MA51103A - Compositions et procédés de traitement de l'atrophie musculaire et de la dystrophie myotonique - Google Patents

Compositions et procédés de traitement de l'atrophie musculaire et de la dystrophie myotonique

Info

Publication number
MA51103A
MA51103A MA051103A MA51103A MA51103A MA 51103 A MA51103 A MA 51103A MA 051103 A MA051103 A MA 051103A MA 51103 A MA51103 A MA 51103A MA 51103 A MA51103 A MA 51103A
Authority
MA
Morocco
Prior art keywords
compositions
treatment
methods
muscle atrophy
myotonic dystrophy
Prior art date
Application number
MA051103A
Other languages
English (en)
Inventor
Rob Burke
David Sai-Ho Chu
Michael Caramian COCHRAN
Beatrice Diana Darimont
Venkata Ramana Doppalapudi
Andrew John Geall
Michael Hood
Barbora Malecova
Gulin Erdogan Marelius
Yunyu Shi
Original Assignee
Avidity Biosciences Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Avidity Biosciences Inc filed Critical Avidity Biosciences Inc
Publication of MA51103A publication Critical patent/MA51103A/fr

Links

Classifications

    • 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
    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • 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
    • 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
    • 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
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/11—Protein-serine/threonine kinases (2.7.11)
    • C12Y207/11001—Non-specific serine/threonine protein kinase (2.7.11.1), i.e. casein kinase or checkpoint kinase
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—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
    • C12N2310/35—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
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • 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
    • C12N2320/31—Combination therapy
    • 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

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)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Immunology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Cell Biology (AREA)
  • Plant Pathology (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Mycology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
MA051103A 2017-12-06 2018-12-06 Compositions et procédés de traitement de l'atrophie musculaire et de la dystrophie myotonique MA51103A (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762595545P 2017-12-06 2017-12-06
US201862725883P 2018-08-31 2018-08-31

Publications (1)

Publication Number Publication Date
MA51103A true MA51103A (fr) 2020-10-14

Family

ID=66751772

Family Applications (1)

Application Number Title Priority Date Filing Date
MA051103A MA51103A (fr) 2017-12-06 2018-12-06 Compositions et procédés de traitement de l'atrophie musculaire et de la dystrophie myotonique

Country Status (12)

Country Link
US (14) US10881743B2 (fr)
EP (1) EP3720448A4 (fr)
JP (3) JP7506601B2 (fr)
KR (5) KR102527941B1 (fr)
CN (4) CN118638787A (fr)
AU (2) AU2018378812B2 (fr)
CA (1) CA3083526A1 (fr)
IL (2) IL319835A (fr)
MA (1) MA51103A (fr)
MX (3) MX2020005860A (fr)
SG (1) SG11202005022YA (fr)
WO (1) WO2019113393A1 (fr)

Families Citing this family (70)

