CA3108282A1 - Muscle targeting complexes and uses thereof for treating dystrophinopathies - Google Patents

Muscle targeting complexes and uses thereof for treating dystrophinopathies Download PDF

Info

Publication number
CA3108282A1
CA3108282A1 CA3108282A CA3108282A CA3108282A1 CA 3108282 A1 CA3108282 A1 CA 3108282A1 CA 3108282 A CA3108282 A CA 3108282A CA 3108282 A CA3108282 A CA 3108282A CA 3108282 A1 CA3108282 A1 CA 3108282A1
Authority
CA
Canada
Prior art keywords
complex
antibody
muscle
oligonucleotide
transferrin receptor
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.)
Pending
Application number
CA3108282A
Other languages
English (en)
French (fr)
Inventor
Romesh R. Subramanian
Mohammed T. QATANANI
Timothy Weeden
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Dyne Therapeutics Inc
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 CA3108282A1 publication Critical patent/CA3108282A1/en
Pending legal-status Critical Current

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal 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
    • A61K47/51—Medicinal 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
    • A61K47/68—Medicinal 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
    • A61K47/6801—Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
    • A61K47/6803—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
    • A61K47/6807—Drugs 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal 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
    • A61K47/51—Medicinal 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
    • A61K47/54—Medicinal 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
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal 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
    • A61K47/51—Medicinal 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
    • A61K47/68—Medicinal 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
    • A61K47/6835—Medicinal 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 the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site
    • A61K47/6849—Medicinal 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 the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site the antibody targeting a receptor, a cell surface antigen or a cell surface determinant
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2881—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against CD71
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • C12N15/1137—Non-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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y204/00—Glycosyltransferases (2.4)
    • C12Y204/02—Pentosyltransferases (2.4.2)
    • C12Y204/02008—Hypoxanthine phosphoribosyltransferase (2.4.2.8)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/20—Immunoglobulins specific features characterized by taxonomic origin
    • C07K2317/24—Immunoglobulins specific features characterized by taxonomic origin containing regions, domains or residues from different species, e.g. chimeric, humanized or veneered
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/30—Immunoglobulins specific features characterized by aspects of specificity or valency
    • C07K2317/33—Crossreactivity, e.g. for species or epitope, or lack of said crossreactivity
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/40—Immunoglobulins specific features characterized by post-translational modification
    • C07K2317/41—Glycosylation, sialylation, or fucosylation
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
    • C07K2317/55—Fab or Fab'
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/60—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
    • C07K2317/62—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
    • C07K2317/622—Single chain antibody (scFv)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/70—Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
    • C07K2317/77—Internalization into the cell
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/90—Immunoglobulins specific features characterized by (pharmaco)kinetic aspects or by stability of the immunoglobulin
    • C07K2317/92—Affinity (KD), association rate (Ka), dissociation rate (Kd) or EC50 value
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/50—Fusion polypeptide containing protease site
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Immunology (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Virology (AREA)
  • Cell Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CA3108282A 2018-08-02 2019-08-02 Muscle targeting complexes and uses thereof for treating dystrophinopathies Pending CA3108282A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862714031P 2018-08-02 2018-08-02
US62/714,031 2018-08-02
US201962855766P 2019-05-31 2019-05-31
US62/855,766 2019-05-31
PCT/US2019/044949 WO2020028832A1 (en) 2018-08-02 2019-08-02 Muscle targeting complexes and uses thereof for treating dystrophinopathies

Publications (1)

Publication Number Publication Date
CA3108282A1 true CA3108282A1 (en) 2020-02-06

Family

ID=69232665

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3108282A Pending CA3108282A1 (en) 2018-08-02 2019-08-02 Muscle targeting complexes and uses thereof for treating dystrophinopathies

Country Status (11)

Country Link
US (9) US20210308273A1 (enExample)
EP (1) EP3829595A4 (enExample)
JP (2) JP7611825B2 (enExample)
KR (1) KR20210081322A (enExample)
CN (1) CN112955153A (enExample)
AU (1) AU2019312692B2 (enExample)
CA (1) CA3108282A1 (enExample)
IL (1) IL280527A (enExample)
MX (1) MX2021001281A (enExample)
SG (1) SG11202100934PA (enExample)
WO (1) WO2020028832A1 (enExample)

