SG11202100934PA - Muscle targeting complexes and uses thereof for treating dystrophinopathies - Google Patents
Muscle targeting complexes and uses thereof for treating dystrophinopathiesInfo
- 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
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 [IGs], e.g. monoclonal or polyclonal antibodies
- C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
- C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
- C07K16/2881—Immunoglobulins [IGs], 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 N.A.
-
- 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)
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 |
Publications (1)
Publication Number | Publication Date |
---|---|
SG11202100934PA true SG11202100934PA (en) | 2021-02-25 |
Family
ID=69232665
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SG11202100934PA SG11202100934PA (en) | 2018-08-02 | 2019-08-02 | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
Country Status (11)
Country | Link |
---|---|
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 (24)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US11911484B2 (en) | 2018-08-02 | 2024-02-27 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
KR20210081322A (en) | 2018-08-02 | 2021-07-01 | 다인 세라퓨틱스, 인크. | Muscle targeting complexes and their use for treating dystrophinopathy |
CA3108289A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
SG11202106593UA (en) | 2018-12-21 | 2021-07-29 | Avidity Biosciences Inc | Anti-transferrin receptor antibodies and uses thereof |
IL293997A (en) * | 2019-12-19 | 2022-08-01 | Nippon Shinyaku Co Ltd | Antisense nucleic acid enabling exon skipping |
JP2023537798A (en) * | 2020-03-19 | 2023-09-06 | アビディティー バイオサイエンシーズ,インク. | Compositions and methods for treating facioscapulohumeral muscular dystrophy |
BR112023001125A2 (en) * | 2020-07-23 | 2023-02-14 | Dyne Therapeutics Inc | MUSCLE TARGETING COMPLEXES AND THEIR USES TO TREAT DYSTROPHINOPATHIES |
CN116194470A (en) * | 2020-07-23 | 2023-05-30 | 达因疗法公司 | Muscle targeting complexes and uses thereof |
EP4330395A1 (en) | 2021-04-30 | 2024-03-06 | Sarepta Therapeutics, Inc. | Treatment methods for muscular dystrophy |
IL309626A (en) | 2021-06-23 | 2024-02-01 | Nippon Shinyaku Co Ltd | Combination of antisense oligomers |
US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
IL309909A (en) * | 2021-07-09 | 2024-03-01 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
WO2023283619A2 (en) * | 2021-07-09 | 2023-01-12 | 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 |
US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
US11648318B2 (en) | 2021-07-09 | 2023-05-16 | Dyne Therapeutics, Inc. | Anti-transferrin receptor (TFR) antibody and uses thereof |
US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
JPWO2023026994A1 (en) | 2021-08-21 | 2023-03-02 | ||
WO2023027125A1 (en) | 2021-08-24 | 2023-03-02 | ペプチドリーム株式会社 | 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 |
AU2022345098A1 (en) | 2021-09-16 | 2024-04-04 | 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 (219)
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 |
CA2400659A1 (en) | 2000-02-22 | 2001-08-30 | Banyu Pharmaceutical Co., Ltd. | Novel imidazoline compounds |
KR20020097241A (en) | 2000-05-04 | 2002-12-31 | 에이브이아이 바이오파마 인코포레이티드 | 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 |
EP2402753A1 (en) | 2002-03-11 | 2012-01-04 | Momenta Pharmaceuticals, Inc. | Analysis of sulfated polysaccharides |
JP4777777B2 (en) | 2002-11-25 | 2011-09-21 | 雅文 松尾 | ENA nucleic acid medicine which modifies splicing of mRNA precursor |