* 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
KR20240172236A (ko) 2017-01-06 2024-12-09 어비디티 바이오사이언시스 인크. 핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
SG11202003126VA (en) 2017-10-04 2020-05-28 Avidity Biosciences Inc Nucleic acid-polypeptide compositions and uses thereof
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
EP3735252A4 (fr) * 2018-01-04 2021-10-06 Avidity Biosciences, Inc. Molécules d'acide nucléique hétéroduplex et leurs utilisations
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
WO2020028832A1 (fr) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Complexes de ciblage musculaire et leurs utilisations pour le traitement de dystrophinopathies
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
US12370264B1 (en) 2018-08-02 2025-07-29 Dyne Therapeutics, Inc. Complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of delivering oligonucleotide to a subject
EP3830259A4 (fr) 2018-08-02 2022-05-04 Dyne Therapeutics, Inc. Complexes de ciblage musculaire et leurs utilisations pour le traitement de la dystrophie musculaire facio-scapulo-humérale
US12097263B2 (en) 2018-08-02 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
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
JP7581252B2 (ja) 2019-06-06 2024-11-12 アビディティー バイオサイエンシーズ，インク． Ｕｎａアミダイトおよびその使用
WO2020247782A1 (fr) 2019-06-06 2020-12-10 Avidity Biosciences, Inc. Compositions d'acide nucléique-polypeptide et utilisations de celles-ci
EP3997109A4 (fr) * 2019-07-10 2023-11-01 University of Washington Immuno-ciblage non viral
JP7692829B2 (ja) * 2019-07-30 2025-06-16 塩野義製薬株式会社 Ｍｕｒｆ１を標的とする核酸医薬
JP2022545265A (ja) * 2019-08-20 2022-10-26 ニューリタス リミテッド 筋萎縮を治療するためのペプチド
US20240148891A1 (en) * 2019-10-18 2024-05-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
WO2021142217A1 (fr) * 2020-01-10 2021-07-15 Dyne Thereapeutics, Inc. Complexes de ciblage musculaire et leurs utilisations pour la modulation de milck1
WO2021142269A1 (fr) * 2020-01-10 2021-07-15 Dyne Therapeutics, Inc. Complexes de ciblage de muscle et leurs utilisations pour la modulation de gènes associés à l'atrophie musculaire
CN122124274A (zh) * 2020-01-10 2026-06-02 达因疗法公司 肌肉靶向复合物及其用于治疗面肩肱型肌营养不良的用途
WO2021142331A1 (fr) * 2020-01-10 2021-07-15 Dyne Therapeutics, Inc. Complexes de ciblage de muscle et leurs utilisations pour la modulation de gènes associés à une maladie du muscle cardiaque
CN115427448A (zh) * 2020-01-10 2022-12-02 达因疗法公司 肌肉靶向复合物及其用途
EP4087653A4 (fr) * 2020-01-10 2024-06-19 Dyne Therapeutics, Inc. Complexes de ciblage musculaire et utilisations associées pour le traitement de la dystrophie myotonique
US20230111212A1 (en) * 2020-01-10 2023-04-13 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
JP7858532B2 (ja) * 2020-01-10 2026-05-14 ダイン セラピューティクス，インコーポレーテッド 筋肉標的複合体および筋肉の健康に関連する遺伝子のモジュレーションのためのその使用
US20230117883A1 (en) * 2020-01-24 2023-04-20 Dyne Therapeutics, Inc. Muscle-targeting complexes and uses thereof in treating muscle atrophy
WO2021154477A1 (fr) 2020-01-31 2021-08-05 Dyne Therapeutics, Inc. Anticorps anti-récepteur de la transferrine (tfr) et utilisations associées
IL295022A (en) * 2020-01-31 2022-09-01 Dyne Therapeutics Inc Antibody against transferrin receptor and uses thereof
JP2023515664A (ja) * 2020-03-06 2023-04-13 アリゴス セラピューティクス，インコーポレーテッド 修飾された短い干渉核酸（ｓｉＮＡ）分子およびそれらの使用
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
KR102927945B1 (ko) * 2020-03-27 2026-02-23 어비디티 바이오사이언시스 인크. 근이영양증의 치료용 조성물 및 방법
US20230330246A1 (en) * 2020-05-05 2023-10-19 Avidity Biosciences, Inc. Compositions and methods of treating pompe disease