Families Citing this family (41)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102775461B1 (ko) 2016-04-01 2025-02-28 어비디티 바이오사이언시스 인크. 핵산-폴리펩타이드 조성물 및 이의 용도
EP3830259A4 (en) 2018-08-02 2022-05-04 Dyne Therapeutics, Inc. MUSCLE TARGETED COMPLEXES AND THEIR USES IN THE TREATMENT OF FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
WO2020028832A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
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
IL312067B2 (en) 2018-12-21 2025-08-01 Avidity Biosciences Inc Anti-transferrin receptor antibodies and uses thereof
EP4079329A4 (en) * 2019-12-19 2024-04-17 Nippon Shinyaku Co., Ltd. ANTISENSE NUCLEIC ACID ENABLING EXON SKIPPING
WO2021154477A1 (en) 2020-01-31 2021-08-05 Dyne Therapeutics, Inc. Anti-transferrin receptor (tfr) antibody and uses thereof
TW202144572A (zh) 2020-03-19 2021-12-01 美商亞維代堤生物科學公司 治療臉肩胛肱骨肌肉失養症之組合物及方法
MX2023000984A (es) * 2020-07-23 2023-05-03 Dyne Therapeutics Inc Complejos dirigidos al musculo y usos de los mismos para el tratamiento de distrofinopatias.
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
AU2021313058A1 (en) * 2020-07-23 2023-03-30 Dyne Therapeutics, Inc. Muscle-targeting complexes and uses thereof
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11672872B2 (en) 2021-07-09 2023-06-13 Dyne Therapeutics, Inc. Anti-transferrin receptor antibody and uses thereof
US11633498B2 (en) 2021-07-09 2023-04-25 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
KR20230117182A (ko) 2020-12-04 2023-08-07 다인 세라퓨틱스, 인크. 항체-올리고뉴클레오티드 복합체 및 그의 용도
WO2022232478A1 (en) 2021-04-30 2022-11-03 Sarepta Therapeutics, Inc. Treatment methods for muscular dystrophy
JPWO2022270585A1 (enExample) 2021-06-23 2022-12-29
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
CA3226298A1 (en) * 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
WO2023283623A1 (en) 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating dystrophinopathies
EP4366774A4 (en) * 2021-07-09 2025-11-19 Dyne Therapeutics Inc MUSCLE TARGETTING COMPLEXES AND THEIR USES FOR MODULATING GENES ASSOCIATED WITH MUSCLE HEALTH
US20240318176A1 (en) * 2021-07-09 2024-09-26 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
CN118401553A (zh) * 2021-07-09 2024-07-26 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
CN118591391A (zh) * 2021-07-09 2024-09-03 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
WO2023026994A1 (ja) 2021-08-21 2023-03-02 武田薬品工業株式会社 ヒトトランスフェリンレセプター結合ペプチド－薬物コンジュゲート
US20240366775A1 (en) 2021-08-24 2024-11-07 Peptidream Inc. Human transferrin receptor binding antibody-peptide conjugate
CA3230796A1 (en) 2021-09-01 2023-03-09 Biogen Ma Inc. Anti-transferrin receptor antibodies and uses thereof
IL311507A (en) * 2021-09-16 2024-05-01 Dyne Therapeutics Inc Dosing of muscle targeting complexes for the treatment of dystrophinopathy
EP4401792A4 (en) 2021-09-16 2025-12-03 Avidity Biosciences Inc COMPOSITIONS AND METHODS OF TREATMENT OF FACIO-SCAPULO-HUMERAL MUSCULAR DYSTROPHY
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
IL316143A (en) 2022-04-15 2024-12-01 Dyne Therapeutics Inc Muscle targeting complexes and formulations for the treatment of myotonic dystrophy
AU2024230817A1 (en) * 2023-02-27 2025-09-11 Dyne Therapeutics, Inc. Methods and compositions for inhibiting progression of intramuscular fibrosis
JP2026516311A (ja) 2023-04-24 2026-05-21 バイオマリン ファーマシューティカル インコーポレイテッド Ｓｔｘｂｐ１障害を治療するための組成物および方法
KR20260044219A (ko) 2023-07-21 2026-04-01 매로우 테라퓨틱스, 인크. 조혈 세포 표적화 접합체 및 관련 방법
CN120648680A (zh) * 2024-03-15 2025-09-16 迦进生物医药(上海)有限公司 治疗杜兴型肌营养不良症和贝克型肌营养不良症的药物

Family Cites Families (275)