US20040191243A1 (en) | 2002-12-13 | 2004-09-30 | Bei Chen | System and method for stabilizing antibodies with histidine |
DE60335911D1 (en) | 2002-12-20 | 2011-03-10 | Daiichi Sankyo Co Ltd | ISOCHINOLINE DERIVATIVES AND THEIR USE AS MEDICAMENTS |
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 |
EP3228711A1 (en) | 2004-06-28 | 2017-10-11 | 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 |
DK1812031T3 (en) | 2004-11-01 | 2015-09-14 | Univ California | Compositions and methods for the 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 |
US20060252107A1 (en) | 2005-02-22 | 2006-11-09 | Acucela, Inc. | Compositions and methods for diagnosing and treating retinal diseases |
US8716033B2 (en) | 2006-02-10 | 2014-05-06 | Life Technologies Corporation | Oligosaccharide modification and labeling of 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 |
MX2009006082A (en) | 2006-12-08 | 2009-08-18 | Lexicon Pharmaceuticals Inc | Monoclonal antibodies against angptl3. |
RS56743B1 (en) | 2007-08-21 | 2018-03-30 | Amgen Inc | Human c-fms antigen binding proteins |
PT2203173E (en) | 2007-10-26 | 2016-03-15 | 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 |
CN105779453A (en) | 2008-10-24 | 2016-07-20 | 萨雷普塔治疗公司 | Multiple exon skipping compositions for DMD |
SI2607484T1 (en) | 2008-10-27 | 2016-11-30 | Biomarin Technologies B.V. | Methods and means for efficient skipping of exon 45 in Duchenne Muscular Dystrophy pre-mRNA |
CN102459595A (en) | 2009-04-24 | 2012-05-16 | 普罗森那技术公司 | Oligonucleotide comprising an inosine for treating dmd |
WO2010129861A2 (en) | 2009-05-08 | 2010-11-11 | Curna, Inc. | Treatment of dystrophin family related diseases by inhibition of natural antisense transcript to dmd family |
FR2947269B1 (en) | 2009-06-29 | 2013-01-18 | Sanofi Aventis | NEW ANTICANCER COMPOUNDS |
US8802437B2 (en) | 2009-09-24 | 2014-08-12 | Cellectis | Meganuclease reagents of uses thereof for treating genetic diseases caused by frame shift/non sense mutations |
JP5963678B2 (en) | 2009-11-12 | 2016-08-03 | ジ ユニバーシティ オブ ウェスタン オーストラリア | Antisense molecules and methods for treating disease states |
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 |
WO2011130598A1 (en) | 2010-04-15 | 2011-10-20 | Spirogen Limited | Pyrrolobenzodiazepines and conjugates thereof |
ES2527536T7 (en) | 2010-04-27 | 2016-03-08 | Synaffix B.V. | Fused cyclooctin compounds and their use in metal-free click reactions |
CA2799501C (en) | 2010-05-28 | 2022-02-15 | Sarepta Therapeutics, Inc. | 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. |
SG186451A1 (en) | 2010-07-12 | 2013-01-30 | Covx Technologies Ireland Ltd | Multifunctional antibody conjugates |
TWI541024B (en) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | Antisense nucleic acid |
US20120225034A1 (en) | 2010-09-02 | 2012-09-06 | Universite De Mons | Agents useful in treating facioscapulohumeral muscular dystrophy |
EP2646470B1 (en) | 2010-11-30 | 2017-03-01 | F. Hoffmann-La Roche AG | Low affinity anti-transferrin receptor antibodies and their use to transfer therapeutic scfv across the blood brain barrier |
UA115641C2 (en) | 2010-12-20 | 2017-11-27 | Дженентек, Інк. | DETERMINED ANTIBODY Binds Mesothelin and Immunoconjugate Containing It |
JP2014507143A (en) | 2011-02-08 | 2014-03-27 | ザ シャーロット−メクレンバーグ ホスピタル オーソリティ ドゥーイング/ビジネス/アズ キャロライナズ ヘルスケア システム | Antisense oligonucleotide |
BR112013024717A2 (en) | 2011-04-07 | 2017-08-08 | Genentech Inc | isolated antibody, isolated nucleic acid, host cell, immunoconjugate, pharmaceutical formulation, method of treating an individual who has cancer and method of inhibiting cell proliferation in an individual |
