TW202144572A - 治療臉肩胛肱骨肌肉失養症之組合物及方法 - Google Patents

治療臉肩胛肱骨肌肉失養症之組合物及方法 Download PDF

Info

Publication number
TW202144572A
TW202144572A TW110109011A TW110109011A TW202144572A TW 202144572 A TW202144572 A TW 202144572A TW 110109011 A TW110109011 A TW 110109011A TW 110109011 A TW110109011 A TW 110109011A TW 202144572 A TW202144572 A TW 202144572A
Authority
TW
Taiwan
Prior art keywords
cases
polynucleic acid
acid molecule
sequence
seq
Prior art date
Application number
TW110109011A
Other languages
English (en)
Chinese (zh)
Inventor
巴博拉 梅兒可娃
羅伯 布奇
碧翠絲 黛安娜 達利曼特
卡諾 大衛 薩拉
Original Assignee
美商亞維代堤生物科學公司
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 美商亞維代堤生物科學公司 filed Critical 美商亞維代堤生物科學公司
Publication of TW202144572A publication Critical patent/TW202144572A/zh

Links

Images

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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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
    • 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
    • A61K47/6883—Polymer-drug antibody conjugates, e.g. mitomycin-dextran-Ab; DNA-polylysine-antibody complex or conjugate used for therapy
    • 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
    • A61K47/6889—Conjugates wherein the antibody being the modifying agent and wherein the linker, binder or spacer confers particular properties to the conjugates, e.g. peptidic enzyme-labile linkers or acid-labile linkers, providing for an acid-labile immuno conjugate wherein the drug may be released from its antibody conjugated part in an acidic, e.g. tumoural or environment
    • 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
    • A61K47/69—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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6921—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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere
    • A61K47/6927—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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores
    • A61K47/6929—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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores the form being a nanoparticle, e.g. an immuno-nanoparticle
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/06—Anabolic agents
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K19/00—Hybrid peptides, i.e. peptides covalently bound to nucleic acids, or non-covalently bound protein-protein complexes
    • 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
    • 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
    • C07K2317/40—Immunoglobulins specific features characterized by post-translational modification
    • 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
    • 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
    • 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
    • C12N2310/313—Phosphorodithioates
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • 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
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/332—Abasic residue
    • 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
    • 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
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Molecular Biology (AREA)
  • Immunology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Cell Biology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Endocrinology (AREA)
  • Nanotechnology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
TW110109011A 2020-03-19 2021-03-12 治療臉肩胛肱骨肌肉失養症之組合物及方法 TW202144572A (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202062992071P 2020-03-19 2020-03-19
US62/992,071 2020-03-19
US202063066655P 2020-08-17 2020-08-17
US63/066,655 2020-08-17

Publications (1)

Publication Number Publication Date
TW202144572A true TW202144572A (zh) 2021-12-01

Family

ID=77772141

Family Applications (1)

Application Number Title Priority Date Filing Date
TW110109011A TW202144572A (zh) 2020-03-19 2021-03-12 治療臉肩胛肱骨肌肉失養症之組合物及方法

Country Status (11)

Country Link
US (8) US11555190B2 (enExample)
EP (1) EP4121063A4 (enExample)
JP (2) JP7788060B2 (enExample)
KR (1) KR20220156867A (enExample)
CN (1) CN115666589A (enExample)
AU (1) AU2021237465A1 (enExample)
CA (1) CA3172111A1 (enExample)
IL (2) IL296387B2 (enExample)
MX (1) MX2022011499A (enExample)
TW (1) TW202144572A (enExample)
WO (1) WO2021188390A1 (enExample)

Families Citing this family (12)

* 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
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
CN116096890A (zh) 2020-04-02 2023-05-09 米雷楚莱股份有限公司 使用工程化寡核苷酸的靶向抑制
AU2021342155A1 (en) 2020-09-11 2023-04-13 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of DUX4, compositions thereof, and methods of use
EP4401792A4 (en) 2021-09-16 2025-12-03 Avidity Biosciences Inc COMPOSITIONS AND METHODS OF TREATMENT OF FACIO-SCAPULO-HUMERAL MUSCULAR DYSTROPHY
KR20240125036A (ko) * 2021-12-23 2024-08-19 마이레큘, 인크. 폴리뉴클레오타이드의 전달을 위한 조성물
US12491257B2 (en) 2023-06-27 2025-12-09 Avidity Biosciences, Inc. Compositions and methods of using PRKAG2-targeting antibody-oligonucleotide conjugates
AU2024308906A1 (en) 2023-06-30 2026-02-05 Atrium Therapeutics, Inc. Compositions and methods of using pln-targeting antibody-oligonucleotide conjugates
KR20260044219A (ko) 2023-07-21 2026-04-01 매로우 테라퓨틱스, 인크. 조혈 세포 표적화 접합체 및 관련 방법
US20260159835A1 (en) * 2024-12-05 2026-06-11 Soufflé Therapeutics, Inc. Oligonucleotide-mediated knockdown of dux4