* 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
WO1989007970A1 (en) 1988-03-02 1989-09-08 Uab Research Foundation Reversible mechanochemical engines comprised of bioelastomers capable of modulable inverse temperature transitions for the interconversion of chemical and 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
AU5752601A (en) 2000-05-04 2001-11-12 Avi Biopharma 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 (ja) 2001-03-02 2002-09-10 Bio Applied Systems:Kk 抗ビスフェノールａ抗体をコードする遺伝子、組換えタンパク質およびその製造方法
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
EP1432724A4 (en) 2002-02-20 2006-02-01 Sirna Therapeutics Inc RNA inhibition mediated inhibition of MAP KINASE GENES
US7575886B2 (en) 2002-03-11 2009-08-18 Momenta Pharmaceuticals, Inc. Analysis of sulfated polysaccharides
EP2530155B1 (en) 2002-11-25 2016-03-16 Masafumi Matsuo ENA nucleic acid drugs modifying splicing in mRNA precursor
AU2003293543A1 (en) 2002-12-13 2004-07-09 Abgenix, Inc. System and method for stabilizing antibodies with histidine
WO2004058717A1 (en) 2002-12-20 2004-07-15 X-Ceptor Therapeutics, Inc. Isoquinolinone derivatives and their use as therapeutic agents
AU2003225410A1 (en) 2003-03-21 2004-10-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
AU2004299053B2 (en) 2003-12-15 2011-01-06 Alexion Pharmaceuticals, Inc. Novel anti-DC-SIGN antibodies
CA2566519C (en) 2004-05-14 2020-04-21 Rosetta Genomics Ltd. Micrornas and uses thereof
ES2627917T3 (es) 2004-06-28 2017-08-01 The University Of Western Australia Oligonucleótidos no codificantes para la inducción de la omisión exónica y métodos de uso de los mismos
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
US7807619B2 (en) 2004-11-01 2010-10-05 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
US7736647B2 (en) 2005-06-15 2010-06-15 Monoclonal Antibodies Therapeutics Anti-CD71 monoclonal antibodies and uses thereof for treating malignant tumor cells
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)
EP1857548A1 (en) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
ZA200904022B (en) 2006-12-08 2010-08-25 Lexicon Pharmaceuticals Inc Monoclonal antibodies against ANGPTL3
JP5718640B2 (ja) 2007-08-21 2015-05-13 アムジエン・インコーポレーテツド ヒトｃ−ｆｍｓ抗原結合性タンパク質
NZ584793A (en) 2007-10-26 2012-05-25 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. 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
US20110130346A1 (en) 2008-05-30 2011-06-02 Isis Innovation Limited Peptide conjugates for delvery of biologically active compounds
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
EP3133160B1 (en) 2008-10-24 2018-12-12 Sarepta Therapeutics, Inc. Exon skipping compositions for dmd
SI2607484T1 (sl) 2008-10-27 2016-11-30 Biomarin Technologies B.V. Metode in sredstva za učinkovito preskakovanje eksona 45 v pre-mRNA Duchennove mišične distrofije
CN102459595A (zh) 2009-04-24 2012-05-16 普罗森那技术公司 用于治疗dmd的包含肌苷的寡核苷酸
EP2427554B1 (en) 2009-05-08 2016-11-16 CuRNA, Inc. Treatment of dystrophin family related diseases by inhibition of natural antisense transcript to dmd family
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
HUE040445T2 (hu) 2009-11-12 2019-03-28 Univ Western Australia Antiszensz molekulák és eljárások betegségek kezelésére
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
PT2528625E (pt) 2010-04-15 2013-10-17 Spirogen Sarl Pirrolobenzodiazepinas e conjugados das mesmas
US8859629B2 (en) 2010-04-27 2014-10-14 Synaffix B.V. Fused cyclooctyne compounds and their use in metal-free click reactions
KR101981705B1 (ko) 2010-05-28 2019-05-24 사렙타 쎄러퓨틱스, 인코퍼레이티드 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체
IT1400425B1 (it) 2010-06-08 2013-05-31 Amsterdam Molecular Therapeutics Bv Modified snrnas for use in therapy.
SG186451A1 (en) 2010-07-12 2013-01-30 Covx Technologies Ireland Ltd Multifunctional antibody conjugates
ES2756326T3 (es) 2010-07-19 2020-04-27 Ionis Pharmaceuticals Inc Modulación de la expresión de distrofia miotónica-proteína quinasa (DMPK)
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
EP3118316A1 (en) 2010-09-02 2017-01-18 Université de Mons Agents useful in treating facioscapulohumeral muscular dystrophy
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
AU2011349443B2 (en) 2010-12-20 2015-12-24 Genentech, Inc. Anti-mesothelin antibodies and immunoconjugates
BR112013020273A2 (pt) 2011-02-08 2016-10-18 Charlotte Mecklenburg Hospital oligonucleotídeos antissenso
CA2828890A1 (en) 2011-04-07 2012-10-11 Genentech, Inc. Anti-fgfr4 antibodies and methods of use
WO2012178173A1 (en) 2011-06-24 2012-12-27 Centrose, Llc Extracellular targeted drug conjugates
JP2014534806A (ja) 2011-08-23 2014-12-25 ロシュ グリクアート アーゲー 抗ｍｃｓｐ抗体
US20130066063A1 (en) 2011-09-09 2013-03-14 John Cooke Hodges Bicyclo[6.1.0]non-4-yne regents for chemical modification of oligonucleotides
EP2788026A4 (en) 2011-12-05 2015-08-05 Univ Duke V1V2 IMMUNOGENIC
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