CN103732259A (en) | 2011-06-24 | 2014-04-16 | 森彻斯有限公司 | Extracellular targeted drug conjugates |
US9309306B2 (en) | 2011-08-23 | 2016-04-12 | Roche Glycart Ag | Anti-MCSP antibodies |
EP2753607B1 (en) | 2011-09-09 | 2016-04-13 | Berry And Associates, Inc. | Bicyclo[6.1.0]non-4-yne reagents for chemical modification of oligonucleotides |
WO2013085550A2 (en) | 2011-12-05 | 2013-06-13 | Duke University | V1v2 immunogens |
US9725716B2 (en) | 2011-12-06 | 2017-08-08 | Ohio State Innovation Foundation and Research Institute at Nationwide Children's Hospital | Non-ionic, low osmolar contrast agents for delivery of antisense oligonucleotides and treatment of disease |
WO2013100190A1 (en) | 2011-12-28 | 2013-07-04 | 日本新薬株式会社 | Antisense nucleic acid |
WO2013103800A1 (en) | 2012-01-06 | 2013-07-11 | Bioalliance C.V. | Anti-transferrin receptor antibodies and methods using same |
EP2802355B1 (en) | 2012-01-09 | 2018-09-05 | CovX Technologies Ireland Limited | Mutant antibodies and conjugation thereof |
PE20190658A1 (en) | 2012-02-24 | 2019-05-08 | Abbvie Stemcentrx Llc | NEW MODULATORS AND EMPLOYMENT METHODS |
CA2907072A1 (en) | 2012-03-16 | 2013-09-19 | Valerion Therapeutics, Llc | Antisense conjugates for decreasing expression of dmpk |
BR112014026285B1 (en) | 2012-04-23 | 2021-08-31 | Biomarin Technologies B.V | RNA MODULATOR OLIGONUCLEOTIDES WITH IMPROVED CHARACTERISTICS FOR THE TREATMENT OF NEUROMUSCULAR DISORDERS |
BR112014033004B1 (en) | 2012-07-03 | 2021-10-19 | Biomarin Technologies B.V. | OLIGONUCLEOTIDE FOR THE TREATMENT OF PATIENTS WITH MUSCLE DYSTROPHY |
EP2684892A1 (en) | 2012-07-13 | 2014-01-15 | Association Française contre les Myopathies | Compositions and methods for duchenne muscular dystrophy gene therapy |
US20150247141A1 (en) | 2012-09-14 | 2015-09-03 | Rana Therapeutics, Inc. | Multimeric oligonucleotide compounds |
HUE038285T2 (en) | 2012-10-23 | 2018-10-29 | Synaffix Bv | Modified antibody, antibody-conjugate and process for the preparation thereof |
EP2922818B1 (en) | 2012-11-24 | 2018-09-05 | Hangzhou Dac Biotech Co., Ltd | Hydrophilic linkers and their uses for conjugation of drugs to cell binding molecules |
JP6462591B2 (en) | 2013-02-22 | 2019-01-30 | アッヴィ・ステムセントルクス・エル・エル・シー | Novel antibody conjugates and uses thereof |
US20160040162A1 (en) | 2013-03-14 | 2016-02-11 | Sarepta Therapeutics, Inc. | Exon skipping compositions for treating muscular dystrophy |
MX2015013117A (en) | 2013-03-15 | 2016-07-14 | Sarepta Therapeutics Inc | Improved compositions for treating muscular dystrophy. |
CA2908743A1 (en) | 2013-05-20 | 2014-11-27 | 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 |
EP4389903A2 (en) | 2013-06-05 | 2024-06-26 | Duke University | Rna-guided gene editing and gene regulation |
CN105873612A (en) | 2013-08-28 | 2016-08-17 | 施特姆森特克斯股份有限公司 | Engineered anti-DLL3 conjugates and methods of use |
WO2015042581A1 (en) | 2013-09-23 | 2015-03-26 | President And Fellows Of Harvard College | Allele-specific rna silencing for the treatment of hypertrophic cardiomyopathy |
WO2015057064A1 (en) | 2013-10-14 | 2015-04-23 | Synaffix B.V. | Modified glycoprotein, protein-conjugate and process for the preparation thereof |
US20160235861A1 (en) | 2013-10-14 | 2016-08-18 | SynAffix. B.V. | Glycoengineered antibody, antibody-conjugate and methods for their preparation |
US10344284B2 (en) | 2013-11-14 | 2019-07-09 | Lina A. Shehadeh | Methods and compositions employing an osteopontin aptamer to deliver nucleic acids into smooth muscle, endothelial, cardiac and progenitor/stem cells |
WO2015112016A1 (en) | 2014-01-24 | 2015-07-30 | Synaffix B.V. | Process for the cycloaddition of a halogenated 1,3-dipole compound with a (hetero)cycloalkyne |
