSSIFIANS
JP2021533200A (ja) 2018-08-02 2021-12-02 ダイン セラピューティクス, インコーポレーテッドDyne Therapeutics, Inc. 顔面・肩甲・上腕筋ジストロフィーを処置するための筋標的化複合体およびそれらの使用
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
EA202190417A1 (ru) 2018-08-02 2021-06-23 Дайн Терапьютикс, Инк. Мышечно-специфические комплексы и их применение для лечения болезни помпе
US20210308273A1 (en) 2018-08-02 2021-10-07 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
KR20210086601A (ko) 2018-08-02 2021-07-08 다인 세라퓨틱스, 인크. 근육 표적화 복합체 및 프리드라이히 운동실조를 치료하기 위한 그의 용도
CN112930197A (zh) 2018-08-02 2021-06-08 达因疗法公司 肌肉靶向复合物及其用途
US11708569B2 (en) 2018-08-29 2023-07-25 University Of Copenhagen Modified recombinant lysosomal alpha-galactosidase A and aspartylglucoaminidase having low mannose-6-phosphate and high sialic acid
IT201800009682A1 (it) 2018-10-22 2020-04-22 Ice Spa Coniugati di acidi biliari e loro derivati per la veicolazione di molecole attive
EP3876998A1 (en) 2018-11-05 2021-09-15 Synaffix B.V. Antibody-conjugates for targeting of tumours expressing trop-2
JP2022513719A (ja) 2018-12-06 2022-02-09 ウェイブ ライフ サイエンシズ リミテッド オリゴヌクレオチド組成物及びその方法
IL319265A (en) 2018-12-21 2025-04-01 Avidity Biosciences Inc Anti-transferrin receptor antibodies and their uses
JP2022516515A (ja) 2018-12-31 2022-02-28 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル Rna標的化crispr-cas13bを使用するdux4 rna発現停止
BR112021018801A2 (pt) 2019-03-28 2021-11-23 Sarepta Therapeutics Inc Métodos para tratar a distrofia muscular com casimersen
CN113677374A (zh) 2019-04-08 2021-11-19 国立大学法人东京医科齿科大学 肌疾病治疗用药物组合物
US20220193246A1 (en) 2019-04-18 2022-06-23 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
CA3137715A1 (en) 2019-04-25 2020-10-29 Avidity Biosciences, Inc. Nucleic acid compositions and methods of multi-exon skipping
KR20220019259A (ko) 2019-06-06 2022-02-16 어비디티 바이오사이언시스 인크. Una 아미다이트 및 이의 용도
JP7592034B2 (ja) 2019-06-06 2024-11-29 アビディティー バイオサイエンシーズ,インク. 核酸ポリペプチド組成物およびその使用
WO2020247738A1 (en) 2019-06-07 2020-12-10 Dyne Therapeutics, Inc. Methods of preparing protein-oligonucleotide complexes
EP3987029A1 (en) 2019-06-19 2022-04-27 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy
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
WO2021076856A1 (en) 2019-10-18 2021-04-22 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
EP4065225A1 (en) 2019-11-27 2022-10-05 DTX Pharma, Inc. Compounds and methods for the treatment of duchenne muscular dystrophy
WO2021127457A1 (en) 2019-12-20 2021-06-24 Codexis, Inc. Engineered acid alpha-glucosidase variants
EP4087878A4 (en) 2020-01-10 2024-06-05 Dyne Therapeutics, Inc. MUSCLE-TARGETED COMPLEXES AND USES THEREOF IN THE TREATMENT OF DYSTROPHINOPATHIES
BR112022013686A2 (pt) 2020-01-10 2022-09-06 Dyne Therapeutics Inc Complexos de direcionamento a músculo e usos dos mesmos para tratar distrofia miotônica
WO2021142217A1 (en) 2020-01-10 2021-07-15 Dyne Thereapeutics, Inc. Muscle targeting complexes and uses thereof for modulation of milck1
CN115335062A (zh) 2020-01-10 2022-11-11 达因疗法公司 肌肉靶向复合物及其用于调节与肌肉健康相关的基因的用途
US20230203180A1 (en) 2020-01-10 2023-06-29 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for modulation of genes associated with muscle health
WO2021142260A1 (en) 2020-01-10 2021-07-15 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for modulation of acvr1
WO2021142269A1 (en) 2020-01-10 2021-07-15 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for modulation of genes associated with muscle atrophy
US20230088865A1 (en) 2020-01-10 2023-03-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
WO2021142331A1 (en) 2020-01-10 2021-07-15 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for modulation of genes associated with cardiac muscle disease
CA3163608A1 (en) 2020-01-10 2021-07-15 Romesh R. Subramanian Muscle-targeting complexes and uses thereof
WO2021150382A1 (en) 2020-01-24 2021-07-29 Dyne Therapeutics, Inc. Muscle-targeting complexes and uses thereof in treating muscle atrophy
WO2021154477A1 (en) 2020-01-31 2021-08-05 Dyne Therapeutics, Inc. Anti-transferrin receptor (tfr) antibody and uses thereof
MX2022009418A (es) 2020-01-31 2022-08-25 Dyne Therapeutics Inc Anticuerpo del receptor anti-transferrina (tfr) y usos del mismo.