CN108588073A (zh) 2011-12-28 2018-09-28 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
US20130323234A1 (en) 2012-01-09 2013-12-05 Covx Technologies Ireland Limited Mutant antibodies and conjugation thereof
CA2862628C (en) 2012-01-27 2021-08-24 Prosensa Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
CN104520324A (zh) 2012-02-24 2015-04-15 施特姆森特Rx股份有限公司 Dll3调节剂及其使用方法
US9610362B2 (en) 2012-03-16 2017-04-04 Valerion Therapeutics, Llc Antisense conjugates for decreasing expression of DMPK
PT2841578T (pt) 2012-04-23 2017-08-10 Biomarin Tech Bv Resumo
CN104603271B (zh) 2012-07-03 2019-07-19 马林生物科技有限公司 用于治疗肌肉萎缩症患者的寡核苷酸
EP2684892A1 (en) 2012-07-13 2014-01-15 Association Française contre les Myopathies Compositions and methods for duchenne muscular dystrophy gene therapy
EP4678239A3 (en) 2012-08-01 2026-04-08 Ikaika Therapeutics, Inc. Mitigating tissue damage and fibrosis via latent transforming growth factor beta binding protein (ltbp4)
US20150247141A1 (en) 2012-09-14 2015-09-03 Rana Therapeutics, Inc. Multimeric oligonucleotide compounds
HRP20180268T4 (hr) 2012-10-23 2026-03-27 Synaffix B.V. Modificirano antitijelo, konjugat antitijela i postupak za njihovu pripremu
US10131682B2 (en) 2012-11-24 2018-11-20 Hangzhou Dac Biotech Co., Ltd. Hydrophilic linkers and their uses for conjugation of drugs to a cell binding molecules
ES2731681T3 (es) 2013-02-22 2019-11-18 Abbvie Stemcentrx Llc Conjugados de anticuerpo anti-DLL3 y PBD y usos de los mismos
US9217148B2 (en) 2013-03-14 2015-12-22 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
EP3760720A1 (en) 2013-03-14 2021-01-06 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
HK1220154A1 (zh) 2013-03-15 2017-04-28 Sarepta Therapeutics, Inc. 改进的用於治疗肌营养不良的组合物
SG11201509566RA (en) 2013-05-20 2015-12-30 Genentech Inc Anti-transferrin receptor antibodies and methods of use
EP3004062B1 (en) 2013-05-24 2017-07-19 SynAffix B.V. Substituted azadibenzocyclooctyne compounds and their use in metal-free click reactions
WO2014197748A2 (en) 2013-06-05 2014-12-11 Duke University 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
AU2014321211A1 (en) 2013-09-23 2016-05-05 President And Fellows Of Harvard College Allele-specific RNA silencing for the treatment of hypertrophic cardiomyopathy
WO2015057065A1 (en) 2013-10-14 2015-04-23 Synaffix B.V. Glycoengineered antibody, antibody-conjugate and methods for their preparation
WO2015057064A1 (en) 2013-10-14 2015-04-23 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
CN106660967A (zh) 2014-01-24 2017-05-10 西纳福克斯股份有限公司 (杂)芳基1，3‑偶极化合物与(杂)环炔的环加成方法
JP2017513921A (ja) 2014-03-03 2017-06-01 アーチャルジー、スジャータ ジストロフィン異常症を治療するためのキメラジストロフィン−ｖｓｖ−ｇタンパク質
BR112016020618B1 (pt) 2014-03-12 2023-04-04 Nippon Shinyaku Co., Ltd. Oligômero antissentido, composição farmacêutica, e, uso de um oligômero antissentido
AR099625A1 (es) 2014-03-21 2016-08-03 Lilly Co Eli Anticuerpos de il-21
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
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
AR100560A1 (es) 2014-05-23 2016-10-12 Genzyme Corp Inhibición o regulación por disminución de la glucógeno sintasa mediante la creación de codones de terminación prematuros utilizando oligonucléotidos antisentido
PT3159409T (pt) 2014-06-17 2020-01-21 Nippon Shinyaku Co Ltd Ácido nucleico anti-sentido para utilização no tratamento da distrofia muscular de duchenne
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
GB201416960D0 (en) 2014-09-25 2014-11-12 Antikor Biopharma Ltd Biological materials and uses thereof
US9714288B2 (en) 2014-09-30 2017-07-25 The Regents Of The University Of California Antisense compounds and uses thereof
EP3733209A1 (en) 2014-10-03 2020-11-04 Synaffix B.V. Sulfamide linker, conjugates thereof, and methods of preparation
CA2967830A1 (en) 2014-11-14 2016-05-19 Ossianix, Inc. Tfr selective binding compounds and related methods
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
AU2015349890B2 (en) 2014-11-21 2018-08-30 Merck Sharp & Dohme Llc Insulin receptor partial agonists
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
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
KR102644053B1 (ko) 2015-05-19 2024-03-06 사렙타 쎄러퓨틱스 인코퍼레이티드 펩티드 올리고뉴클레오티드 콘주게이트
US10758607B2 (en) 2015-06-17 2020-09-01 The Research Institute At Nationwide Children's Hospital Respiratory syncytial virus having cleavage-resistant G protein and related materials and methods
CA2990785C (en) 2015-06-24 2024-11-12 Jcr Pharmaceuticals Co., Ltd. ANTI-HUMAN TRANSFERRIN RECEPTOR ANTIBODIES CROSSING THE BLOOD-BRAIN BARRIER
IL257065B (en) 2015-07-22 2022-07-01 Inatherys Antibodies to tfr for use in the treatment of proliferative and inflammatory diseases
HUE050061T2 (hu) 2015-09-15 2020-12-28 Nippon Shinyaku Co Ltd Antiszensz nukleinsav
EP3332008A4 (en) 2015-09-23 2019-01-16 Université Laval CHANGE OF THE DYSTROPHINE GENE AND USES THEREOF