MX2023000958A (es) * 2020-07-23 2023-04-14 Dyne Therapeutics Inc Complejos dirigidos al musculo y usos de estos para el tratamiento de la distrofia miotonica.
US11672872B2 (en) 2021-07-09 2023-06-13 Dyne Therapeutics, Inc. Anti-transferrin receptor antibody and uses thereof
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11633498B2 (en) 2021-07-09 2023-04-25 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
EP4185320A4 (fr) * 2020-07-23 2025-10-08 Dyne Therapeutics Inc Complexes de ciblage musculaire et leurs utilisations dans le traitement de l'atrophie musculaire
AU2021342155A1 (en) * 2020-09-11 2023-04-13 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of DUX4, compositions thereof, and methods of use
AU2021368859A1 (en) * 2020-10-28 2023-06-22 The Florey Institute Of Neuroscience And Mental Health Peptide-based delivery of agents
AU2021414227A1 (en) * 2020-12-31 2023-07-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
WO2023283623A1 (fr) 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Complexes ciblant le muscle et formulations de traitement de dystrophinopathies
CA3230796A1 (fr) 2021-09-01 2023-03-09 Biogen Ma Inc. Anticorps anti-recepteurs de la transferrine et leurs utilisations
US11833221B2 (en) * 2021-09-01 2023-12-05 Ionis Pharmaceuticals, Inc. Oligomeric compounds for reducing DMPK expression
EP4401792A4 (fr) 2021-09-16 2025-12-03 Avidity Biosciences Inc Compositions et méthodes de traitement d'une dystrophie musculaire facio-scapulo-humérale
KR102389889B1 (ko) 2021-11-23 2022-04-25 (주)에이피테크놀로지 2'-fl을 함유하는 근위축 또는 근감소증 개선, 예방 또는 치료용 조성물
JP7889887B2 (ja) * 2021-12-02 2026-07-14 リンノ ファーマシューティカルズ インコーポレイテッド トランスフェリン結合抗体及びその使用
US12071621B2 (en) 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping
IL316143A (en) 2022-04-15 2024-12-01 Dyne Therapeutics Inc Muscle targeting complexes and formulations for the treatment of myotonic dystrophy
WO2024063570A1 (fr) * 2022-09-23 2024-03-28 주식회사 셀리버리 Oligonucléotide antisens fusionné avec un peptide à pénétration cellulaire qui cible la protéine kinase de la dystrophie myotonique et son utilisation
JP2025535376A (ja) * 2022-10-19 2025-10-24 アローヘッド ファーマシューティカルズ インコーポレイテッド ＤＭ１タンパク質キナーゼ（ＤＭＰＫ）の発現を阻害するためのＲＮＡｉ剤、その組成物および使用方法
WO2024187057A2 (fr) 2023-03-08 2024-09-12 Biogen Ma Inc. Anticorps anti-récepteurs de la transferrine et leurs utilisations
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
US20250064958A1 (en) * 2023-08-22 2025-02-27 Eli Lilly And Company AMYLOID PRECURSOR PROTEIN (APP) RNAi AGENTS
WO2025072685A1 (fr) * 2023-09-28 2025-04-03 Wave Life Sciences Ltd. Compositions oligonucléotidiques et méthodes associées
IL327952A (en) 2023-10-26 2026-06-01 Biogen Ma Inc Anti-transferrin receptor antibodies and uses thereof
WO2025147541A1 (fr) * 2024-01-02 2025-07-10 Dyne Therapeutics, Inc. Méthodes et compositions pour traiter une dystrophie myotonique
CN120648679A (zh) * 2024-03-15 2025-09-16 迦进生物医药(上海)有限公司 抑制dmpk表达的多核苷酸分子及其用途
WO2025207959A1 (fr) 2024-03-27 2025-10-02 Biogen Ma Inc. Anticorps anti-récepteurs de la transferrine et leurs utilisations
TW202615058A (zh) * 2024-06-12 2026-04-16 美商艾拉倫製藥股份有限公司 失養強直性肌肉蛋白質激酶（ｄｍｐｋ）ｉｒｎａ組成物及其使用方法
WO2026030210A1 (fr) * 2024-07-29 2026-02-05 Arrowhead Pharmaceuticals, Inc. Plateformes d'administration à des cardiomyocytes et procédés d'utilisation
US20260216357A1 (en) * 2024-10-28 2026-07-30 Atrium Therapeutics, Inc. Lipid antibody oligonucleotide conjugates compositions and uses thereof
CN122303231A (zh) * 2024-12-31 2026-06-30 北京尧景基因技术有限公司 抑制MSTN基因表达的siRNA及其缀合物和应用
WO2026163078A1 (fr) * 2025-01-29 2026-08-06 Novartis Ag Composés ciblant dmpk pour le traitement de la dystrophie myotonique

