SG11202100934PA - Muscle targeting complexes and uses thereof for treating dystrophinopathies - Google Patents

Muscle targeting complexes and uses thereof for treating dystrophinopathies

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Publication number
SG11202100934PA
SG11202100934PA SG11202100934PA SG11202100934PA SG11202100934PA SG 11202100934P A SG11202100934P A SG 11202100934PA SG 11202100934P A SG11202100934P A SG 11202100934PA SG 11202100934P A SG11202100934P A SG 11202100934PA SG 11202100934P A SG11202100934P A SG 11202100934PA
Authority
SG
Singapore
Prior art keywords
muscle targeting
targeting complexes
dystrophinopathies
treating
treating dystrophinopathies
Prior art date
Application number
SG11202100934PA
Inventor
Romesh Subramanian
Mohammed Qatanani
Timothy Weeden
Original Assignee
Dyne Therapeutics Inc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Dyne Therapeutics Inc filed Critical Dyne Therapeutics Inc
Publication of SG11202100934PA publication Critical patent/SG11202100934PA/en

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    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/68Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • A61K47/6801Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
    • A61K47/6803Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
    • A61K47/6807Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates the drug or compound being a sugar, nucleoside, nucleotide, nucleic acid, e.g. RNA antisense
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/10Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
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    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
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    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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    • C12Y204/00Glycosyltransferases (2.4)
    • C12Y204/02Pentosyltransferases (2.4.2)
    • C12Y204/02008Hypoxanthine phosphoribosyltransferase (2.4.2.8)
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C07K2317/62Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
    • C07K2317/622Single chain antibody (scFv)
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    • C07K2317/00Immunoglobulins specific features
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SG11202100934PA 2018-08-02 2019-08-02 Muscle targeting complexes and uses thereof for treating dystrophinopathies SG11202100934PA (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862714031P 2018-08-02 2018-08-02
US201962855766P 2019-05-31 2019-05-31
PCT/US2019/044949 WO2020028832A1 (en) 2018-08-02 2019-08-02 Muscle targeting complexes and uses thereof for treating dystrophinopathies

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SG11202100934PA true SG11202100934PA (en) 2021-02-25

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US (6) US20210308273A1 (en)
EP (1) EP3829595A4 (en)
JP (1) JP2021532831A (en)
KR (1) KR20210081322A (en)
CN (1) CN112955153A (en)
AU (1) AU2019312692A1 (en)
CA (1) CA3108282A1 (en)
IL (1) IL280527A (en)
MX (1) MX2021001281A (en)
SG (1) SG11202100934PA (en)
WO (1) WO2020028832A1 (en)

Families Citing this family (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20210081324A (en) 2018-08-02 2021-07-01 다인 세라퓨틱스, 인크. Muscle targeting complexes and their use for treating facioscapulohumeral muscular dystrophy
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
CA3108282A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
JP2022513482A (en) 2018-12-21 2022-02-08 アビディティー バイオサイエンシーズ,インク. Anti-transferrin receptor antibody and its use
KR20220118459A (en) * 2019-12-19 2022-08-25 니뽄 신야쿠 가부시키가이샤 Antisense nucleic acids that enable exon skipping
CN115666589A (en) 2020-03-19 2023-01-31 艾维迪提生物科学公司 Compositions and methods for treating facioscapulohumeral muscular dystrophy
US20230285586A1 (en) * 2020-07-23 2023-09-14 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
CA3186755A1 (en) * 2020-07-23 2022-01-27 Romesh R. Subramanian Muscle-targeting complexes and uses thereof
EP4330395A1 (en) 2021-04-30 2024-03-06 Sarepta Therapeutics, Inc. Treatment methods for muscular dystrophy
EP4361269A1 (en) 2021-06-23 2024-05-01 Nippon Shinyaku Co., Ltd. Combination of antisense oligomers
US11672872B2 (en) 2021-07-09 2023-06-13 Dyne Therapeutics, Inc. Anti-transferrin receptor antibody and uses thereof
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11633498B2 (en) 2021-07-09 2023-04-25 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
IL309909A (en) * 2021-07-09 2024-03-01 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating dystrophinopathies
IL309911A (en) * 2021-07-09 2024-03-01 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating dystrophinopathies
WO2023026994A1 (en) 2021-08-21 2023-03-02 武田薬品工業株式会社 Human transferrin receptor binding peptide-drug conjugate
IL311025A (en) 2021-08-24 2024-04-01 Peptidream Inc Human transferrin receptor-binding antibody-peptide conjugate
IL311175A (en) 2021-09-01 2024-04-01 Biogen Ma Inc Anti-transferrin receptor antibodies and uses thereof
CA3231330A1 (en) 2021-09-16 2023-03-23 Avidity Biosciences, Inc. Compositions and methods of treating facioscapulohumeral muscular dystrophy
WO2023178230A1 (en) 2022-03-17 2023-09-21 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer conjugates
US11931421B2 (en) 2022-04-15 2024-03-19 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating myotonic dystrophy

Family Cites Families (200)

* 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
ATE108692T1 (en) 1988-03-02 1994-08-15 Uab Research Foundation REVERSIBLE MECHANICAL-CHEMICAL MACHINES MADE OF BIOELASTOMERS CAPABLE OF ADJUSTABLE INVERSE TEMPERATURE TRANSITIONS TO TRANSFORM CHEMICAL WORK INTO MECHANICAL WORK.