MA46427A (fr) 2015-10-09 2018-08-15 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
JP2018530560A (ja) 2015-10-09 2018-10-18 サレプタ セラピューティクス， インコーポレイテッド デュシェンヌ型筋ジストロフィーおよび関連障害の処置のための組成物および方法
MY207684A (en) 2015-12-08 2025-03-12 Regeneron Pharma Compositions and methods for internalizing enzymes
FR3044926B1 (fr) 2015-12-09 2020-01-31 Genethon Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine
EP4032902A1 (en) 2015-12-18 2022-07-27 Eisai R&D Management Co., Ltd. C-terminal lysine conjugated immunoglobulins
US10874746B2 (en) 2016-02-08 2020-12-29 Synaffix B.V. Sulfamide linkers for use in bioconjugates
WO2017143156A1 (en) 2016-02-19 2017-08-24 Genisphere Llc Nucleic acid carriers and therapeutic methods of use
JP7015244B2 (ja) 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 プロテアーゼ活性化ｔ細胞二重特異性分子
KR102775461B1 (ko) 2016-04-01 2025-02-28 어비디티 바이오사이언시스 인크. 핵산-폴리펩타이드 조성물 및 이의 용도
FR3049951A1 (fr) 2016-04-12 2017-10-13 Univ Rabelais Francois Nouvelles constructions peptidiques et leur utilisation dans le traitement de la toxoplasmose
JP2019516679A (ja) 2016-05-04 2019-06-20 ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． 生物活性剤に関する方法および組成物
TWI737736B (zh) 2016-05-24 2021-09-01 美商薩羅塔治療公司 製備磷醯二胺嗎啉代寡聚物之方法
WO2017205191A1 (en) 2016-05-24 2017-11-30 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
DK3473270T5 (da) 2016-06-20 2024-09-09 Genahead Bio Inc Konjugat af antistof-lægemiddel
AU2017291960B2 (en) 2016-07-05 2024-03-14 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
JP7490211B2 (ja) 2016-07-19 2024-05-27 デューク ユニバーシティ Ｃｐｆ１に基づくゲノム編集の治療適用
BR112019009953A2 (pt) 2016-11-16 2019-10-08 Academisch Ziekenhuis Leiden substâncias direcionadas a vários órgãos ou tecidos selecionados
US10947540B2 (en) 2016-11-29 2021-03-16 Association Institut De Myologie Allele-specific silencing therapy for Dynamin 2-related diseases
AU2017364106A1 (en) 2016-11-28 2019-06-20 The Board Of Regents Of The University Of Texas System Prevention of muscular dystrophy by CRISPR/Cpfl-mediated gene editing
CA3046220A1 (en) 2016-12-08 2018-06-14 The Board Of Regents Of The University Of Texas System Dmd reporter models containing humanized duschene muscular dystrophy mutations
PT4122497T (pt) 2016-12-19 2024-06-12 Sarepta Therapeutics Inc Conjugados oligoméricos de salto de exão para a distrofia muscular
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
JOP20190166A1 (ar) 2017-01-05 2019-07-02 Univ Texas استراتيجية مثلى من أجل تعديلات تخطي إكسون باستخدام crispr/cas9 مع متواليات توجيه ثلاثي
KR20240172236A (ko) 2017-01-06 2024-12-09 어비디티 바이오사이언시스 인크. 핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법
CA3049791A1 (en) 2017-01-27 2018-08-02 Silverback Therapeutics, Inc. Tumor targeting conjugates and methods of use thereof
AU2018281280B2 (en) 2017-06-07 2024-12-19 Regeneron Pharmaceuticals, Inc. Compositions and methods for internalizing enzymes
US20200155702A1 (en) 2017-06-16 2020-05-21 Eli Lilly And Company Engineered Antibody Compounds and Conjuates Thereof
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
EA201991450A1 (ru) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
SG11202002517RA (en) 2017-09-22 2020-04-29 Avidity Biosciences Inc Nucleic acid-polypeptide compositions and methods of inducing exon skipping
JP2020536058A (ja) 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 筋ジストロフィーを処置するための併用療法
JP2020536060A (ja) 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 筋ジストロフィーを処置するための併用療法
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
MA50579A (fr) 2017-11-09 2020-09-16 Crispr Therapeutics Ag Systèmes crispr/cas ou crispr/cpf1 à auto-inactivation (sin) et leurs utilisations
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
CN108220418B (zh) 2017-12-29 2018-11-09 东莞博奥木华基因科技有限公司 基于多重pcr捕获技术的杜氏/贝氏肌营养不良症的检测试剂盒及方法
EP3735252A4 (en) 2018-01-04 2021-10-06 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
EP3746557A1 (en) 2018-01-31 2020-12-09 The Board Of Regents Of The University Of Texas System Compositions and methods for correcting dystrophin mutations in human cardiomyocytes
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
CN112040985A (zh) 2018-02-07 2020-12-04 瑞泽恩制药公司 用于递送治疗性蛋白质的方法和组合物
JP2021521140A (ja) 2018-04-12 2021-08-26 ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． オリゴヌクレオチド組成物及びその使用方法
WO2019209764A2 (en) 2018-04-26 2019-10-31 Sarepta Therapeutics, Inc. Exon skipping oligomers and oligomer conjugates for muscular dystrophy
US20210371860A1 (en) 2018-05-08 2021-12-02 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating myh7 expression
CA3098624A1 (en) 2018-05-11 2019-11-14 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
AU2019266550A1 (en) 2018-05-11 2020-11-26 Alpha Anomeric Sas Oligonucleotides conjugates comprising 7'-5'-alpha-anomeric-bicyclic sugar nucleosides
UY38247A (es) 2018-05-30 2019-12-31 Novartis Ag Anticuerpos frente a entpd2, terapias de combinación y métodos de uso de los anticuerpos y las terapias de combinación