Family Cites Families (214)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US1000075A (en) 1910-07-27 1911-08-08 William R Collins Tunneling-machine.
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
US4828971A (en) 1988-03-24 1989-05-09 Eastman Kodak Company Thermally processable element comprising a backing layer
CA1251835A (fr) 1988-04-05 1989-03-28 Wai-Cheung Tang Multiplexeur a resonateurs images dielectriques
WO1991004753A1 (fr) 1989-10-02 1991-04-18 Cetus Corporation Conjugues d'oligonucleotides non codants et leurs emplois therapeutiques
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
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
KR100361933B1 (ko) 1993-09-08 2003-02-14 라 졸라 파마슈티칼 컴파니 화학적으로정의된비중합성결합가플랫폼분자및그것의콘주게이트
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
CA2192203A1 (fr) 1994-06-17 1995-12-28 Werner Hofheinz Derives n,n'-bis(quinolin-4-yle)-diamine, leur preparation et leur utilisation en tant qu'antipaludeens
US5716824A (en) 1995-04-20 1998-02-10 Ribozyme Pharmaceuticals, Inc. 2'-O-alkylthioalkyl and 2-C-alkylthioalkyl-containing enzymatic nucleic acids (ribozymes)
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 (fr) 1996-03-18 1997-09-25 Board Of Regents, The University Of Texas System Domaines analogues a l'immunoglobuline a demi-vies prolongees
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 (fr) 1996-12-10 1998-06-18 Genetrace Systems Inc. Molecules d'etiquetage massique, non volatiles et liberables
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
US6194551B1 (en) 1998-04-02 2001-02-27 Genentech, Inc. Polypeptide variants
US6528624B1 (en) 1998-04-02 2003-03-04 Genentech, Inc. Polypeptide variants
CA2323757C (fr) 1998-04-02 2011-08-02 Genentech, Inc. Variants d'anticorps et fragments de ceux-ci
CA2326823A1 (fr) 1998-04-20 1999-10-28 Ribozyme Pharmaceuticals, Inc. Molecules d'acides nucleiques presentant de nouvelles compositions chimiques capables de moduler l'expression genique
GB9816575D0 (en) 1998-07-31 1998-09-30 Zeneca Ltd Novel compounds
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
AU3703100A (en) 1999-02-22 2000-09-14 Georgetown University Antibody fragment-targeted immunoliposomes for systemic gene delivery
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 (fr) 1999-12-22 2001-06-22 Itty Atcravi Vehicules recreatifs
AU767394C (en) 1999-12-29 2005-04-21 Immunogen, Inc. Cytotoxic agents comprising modified doxorubicins and daunorubicins and their therapeutic use
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
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
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)
EP2796553B1 (fr) 2000-03-30 2019-06-19 Whitehead Institute for Biomedical Research Médiateurs spécifiques de la séquence d'ARN d'interférence d'ARN
KR20080023768A (ko) 2000-03-30 2008-03-14 화이트헤드 인스티튜트 포 바이오메디칼 리서치 Ｒｎａ 간섭의 ｒｎａ 서열 특이적인 매개체
AU5752601A (en) 2000-05-04 2001-11-12 Avi Biopharma Inc Splice-region antisense composition and method
JP2002095476A (ja) * 2000-09-20 2002-04-02 Eisai Co Ltd トランスフェリン受容体を介した遺伝子導入法
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
US6942972B2 (en) 2001-10-24 2005-09-13 Beckman Coulter, Inc. Efficient synthesis of protein-oligonucleotide conjugates
US20060009409A1 (en) 2002-02-01 2006-01-12 Woolf Tod M Double-stranded oligonucleotides
EP1432724A4 (fr) 2002-02-20 2006-02-01 Sirna Therapeutics Inc Inhibition a mediation par interference d'arn de genes de map kinase
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)
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)
US20040023220A1 (en) 2002-07-23 2004-02-05 Lawrence Greenfield Integrated method for PCR cleanup and oligonucleotide removal
EP1857547B2 (fr) 2002-08-05 2020-12-02 Silence Therapeutics GmbH Autres nouvelles formes d'ARN interférants
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)
WO2006006948A2 (fr) 2002-11-14 2006-01-19 Dharmacon, Inc. Methodes et compositions permettant de selectionner des arnsi presentant une fonctionnalite amelioree
US7250496B2 (en) 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
EP2305813A3 (fr) 2002-11-14 2012-03-28 Dharmacon, Inc. SIRNA fonctionnel et hyperfonctionnel