WO1991004753A1 (en) 1989-10-02 1991-04-18 Cetus Corporation Conjugates of antisense oligonucleotides and therapeutic uses thereof
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
GB9722131D0 (en) 1997-10-20 1997-12-17 Medical Res Council Method
EP1264826B1 (en) 2000-02-22 2005-03-30 Banyu Pharmaceutical Co., Ltd. Novel imidazoline compounds
EP1282699B1 (en) 2000-05-04 2012-11-21 Sarepta Therapeutics, Inc. Splice-region antisense composition and method
EP1191097A1 (en) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
JP2002253259A (en) 2001-03-02 2002-09-10 Bio Applied Systems:Kk Gene encoding anti-bisphenol a antibody, recombinant protein and method for producing the same
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
EP2284535A1 (en) 2002-03-11 2011-02-16 Momenta Pharmaceuticals, Inc. Low molecular weight heparins
ES2509140T3 (en) 2002-11-25 2014-10-17 Masafumi Matsuo ENA nucleic acid drugs that modify splicing in mRNA precursors
WO2004055164A2 (en) 2002-12-13 2004-07-01 Abgenix, Inc. System and method for stabilizing antibodies with histidine
AU2003301020A1 (en) 2002-12-20 2004-07-22 Sankyo Company, Limited Isoquinolinone derivatives and their use as therapeutic agents
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
EP2241331A3 (en) 2003-12-15 2011-03-09 Alexion Pharmaceuticals, Inc. Novel anti-DC-SIGN antibodies
WO2005111211A2 (en) 2004-05-14 2005-11-24 Rosetta Genomics Ltd. Micronas and uses thereof
SI2206781T1 (en) 2004-06-28 2016-05-31 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 (en) 2004-08-17 2010-07-30 Genethon ADENO-ASSOCIATED VIRAL VECTOR FOR PRODUCING EXON JUMP IN A GENE ENCODING A PROTEIN WITH DISPENSABLE DOMAINS
WO2006050262A2 (en) 2004-11-01 2006-05-11 The Regents Of The University Of California Compositions and methods for modification of biomolecules
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
EP2623986B1 (en) 2006-02-10 2017-06-14 Life Technologies 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)
EP1857548A1 (en) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
PT2121751T (en) 2006-12-08 2017-04-18 Lexicon Pharmaceuticals Inc Monoclonal antibodies against angptl3
EP3330292A1 (en) 2007-08-21 2018-06-06 Amgen, Inc Human c-fms antigen binding proteins
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
WO2009054725A2 (en) 2007-10-26 2009-04-30 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
EP2119783A1 (en) * 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
WO2009144481A2 (en) 2008-05-30 2009-12-03 Isis Innovation Limited Conjugates for delivery of biologically active compounds
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
WO2010048586A1 (en) 2008-10-24 2010-04-29 Avi Biopharma, Inc. Multiple exon skipping compositions for dmd
AU2009310557B2 (en) 2008-10-27 2014-09-11 Academisch Ziekenhuis Leiden Methods and means for efficient skipping of exon 45 in Duchenne Muscular Dystrophy pre-mRNA
JP2012524540A (en) 2009-04-24 2012-10-18 プロセンサ テクノロジーズ ビー.ブイ. Oligonucleotides containing inosine for treating DMD
FR2947269B1 (en) 2009-06-29 2013-01-18 Sanofi Aventis NEW ANTICANCER COMPOUNDS
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
KR102487132B1 (en) 2009-11-12 2023-01-10 더 유니버시티 오브 웨스턴 오스트레일리아 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
US8734799B2 (en) 2010-04-13 2014-05-27 The Regents Of The University Of California Unconjugated anti-TfR antibodies and compositions thereof for the treatment of cancer
EP3604264A1 (en) 2010-04-27 2020-02-05 SynAffix B.V. Fused cyclooctyne compounds
KR102095478B1 (en) 2010-05-28 2020-04-01 사렙타 쎄러퓨틱스, 인코퍼레이티드 Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
IT1400425B1 (en) 2010-06-08 2013-05-31 Amsterdam Molecular Therapeutics Bv MODIFIED SNRNAS FOR USE IN THERAPY.