Family Cites Families (253)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPS5892644A (ja) 1981-11-26 1983-06-02 Taiho Yakuhin Kogyo Kk 新規なニトロ安息香酸アミド誘導体およびそれを含有する放射線増感剤
US4694778A (en) 1984-05-04 1987-09-22 Anicon, Inc. Chemical vapor deposition wafer boat
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
JP2528107B2 (ja) 1985-03-15 1996-08-28 サマ−トン,ジエ−ムス ポリヌクレオチド測定試薬と方法
US4866132A (en) 1986-04-17 1989-09-12 The Research Foundation Of State University Of New York Novel radiopaque barium polymer complexes, compositions of matter and articles prepared therefrom
US5616584A (en) 1986-09-25 1997-04-01 Sri International 1,2,4-benzotriazine oxides as radiosensitizers and selective cytotoxic agents
US4921963A (en) 1987-04-13 1990-05-01 British Columbia Cancer Foundation Platinum complexes with one radiosensitizing ligand
JPH0819111B2 (ja) 1987-10-22 1996-02-28 ポーラ化成工業株式会社 2―ニトロイミダゾール誘導体及びこれを有効成分とする放射線増感剤
US4828971A (en) 1988-03-24 1989-05-09 Eastman Kodak Company Thermally processable element comprising a backing layer
CA1251835A (en) 1988-04-05 1989-03-28 Wai-Cheung Tang Dielectric image-resonator multiplexer
US5256334A (en) 1988-09-08 1993-10-26 The Research Foundation Of The State University Of New York Homogeneous radiopaque polymer-organobismuth composites
WO1991004753A1 (en) 1989-10-02 1991-04-18 Cetus Corporation Conjugates of antisense oligonucleotides and therapeutic uses thereof
US5208020A (en) 1989-10-25 1993-05-04 Immunogen Inc. Cytotoxic agents comprising maytansinoids and their therapeutic use
AU649074B2 (en) 1990-10-12 1994-05-12 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Modified ribozymes
DE4216134A1 (de) 1991-06-20 1992-12-24 Europ Lab Molekularbiolog Synthetische katalytische oligonukleotidstrukturen
JPH07509444A (ja) 1991-11-26 1995-10-19 アルカーメス・インコーポレーテツド トランスフェリンセレプター特異的抗体−神経薬又は診断薬複合体の製造法
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
EP0654993A1 (en) 1992-08-17 1995-05-31 Quadra Logic Technologies Inc. Method for destroying or inhibiting growth of unwanted cells or tissues
US5346981A (en) 1993-01-13 1994-09-13 Miles Inc. Radiopaque polyurethanes
KR100361933B1 (ko) 1993-09-08 2003-02-14 라 졸라 파마슈티칼 컴파니 화학적으로정의된비중합성결합가플랫폼분자및그것의콘주게이트
US5599796A (en) 1993-12-02 1997-02-04 Emory University Treatment of urogenital cancer with boron neutron capture therapy
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
CA2192203A1 (en) 1994-06-17 1995-12-28 Werner Hofheinz N,n'-bis(quinolin-4-yl)-diamine derivatives, their preparation and their use as antimalarials
US5716824A (en) 1995-04-20 1998-02-10 Ribozyme Pharmaceuticals, Inc. 2'-O-alkylthioalkyl and 2-C-alkylthioalkyl-containing enzymatic nucleic acids (ribozymes)
WO1996019487A1 (en) 1994-12-22 1996-06-27 Nissan Chemical Industries, Ltd. Organobismuth derivatives and process for producing the same
US5700825A (en) 1995-03-31 1997-12-23 Florida State University Radiosensitizing diamines and their pharmaceutical preparations
US5889136A (en) 1995-06-09 1999-03-30 The Regents Of The University Of Colorado Orthoester protecting groups in RNA synthesis
AU1430097A (en) 1996-01-16 1997-08-11 Ribozyme Pharmaceuticals, Inc. Synthesis of methoxy nucleosides and enzymatic nucleic acid molecules
CA2249195A1 (en) 1996-03-18 1997-09-25 Board Of Regents, The University Of Texas System Immunoglobin-like domains with increased half lives
US9096636B2 (en) 1996-06-06 2015-08-04 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds and their use in gene modulation
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US5849902A (en) 1996-09-26 1998-12-15 Oligos Etc. Inc. Three component chimeric antisense oligonucleotides
CA2274587A1 (en) 1996-12-10 1998-06-18 Genetrace Systems Inc. Releasable nonvolatile mass-label molecules
US20030073207A1 (en) 1997-01-31 2003-04-17 Saghir Akhtar Enzymatic nucleic acid treatment of diseases or conditions related to levels of epidermal growth factor receptors
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
US6111086A (en) 1998-02-27 2000-08-29 Scaringe; Stephen A. Orthoester protecting groups
CA2323757C (en) 1998-04-02 2011-08-02 Genentech, Inc. Antibody variants and fragments thereof
US6194551B1 (en) 1998-04-02 2001-02-27 Genentech, Inc. Polypeptide variants
US6528624B1 (en) 1998-04-02 2003-03-04 Genentech, Inc. Polypeptide variants
CA2326823A1 (en) 1998-04-20 1999-10-28 Ribozyme Pharmaceuticals, Inc. Nucleic acid molecules with novel chemical compositions capable of modulating gene expression
GB9816575D0 (en) 1998-07-31 1998-09-30 Zeneca Ltd Novel compounds