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 (fr) 2003-04-02 2011-12-07 Dharmacon, Inc. Polynucleotides modifies utilisables pour l'interference arn
US20040224893A1 (en) * 2003-05-06 2004-11-11 Li-Hsien Wang Methods of using IL-1 antagonists to treat neointimal hyperplasia
CA2528012C (fr) 2003-06-02 2015-11-24 University Of Massachusetts Procedes et compositions de commande de l'efficacite permettant de rendre silencieux un arn
US7750144B2 (en) 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
CA2527958C (fr) 2003-06-02 2014-04-08 University Of Massachusetts Methodes et compositions en vue d'ameliorer l'efficacite et la specificicite de l'arni
ES2905724T3 (es) 2003-06-13 2022-04-11 Alnylam Europe Ag Acido ribonucleico bicatenario con elevada eficacia en un organismo
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
US7494925B2 (en) 2004-02-23 2009-02-24 Micron Technology, Inc. Method for making through-hole conductors for semiconductor substrates
US8389710B2 (en) 2004-02-27 2013-03-05 Operational Technologies Corporation Therapeutic nucleic acid-3′-conjugates
CA2559955C (fr) 2004-03-15 2016-02-16 City Of Hope Procedes et compositions pour l'inhibition specifique de l'expression genetique par l'arn double brin
EP1765847A4 (fr) 2004-05-27 2010-10-20 Alnylam Pharmaceuticals Inc Acide ribonucleique double brin resistant aux nucleases
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
EP1851248B1 (fr) 2005-02-10 2016-08-10 Baylor Research Institute Anticorps monoclonaux anti-interferon alpha et procedes d'utilisation
BRPI0611474A2 (pt) 2005-05-26 2010-09-14 Ensemble Discovery Corp biodetecção por quìmica modelada com ácido nucléico
US20070054259A1 (en) * 2005-06-17 2007-03-08 City Of Hope Modulation of hnRNP H and treatment of DM1
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 (fr) 2005-08-30 2007-03-08 Isis Pharmaceuticals, Inc. Composes oligomeriques chimeriques pour la modulation de l'epissage
WO2007121272A2 (fr) * 2006-04-11 2007-10-25 The Board Of Regents Of The University Of Texas System Procédé et compositions concernant des troubles à base de séquences nucléotidiques répétées
LT2735568T (lt) 2006-05-10 2017-11-27 Sarepta Therapeutics, Inc. Oligonukleotido analogai, turintys katijonines jungtis tarp subvienetų
EP1857548A1 (fr) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Procédé et moyen permettant d'induire un saut d'exon
WO2012024613A1 (fr) 2010-08-19 2012-02-23 Wintrance Corporation Conjugués synergiques biomolécule-polymère
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
MX2008015138A (es) 2006-06-02 2009-01-28 Aveo Pharmaceuticals Inc Proteinas de union al factor de crecimiento de hepatocitos (fch).
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
EP3121281B1 (fr) 2007-08-27 2021-03-31 1Globe Health Institute LLC Compositions d'arn interférent asymétrique et leurs utilisations
DK2185816T3 (en) 2007-08-29 2018-03-12 Mhi Vestas Offshore Wind As MONOPILE FOUNDATION FOR OFFSHORE WINDMILL
EP2190995A2 (fr) 2007-09-18 2010-06-02 Intradigm Corporation Compositions comprenant un arnsi de k-ras et procédés d utilisation
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
KR20100107501A (ko) 2008-01-18 2010-10-05 메디뮨 엘엘씨 부위 특이적 접합을 위한 시스테인 조작 항체
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 (fr) 2008-02-11 2020-04-29 Phio Pharmaceuticals Corp. Polynucléotides d'arni modifiés et leurs utilisations
WO2009117531A1 (fr) 2008-03-18 2009-09-24 Seattle Genetics, Inc. Conjugués auristatine-lieur de médicament
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
US8248352B2 (en) 2008-04-25 2012-08-21 Lg Display Co., Ltd. Driving circuit of liquid crystal display
EP2119783A1 (fr) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Procédé pour l'omission efficace de l'exon (44) dans la dystrophie musculaire de Duchenne et moyens connexes
US20110130346A1 (en) 2008-05-30 2011-06-02 Isis Innovation Limited Peptide conjugates for delvery of biologically active compounds
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 (fr) 2008-09-02 2016-12-28 Alnylam Pharmaceuticals, Inc. Compositions et procédés d'inhibition combinée d'expression du gène mutant EGFR et IL-6
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