EP4121063A4 (en) 2020-03-19 2024-07-03 Avidity Biosciences, Inc. COMPOSITIONS AND METHODS FOR TREATING FACIO-SCAPULO-HUMERAL MUSCULAR DYSTROPHY
JP7748962B2 (ja) 2020-03-27 2025-10-03 アビディティー バイオサイエンシーズ,インク. 筋ジストロフィーを処置するための組成物および方法
EP4146229A4 (en) 2020-05-05 2025-07-23 Avidity Biosciences Inc COMPOSITIONS AND METHODS FOR TREATING POMPE DISEASE
KR20230041760A (ko) 2020-07-23 2023-03-24 다인 세라퓨틱스, 인크. 항-트랜스페린 수용체 (tfr) 항체 및 그의 용도
JP2023535078A (ja) 2020-07-23 2023-08-15 ダイン セラピューティクス,インコーポレーテッド 筋緊張性ジストロフィーを処置するための筋標的化複合体およびその使用
EP4185320A4 (en) 2020-07-23 2025-10-08 Dyne Therapeutics Inc MUSCLE TARGETING COMPLEXES AND THEIR USES IN THE TREATMENT OF MUSCLE ATROPHY
EP4185314A4 (en) 2020-07-23 2024-08-07 Dyne Therapeutics, Inc. MUSCLE-TARGETED COMPLEXES AND USES THEREOF FOR THE TREATMENT OF FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
JP2023535073A (ja) 2020-07-23 2023-08-15 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それらの使用
AU2021313058A1 (en) 2020-07-23 2023-03-30 Dyne Therapeutics, Inc. Muscle-targeting complexes and uses thereof
EP4208548A4 (en) 2020-09-01 2025-10-22 Ultragenyx Pharmaceutical Inc DUX4 INHIBITORS AND METHODS OF USE
KR20230079068A (ko) 2020-09-03 2023-06-05 다인 세라퓨틱스, 인크. 단백질-올리고뉴클레오티드 복합체의 제조 방법
WO2022056266A2 (en) 2020-09-11 2022-03-17 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of dux4, compositions thereof, and methods of use
US20240026356A1 (en) 2020-11-30 2024-01-25 Research Institute At Nationwide Children's Hospital Compositions and methods for treating facioscapulohumeral muscular dystrophy (fshd)
KR20230117182A (ko) 2020-12-04 2023-08-07 다인 세라퓨틱스, 인크. 항체-올리고뉴클레오티드 복합체 및 그의 용도
EP4271816A4 (en) 2020-12-31 2025-04-02 Dyne Therapeutics, Inc. MUSCLE TARGETING COMPLEXES AND RELATED USES FOR THE TREATMENT OF MYOTONIC DYSTROPHY
WO2022147207A1 (en) 2020-12-31 2022-07-07 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US20240336915A1 (en) 2021-01-22 2024-10-10 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing dux4 expression
MX2023011612A (es) 2021-03-31 2023-12-15 Entrada Therapeutics Inc Peptidos de penetracion celular ciclicos.
EP4314298A1 (en) 2021-04-02 2024-02-07 Avidity Biosciences, Inc. Antibody-oligonucleotide conjugate and antibody-peptide-oligonucleotide conjugate compositions and methods of inducing exon skipping
WO2022217366A1 (en) 2021-04-14 2022-10-20 The Royal Institution For The Advancement Of Learning/Mcgill University Modified oligonucleotides for the treatment of duchenne muscular dystrophy
AU2022267324A1 (en) 2021-04-30 2023-12-14 Sarepta Therapeutics, Inc. Treatment methods for muscular dystrophy
WO2022271543A2 (en) 2021-06-21 2022-12-29 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating friedreich's ataxia
EP4359534A1 (en) 2021-06-21 2024-05-01 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating pompe disease
AU2022298028A1 (en) 2021-06-23 2023-12-21 National Center Of Neurology And Psychiatry Combination of antisense oligomers
US11648318B2 (en) 2021-07-09 2023-05-16 Dyne Therapeutics, Inc. Anti-transferrin receptor (TFR) antibody and uses thereof
EP4366784A1 (en) 2021-07-09 2024-05-15 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating dystrophinopathies
KR20240035826A (ko) 2021-07-09 2024-03-18 다인 세라퓨틱스, 인크. 근육 표적화 복합체 및 디스트로핀병증을 치료하기 위한 그의 용도
JP2024525613A (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 筋標的化複合体およびジストロフィノパチーを処置するためのその使用
CN118355035A (zh) 2021-07-09 2024-07-16 达因疗法公司 肌肉靶向复合物及其用于治疗肌养蛋白病的用途
EP4367142A4 (en) 2021-07-09 2025-08-27 Dyne Therapeutics Inc MUSCLE TARGETING COMPLEXES AND THEIR USES FOR THE TREATMENT OF DYSTROPHINOPATHIES
EP4367247A4 (en) 2021-07-09 2025-08-27 Dyne Therapeutics Inc MUSCLE TARGETING COMPLEXES AND THEIR USES IN THE TREATMENT OF DYSTROPHINOPATHIES