ES2260570T3 (es) 1998-08-27 2006-11-01 Spirogen Limited Pirrolobenziodiazepinas dimericas.
GB9818730D0 (en) 1998-08-27 1998-10-21 Univ Portsmouth Collections of compounds
GB9818731D0 (en) 1998-08-27 1998-10-21 Univ Portsmouth Compounds
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
US6849272B1 (en) 1999-04-21 2005-02-01 Massachusetts Institute Of Technology Endosomolytic agents and cell delivery systems
DE10160151A1 (de) 2001-01-09 2003-06-26 Ribopharma Ag Verfahren zur Hemmung der Expression eines vorgegebenen Zielgens
DE10100586C1 (de) 2001-01-09 2002-04-11 Ribopharma Ag Verfahren zur Hemmung der Expression eines Ziegens
US7829693B2 (en) 1999-11-24 2010-11-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a target gene
CA2328356A1 (en) 1999-12-22 2001-06-22 Itty Atcravi Recreational vehicles
AU767394C (en) 1999-12-29 2005-04-21 Immunogen, Inc. Cytotoxic agents comprising modified doxorubicins and daunorubicins and their therapeutic use
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
US8202979B2 (en) 2002-02-20 2012-06-19 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid
US8273866B2 (en) 2002-02-20 2012-09-25 Merck Sharp & Dohme Corp. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SINA)
KR20080023768A (ko) 2000-03-30 2008-03-14 화이트헤드 인스티튜트 포 바이오메디칼 리서치 Rna 간섭의 rna 서열 특이적인 매개체
EP2796553B1 (en) 2000-03-30 2019-06-19 Whitehead Institute for Biomedical Research RNA sequence-specific mediators of RNA interference
AU5752601A (en) 2000-05-04 2001-11-12 Avi Biopharma Inc Splice-region antisense composition and method
CN100523215C (zh) 2000-12-01 2009-08-05 马普科技促进协会 介导rna干涉的小rna分子
US8546143B2 (en) 2001-01-09 2013-10-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a target gene
US6884869B2 (en) 2001-04-30 2005-04-26 Seattle Genetics, Inc. Pentapeptide compounds and uses related thereto
DE10133858A1 (de) 2001-07-12 2003-02-06 Aventis Pharma Gmbh Synthetische doppelsträngige Oligonucleotide zur gezielten Hemmung der Genexpression
AU2002368077B2 (en) 2001-07-12 2010-03-04 Jefferson Foote Super humanized antibodies
US6942972B2 (en) 2001-10-24 2005-09-13 Beckman Coulter, Inc. Efficient synthesis of protein-oligonucleotide conjugates
US6716821B2 (en) 2001-12-21 2004-04-06 Immunogen Inc. Cytotoxic agents bearing a reactive polyethylene glycol moiety, cytotoxic conjugates comprising polyethylene glycol linking groups, and methods of making and using the same
US20060009409A1 (en) 2002-02-01 2006-01-12 Woolf Tod M Double-stranded oligonucleotides
EP1432724A4 (en) 2002-02-20 2006-02-01 Sirna Therapeutics Inc RNA inhibition mediated inhibition of MAP KINASE GENES
US9181551B2 (en) 2002-02-20 2015-11-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US9657294B2 (en) 2002-02-20 2017-05-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US20040023220A1 (en) 2002-07-23 2004-02-05 Lawrence Greenfield Integrated method for PCR cleanup and oligonucleotide removal
ES2369542T3 (es) 2002-07-31 2011-12-01 Seattle Genetics, Inc. Conjugados de auristatina y su uso para tratar cáncer, una enfermedad autoinmune o una enfermedad infecciosa.
EP1857547B2 (en) 2002-08-05 2020-12-02 Silence Therapeutics GmbH Further novel forms of interfering RNA molecules
US7923547B2 (en) 2002-09-05 2011-04-12 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US7250496B2 (en) 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
EP2305813A3 (en) 2002-11-14 2012-03-28 Dharmacon, Inc. Fuctional and hyperfunctional sirna
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
US8084582B2 (en) 2003-03-03 2011-12-27 Xencor, Inc. Optimized anti-CD20 monoclonal antibodies having Fc variants
US20070167384A1 (en) 2003-04-02 2007-07-19 Dharmacon, Inc. Modified polynucleotides for use in rna interference
EP1608733B1 (en) 2003-04-02 2011-12-07 Dharmacon, Inc. Modified polynucleotides for use in rna interference
US20040224893A1 (en) 2003-05-06 2004-11-11 Li-Hsien Wang Methods of using IL-1 antagonists to treat neointimal hyperplasia
US7276497B2 (en) 2003-05-20 2007-10-02 Immunogen Inc. Cytotoxic agents comprising new maytansinoids
US7750144B2 (en) 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
CA2528012C (en) 2003-06-02 2015-11-24 University Of Massachusetts Methods and compositions for controlling efficacy of rna silencing
CA2527958C (en) 2003-06-02 2014-04-08 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of rnai
ES2905724T3 (es) 2003-06-13 2022-04-11 Alnylam Europe Ag Acido ribonucleico bicatenario con elevada eficacia en un organismo
GB0416511D0 (en) 2003-10-22 2004-08-25 Spirogen Ltd Pyrrolobenzodiazepines
