JP7788855B2 - 筋ジストロフィーの治療のための併用療法 - Google Patents

筋ジストロフィーの治療のための併用療法

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JP7788855B2
JP7788855B2 JP2021533367A JP2021533367A JP7788855B2 JP 7788855 B2 JP7788855 B2 JP 7788855B2 JP 2021533367 A JP2021533367 A JP 2021533367A JP 2021533367 A JP2021533367 A JP 2021533367A JP 7788855 B2 JP7788855 B2 JP 7788855B2
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sequence
mir
recombinant aav
vector
gene
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JPWO2020123645A5 (https=
JP2022513456A5 (https=
JP2022513456A (ja
Inventor
ラム，センディル
シュナイダー，ジョエル
モーガン，キャシー・イェ
ツェン，ウェン・アレン
オズソロック，ファティ
ソウステック－クレーマ，メーガン
レイズ，エリック
マンダーバ，サラット
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ソリッド・バイオサイエンシーズ・インコーポレーテッド
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    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/39—Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin, cold insoluble globulin [CIG]
    • 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
    • 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
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions
    • 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
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707—Muscular dystrophy
    • C07K14/4708—Duchenne dystrophy
    • 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
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    • 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
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    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/122—Hairpin
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/141—MicroRNAs, miRNAs
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/51—Physical structure in polymeric form, e.g. multimers, concatemers
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • C—CHEMISTRY; METALLURGY
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Health & Medical Sciences (AREA)
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  • Public Health (AREA)
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  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
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  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Manufacturing & Machinery (AREA)
  • Immunology (AREA)
  • Toxicology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2021533367A 2018-12-12 2019-12-11 筋ジストロフィーの治療のための併用療法 Active JP7788855B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862778646P 2018-12-12 2018-12-12
US62/778,646 2018-12-12
PCT/US2019/065718 WO2020123645A1 (en) 2018-12-12 2019-12-11 Combination therapy for treating muscular dystrophy

Publications (4)

Publication Number Publication Date
JP2022513456A JP2022513456A (ja) 2022-02-08
JPWO2020123645A5 JPWO2020123645A5 (https=) 2022-12-20
JP2022513456A5 JP2022513456A5 (https=) 2022-12-20
JP7788855B2 true JP7788855B2 (ja) 2025-12-19

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JP2021533367A Active JP7788855B2 (ja) 2018-12-12 2019-12-11 筋ジストロフィーの治療のための併用療法

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US (1) US20220031865A1 (https=)
EP (1) EP3893940A4 (https=)
JP (1) JP7788855B2 (https=)
KR (1) KR20210124969A (https=)
CN (1) CN113646004A (https=)
AU (1) AU2019395388A1 (https=)
CA (1) CA3123003A1 (https=)
SG (1) SG11202105873SA (https=)
WO (1) WO2020123645A1 (https=)

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WO2019012336A2 (en) 2017-03-17 2019-01-17 Newcastle University ADENO-ASSOCIATED VIRAL VECTOR DELIVERY OF A MICRO-DYSTROPHIN FRAGMENT FOR TREATING MUSCLE DYSTROPHY
MX2020007876A (es) 2018-01-31 2020-12-03 Res Inst Nationwide Childrens Hospital Terapia genica para la distrofia muscular de cinturas tipo 2c.
CN120393053A (zh) 2018-06-18 2025-08-01 全国儿童医院研究所 用于治疗肌营养不良的肌肉特异性微小肌营养不良蛋白的腺相关病毒载体递送
MX2020014119A (es) 2018-06-29 2021-06-18 Res Inst Nationwide Childrens Hospital Productos y métodos de virus adenoasociado recombinante para el tratamiento de la distrofia muscular de cinturas 2a.
MX2021010356A (es) 2019-02-26 2022-03-04 Res Inst Nationwide Childrens Hospital SUMINISTRO DE ß-SARCOGLICANO MEDIANTE VECTORES DE VIRUS ADENOASOCIADO Y EL TRATAMIENTO DE LA DISTROFIA MUSCULAR.
PT4017871T (pt) 2019-08-21 2024-04-22 Res Inst Nationwide Childrens Hospital Distribuição de vetor de vírus adeno-associado de alfasarcoglicano e o tratamento da distrofia muscular
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EP4108263A3 (en) 2021-06-02 2023-03-22 Research Institute at Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a
US20240390522A1 (en) * 2021-09-28 2024-11-28 University Of Florida Research Foundation, Incorporated Gene therapy for duchenne muscular dystrophy
JP2023059858A (ja) 2021-10-15 2023-04-27 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 自己相補的アデノ随伴ウイルスベクター及び筋ジストロフィーの治療におけるその使用
AR129014A1 (es) * 2022-04-05 2024-07-03 Astellas Gene Therapies Inc Composiciones y métodos para el tratamiento de distrofias musculares
WO2023225592A2 (en) * 2022-05-18 2023-11-23 Inadcure Foundation Inc. Gene therapies for treatment of infantile neuroaxonal dystrophy
IL320023A (en) * 2022-10-06 2025-06-01 Solid Biosciences Inc Dual cell-to-cell gene transfer vector
CN117959464A (zh) * 2022-10-27 2024-05-03 苏州新芽基因生物技术有限公司 用于疾病治疗的基因编辑器和抗纤维化抑制剂的核酸药物组合物
CN116218849B (zh) * 2023-01-18 2024-01-23 昆明理工大学 针对utrophin激活治疗杜氏肌营养不良的sgRNA和应用
WO2024220592A2 (en) * 2023-04-18 2024-10-24 Research Institute At Nationwide Children's Hospital, Inc. Gene therapy for treating limb girdle muscular dystrophy r9 and congenital muscular dystrophy 1c
WO2024229164A2 (en) * 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
CN116926125B (zh) * 2023-09-07 2024-06-11 昆明理工大学 一种抑制炎症与基因编辑同时进行的基因载体
WO2025085731A1 (en) * 2023-10-18 2025-04-24 Children's National Medical Center TRANSFORMING GROWTH FACTOR ß (TGF-ß) OLIGONUCLEOTIDE COMBINATION THERAPY FOR TREATING MUSCULOSKELETAL DISEASES

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