CA2804185C (en) 2010-07-12 2017-03-21 Covx Technologies Ireland Limited Multifunctional antibody conjugates
TWI541024B (en) 2010-09-01 2016-07-11 日本新藥股份有限公司 Antisense nucleic acid
EP3118316A1 (en) 2010-09-02 2017-01-18 Université de Mons Agents useful in treating facioscapulohumeral muscular dystrophy
ES2631356T3 (en) 2010-11-30 2017-08-30 F. Hoffmann-La Roche Ag Low affinity transferrin receptor anti-antibodies and their use to transfer therapeutic scFv through the blood brain barrier
EA028744B1 (en) 2010-12-20 2017-12-29 Дженентек, Инк. Anti-mesothelin antibodies and immunoconjugates
KR20140052963A (en) 2011-02-08 2014-05-07 더 샬롯테-맥클렌버그 하스피털 오쏘러티 디/비/에이 카롤리나스 헬스케어 시스템 Antisense oligonucleotides
RU2013143358A (en) 2011-04-07 2015-05-20 Дженентек, Инк. ANTI-FGFR4 ANTIBODIES AND WAYS OF THEIR APPLICATION
US20140193436A1 (en) 2011-06-24 2014-07-10 Centrose, Llc Extracellular targeted drug conjugates
BR112014003999A2 (en) 2011-08-23 2017-06-13 Roche Glycart Ag isolated antibody that binds to a near epitope near the human mcsp membrane, isolated nucleic acid, host cell, method of producing an antibody, immunoconjugate, pharmaceutical formulation, use of the antibody, method of treating cancer patients, unduction method of cell lysis in individuals and mcsp immunohistochemical test
WO2013036748A1 (en) 2011-09-09 2013-03-14 Berry, Lana, L. Bicyclo[6.1.0]non-4-yne reagents for chemical modification of oligonucleotides
CA2858347C (en) 2011-12-05 2020-07-07 Duke University V1v2 immunogens
CA2858576A1 (en) 2011-12-06 2013-06-13 Research Institute At Nationwide Children's Hospital Non-ionic, low osmolar contrast agents for delivery of antisense oligonucleotides and treatment of disease
CA2861247C (en) 2011-12-28 2021-11-16 Nippon Shinyaku Co., Ltd. Antisense nucleic acids
US20130177579A1 (en) 2012-01-06 2013-07-11 Bioalliance C.V. Anti-transferrin receptor antibodies and methods using same
SI2817338T1 (en) 2012-02-24 2017-11-30 Abbvie Stemcentrx Llc Dll3 modulators and methods of use
US9610362B2 (en) 2012-03-16 2017-04-04 Valerion Therapeutics, Llc Antisense conjugates for decreasing expression of DMPK
RS56319B1 (en) 2012-04-23 2017-12-29 Biomarin Tech Bv Rna modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders
JP6460983B2 (en) 2012-07-03 2019-01-30 バイオマリン テクノロジーズ ベー.フェー. Oligonucleotides for the treatment of patients with muscular dystrophy
EP2684892A1 (en) 2012-07-13 2014-01-15 Association Française contre les Myopathies Compositions and methods for duchenne muscular dystrophy gene therapy
EP2895200B1 (en) 2012-09-14 2019-11-06 Translate Bio MA, Inc. Multimeric oligonucleotide compounds
RS64329B1 (en) 2012-10-23 2023-08-31 Synaffix Bv Modified antibody, antibody-conjugate and process for the preparation thereof
ES2701076T3 (en) 2012-11-24 2019-02-20 Hangzhou Dac Biotech Co Ltd Hydrophilic linkers and their uses for the conjugation of drugs to molecules that bind to cells
DK2958944T3 (en) 2013-02-22 2019-06-24 Abbvie Stemcentrx Llc ANTI-DLL3 ANTISTOF PBD CONJUGATES AND USES THEREOF
PL2970964T3 (en) 2013-03-14 2019-06-28 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
CA2906812A1 (en) 2013-03-15 2014-09-18 Sarepta Therapeutics, Inc. Improved compositions for treating muscular dystrophy