EP2342340A1 (fr) 2008-09-22 2011-07-13 Rxi Pharmaceuticals Corporation Emploi d'arni dans des applications dermatologiques
EP3133160B1 (fr) 2008-10-24 2018-12-12 Sarepta Therapeutics, Inc. Compositions de saut d'exon pour 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
KR101602431B1 (ko) 2008-11-05 2016-03-10 삼성전자 주식회사 냉장고
BRPI0923225A2 (pt) 2008-12-02 2016-10-04 Chiralgen Ltd metodo para sintese de acidos nucleicos modificados no atomo de fosforo
WO2010093788A2 (fr) 2009-02-11 2010-08-19 Dicerna Pharmaceuticals, Inc. Molécules d'arn interférence substrats de dicer multiplexes ayant des séquences de jonction
US8481698B2 (en) 2009-03-19 2013-07-09 The President And Fellows Of Harvard College Parallel proximity ligation event analysis
WO2010115033A2 (fr) 2009-04-02 2010-10-07 Regents Of The University Of Minnesota Polypeptides associés à des expansions de répétitions nucléotidiques et leurs utilisations
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 (fr) 2009-05-05 2010-11-11 Altermune Technologies, Llc Immunité chimiquement programmable
EP2437785B1 (fr) 2009-06-04 2015-02-25 Novartis AG Procédés d'identification de sites pour la conjugaison d'igg
BRPI1015234A2 (en) 2009-06-22 2018-02-20 Medimmune Llc fc regions designed for site specific 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
EP2456470A1 (fr) 2009-07-22 2012-05-30 Cenix Bioscience GmbH Système d'administration et conjugués pour l'administration de composés par des voies de transport intracellulaire naturelles
EP2473607A2 (fr) 2009-08-31 2012-07-11 INSERM - Institut National de la Santé et de la Recherche Médicale Thérapie par saut d'exon pour l'amélioration fonctionnelle de la dystrophine semi-fonctionnelle dans la dystrophie musculaire de duchenne et becker
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
MX368648B (es) 2010-04-15 2019-10-09 Seattle Genetics Inc Pirrolobenzodiazepinas usadas para tratar enfermedades proliferativas.
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
WO2011139702A2 (fr) 2010-04-28 2011-11-10 Isis Pharmaceuticals, Inc. Nucléosides modifiés et composés oligomères préparés à partir de ceux-ci
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
ES2756326T3 (es) 2010-07-19 2020-04-27 Ionis Pharmaceuticals Inc Modulación de la expresión de distrofia miotónica-proteína quinasa (DMPK)
WO2012058210A1 (fr) 2010-10-29 2012-05-03 Merck Sharp & Dohme Corp. INHIBITION FACILITÉE PAR L'INTERFÉRENCE D'ARN DE L'EXPRESSION D'UN GÈNE AU MOYEN D'ACIDES NUCLÉIQUES INTERFÉRENTS COURTS (siNA)
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
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
CA2839593A1 (fr) 2011-07-15 2013-01-24 Sarepta Therapeutics, Inc. Procedes et compositions pour manipulation de la traduction d'isoformes de proteines a partir de sites initiateurs alternatifs d'initiation
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
US9418216B2 (en) 2011-07-21 2016-08-16 Microsoft Technology Licensing, Llc Cloud service authentication
US10023861B2 (en) 2011-08-29 2018-07-17 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
CA2850373C (fr) 2011-10-14 2019-07-16 Seattle Genetics, Inc. Pyrrolobenzodiazepines et conjugues cibles
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
GB201121069D0 (en) * 2011-12-07 2012-01-18 Isis Innovation Delivery system
EP3539982B1 (fr) 2011-12-23 2025-02-19 Pfizer Inc. Régions constantes d'anticorps modifiés pour conjugaison spécifique à un site, et leurs procédés et utilisations
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
US9610362B2 (en) * 2012-03-16 2017-04-04 Valerion Therapeutics, Llc Antisense conjugates for decreasing expression of DMPK
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
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
US9504756B2 (en) 2012-05-15 2016-11-29 Seattle Genetics, Inc. Self-stabilizing linker conjugates
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
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
DK2839860T3 (da) 2012-10-12 2019-06-17 Medimmune Ltd Pyrrolobenzodiazepiner og konjugater deraf
CA2889044A1 (fr) 2012-11-15 2014-05-22 Roche Innovation Center Copenhagen A/S Composes conjugues antisens anti-apob