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
CA3226361A1 (en) 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for modulation of genes associated with muscle health
US11633498B2 (en) 2021-07-09 2023-04-25 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
WO2023283629A1 (en) 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations 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
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
AU2022306307A1 (en) 2021-07-09 2024-02-01 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
JPWO2023022229A1 (enExample) 2021-08-19 2023-02-23
JPWO2023026994A1 (enExample) 2021-08-21 2023-03-02
CA3230279A1 (en) 2021-09-01 2023-03-09 Xiang Li Compositions and methods for skipping exon 45 in duchenne muscular dystrophy
KR20240055874A (ko) 2021-09-16 2024-04-29 어비디티 바이오사이언시스 인크. 안면견갑상완 근이영양증을 치료하는 조성물 및 방법
MX2024003373A (es) 2021-09-16 2024-04-04 Dyne Therapeutics Inc Dosis de complejos dirigidos a musculos para el tratamiento de distrofinopatias.
IL312142A (en) 2021-11-01 2024-06-01 Dyne Therapeutics Inc Muscle targeting complexes for the treatment of dystrophinopathy
EP4429711A1 (en) 2021-11-12 2024-09-18 Dyne Therapeutics, Inc. Muscle targeting complexes for treating facioscapulohumeral muscular dystrophy
EP4452331B8 (en) 2021-12-22 2025-09-17 Sapreme Technologies B.V. Targeted saponin-nucleic acid conjugates for treatment of muscle wasting disorders
WO2023121444A1 (en) 2021-12-22 2023-06-29 Sapreme Technologies B.V. Compositions comprising therapeutic nucleic acid and targeted saponin for the treatment of muscle-wasting disorders
JP2025500481A (ja) 2021-12-22 2025-01-09 サプリーム テクノロジーズ,ベー.フェー. 筋消耗性障害を治療するための治療用核酸とサポニンとを含む組成物
EP4469576A1 (en) 2022-01-27 2024-12-04 The Governors of the University of Alberta Pre-mrna slice modulating peptide-conjugated antisense therapeutics for the treatment of diseases
WO2023154807A2 (en) 2022-02-09 2023-08-17 Locanabio, Inc. Compositions and methods for modulating pre-mrna splicing
WO2023168427A1 (en) 2022-03-03 2023-09-07 Yale University Compositions and methods for delivering therapeutic polynucleotides for exon skipping
JP2025079347A (ja) 2022-03-11 2025-05-22 日本新薬株式会社 キャリアペプチドが連結された核酸
EP4493693A1 (en) 2022-03-17 2025-01-22 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer conjugates
US12071621B2 (en) 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping
WO2023201318A1 (en) 2022-04-15 2023-10-19 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating myotonic dystrophy
IL316206A (en) 2022-04-15 2024-12-01 Dyne Therapeutics Inc Dosage of muscle targeting complexes for the treatment of myotonic dystrophy
KR20250004770A (ko) 2022-04-15 2025-01-08 다인 세라퓨틱스, 인크. 근긴장성 이영양증을 치료하기 위한 근육 표적화 복합체 및 제제
KR20250004769A (ko) 2022-04-15 2025-01-08 다인 세라퓨틱스, 인크. 근긴장성 이영양증을 치료하기 위한 근육 표적화 복합체 및 그의 용도
WO2024011150A2 (en) 2022-07-06 2024-01-11 Dyne Therapeutics, Inc. Cns targeting complexes and uses thereof
WO2024011135A2 (en) 2022-07-06 2024-01-11 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
WO2024064237A2 (en) 2022-09-21 2024-03-28 Sarepta Therapeutics, Inc. Dmd antisense oligonucleotide-mediated exon skipping efficiency
WO2024097644A1 (en) 2022-10-31 2024-05-10 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for skipping exon 45 of a dmd gene
CN120529923A (zh) 2022-11-21 2025-08-22 肽世代公司 细胞穿透肽、其缀合物及其使用方法
AU2024208393A1 (en) 2023-01-10 2025-07-10 Ausperbio Therapeutics Inc. Modified multi-segmented antisense oligonucleotides for use
KR20250159196A (ko) 2023-02-27 2025-11-10 다인 세라퓨틱스, 인크. 근육내 섬유증의 진행을 억제하기 위한 방법 및 조성물

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