JP6612214B2 (en) 2013-05-20 2019-11-27 ジェネンテック, インコーポレイテッド Anti-transferrin receptor antibodies and methods of use
US20160107999A1 (en) 2013-05-24 2016-04-21 Synaffix B.V. Substituted azadibenzocyclooctyne compounds and their use in metal-free click reactions
CN114230675A (en) 2013-06-05 2022-03-25 杜克大学 RNA-guided gene editing and gene regulation
CA2922544A1 (en) 2013-08-28 2015-03-05 Stemcentrx, Inc. Engineered anti-dll3 conjugates and methods of use
WO2015057065A1 (en) 2013-10-14 2015-04-23 Synaffix B.V. Glycoengineered antibody, antibody-conjugate and methods for their preparation
EP3058083B1 (en) 2013-10-14 2018-04-11 SynAffix B.V. Modified glycoprotein, protein-conjugate and process for the preparation thereof
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
EP3097080A1 (en) 2014-01-24 2016-11-30 SynAffix B.V. Process for the cycloaddition of a halogenated 1,3-dipole compound with a (hetero)cycloalkyne
JP2017505770A (en) 2014-01-24 2017-02-23 シンアフィックス ビー.ブイ. Process for cycloaddition of (hetero) aryl 1,3-dipole compounds with (hetero) cycloalkynes
AU2015227733B2 (en) 2014-03-12 2019-10-31 National Center Of Neurology And Psychiatry Antisense nucleic acids
AR099625A1 (en) 2014-03-21 2016-08-03 Lilly Co Eli IL-21 ANTIBODIES
JP6750148B2 (en) 2014-04-25 2020-09-02 公益財団法人野口研究所 Process for producing sugar chain cleaving antibody and uniform sugar chain antibody
TWI713450B (en) 2014-05-23 2020-12-21 美商健臻公司 Inhibiting or downregulating glycogen synthase by creating premature stop codons using antisense oligonucleotides
RS59764B1 (en) 2014-06-17 2020-02-28 Nippon Shinyaku Co Ltd Antisense nucleic acid for use in the treatment of duchenne's muscular dystrophy
EP3177633B1 (en) 2014-08-04 2022-07-20 SynAffix B.V. Process for the modification of a glycoprotein using a beta-(1,4)-n-acetylgalactosaminyltransferase or a mutant thereof
JP6733993B2 (en) 2014-10-03 2020-08-05 シンアフィックス ビー.ブイ. Sulfamide linker, conjugate of sulfamide linker, and method of preparation
AU2015346045B2 (en) 2014-11-14 2021-08-26 Ossianix, Inc. Variable new antigen receptors (VNARs) directed against transferrin receptor (TfR) and their use
JP6779876B2 (en) 2014-11-19 2020-11-04 ジェネンテック, インコーポレイテッド Anti-transferrin receptor antibody and how to use it
CA3014641C (en) 2014-11-21 2020-08-18 Songnian Lin Insulin receptor partial agonists
WO2016170186A1 (en) 2015-04-23 2016-10-27 Synaffix B.V. PROCESS FOR THE MODIFICATION OF A GLYCOPROTEIN USING A GLYCOSYLTRANSFERASE THAT IS OR IS DERIVED FROM A β(1,4)-N-ACETYLGALACTOSAMINYLTRANSFERASE
EP4029880A1 (en) 2015-05-04 2022-07-20 CytomX Therapeutics, Inc. Activatable anti-cd71 antibodies, and methods of use thereof
EP4306538A3 (en) 2015-05-19 2024-05-01 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
EP3325509B1 (en) 2015-07-22 2020-12-16 Inatherys Anti-tfr antibodies and their use in treating proliferative and inflammatory disorders
US10144931B2 (en) 2015-09-15 2018-12-04 Nippon Shinyaku Co., Ltd. Antisense nucleic acids
EP3332008A4 (en) 2015-09-23 2019-01-16 Université Laval Modification of the dystrophin gene and uses thereof
MX2018003992A (en) 2015-10-09 2018-08-01 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof.