EP3925951A1 (en) | 2014-01-24 | 2021-12-22 | SynAffix B.V. | Process for the cycloaddition of a(hetero)aryl 1,3-dipole compound with a (hetero)cycloalkyne |
TR201901939T4 (en) | 2014-03-12 | 2019-03-21 | Nat Center Neurology & Psychiatry | Antisense nucleic acid. |
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 |
EP3146050B1 (en) | 2014-05-23 | 2020-08-12 | Genzyme Corporation | Inhibiting or downregulating glycogen synthase by creating premature stop codons using antisense oligonucleotides |
SG10201912858VA (en) | 2014-06-17 | 2020-02-27 | Nippon Shinyaku Co Ltd | Antisense nucleic acids |
JP2017528124A (en) | 2014-08-04 | 2017-09-28 | シンアフィックス ビー.ブイ. | Method for modifying glycoprotein using 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 |
EP3218411B1 (en) | 2014-11-14 | 2022-01-12 | Ossianix, Inc. | Variable new antigen receptors (vnars) directed against transferrin receptor (tfr) and their use |
EP3221362B1 (en) | 2014-11-19 | 2019-07-24 | F.Hoffmann-La Roche Ag | Anti-transferrin receptor antibodies and methods of use |
EP3666792A3 (en) | 2014-11-21 | 2020-07-29 | Merck Sharp & Dohme Corp. | 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 |
LT3292149T (en) | 2015-05-04 | 2022-03-10 | Cytomx Therapeutics, Inc. | Activatable anti-cd71 antibodies, and methods of use thereof |
KR102644053B1 (en) | 2015-05-19 | 2024-03-06 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | Peptide Oligonucleotide Conjugate |
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 |
EP3325509B1 (en) | 2015-07-22 | 2020-12-16 | Inatherys | Anti-tfr antibodies and their use in treating proliferative and inflammatory disorders |
EP3778895A1 (en) | 2015-09-15 | 2021-02-17 | Nippon Shinyaku Co., Ltd. | Antisense nucleic acid |
EP3332008A4 (en) | 2015-09-23 | 2019-01-16 | Université Laval | Modification of the dystrophin gene and uses thereof |
MA45819A (en) | 2015-10-09 | 2018-08-15 | Sarepta Therapeutics Inc | COMPOSITIONS AND METHODS FOR TREATING DUCHENNE MUSCLE DYSTROPHY AND RELATED DISORDERS |
KR20180056766A (en) | 2015-10-09 | 2018-05-29 | 웨이브 라이프 사이언시스 리미티드 | Nucleotide compositions and methods thereof |
EP3386534B1 (en) | 2015-12-08 | 2020-09-23 | 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 |
BR112018012388A2 (en) | 2015-12-18 | 2018-12-04 | 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 |
JP7366541B2 (en) | 2016-02-19 | 2023-10-23 | コード バイオセラピューティクス インコーポレイテッド | Nucleic acid carriers and therapeutic uses |
LT3433280T (en) | 2016-03-22 | 2023-07-10 | F. Hoffmann-La Roche Ag | Protease-activated t cell bispecific molecules |
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 |
AU2017271524B2 (en) | 2016-05-24 | 2021-06-24 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
EP3922260A3 (en) | 2016-05-24 | 2022-06-15 | Merck Sharp & Dohme Corp. | Insulin receptor partial agonists and glp-1 analogues |
WO2017210647A1 (en) | 2016-06-03 | 2017-12-07 | Wave Life Sciences Ltd. | Oligonucleotides, compositions and methods thereof |
CN109310765A (en) | 2016-06-20 | 2019-02-05 | 领先基因生物技术股份有限公司 | Antibody-drug conjugates |
IL301091A (en) | 2016-07-05 | 2023-05-01 | Biomarin Tech Bv | Pre-mrna splice switching or modulating oligonucleotides comprising bicyclic scaffold moieties, with improved characteristics for the treatment of genetic disorders |
EP3485015A4 (en) | 2016-07-15 | 2020-07-29 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulation of dystrophin transcript |
EP3487523B1 (en) | 2016-07-19 | 2023-09-06 | Duke University | Therapeutic applications of cpf1-based genome editing |
MX2019005671A (en) | 2016-11-16 | 2020-07-20 | Academisch Ziekenhuis Leiden | Substances for targeting various selected organs or tissues. |