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
US9319764B2 (en) 2013-03-08 2016-04-19 Merry Electronics Co., Ltd. MEMS microphone packaging structure
US9217148B2 (en) 2013-03-14 2015-12-22 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
EP3964237B1 (fr) 2013-03-15 2026-02-18 Regeneron Pharmaceuticals, Inc. Molécules biologiquement actives, leurs conjugués, et utilisations thérapeutiques
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
EP2777714A1 (fr) 2013-03-15 2014-09-17 NBE-Therapeutics LLC Procédé de production d'un conjugué de ligand immunologique/charge utile à l'aide d'une enzyme transpeptidase spécifique pour une séquence
US9577459B2 (en) 2013-03-15 2017-02-21 Sequana Medical Ag Systems and methods for regulating inductive energy transfer to an implantable system
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 (fr) 2013-04-09 2014-10-16 Duke University Arn 2'fluoro-modifiés en tant qu'immunostimulateurs
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 (fr) 2013-05-31 2019-04-17 Zymeworks Inc. Hétéromultimères à fonction effectrice réduite ou silencieuse
US11229711B2 (en) 2013-06-06 2022-01-25 Magenta Therapeutics, Inc. Linkers for antibody-drug conjugates and related compounds, 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 (fr) 2013-09-09 2015-03-11 University of Vienna Oligonucléotides antisens ayant des propriétés pharmacocinétiques améliorées
WO2015038426A1 (fr) 2013-09-13 2015-03-19 Asana Biosciences, Llc Lieurs auto-immolables contenant des dérivés d'acide mandélique, conjugués médicament-ligand pour thérapies ciblées, et leurs utilisations
AU2014337317A1 (en) 2013-10-15 2016-09-15 Sorrento Therapeutics Inc. Drug-conjugates with a targeting molecule and two different drugs
PT3057585T (pt) 2013-10-15 2020-10-21 Seagen Inc Fármaco-ligadores peguilados para farmacocinética melhorada de conjugados de fármaco-ligando
WO2015069587A2 (fr) 2013-11-06 2015-05-14 Merck Sharp & Dohme Corp. Conjugués contenant des peptides pour double distribution moléculaire d'oligonucléotides
US10378017B2 (en) 2013-12-02 2019-08-13 Brandeis University High temperature selection of nucleotide-supported carbohydrate vaccines and resulting glycosylated oligonucleotides
EP3094728B1 (fr) 2014-01-16 2022-03-09 Wave Life Sciences Ltd. Conception chirale
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
EP3286318A2 (fr) 2015-04-22 2018-02-28 Mina Therapeutics Limited Compositions de petits arn activeurs arnsa et méthodes d'utilisation
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
WO2017148880A1 (fr) 2016-03-01 2017-09-08 F. Hoffmann-La Roche Ag Variantes de l'obinutuzumab dont l'induction de l'apoptose est modifiée
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
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
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
WO2018098328A1 (fr) 2016-11-23 2018-05-31 Alnylam Pharmaceuticals, Inc. Agents arn modifiés à effet hors cible réduit
KR20240172236A (ko) 2017-01-06 2024-12-09 어비디티 바이오사이언시스 인크. 핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법
IT201700008383A1 (it) 2017-01-26 2018-07-26 Explea S R L Dispositivo per la produzione di energia elettrica utilizzante l’azione combinata di campi magnetici pulsati e di correnti fluidodinamiche agenti su una lamina flessibile dotata di un elemento piezoelettrico ed impianto formato da più elementi modulari collegati elettricamente fra loro e composti ciascuno da una pluralità di tali dispositivi
WO2018204872A2 (fr) 2017-05-05 2018-11-08 Fusion Pharmaceuticals Inc. Anticorps monoclonaux anti-igf-1r et utilisations de ceux-ci
SG11202002517RA (en) * 2017-09-22 2020-04-29 Avidity Biosciences Inc Nucleic acid-polypeptide compositions and methods of inducing exon skipping
SG11202003126VA (en) * 2017-10-04 2020-05-28 Avidity Biosciences Inc Nucleic acid-polypeptide compositions and uses thereof
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
AU2019315593B2 (en) 2018-08-02 2026-05-21 Dyne Therapeutics, Inc. Muscle-targeting complexes and uses thereof
CN112955154A (zh) 2018-08-02 2021-06-11 达因疗法公司 肌肉靶向复合物及其用于治疗强直性肌营养不良的用途
KR102927945B1 (ko) 2020-03-27 2026-02-23 어비디티 바이오사이언시스 인크. 근이영양증의 치료용 조성물 및 방법