EP3858993A1 (en) 2015-10-09 2021-08-04 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders
EP4074331A1 (en) 2015-12-08 2022-10-19 Regeneron Pharmaceuticals, Inc. Compositions and methods for internalizing enzymes
FR3044926B1 (en) 2015-12-09 2020-01-31 Genethon EFFICIENT GENE THERAPY TOOLS FOR JUMPING DYSTROPHIN EXON 53
EP3413922A1 (en) 2016-02-08 2018-12-19 SynAffix B.V. Improved sulfamide linkers for use in bioconjugates
US20210213138A1 (en) 2016-02-19 2021-07-15 Genisphere Llc Nucleic Acid Carriers And Therapeutic Methods of Use
JP7015244B2 (en) 2016-03-22 2022-02-02 エフ・ホフマン-ラ・ロシュ・アクチェンゲゼルシャフト Protease-activated T cell bispecific molecule
MA45328A (en) 2016-04-01 2019-02-06 Avidity Biosciences Llc NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND USES THEREOF
FR3049951A1 (en) 2016-04-12 2017-10-13 Univ Rabelais Francois NOVEL PEPTIDE CONSTRUCTS AND THEIR USE IN THE TREATMENT OF TOXOPLASMOSIS
MA45290A (en) 2016-05-04 2019-03-13 Wave Life Sciences Ltd PROCESSES AND COMPOSITIONS OF BIOLOGICALLY ACTIVE AGENTS
WO2017205191A1 (en) 2016-05-24 2017-11-30 Merck Sharp & Dohme Corp. Insulin receptor partial agonists and glp-1 analogues
MA45188A (en) 2016-06-03 2019-04-10 Wave Life Sciences Ltd OLIGONUCLEOTIDES, ASSOCIATED COMPOSITIONS AND METHODS
EP4212176A1 (en) * 2016-06-20 2023-07-19 Genahead Bio, Inc. Antibody-drug conjugate
WO2018007475A1 (en) 2016-07-05 2018-01-11 Biomarin Technologies B.V. Pre-mrna splice switching or modulating oligonucleotides comprising bicyclic scaffold moieties, with improved characteristics for the treatment of genetic disorders
US20190330626A1 (en) * 2016-07-15 2019-10-31 Ionis Pharmaceuticals, Inc. Compounds and methods for use in dystrophin transcript
US20190151476A1 (en) 2016-07-19 2019-05-23 Duke University Therapeutic applications of cpf1-based genome editing
WO2018091544A1 (en) 2016-11-16 2018-05-24 Biomarin Pharmaceutical, Inc. 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
EP3545090A1 (en) 2016-11-28 2019-10-02 The Board of Regents of The University of Texas System Prevention of muscular dystrophy by crispr/cpf1-mediated gene editing
US20190364862A1 (en) 2016-12-08 2019-12-05 The Board Of Regents Of The University Of Texas System Dmd reporter models containing humanized duchenne muscular dystrophy mutations
JOP20190166A1 (en) 2017-01-05 2019-07-02 Univ Texas Optimized strategy for exon skipping modifications using crispr/cas9 with triple guide sequences
MX2019008199A (en) 2017-01-06 2019-11-25 Avidity Biosciences Llc Nucleic acid-polypeptide compositions and methods of inducing exon skipping.
AU2018281280A1 (en) 2017-06-07 2020-01-16 Regeneron Pharmaceuticals, Inc. Compositions and methods for internalizing enzymes
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
MX2020003130A (en) 2017-09-22 2020-09-25 Avidity Biosciences Inc Nucleic acid-polypeptide compositions and methods of inducing exon skipping.