US10947540B2 (en) | 2016-11-29 | 2021-03-16 | Association Institut De Myologie | Allele-specific silencing therapy for Dynamin 2-related diseases |
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 |
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 |
KR102497564B1 (en) | 2016-12-26 | 2023-02-07 | 제이씨알 파마 가부시키가이샤 | Novel anti-human transferrin receptor antibody capable of penetrating blood-brain barrier |
JOP20190166A1 (en) | 2017-01-05 | 2019-07-02 | Univ Texas | Optimized strategy for exon skipping modifications using crispr/cas9 with triple guide sequences |
SG10202107429WA (en) | 2017-01-06 | 2021-08-30 | Avidity Biosciences Inc | Nucleic acid-polypeptide compositions and methods of inducing exon skipping |
WO2018140831A2 (en) | 2017-01-27 | 2018-08-02 | Silverback Therapeutics, Inc. | Tumor targeting conjugates and methods of use thereof |
EP3635009A1 (en) | 2017-06-07 | 2020-04-15 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for internalizing enzymes |
CA3066920A1 (en) | 2017-06-16 | 2018-12-20 | Eli Lilly And Company | Engineered antibody compounds and conjugates thereof |
GB201711809D0 (en) | 2017-07-21 | 2017-09-06 | Governors Of The Univ Of Alberta | Antisense oligonucleotide |
JP2020537497A (en) | 2017-09-22 | 2020-12-24 | アビディティー バイオサイエンシーズ,インク. | Nucleic Acid Polypeptide Compositions and Methods to Induce 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 |
MX2020003596A (en) | 2017-10-04 | 2020-07-22 | Avidity Biosciences Inc | Nucleic acid-polypeptide compositions and uses thereof. |
US20210363521A1 (en) | 2017-11-09 | 2021-11-25 | Vertex Pharmaceuticals Incorporated | CRISPR/CAS Systems For Treatment of DMD |
CA3083526A1 (en) | 2017-12-06 | 2019-06-13 | Andrew John Geall | Compositions and methods of treating muscle atrophy and myotonic dystrophy |
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 |
JP2021511803A (en) | 2018-01-31 | 2021-05-13 | ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム | Compositions and Methods for Correcting Dystrophin Mutations in Human Cardiomyocytes |
JP2019137675A (en) | 2018-02-05 | 2019-08-22 | Jcrファーマ株式会社 | Method for delivering drug to muscle |
JP2021512899A (en) | 2018-02-07 | 2021-05-20 | リジェネロン・ファーマシューティカルズ・インコーポレイテッドRegeneron Pharmaceuticals, Inc. | Methods and Compositions for Therapeutic Protein Delivery |
MX2020010687A (en) | 2018-04-12 | 2021-01-20 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods of use thereof. |
WO2019215175A1 (en) | 2018-05-08 | 2019-11-14 | Roche Innovation Center Copenhagen A/S | Oligonucleotides for modulating myh7 expression |
AU2019266550A1 (en) | 2018-05-11 | 2020-11-26 | Alpha Anomeric Sas | Oligonucleotides conjugates comprising 7'-5'-alpha-anomeric-bicyclic sugar nucleosides |
UY38247A (en) | 2018-05-30 | 2019-12-31 | Novartis Ag | ANTIBODIES AGAINST ENTPD2, COMBINATION THERAPIES AND METHODS OF USE OF ANTIBODIES AND COMBINATION THERAPIES |
US20220251551A1 (en) | 2018-06-13 | 2022-08-11 | Sarepta Therapeutics, Inc. | Exon skipping oligomers for muscular dystrophy |
EP3810775A1 (en) | 2018-06-21 | 2021-04-28 | 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 |
US20210322562A1 (en) | 2018-08-02 | 2021-10-21 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating centronuclear myopathy |
JP2021533196A (en) | 2018-08-02 | 2021-12-02 | ダイン セラピューティクス, インコーポレーテッドDyne Therapeutics, Inc. | Muscle-targeted complexes and their use to treat progressive ossifying fibrodysplasia |
US11911484B2 (en) | 2018-08-02 | 2024-02-27 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