US8133733B2 (en) 2003-10-24 2012-03-13 Gencia Corporation Nonviral vectors for delivering polynucleotides to target tissues
ES2605443T3 (es) 2003-11-06 2017-03-14 Seattle Genetics, Inc. Conjugados de auristatina con anticuerpos anti-HER2 o anti-CD22 y su uso en terapia
US8389710B2 (en) 2004-02-27 2013-03-05 Operational Technologies Corporation Therapeutic nucleic acid-3′-conjugates
EP1723152B1 (en) 2004-03-09 2015-02-11 Spirogen Sàrl Pyrrolobenzodiazepines
CA2559955C (en) 2004-03-15 2016-02-16 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded rna
EP1765847A4 (en) 2004-05-27 2010-10-20 Alnylam Pharmaceuticals Inc NUCLEASERESISTENT DOUBLE-STRANDED RIBONUCLEIC ACID
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
JP2008519863A (ja) 2004-11-12 2008-06-12 シアトル ジェネティクス インコーポレイティッド N末端にアミノ安息香酸単位を有するオーリスタチン
US7429482B2 (en) 2005-01-13 2008-09-30 United States Of America As Represented By The Department Of Veterans Affairs Screening tools for discovery of novel anabolic agents
ES2338696T3 (es) 2005-04-21 2010-05-11 Spirogen Limited Pirrolobenzodiazepinas.
BRPI0611474A2 (pt) 2005-05-26 2010-09-14 Ensemble Discovery Corp biodetecção por quìmica modelada com ácido nucléico
WO2007008848A2 (en) 2005-07-07 2007-01-18 Seattle Genetics, Inc. Monomethylvaline compounds having phenylalanine carboxy modifications at the c-terminus
CN101287835A (zh) 2005-08-17 2008-10-15 株式会社百奥尼 用于siRNA细胞内输送的siRNA-亲水性聚合物结合物及其方法
WO2007028065A2 (en) 2005-08-30 2007-03-08 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds for modulation of splicing
DK1813614T3 (da) 2006-01-25 2012-01-23 Sanofi Sa Cytotoksiske midler, der omfatter nye tomaymycinderivater
US7750116B1 (en) 2006-02-18 2010-07-06 Seattle Genetics, Inc. Antibody drug conjugate metabolites
LT2735568T (lt) 2006-05-10 2017-11-27 Sarepta Therapeutics, Inc. Oligonukleotido analogai, turintys katijonines jungtis tarp subvienetų
EP1857548A1 (en) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
WO2012024613A1 (en) 2010-08-19 2012-02-23 Wintrance Corporation Synergistic biomolecule-polymer conjugates
US8598333B2 (en) 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
US7521232B2 (en) 2006-05-31 2009-04-21 Icx Nomadics, Inc. Emissive species for clinical imaging
BRPI0714381A2 (pt) 2006-07-10 2013-03-05 Biogen Idec Inc composiÇÕes e mÉtodos para inibir crescimento de cÂnceres deficientes em smad4
JP6047270B2 (ja) 2006-08-11 2016-12-21 バイオマリン テクノロジーズ ベー.フェー. Dnaリピートの不安定性に関連した遺伝的障害を治療するための方法及び手段
CN101500548A (zh) 2006-08-18 2009-08-05 弗·哈夫曼-拉罗切有限公司 用于体内递送多核苷酸的多缀合物
EP2148886B1 (en) 2007-05-10 2014-02-19 R & D Biopharmaceuticals Gmbh Tubulysine derivatives
PT2019104E (pt) 2007-07-19 2013-12-03 Sanofi Sa Agentes citotóxicos compreendendo novos derivados de tomaimicina e sua utilização terapêutica
EP3121281B1 (en) 2007-08-27 2021-03-31 1Globe Health Institute LLC Compositions of asymmetric interfering rna and uses thereof
EP2190995A2 (en) 2007-09-18 2010-06-02 Intradigm Corporation Compositions comprising k-ras sirna and methods of use
JP5723154B2 (ja) 2007-09-19 2015-05-27 アプライド バイオシステムズ リミテッド ライアビリティー カンパニー RNAiにおけるオフターゲット表現型の影響を減少させるためのSiRNA配列非依存性修飾フォーマットおよびその安定化型
NZ584793A (en) 2007-10-26 2012-05-25 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
US20110081362A1 (en) 2008-01-31 2011-04-07 The Brigham And Women's Hospital, Inc. Treatment of cancer
US20110065774A1 (en) 2008-01-31 2011-03-17 Alnylam Pharmaceuticals Chemically modified oligonucleotides and uses thereof
LT2237799T (lt) 2008-02-01 2019-07-25 Ascendis Pharma A/S Provaistas, apimantis besiskaidantį linkerį
EP3643782A1 (en) 2008-02-11 2020-04-29 Phio Pharmaceuticals Corp. Modified rnai polynucleotides and uses thereof
WO2009117531A1 (en) 2008-03-18 2009-09-24 Seattle Genetics, Inc. Auristatin drug linker conjugates
US8575123B2 (en) 2008-04-11 2013-11-05 Tekmira Pharmaceuticals Corporation Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
US8703921B2 (en) 2008-04-15 2014-04-22 The United States Of America, As Represented By The Secretary, Department Of Health & Human Services Compositions and methods for delivering inhibitory oligonucleotides
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
GB0813432D0 (en) 2008-07-22 2008-08-27 Spirogen Ltd Pyrrolobenzodiazepines
AU2009279458B2 (en) 2008-08-08 2015-07-02 Code Biotherapeutics, Inc. Long-acting DNA dendrimers and methods thereof
ES2915692T3 (es) 2008-08-13 2022-06-24 California Inst Of Techn Nanopartículas portadoras y composiciones, métodos y sistemas relacionados