EA201991450A1 (en) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. OLIGOMER CONJUGATES FOR EXONISM SKIP IN MUSCULAR DYSTROPHY
JP2020536060A (en) 2017-09-28 2020-12-10 サレプタ セラピューティクス, インコーポレイテッド Combination therapy to treat muscular dystrophy
MA50753A (en) 2017-10-04 2020-08-12 Avidity Biosciences Inc NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND USES THEREOF
EP3707155A2 (en) 2017-11-09 2020-09-16 Vertex Pharmaceuticals Incorporated Crispr/cas systems for treatment of dmd
EP3720448A4 (en) 2017-12-06 2021-11-03 Avidity Biosciences, Inc. Compositions and methods of treating muscle atrophy and myotonic dystrophy
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
US20200370042A1 (en) 2018-01-31 2020-11-26 The Board Of Regents Of The University Of Texas System Compositions and methods for correcting dystrophin mutations in human cardiomyocytes
CN112040985A (en) 2018-02-07 2020-12-04 瑞泽恩制药公司 Methods and compositions for delivery of therapeutic proteins
CA3096667A1 (en) 2018-04-12 2019-10-17 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
EP3790968A1 (en) 2018-05-11 2021-03-17 Alpha Anomeric SAS Oligonucleotides conjugates comprising 7'-5'-alpha-anomeric-bicyclic sugar nucleosides
TW202015726A (en) 2018-05-30 2020-05-01 瑞士商諾華公司 Entpd2 antibodies, combination therapies, and methods of using the antibodies and combination therapies
JP2021526796A (en) 2018-06-13 2021-10-11 サレプタ セラピューティクス, インコーポレイテッド Exon skipping oligomer for muscular dystrophy
US20210261962A1 (en) 2018-06-21 2021-08-26 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
AU2019314538A1 (en) 2018-08-02 2021-03-11 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
KR20210081324A (en) 2018-08-02 2021-07-01 다인 세라퓨틱스, 인크. Muscle targeting complexes and their use for treating facioscapulohumeral muscular dystrophy
CA3108282A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
CA3108328A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating fibrodysplasia ossificans progressiva
WO2020028842A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating hypertrophic cardiomyopathy
EA202190421A1 (en) 2018-08-02 2021-06-25 Дайн Терапьютикс, Инк. MUSCLE-SPECIFIC COMPLEXES AND THEIR APPLICATION FOR TREATMENT OF MUSCLE ATROPHY
EP3829649A4 (en) 2018-08-02 2022-05-04 Dyne Therapeutics, Inc. Muscle-targeting complexes and uses thereof
US20210322562A1 (en) 2018-08-02 2021-10-21 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating centronuclear myopathy
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
EP3830127A4 (en) 2018-08-02 2022-06-29 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating pompe disease
EA202190419A1 (en) 2018-08-02 2021-06-24 Дайн Терапьютикс, Инк. MUSCLE-SPECIFIC COMPLEXES AND THEIR APPLICATION FOR TREATMENT OF FREDREICH'S ATAXIA
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
IT201800009682A1 (en) 2018-10-22 2020-04-22 Ice Spa CONJUGATES OF BILIARY ACIDS AND THEIR DERIVATIVES FOR THE CARRIAGE OF ACTIVE MOLECULES
CN113260384A (en) 2018-11-05 2021-08-13 西纳福克斯股份有限公司 Antibody conjugates for targeting TROP-2 expressing tumors
JP2022513482A (en) 2018-12-21 2022-02-08 アビディティー バイオサイエンシーズ,インク. Anti-transferrin receptor antibody and its use
AU2020263487A1 (en) 2019-04-25 2021-12-16 Avidity Biosciences, Inc. Nucleic acid compositions and methods of multi-exon skipping
EP3980436A4 (en) 2019-06-06 2023-12-20 Avidity Biosciences, Inc. Nucleic acid-polypeptide compositions and uses thereof
JP2022535911A (en) 2019-06-06 2022-08-10 アビディティー バイオサイエンシーズ,インク. UNA amidites and uses thereof
US20220306685A1 (en) 2019-06-07 2022-09-29 Dyne Therapeutics, Inc. Methods of preparing protein-oligonucleotide complexes
WO2021003573A1 (en) 2019-07-09 2021-01-14 The Governors Of The University Of Alberta Exons 45-55 skipping using mutation-tailored cocktails of antisense morpholinos in the dmd gene
WO2021068761A1 (en) 2019-10-10 2021-04-15 苏州亲为药业有限公司 Humanized monoclonal antibody targeting bcma and having human monkey cross-reactivity
WO2021076856A1 (en) 2019-10-18 2021-04-22 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
EP4087876A4 (en) 2020-01-10 2024-03-27 Dyne Therapeutics Inc Muscle-targeting complexes and uses thereof