CN113166240A (en) | 2018-08-02 | 2021-07-23 | 达因疗法公司 | Muscle-targeting complexes and their use for the treatment of friedreich's ataxia |
US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
EA202190421A1 (en) | 2018-08-02 | 2021-06-25 | Дайн Терапьютикс, Инк. | MUSCLE-SPECIFIC COMPLEXES AND THEIR APPLICATION FOR TREATMENT OF MUSCLE ATROPHY |
KR20210081322A (en) | 2018-08-02 | 2021-07-01 | 다인 세라퓨틱스, 인크. | Muscle targeting complexes and their use for treating dystrophinopathy |
CA3108335A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle-targeting complexes and uses thereof |
CA3108289A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
EP3829594A4 (en) | 2018-08-02 | 2022-05-11 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating hypertrophic cardiomyopathy |
US20210308272A1 (en) | 2018-08-02 | 2021-10-07 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
EA202190417A1 (en) | 2018-08-02 | 2021-06-23 | Дайн Терапьютикс, Инк. | MUSCLE-SPECIFIC COMPLEXES AND THEIR APPLICATION FOR TREATMENT OF PUMP'S DISEASE |
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 |
SG11202106593UA (en) | 2018-12-21 | 2021-07-29 | Avidity Biosciences Inc | Anti-transferrin receptor antibodies and uses thereof |
EP3959319A4 (en) | 2019-04-25 | 2023-06-07 | Avidity Biosciences, Inc. | Nucleic acid compositions and methods of multi-exon skipping |
AU2020287880A1 (en) | 2019-06-06 | 2022-01-20 | Avidity Biosciences, Inc. | UNA amidites and uses thereof |
EP3980436A4 (en) | 2019-06-06 | 2023-12-20 | Avidity Biosciences, Inc. | Nucleic acid-polypeptide compositions and uses thereof |
EP3980073A4 (en) | 2019-06-07 | 2023-11-01 | Dyne Therapeutics, Inc. | Methods of preparing protein-oligonucleotide complexes |
EP3997228A4 (en) | 2019-07-09 | 2024-05-15 | Univ Alberta | Exons 45-55 skipping using mutation-tailored cocktails of antisense morpholinos in the dmd gene |
CN112646032A (en) | 2019-10-10 | 2021-04-13 | 苏州亲为药业有限公司 | BCMA-targeted humanized monoclonal antibodies with human monkey crossover |
EP4045060A4 (en) | 2019-10-18 | 2023-09-13 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
WO2021142217A1 (en) | 2020-01-10 | 2021-07-15 | Dyne Thereapeutics, Inc. | Muscle targeting complexes and uses thereof for modulation of milck1 |
IL294478A (en) | 2020-01-10 | 2022-09-01 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
JP2023509793A (en) | 2020-01-10 | 2023-03-09 | 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 |
EP4087586A4 (en) | 2020-01-10 | 2024-05-08 | 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 |
US20230203180A1 (en) | 2020-01-10 | 2023-06-29 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for modulation of genes associated with muscle health |
WO2021142260A1 (en) | 2020-01-10 | 2021-07-15 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for modulation of acvr1 |
US20230226212A1 (en) | 2020-01-10 | 2023-07-20 | Dyne Therapeutics, Inc. | Muscle-targeting complexes and uses thereof |
CN115349013A (en) | 2020-01-10 | 2022-11-15 | 达因疗法公司 | Muscle targeting complexes and their use for the treatment of facioscapulohumeral muscular dystrophy |
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 |
US20230113823A1 (en) | 2020-01-31 | 2023-04-13 | Dyne Therapeutics, Inc. | Anti-transferrin receptor (tfr) antibody and uses thereof |
US20230103793A1 (en) | 2020-01-31 | 2023-04-06 | Dyne Therapeutics, Inc. | Anti-transferrin receptor (tfr) antibody and uses thereof |
WO2022020109A1 (en) | 2020-07-23 | 2022-01-27 | Dyne Therapeutics, Inc. | Muscle-targeting complexes and uses thereof in treating muscle atrophy |
EP4185315A1 (en) | 2020-07-23 | 2023-05-31 | Dyne Therapeutics, Inc. | Anti-transferrin receptor (tfr) antibody and uses thereof |