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
EP3830127A4 (en) 2018-08-02 2022-06-29 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating pompe disease
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
CN112955154A (zh) 2018-08-02 2021-06-11 达因疗法公司 肌肉靶向复合物及其用于治疗强直性肌营养不良的用途
US20210322562A1 (en) 2018-08-02 2021-10-21 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating centronuclear myopathy
EP3829594A4 (en) 2018-08-02 2022-05-11 Dyne Therapeutics, Inc. MUSCLE TARGETING COMPLEXES AND THEIR USES FOR THE TREATMENT OF HYPERTROPHIC CARDIOMYOPATHY
CA3108386A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating friedreich's ataxia
CA3108328A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating fibrodysplasia ossificans progressiva
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
US12097263B2 (en) 2018-08-02 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
WO2020028832A1 (en) 2018-08-02 2020-02-06 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
EP3830259A4 (en) 2018-08-02 2022-05-04 Dyne Therapeutics, Inc. MUSCLE TARGETED COMPLEXES AND THEIR USES IN THE TREATMENT OF FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
AU2019315593B2 (en) 2018-08-02 2026-05-21 Dyne Therapeutics, Inc. Muscle-targeting complexes and uses thereof
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
CN109306375A (zh) 2018-10-16 2019-02-05 上海瀚垚生物医学科技有限公司 EvaGreen法数字PCR检测DMD拷贝数变异的引物、检测试剂盒及其检测方法
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
AU2019392928A1 (en) 2018-12-06 2021-06-17 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
IL312067B2 (en) 2018-12-21 2025-08-01 Avidity Biosciences Inc Anti-transferrin receptor antibodies and uses thereof
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
EP3946376A1 (en) 2019-03-28 2022-02-09 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy with 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
WO2020214763A1 (en) 2019-04-18 2020-10-22 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
WO2020219820A1 (en) 2019-04-25 2020-10-29 Avidity Biosciences, Inc. Nucleic acid compositions and methods of multi-exon skipping
JP7581252B2 (ja) 2019-06-06 2024-11-12 アビディティー バイオサイエンシーズ，インク． Ｕｎａアミダイトおよびその使用
WO2020247782A1 (en) 2019-06-06 2020-12-10 Avidity Biosciences, Inc. Nucleic acid-polypeptide compositions and uses thereof
CN114025805B (zh) 2019-06-07 2025-01-24 达因疗法公司 制备蛋白质-寡核苷酸复合物的方法
EP3987029A1 (en) 2019-06-19 2022-04-27 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy
EP3997228A4 (en) 2019-07-09 2024-05-15 The Governors of the University of Alberta SKIP OF EXONS 45 TO 55 USING MUTATION-ADAPTED COCKTAILS, OF ANTISENS MORPHOLINOS, IN THE DMD GENE
CN120775052A (zh) 2019-10-10 2025-10-14 再凌生物医药(香港)有限公司 靶向bcma的具有人猴交叉的人源化单克隆抗体
US20240148891A1 (en) 2019-10-18 2024-05-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
JP2023503644A (ja) 2019-11-27 2023-01-31 ディー・ティー・エックス・ファーマ・インコーポレイテッド デュシェンヌ型筋ジストロフィーの治療のための化合物および方法
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
WO2021142260A1 (en) 2020-01-10 2021-07-15 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for modulation of acvr1
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
EP4087653A4 (en) 2020-01-10 2024-06-19 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
CN115427448A (zh) 2020-01-10 2022-12-02 达因疗法公司 肌肉靶向复合物及其用途
CN122124274A (zh) 2020-01-10 2026-06-02 达因疗法公司 肌肉靶向复合物及其用于治疗面肩肱型肌营养不良的用途
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
US20230111212A1 (en) 2020-01-10 2023-04-13 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
JP7858532B2 (ja) 2020-01-10 2026-05-14 ダイン セラピューティクス，インコーポレーテッド 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
WO2021142217A1 (en) 2020-01-10 2021-07-15 Dyne Thereapeutics, Inc. Muscle targeting complexes and uses thereof for modulation of milck1
US20230117883A1 (en) 2020-01-24 2023-04-20 Dyne Therapeutics, Inc. Muscle-targeting complexes and uses thereof in treating muscle atrophy
IL295022A (en) 2020-01-31 2022-09-01 Dyne Therapeutics Inc Antibody against transferrin receptor and uses thereof
WO2021154477A1 (en) 2020-01-31 2021-08-05 Dyne Therapeutics, Inc. Anti-transferrin receptor (tfr) antibody and uses thereof
KR102927945B1 (ko) 2020-03-27 2026-02-23 어비디티 바이오사이언시스 인크. 근이영양증의 치료용 조성물 및 방법
US20230330246A1 (en) 2020-05-05 2023-10-19 Avidity Biosciences, Inc. Compositions and methods of treating pompe disease
CN116348139A (zh) 2020-07-23 2023-06-27 达因疗法公司 肌肉靶向复合物及其用于治疗面肩肱型肌营养不良的用途
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses 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
EP4185320A4 (en) 2020-07-23 2025-10-08 Dyne Therapeutics Inc MUSCLE TARGETING COMPLEXES AND THEIR USES IN THE TREATMENT OF MUSCLE ATROPHY
US11672872B2 (en) 2021-07-09 2023-06-13 Dyne Therapeutics, Inc. Anti-transferrin receptor antibody and uses thereof
AU2021313058A1 (en) 2020-07-23 2023-03-30 Dyne Therapeutics, Inc. Muscle-targeting complexes and uses thereof