EP2690175B1 (en) 2008-09-02 2016-12-28 Alnylam Pharmaceuticals, Inc. Compositions and methods for combined inhibition of mutant EGFR gene and IL-6 expression
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
EP2342340A1 (en) 2008-09-22 2011-07-13 Rxi Pharmaceuticals Corporation Rna interference in skin indications
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
BRPI0923225A2 (pt) 2008-12-02 2016-10-04 Chiralgen Ltd metodo para sintese de acidos nucleicos modificados no atomo de fosforo
CA3014224C (en) 2009-02-05 2022-05-24 Immunogen, Inc. Condensed benzodiazepine-indoline derivatives and processes to prepare said derivatives
WO2010093788A2 (en) 2009-02-11 2010-08-19 Dicerna Pharmaceuticals, Inc. Multiplex dicer substrate rna interference molecules having joining sequences
US8481698B2 (en) 2009-03-19 2013-07-09 The President And Fellows Of Harvard College Parallel proximity ligation event analysis
WO2010115033A2 (en) 2009-04-02 2010-10-07 Regents Of The University Of Minnesota Nucleotide repeat expansion-associated polypeptides and uses thereof
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
WO2010129666A1 (en) 2009-05-05 2010-11-11 Altermune Technologies, Llc Chemically programmable immunity
EP2437785B1 (en) 2009-06-04 2015-02-25 Novartis AG METHODS FOR IDENTIFICATION OF SITES FOR IgG CONJUGATION
US20110269665A1 (en) 2009-06-26 2011-11-03 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
US9050375B2 (en) 2009-07-06 2015-06-09 Hoffmann-La Roche, Inc. Bi-specific digoxigenin binding antibodies
IT1394860B1 (it) 2009-07-22 2012-07-20 Kemotech S R L Composti farmaceutici
EP2456470A1 (en) 2009-07-22 2012-05-30 Cenix Bioscience GmbH Delivery system and conjugates for compound delivery via naturally occurring intracellular transport routes
EP2473607A2 (en) 2009-08-31 2012-07-11 INSERM - Institut National de la Santé et de la Recherche Médicale Exon skipping therapy for functional amelioration of semi functional dystrophin in becker and duchenne muscular dystrophy
MX2012004029A (es) 2009-10-07 2012-05-08 Macrogenics Inc Polipeptidos que contienen la region fc que exhiben funcion efectora mejorada debido a alteraciones del grado de fucosilacion, y metodos para su uso.
HUE040445T2 (hu) 2009-11-12 2019-03-28 Univ Western Australia Antiszensz molekulák és eljárások betegségek kezelésére
SG183273A1 (en) 2010-02-12 2012-09-27 Solulink Inc Preparation and/or purification of oligonucleotide conjugates
CN103068405A (zh) 2010-04-15 2013-04-24 西雅图基因公司 靶向吡咯并苯并二氮杂卓结合物
MX368648B (es) 2010-04-15 2019-10-09 Seattle Genetics Inc Pirrolobenzodiazepinas usadas para tratar enfermedades proliferativas.
WO2011139702A2 (en) 2010-04-28 2011-11-10 Isis Pharmaceuticals, Inc. Modified nucleosides and oligomeric compounds prepared therefrom
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.
JP2013531981A (ja) 2010-06-11 2013-08-15 アンチセンス・ファーマ・ゲーエムベーハー 選択的オリゴヌクレオチド修飾の方法
EP2409983A1 (en) 2010-07-19 2012-01-25 Leibniz-Institut für Pflanzenbiochemie (IPB) Tubulysin analogues
ES2756326T3 (es) 2010-07-19 2020-04-27 Ionis Pharmaceuticals Inc Modulación de la expresión de distrofia miotónica-proteína quinasa (DMPK)
EP2606152B2 (en) * 2010-08-18 2025-03-12 Fred Hutchinson Cancer Center Methods for determining the presence or risk of developing facioscapulohumeral dystrophy (fshd)
EP3118316A1 (en) * 2010-09-02 2017-01-18 Université de Mons Agents useful in treating facioscapulohumeral muscular dystrophy
WO2012058210A1 (en) 2010-10-29 2012-05-03 Merck Sharp & Dohme Corp. RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACIDS (siNA)
US9090629B2 (en) 2010-11-03 2015-07-28 Immunogen, Inc. Cytotoxic agents comprising new ansamitocin derivatives
US8501930B2 (en) 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
MX347298B (es) 2010-12-29 2017-04-21 Arrowhead Res Corp Conjugados de suministro de polinucleotidos in vivo que tienen acoplamientos sensibles a enzima.
BR112013020273A2 (pt) 2011-02-08 2016-10-18 Charlotte Mecklenburg Hospital oligonucleotídeos antissenso
WO2012125850A1 (en) 2011-03-16 2012-09-20 Amgen Inc. Fc variants
US9045750B2 (en) 2011-03-18 2015-06-02 Yuelong Ma Humanized lewis-Y specific antibody-based delivery of dicer substrate siRNA (D-siRNA) against STAT3
LT2691417T (lt) 2011-03-29 2018-10-25 Roche Glycart Ag Antikūno fc variantai
WO2012177837A2 (en) 2011-06-21 2012-12-27 Immunogen, Inc. Novel maytansinoid derivatives with peptide linker and conjugates thereof
CA2839593A1 (en) 2011-07-15 2013-01-24 Sarepta Therapeutics, Inc. Methods and compositions for manipulating translation of protein isoforms from alternative initiation start sites