IL299663A (en) | 2020-07-23 | 2023-03-01 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
WO2022020106A1 (en) | 2020-07-23 | 2022-01-27 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
CN116194470A (en) | 2020-07-23 | 2023-05-30 | 达因疗法公司 | Muscle targeting complexes and uses thereof |
BR112023001125A2 (en) | 2020-07-23 | 2023-02-14 | Dyne Therapeutics Inc | MUSCLE TARGETING COMPLEXES AND THEIR USES TO TREAT DYSTROPHINOPATHIES |
US20230330562A1 (en) | 2020-09-03 | 2023-10-19 | Dyne Therapeutics, Inc. | Methods of preparing protein-oligonucleotide complexes |
EP4211243A2 (en) | 2020-09-11 | 2023-07-19 | Arrowhead Pharmaceuticals, Inc. | Rnai agents for inhibiting expression of dux4, compositions thereof, and methods of use |
JP2023552559A (en) | 2020-12-04 | 2023-12-18 | ダイン セラピューティクス,インコーポレーテッド | Antibody-oligonucleotide complexes and their uses |
AU2021414227A1 (en) | 2020-12-31 | 2023-07-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
EP4271478A1 (en) | 2020-12-31 | 2023-11-08 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
WO2022271549A1 (en) | 2021-06-21 | 2022-12-29 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating pompe disease |
WO2022271543A2 (en) | 2021-06-21 | 2022-12-29 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating friedreich's ataxia |
US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
AU2022309238A1 (en) | 2021-07-09 | 2024-01-25 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
WO2023283619A2 (en) | 2021-07-09 | 2023-01-12 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
EP4366774A2 (en) | 2021-07-09 | 2024-05-15 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for modulation of genes associated with muscle health |
AU2022306307A1 (en) | 2021-07-09 | 2024-02-01 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
US11648318B2 (en) | 2021-07-09 | 2023-05-16 | Dyne Therapeutics, Inc. | Anti-transferrin receptor (TFR) antibody and uses thereof |
EP4366784A1 (en) | 2021-07-09 | 2024-05-15 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations for treating dystrophinopathies |
WO2023283629A1 (en) | 2021-07-09 | 2023-01-12 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations thereof for treating facioscapulohumeral muscular dystrophy |
AU2022307086A1 (en) | 2021-07-09 | 2024-01-25 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
IL309935A (en) | 2021-07-09 | 2024-03-01 | 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 |
US11638761B2 (en) | 2021-07-09 | 2023-05-02 | 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 |
KR20240058160A (en) | 2021-09-16 | 2024-05-03 | 다인 세라퓨틱스, 인크. | Administration of muscle targeting complexes to treat dystrophinopathy |
CN118201640A (en) | 2021-11-01 | 2024-06-14 | 达因疗法公司 | Muscle targeting complexes for the treatment of myodystrophy |
WO2023086864A1 (en) | 2021-11-12 | 2023-05-19 | Dyne Therapeutics, Inc. | Muscle targeting complexes for treating facioscapulohumeral muscular dystrophy |
WO2023201318A1 (en) | 2022-04-15 | 2023-10-19 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations for treating myotonic dystrophy |
WO2023201324A1 (en) | 2022-04-15 | 2023-10-19 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
WO2023201332A1 (en) | 2022-04-15 | 2023-10-19 | Dyne Therapeutics, Inc. | Dosing of muscle targeting complexes for treating myotonic dystrophy |
US11931421B2 (en) | 2022-04-15 | 2024-03-19 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations 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 |
WO2024011150A2 (en) | 2022-07-06 | 2024-01-11 | Dyne Therapeutics, Inc. | Cns targeting complexes and uses thereof |
-
2019