Also Published As

Publication number Publication date
IL274906B2 (en) 2025-09-01
AU2018378812A1 (en) 2020-07-09
US11583591B2 (en) 2023-02-21
US20200405877A1 (en) 2020-12-31
US20220008550A1 (en) 2022-01-13
US20250195676A1 (en) 2025-06-19
AU2025220738A1 (en) 2025-09-11
KR20230059845A (ko) 2023-05-03
KR20240007965A (ko) 2024-01-17
US20220096647A1 (en) 2022-03-31
US20220040322A1 (en) 2022-02-10
EP3720448A1 (fr) 2020-10-14
KR20200097751A (ko) 2020-08-19
US11872287B2 (en) 2024-01-16
JP7506601B2 (ja) 2024-06-26
CN118667812A (zh) 2024-09-20
US11554176B2 (en) 2023-01-17
KR20220129670A (ko) 2022-09-23
US11246941B2 (en) 2022-02-15
KR20250148679A (ko) 2025-10-14
US20220062434A1 (en) 2022-03-03
US20190298847A1 (en) 2019-10-03
CN111902148A (zh) 2020-11-06
KR102623786B1 (ko) 2024-01-11
US20260007766A1 (en) 2026-01-08
MX2025014047A (es) 2026-01-07
US20230201363A1 (en) 2023-06-29
JP7564852B2 (ja) 2024-10-09
EP3720448A4 (fr) 2021-11-03
US20220133900A1 (en) 2022-05-05
KR102867099B1 (ko) 2025-10-01
US11712478B2 (en) 2023-08-01
JP2021507686A (ja) 2021-02-25
US20210187116A1 (en) 2021-06-24
KR102443358B1 (ko) 2022-09-14
US20220040323A1 (en) 2022-02-10
IL274906A (en) 2020-07-30
US11497814B2 (en) 2022-11-15
US11576980B2 (en) 2023-02-14
SG11202005022YA (en) 2020-06-29
US10881743B2 (en) 2021-01-05
US20230047754A1 (en) 2023-02-16
US11253607B2 (en) 2022-02-22
CN118638788A (zh) 2024-09-13
MX2020005860A (es) 2020-09-09
AU2018378812B2 (en) 2025-05-22
JP2025000818A (ja) 2025-01-07
US12263224B2 (en) 2025-04-01
WO2019113393A1 (fr) 2019-06-13
CN111902148B (zh) 2024-07-05
US20230201362A1 (en) 2023-06-29
IL319835A (en) 2025-05-01
CN118638787A (zh) 2024-09-13
IL274906B1 (en) 2025-05-01
CA3083526A1 (fr) 2019-06-13
JP2023011614A (ja) 2023-01-24
MX2024012513A (es) 2024-11-08
KR102527941B1 (ko) 2023-05-02

Similar Documents

Publication Publication Date Title
EP3720448A4 (fr) Compositions et procédés de traitement de l'atrophie musculaire et de la dystrophie myotonique
EP3496739A4 (fr) Compositions et procédés de traitement de l'hypertension pulmonaire
EP3612215A4 (fr) Compositions et procédés pour le traitement d'inflammation pulmonaire
EP3781214A4 (fr) Compositions et procédés de traitement de l'amyotrophie spinale
MA51306A (fr) Compositions et procédés améliorés pour le traitement du déficit en ornithine transcarbamylase
EP3474879A4 (fr) Compositions et procédés de traitement d'un mélanome
EP3704239A4 (fr) Compositions de casz et procédés d'utilisation
EP3390642A4 (fr) Compositions et procédés pour le traitement de rétinite pigmentaire 18 et de rétinite pigmentaire 13
EP3833340A4 (fr) Compositions et procédés de traitement de la presbytie
EP3414321A4 (fr) Compositions améliorant vcn et procédés d'utilisation desdites compositions
EP3645739A4 (fr) Procédés et compositions pour le traitement du mélanome
EP3377042A4 (fr) Substances et procédés de traitement de myopathies à base de titine et d'autres titinopathies
MA53873A (fr) Compositions et procédés pour le traitement de la presbytie
ME03380B (fr) Compositions comprenant des souches bactériennes
MA45270A (fr) Compositions d'oligonucléotides et procédés associés
EP3625229C0 (fr) Formes solides de berbérine ursodésoxycholate, compositions et procédés associés
EP3341391A4 (fr) Compositions et procédés pour le traitement de la douleur
EP3735325A4 (fr) Compositions et procédés de traitement de surface
EP4699658A3 (fr) Méthodes et compositions pour le traitement de la dystrophie musculaire squelettique
MA52199A (fr) Compositions et procédés de traitement de la dystrophie maculaire
EP3684378A4 (fr) Gapmères et procédés d'utilisation de ces derniers pour le traitement de la dystrophie musculaire
EP3448421A4 (fr) Procédés et compositions pour la prévention et le traitement d'adhérences chirurgicales
EP3289064A4 (fr) Compositions et procédés pour la détection d'allergènes
EP4017924A4 (fr) Compositions et procédés de traitement de surface
EP3830196A4 (fr) Compositions et procédés de traitement de surface