CN107365339A (zh) 2011-07-19 2017-11-21 波涛生命科学有限公司 合成官能化核酸的方法
CA2842798C (en) * 2011-07-25 2021-02-23 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of dux4
US10023861B2 (en) 2011-08-29 2018-07-17 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
CA2850373C (en) 2011-10-14 2019-07-16 Seattle Genetics, Inc. Pyrrolobenzodiazepines and targeted conjugates
US9593141B2 (en) 2011-11-09 2017-03-14 President And Fellows Of Harvard College Lin28/let-7 crystal structures, purification protocols, and molecular probes suitable for screening assays and therapeutics
US9796974B2 (en) 2011-11-18 2017-10-24 Alnylam Pharmaceuticals, Inc. Modified RNAi agents
BR112014013526A8 (pt) 2011-12-05 2017-06-13 Igenica Biotherapeutics Inc conjugados de anticorpo-fármaco e compostos, composições e métodos relacionados
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
US10010623B2 (en) 2012-02-16 2018-07-03 Ucl Business Plc Lysosome-cleavable linker
RU2582259C2 (ru) 2012-02-24 2016-04-20 Алтеоген Инк. Модифицированные антитела, с которыми связан мотив, включающий остаток цистеина, конъюгаты этих модифицированных антител с лекарственными веществами и способ их получения
AR090905A1 (es) 2012-05-02 2014-12-17 Merck Sharp & Dohme Conjugados que contienen tetragalnac y peptidos y procedimientos para la administracion de oligonucleotidos, composicion farmaceutica
CN104583406A (zh) 2012-05-02 2015-04-29 诺华股份有限公司 治疗kras相关疾病的有机组合物
CN104640572B (zh) 2012-05-15 2018-04-27 索伦托医疗有限公司 药物偶联物,偶联方法,及其用途
US9504756B2 (en) 2012-05-15 2016-11-29 Seattle Genetics, Inc. Self-stabilizing linker conjugates
DK2872147T3 (da) 2012-07-13 2023-02-20 Wave Life Sciences Ltd Fremgangsmåde til fremstilling af kirale oligonukleotider
JP2015529073A (ja) 2012-08-20 2015-10-05 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 生物可逆性基を有するポリヌクレオチド
KR102058381B1 (ko) 2012-08-24 2019-12-24 강원대학교산학협력단 인간 l1cam에 대한 인간화 항체 및 그의 제조 방법
DK2839860T3 (da) 2012-10-12 2019-06-17 Medimmune Ltd Pyrrolobenzodiazepiner og konjugater deraf
CA2889044A1 (en) 2012-11-15 2014-05-22 Roche Innovation Center Copenhagen A/S Anti apob antisense conjugate compounds
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
KR20210132742A (ko) 2012-12-21 2021-11-04 알트루바이오 인코퍼레이티드 친수성 자기-희생 링커 및 그것의 포합체
US9481905B2 (en) 2013-02-19 2016-11-01 Orizhan Bioscience Limited Method of using neutrilized DNA (N-DNA) as surface probe for high throughput detection platform
MX363787B (es) 2013-03-13 2019-04-03 Medimmune Ltd Pirrolobenzodiazepinas y conjugados de los mismos.
US9217148B2 (en) 2013-03-14 2015-12-22 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
EP3964237B1 (en) 2013-03-15 2026-02-18 Regeneron Pharmaceuticals, Inc. Biologically active molecules, conjugates thereof, and therapeutic uses
EP2777714A1 (en) 2013-03-15 2014-09-17 NBE-Therapeutics LLC Method of producing an immunoligand/payload conjugate by means of a sequence-specific transpeptidase enzyme
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
CN109234274B (zh) 2013-03-27 2021-09-21 伊萨纳治疗有限公司 修饰的TGF-β寡核苷酸
US9295731B2 (en) 2013-04-01 2016-03-29 Mark Quang Nguyen Cleavable drug conjugates, compositions thereof and methods of use
WO2014169049A1 (en) 2013-04-09 2014-10-16 Duke University 2' fluoro-modified rnas as immunostimulators
GB2529356B (en) 2013-04-28 2020-12-23 Genequantum Healthcare Co Ltd Novel linkers, coupling intermediates, conjugates, preparation method and application thereof
SG11201509566RA (en) 2013-05-20 2015-12-30 Genentech Inc Anti-transferrin receptor antibodies and methods of use
EP3004174B1 (en) 2013-05-31 2019-04-17 Zymeworks Inc. Heteromultimers with reduced or silenced effector function
US11229711B2 (en) 2013-06-06 2022-01-25 Magenta Therapeutics, Inc. Linkers for antibody-drug conjugates and related compounds, compositions, and methods of use
US20140363454A1 (en) 2013-06-06 2014-12-11 Igenica Biotherapeutics, Inc. Antibody-Drug Conjugates, Compositions and Methods of Use
TW201536329A (zh) 2013-08-09 2015-10-01 Isis Pharmaceuticals Inc 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法
EP2845607A1 (en) 2013-09-09 2015-03-11 University of Vienna Antisense oligonucleotides with improved pharmacokinetic properties
WO2015038426A1 (en) 2013-09-13 2015-03-19 Asana Biosciences, Llc Self-immolative linkers containing mandelic acid derivatives, drug-ligand conjugates for targeted therapies and uses thereof
PT3057585T (pt) 2013-10-15 2020-10-21 Seagen Inc Fármaco-ligadores peguilados para farmacocinética melhorada de conjugados de fármaco-ligando