MX2023000958A (es) 2020-07-23 2023-04-14 Dyne Therapeutics Inc Complejos dirigidos al musculo y usos de estos para el tratamiento de la distrofia miotonica.
US20230256112A1 (en) 2020-07-23 2023-08-17 Dyne Therapeutics, lnc. Anti-transferrin receptor (tfr) 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
MX2023000984A (es) 2020-07-23 2023-05-03 Dyne Therapeutics Inc Complejos dirigidos al musculo y usos de los mismos para el tratamiento de distrofinopatias.
KR20230079068A (ko) 2020-09-03 2023-06-05 다인 세라퓨틱스, 인크. 단백질-올리고뉴클레오티드 복합체의 제조 방법
AU2021342155A1 (en) 2020-09-11 2023-04-13 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
EP4271478A4 (en) 2020-12-31 2025-03-26 Dyne Therapeutics, Inc. MUSCLE TARGETING COMPLEXES AND THEIR USES FOR TREATING FACIOSCAPULOHUMERAL MYOPATHY
AU2021414227A1 (en) 2020-12-31 2023-07-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
KR20240009393A (ko) 2021-03-31 2024-01-22 엔트라다 테라퓨틱스, 인크. 사이클릭 세포 침투 펩티드
EP4314298A4 (en) 2021-04-02 2026-06-03 Avidity Biosciences Inc ANTIBODY-OLIGONUCLEOTIDE CONJUGATES AND ANTIBODY-PEPTIDE-OLIGONUCLEOTIDE CONJUGATE COMPOSITIONS AND EXON-SKIPPING INDUCTION METHODS
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
WO2022232478A1 (en) 2021-04-30 2022-11-03 Sarepta Therapeutics, Inc. Treatment methods for muscular dystrophy
WO2022240758A1 (en) 2021-05-10 2022-11-17 Entrada Therapeutics, Inc. Compositions and methods for modulating gene expression
EP4359534A4 (en) 2021-06-21 2026-07-22 Dyne Therapeutics Inc MUSCLE TARGETTING COMPLEXES AND THEIR USES IN THE TREATMENT OF POMPE DISEASE
CA3222816A1 (en) 2021-06-21 2022-12-29 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating friedreich's ataxia
JPWO2022270585A1 (enExample) 2021-06-23 2022-12-29
US20240318176A1 (en) 2021-07-09 2024-09-26 Dyne Therapeutics, 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
CN118591391A (zh) 2021-07-09 2024-09-03 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
EP4366774A4 (en) 2021-07-09 2025-11-19 Dyne Therapeutics Inc MUSCLE TARGETTING COMPLEXES AND THEIR USES FOR MODULATING GENES ASSOCIATED WITH MUSCLE HEALTH
CA3226301A1 (en) 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
CN118401553A (zh) 2021-07-09 2024-07-26 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
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
CA3226298A1 (en) 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
WO2023283623A1 (en) 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating dystrophinopathies
EP4389891A4 (en) 2021-08-19 2025-10-29 Univ Nat Corp Tokyo Medical & Dental MODIFIED HETERONUCLEIC ACID CONTAINING MORPHOLINO NUCLEIC ACID
WO2023026994A1 (ja) 2021-08-21 2023-03-02 武田薬品工業株式会社 ヒトトランスフェリンレセプター結合ペプチド－薬物コンジュゲート
EP4395831A1 (en) 2021-09-01 2024-07-10 Entrada Therapeutics, Inc. Compositions and methods for skipping exon 45 in duchenne muscular dystrophy
IL311507A (en) 2021-09-16 2024-05-01 Dyne Therapeutics Inc Dosing of muscle targeting complexes for the treatment of dystrophinopathy
EP4426356A1 (en) 2021-11-01 2024-09-11 Dyne Therapeutics, Inc. Muscle targeting complexes for treating dystrophinopathies
JP2024546436A (ja) 2021-11-12 2024-12-24 ダイン セラピューティクス，インコーポレーテッド 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
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
EP4469576A4 (en) 2022-01-27 2026-04-15 Univ Alberta Antisense therapeutic products conjugate to pre-MIARN splicing modulating peptides 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
WO2023201324A1 (en) 2022-04-15 2023-10-19 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
IL316143A (en) 2022-04-15 2024-12-01 Dyne Therapeutics Inc Muscle targeting complexes and formulations for the treatment of myotonic dystrophy
US20250249117A1 (en) 2022-04-15 2025-08-07 Dyne Therapeutics, Inc. Dosing of muscle targeting complexes for treating myotonic dystrophy
WO2024011135A2 (en) 2022-07-06 2024-01-11 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
AU2023303022A1 (en) 2022-07-06 2025-01-02 Dyne Therapeutics, Inc. Cns targeting complexes and uses thereof
EP4590311A2 (en) 2022-09-21 2025-07-30 Sarepta Therapeutics, Inc. Dmd antisense oligonucleotide-mediated exon skipping efficiency
JP2026503873A (ja) 2022-10-31 2026-02-02 ダイン セラピューティクス，インコーポレーテッド Ｄｍｄ遺伝子のエクソン４５をスキップするための筋肉標的化複合体およびその使用
IL321025A (en) 2022-11-21 2025-07-01 Pepgen Inc Cell-penetrating peptides, conjugates thereof, and methods of their use
WO2024149282A1 (en) 2023-01-10 2024-07-18 Ausperbio Therapeutics Inc. Modified multi-segmented antisense oligonucleotides for use
AU2024230817A1 (en) 2023-02-27 2025-09-11 Dyne Therapeutics, Inc. Methods and compositions for inhibiting progression of intramuscular fibrosis
EP4788835A1 (en) 2023-10-06 2026-08-12 Dyne Therapeutics, Inc. Biomarkers for repeat expansion diseases
WO2025147541A1 (en) 2024-01-02 2025-07-10 Dyne Therapeutics, Inc. Methods and compositions for treating myotonic dystrophy