- 2019-08-02 KR KR1020217005988A patent/KR20210081322A/en unknown
- 2019-08-02 CA CA3108282A patent/CA3108282A1/en active Pending
- 2019-08-02 AU AU2019312692A patent/AU2019312692A1/en active Pending
- 2019-08-02 MX MX2021001281A patent/MX2021001281A/en unknown
- 2019-08-02 EP EP19844892.0A patent/EP3829595A4/en active Pending
- 2019-08-02 US US17/265,024 patent/US20210308273A1/en not_active Abandoned
- 2019-08-02 SG SG11202100934PA patent/SG11202100934PA/en unknown
- 2019-08-02 JP JP2021529254A patent/JP2021532831A/en active Pending
- 2019-08-02 CN CN201980064966.2A patent/CN112955153A/en active Pending
- 2019-08-02 WO PCT/US2019/044949 patent/WO2020028832A1/en active Application Filing
-
2021
- 2021-01-31 IL IL280527A patent/IL280527A/en unknown
-
2022
- 2022-01-21 US US17/580,844 patent/US11369689B2/en active Active
- 2022-05-24 US US17/751,875 patent/US11497815B2/en active Active
- 2022-09-08 US US17/930,522 patent/US11633496B2/en active Active
-
2023
- 2023-03-10 US US18/181,623 patent/US11833217B2/en active Active
- 2023-10-24 US US18/492,894 patent/US12005124B2/en active Active
Also Published As
Publication number | Publication date |
---|---|
US11833217B2 (en) | 2023-12-05 |
IL280527A (en) | 2021-03-01 |
MX2021001281A (en) | 2021-07-15 |
JP2021532831A (en) | 2021-12-02 |
US11497815B2 (en) | 2022-11-15 |
US11633496B2 (en) | 2023-04-25 |
US20210308273A1 (en) | 2021-10-07 |
WO2020028832A1 (en) | 2020-02-06 |
EP3829595A4 (en) | 2022-08-24 |
KR20210081322A (en) | 2021-07-01 |
US20230001002A1 (en) | 2023-01-05 |
US20220288220A1 (en) | 2022-09-15 |
AU2019312692A1 (en) | 2021-03-11 |
US20240066139A1 (en) | 2024-02-29 |
US20230270873A1 (en) | 2023-08-31 |
CN112955153A (en) | 2021-06-11 |
US12005124B2 (en) | 2024-06-11 |
US11369689B2 (en) | 2022-06-28 |
US20220143206A1 (en) | 2022-05-12 |
CA3108282A1 (en) | 2020-02-06 |
EP3829595A1 (en) | 2021-06-09 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
IL280527A (en) | Muscle targeting complexes and uses thereof for treating dystrophinopathies | |
IL280536A (en) | Muscle targeting complexes and uses thereof | |
IL280426A (en) | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy | |
IL280483A (en) | Muscle targeting complexes and uses thereof for treating pompe disease | |
EP4087878A4 (en) | Muscle targeting complexes and uses thereof for treating dystrophinopathies | |
EP3830130A4 (en) | Muscle targeting complexes and uses thereof for treating centronuclear myopathy | |
IL280535A (en) | Muscle targeting complexes and uses thereof for treating friedreich's ataxia | |
IL299662A (en) | Muscle targeting complexes and uses thereof for treating dystrophinopathies | |
IL276808A (en) | Compositions and methods for cancer treatment | |
IL267795A (en) | Combination therapy for the treatment of cancer | |
GB201903546D0 (en) | Cancer treatment | |
IL312142A (en) | Muscle targeting complexes for treating dystrophinopathies | |
IL275949A (en) | Combination therapy for treating or preventing cancer | |
IL287652A (en) | Cancer treatment | |
IL266993A (en) | Combination therapy for the treatment of cancer | |
IL285466A (en) | Cancer treatment | |
ZA202002526B (en) | New alcoxyamino derivatives for treating pain and pain related conditions | |
IL269121A (en) | Usl-311 for use in the treatment of cancer | |
IL288035A (en) | Cancer treatment | |
IL276073A (en) | Combination therapy for treating or preventing cancer | |
IL275860A (en) | Combination therapy for treating or preventing cancer | |
GB201819920D0 (en) | Cancer treatment | |
IL253642A0 (en) | Combination therapy for the treatment of cancer | |
GB201907204D0 (en) | Cancer treatment 3 | |
GB201907150D0 (en) | Cancer treatment 2 |