AU2014337317A1 (en) 2013-10-15 2016-09-15 Sorrento Therapeutics Inc. Drug-conjugates with a targeting molecule and two different drugs
WO2015069587A2 (en) 2013-11-06 2015-05-14 Merck Sharp & Dohme Corp. Peptide containing conjugates for dual molecular delivery of oligonucleotides
US10378017B2 (en) 2013-12-02 2019-08-13 Brandeis University High temperature selection of nucleotide-supported carbohydrate vaccines and resulting glycosylated oligonucleotides
EP3094728B1 (en) 2014-01-16 2022-03-09 Wave Life Sciences Ltd. Chiral design
US10358643B2 (en) 2014-01-30 2019-07-23 Hoffmann-La Roche, Inc. Poly oligomer compound with biocleavable conjugates
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
TWI695011B (zh) 2014-06-18 2020-06-01 美商梅爾莎納醫療公司 抗her2表位之單株抗體及其使用之方法
US10988764B2 (en) 2014-06-23 2021-04-27 Monsanto Technology Llc Compositions and methods for regulating gene expression via RNA interference
IL316808A (en) 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
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
US10538763B2 (en) * 2015-01-16 2020-01-21 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of DUX4
EP3286318A2 (en) 2015-04-22 2018-02-28 Mina Therapeutics Limited Sarna compositions and methods of use
US20180344817A1 (en) 2015-05-01 2018-12-06 Precision Biosciences, Inc. Precise deletion of chromosomal sequences in vivo and treatment of nucleotide repeat expansion disorders using engineered nucleases
KR102644053B1 (ko) 2015-05-19 2024-03-06 사렙타 쎄러퓨틱스 인코퍼레이티드 펩티드 올리고뉴클레오티드 콘주게이트
AU2016282334B2 (en) 2015-06-24 2022-07-21 F. Hoffmann-La Roche Ag Anti-transferrin receptor antibodies with tailored affinity
WO2017007886A2 (en) 2015-07-08 2017-01-12 Idera Pharmaceuticals, Inc. Compositions for inhibiting dux4 gene expression and uses thereof
JP6749589B2 (ja) * 2015-07-31 2020-09-02 国立大学法人北海道大学 抗Eva1タンパク質抗体
WO2017148880A1 (en) 2016-03-01 2017-09-08 F. Hoffmann-La Roche Ag Obinutuzumab variants having altered cell death induction
KR102775461B1 (ko) * 2016-04-01 2025-02-28 어비디티 바이오사이언시스 인크. 핵산-폴리펩타이드 조성물 및 이의 용도
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
DK3473270T5 (da) 2016-06-20 2024-09-09 Genahead Bio Inc Konjugat af antistof-lægemiddel
US11427838B2 (en) 2016-06-29 2022-08-30 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of myotonic dystrophy type 1 (DM1) and other related disorders
CN110234762A (zh) 2016-10-28 2019-09-13 吉尼松公司 用于治疗肌强直性营养不良的组合物和方法
CN110337493B (zh) 2016-10-28 2024-03-12 吉尼松公司 用于治疗肌强直性营养不良的组合物和方法
WO2018098328A1 (en) 2016-11-23 2018-05-31 Alnylam Pharmaceuticals, Inc. Modified rna agents with reduced off-target effect
KR20240172236A (ko) * 2017-01-06 2024-12-09 어비디티 바이오사이언시스 인크. 핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법
JOP20190187A1 (ar) 2017-02-03 2019-08-01 Novartis Ag مترافقات عقار جسم مضاد لـ ccr7
DK3444010T3 (da) * 2017-08-14 2021-03-15 Scandiedge Therapeutics Ab Pklr-inhibering i behandlingen af nafld og hcc
SG11202002517RA (en) 2017-09-22 2020-04-29 Avidity Biosciences Inc Nucleic acid-polypeptide compositions and methods of inducing exon skipping
MA49923A (fr) 2017-10-02 2020-06-24 Res Inst Nationwide Childrens Hospital Système de déciblage de miarn pour interférence spécifique d'un tissu
SG11202003126VA (en) 2017-10-04 2020-05-28 Avidity Biosciences Inc Nucleic acid-polypeptide compositions and uses thereof
ES2927715T3 (es) * 2017-10-05 2022-11-10 Fulcrum Therapeutics Inc Inhibidores de la quinasa p38 reducen la expresión de dux4 y de los genes que le siguen para el tratamiento de la FSHD
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
WO2020028832A1 (en) * 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
EP3830259A4 (en) * 2018-08-02 2022-05-04 Dyne Therapeutics, Inc. MUSCLE TARGETED COMPLEXES AND THEIR USES IN THE TREATMENT OF FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
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
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
TW202104595A (zh) 2019-03-29 2021-02-01 日商田邊三菱製藥股份有限公司 用來調節dux4的表現之化合物、方法及醫藥組成物
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
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
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
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
AR123420A1 (es) 2020-09-01 2022-11-30 Ultragenyx Pharmaceutical Inc Inhibidores de dux4 y métodos de uso de estos
CN117677698A (zh) 2021-07-14 2024-03-08 米雷楚来股份有限公司 用于神经肌肉障碍的寡核苷酸及其组合物
EP4401792A4 (en) 2021-09-16 2025-12-03 Avidity Biosciences Inc COMPOSITIONS AND METHODS OF TREATMENT OF FACIO-SCAPULO-HUMERAL MUSCULAR DYSTROPHY