Also Published As

Publication number Publication date
US20250170261A1 (en) 2025-05-29
US12005124B2 (en) 2024-06-11
US11497815B2 (en) 2022-11-15
US11369689B2 (en) 2022-06-28
US20220143206A1 (en) 2022-05-12
US11833217B2 (en) 2023-12-05
CN112955153A (zh) 2021-06-11
MX2021001281A (es) 2021-07-15
JP7611825B2 (ja) 2025-01-10
US20240293568A1 (en) 2024-09-05
JP2021532831A (ja) 2021-12-02
US12357703B2 (en) 2025-07-15
EP3829595A1 (en) 2021-06-09
US20240066139A1 (en) 2024-02-29
US20220288220A1 (en) 2022-09-15
US20250297024A1 (en) 2025-09-25
SG11202100934PA (en) 2021-02-25
US11633496B2 (en) 2023-04-25
AU2019312692A1 (en) 2021-03-11
US20230270873A1 (en) 2023-08-31
JP2024160045A (ja) 2024-11-11
KR20210081322A (ko) 2021-07-01
AU2019312692B2 (en) 2026-06-11
IL280527A (en) 2021-03-01
WO2020028832A1 (en) 2020-02-06
US12263225B2 (en) 2025-04-01
US20210308273A1 (en) 2021-10-07
US20230001002A1 (en) 2023-01-05
US12478687B2 (en) 2025-11-25
EP3829595A4 (en) 2022-08-24

Similar Documents

Publication Publication Date Title
US12478687B2 (en) United states
US12329825B1 (en) Muscle targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of use thereof to induce exon skipping of exon 44 of dystrophin in a subject
AU2019316103B2 (en) Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US20210322562A1 (en) Muscle targeting complexes and uses thereof for treating centronuclear myopathy
US20210308274A1 (en) Muscle targeting complexes and uses thereof for treating friedreich's ataxia
CA3108285A1 (en) Muscle targeting complexes and uses thereof for treating pompe disease
CA3108328A1 (en) Muscle targeting complexes and uses thereof for treating fibrodysplasia ossificans progressiva
CA3108313A1 (en) Muscle-targeting complexes and uses thereof in treating muscle atrophy
CA3108287A1 (en) Muscle targeting complexes and uses thereof for treating myotonic dystrophy
EP3829594A1 (en) Muscle targeting complexes and uses thereof for treating hypertrophic cardiomyopathy

Legal Events

Date Code Title Description
MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 5TH ANNIV.) - STANDARD

Year of fee payment: 5

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20240726

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT DETERMINED COMPLIANT

Effective date: 20240726

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20240726

D11 Substantive examination requested

Free format text: ST27 STATUS EVENT CODE: A-1-1-D10-D11-D117 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REQUEST FOR EXAMINATION RECEIVED

Effective date: 20240730

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P11-P101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT RECEIVED - VOLUNTARY AMENDMENT

Effective date: 20240730

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20241109

D00 Search and/or examination requested or commenced

Free format text: ST27 STATUS EVENT CODE: A-1-1-D10-D00-D118 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REQUEST FOR EXAMINATION REQUIREMENTS DETERMINED COMPLIANT

Effective date: 20241118

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P11-P102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT DETERMINED COMPLIANT

Effective date: 20241118

P13 Application amended

Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P13-X000 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: APPLICATION AMENDED

Effective date: 20241118

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20241118

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20250410

D11 Substantive examination requested

Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D11-D155 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: ALL REQUIREMENTS FOR EXAMINATION DETERMINED COMPLIANT

Effective date: 20250430

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 6TH ANNIV.) - STANDARD

Year of fee payment: 6

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20250725

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20250725

D15 Examination report completed

Free format text: ST27 STATUS EVENT CODE: A-2-2-D10-D15-D126 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: EXAMINER'S REPORT

Effective date: 20260303

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT RECEIVED - RESPONSE TO EXAMINER'S REQUISITION

Effective date: 20260703

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20260706