IL294478A (en) 2020-01-10 2022-09-01 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating dystrophinopathies
WO2023283531A2 (en) 2021-07-09 2023-01-12 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
BR112022013603A2 (en) 2020-01-10 2022-09-13 Dyne Therapeutics Inc MUSCLE TARGETING COMPLEXES AND USES THEM TO TREAT FACIOSCAPULOUMERAL MUSCULAR DYSTROPHY
AU2021206234A1 (en) 2020-01-10 2022-09-01 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
CA3163283A1 (en) 2020-01-10 2021-07-15 Romesh R. Subramanian Muscle targeting complexes and uses thereof for modulation of genes associated with muscle health
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
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
WO2021142217A1 (en) 2020-01-10 2021-07-15 Dyne Thereapeutics, Inc. Muscle targeting complexes and uses thereof for modulation of milck1
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
US20230117883A1 (en) 2020-01-24 2023-04-20 Dyne Therapeutics, Inc. Muscle-targeting complexes and uses thereof in treating muscle atrophy
CA3163290A1 (en) 2020-01-31 2021-08-05 Romesh R. Subramanian Anti-transferrin receptor (tfr) antibody and uses thereof
WO2021154477A1 (en) 2020-01-31 2021-08-05 Dyne Therapeutics, Inc. Anti-transferrin receptor (tfr) antibody and uses thereof
JP2023535443A (en) 2020-07-23 2023-08-17 ダイン セラピューティクス,インコーポレーテッド Anti-transferrin receptor (TfR) antibodies and uses thereof
US20230285586A1 (en) 2020-07-23 2023-09-14 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US20230346966A1 (en) 2020-07-23 2023-11-02 Dyne Therapeutics, Inc. Muscle-targeting complexes and uses thereof in treating muscle atrophy
JP2023535078A (en) 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 Muscle-targeted complexes and their use for treating myotonic dystrophy
CA3186755A1 (en) 2020-07-23 2022-01-27 Romesh R. Subramanian Muscle-targeting complexes and uses thereof
IL299666A (en) 2020-07-23 2023-03-01 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
EP4208173A1 (en) 2020-09-03 2023-07-12 Dyne Therapeutics, Inc. Methods of preparing protein-oligonucleotide complexes
US20240100177A1 (en) 2020-12-04 2024-03-28 Dyne Therapeutics, Inc. Antibody-oligonucleotide complexes and uses thereof
MX2023007881A (en) 2020-12-31 2023-07-10 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating myotonic dystrophy.
IL304049A (en) 2020-12-31 2023-08-01 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
EP4359008A2 (en) 2021-06-21 2024-05-01 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating friedreich's ataxia
WO2022271549A1 (en) 2021-06-21 2022-12-29 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating pompe disease
IL309911A (en) 2021-07-09 2024-03-01 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
IL309909A (en) 2021-07-09 2024-03-01 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating dystrophinopathies
IL309936A (en) 2021-07-09 2024-03-01 Dyne Therapeutics Inc Muscle targeting complexes and formulations for treating dystrophinopathies
US11672872B2 (en) 2021-07-09 2023-06-13 Dyne Therapeutics, Inc. Anti-transferrin receptor antibody and uses thereof
CA3226367A1 (en) 2021-07-09 2023-01-12 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
WO2023283615A1 (en) 2021-07-09 2023-01-12 Dynetherapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
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
IL309937A (en) 2021-07-09 2024-03-01 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating dystrophinopathies
KR20240032953A (en) 2021-07-09 2024-03-12 다인 세라퓨틱스, 인크. Muscle targeting complex and its use to treat myotonic dystrophy
WO2023044398A1 (en) 2021-09-16 2023-03-23 Dyne Therapeutics, Inc. Dosing of muscle targeting complexes for treating dystrophinopathies
AU2022377070A1 (en) 2021-11-01 2024-04-11 Dyne Therapeutics, Inc. Muscle targeting complexes for treating dystrophinopathies
WO2023086864A1 (en) 2021-11-12 2023-05-19 Dyne Therapeutics, Inc. Muscle targeting complexes for treating facioscapulohumeral muscular dystrophy

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US11497815B2 (en) 2022-11-15
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US20230270873A1 (en) 2023-08-31
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