Also Published As

Publication number Publication date
US12104156B2 (en) 2024-10-01
KR20220156867A (ko) 2022-11-28
CA3172111A1 (en) 2021-09-23
US20210369762A1 (en) 2021-12-02
JP2026041765A (ja) 2026-03-10
IL296387B1 (en) 2024-04-01
US12049629B2 (en) 2024-07-30
WO2021188390A1 (en) 2021-09-23
JP7788060B2 (ja) 2025-12-18
US11999955B2 (en) 2024-06-04
IL310900A (en) 2024-04-01
US20230279395A1 (en) 2023-09-07
US12529056B2 (en) 2026-01-20
JP2023537798A (ja) 2023-09-06
US20250340880A1 (en) 2025-11-06
IL296387B2 (en) 2024-08-01
US20230287420A1 (en) 2023-09-14
US11555190B2 (en) 2023-01-17
AU2021237465A1 (en) 2022-10-13
US20240309382A1 (en) 2024-09-19
US20230340474A1 (en) 2023-10-26
US11525137B2 (en) 2022-12-13
EP4121063A1 (en) 2023-01-25
EP4121063A4 (en) 2024-07-03
MX2022011499A (es) 2022-10-07
US20210301290A1 (en) 2021-09-30
CN115666589A (zh) 2023-01-31
US20240287522A1 (en) 2024-08-29
IL296387A (en) 2022-11-01

Similar Documents

Publication Publication Date Title
JP7748962B2 (ja) 筋ジストロフィーを処置するための組成物および方法
JP7788060B2 (ja) 顔面肩甲上腕型筋ジストロフィーを処置するための組成物および方法
US20230330246A1 (en) Compositions and methods of treating pompe disease
JP7742485B2 (ja) 顔面肩甲上腕型筋ジストロフィーを処置する組成物および方法
HK40110738A (en) Compositions and methods of treating facioscapulohumeral muscular dystrophy
HK40087592A (en) Compositions and methods of treating facioscapulohumeral muscular dystrophy