DK3442602T3 - Adeno-associeret virusvektors frigørelse af mikrodystrofin til behandling af muskeldystrofi - Google Patents

Adeno-associeret virusvektors frigørelse af mikrodystrofin til behandling af muskeldystrofi Download PDF

Info

Publication number
DK3442602T3
DK3442602T3 DK17783236.7T DK17783236T DK3442602T3 DK 3442602 T3 DK3442602 T3 DK 3442602T3 DK 17783236 T DK17783236 T DK 17783236T DK 3442602 T3 DK3442602 T3 DK 3442602T3
Authority
DK
Denmark
Prior art keywords
microdystrophin
adeno
treatment
associated virus
muscular dystrophy
Prior art date
Application number
DK17783236.7T
Other languages
Danish (da)
English (en)
Inventor
Louise Rodino-Klapac
Jerry R Mendell
Kristin N Heller
Original Assignee
Res Inst Nationwide Childrens Hospital
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 Res Inst Nationwide Childrens Hospital filed Critical Res Inst Nationwide Childrens Hospital
Application granted granted Critical
Publication of DK3442602T3 publication Critical patent/DK3442602T3/da

Links

Classifications

    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • 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
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A61K38/1719—Muscle proteins, e.g. myosin or actin
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/04—Drugs for skeletal disorders for non-specific disorders of the connective tissue
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/65—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression using markers
    • 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
    • C12N15/63—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
    • 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
    • C12N15/63—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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • 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]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Immunology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Toxicology (AREA)
  • Dermatology (AREA)
  • Mycology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
DK17783236.7T 2016-04-15 2017-04-14 Adeno-associeret virusvektors frigørelse af mikrodystrofin til behandling af muskeldystrofi DK3442602T3 (da)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662323163P 2016-04-15 2016-04-15
US201762473253P 2017-03-17 2017-03-17
PCT/US2017/027636 WO2017181015A1 (en) 2016-04-15 2017-04-14 Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy

Publications (1)

Publication Number Publication Date
DK3442602T3 true DK3442602T3 (da) 2025-05-19

Family

ID=60041940

Family Applications (1)

Application Number Title Priority Date Filing Date
DK17783236.7T DK3442602T3 (da) 2016-04-15 2017-04-14 Adeno-associeret virusvektors frigørelse af mikrodystrofin til behandling af muskeldystrofi

Country Status (28)

Country Link
US (6) US11406717B2 (OSRAM)
EP (5) EP3442602B1 (OSRAM)
JP (6) JP7153562B2 (OSRAM)
KR (3) KR20250061772A (OSRAM)
CN (2) CN109069672B (OSRAM)
AU (4) AU2017250791B2 (OSRAM)
BR (1) BR112018071182A2 (OSRAM)
CA (2) CA3020999A1 (OSRAM)
CO (1) CO2018012084A2 (OSRAM)
DK (1) DK3442602T3 (OSRAM)
ES (3) ES3026989T3 (OSRAM)
FI (1) FI3442602T3 (OSRAM)
HR (1) HRP20250541T1 (OSRAM)
HU (1) HUE071243T2 (OSRAM)
IL (3) IL262194B2 (OSRAM)
LT (1) LT3442602T (OSRAM)
MA (2) MA44683B1 (OSRAM)
MD (1) MD3442602T2 (OSRAM)
MX (2) MX2018012604A (OSRAM)
MY (2) MY209041A (OSRAM)
NZ (2) NZ787316A (OSRAM)
PL (1) PL3442602T3 (OSRAM)
PT (1) PT3442602T (OSRAM)
RS (1) RS66847B1 (OSRAM)
SG (1) SG11201808941PA (OSRAM)
SI (1) SI3442602T1 (OSRAM)
WO (3) WO2017181015A1 (OSRAM)
ZA (2) ZA201806746B (OSRAM)

Families Citing this family (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HK1257955A1 (zh) 2015-11-16 2019-11-01 The Research Institute At Nationwide Children's Hospital 用於治疗肌联蛋白类肌病和其他肌联蛋白病变的材料和方法
MA45477A (fr) 2016-04-15 2019-02-20 Res Inst Nationwide Childrens Hospital Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire
IL311713B2 (en) * 2017-03-17 2025-10-01 Res Inst Nationwide Childrens Hospital Muscle-specific delivery of microdystrophin using an adeno-associated virus vector for the treatment of muscular dystrophy
JP7162021B2 (ja) 2017-03-17 2022-10-27 ニューカッスル ユニバーシティ 筋ジストロフィーを治療するためのマイクロジストロフィン断片のアデノ随伴ウイルスベクター送達
WO2019078916A1 (en) * 2017-10-18 2019-04-25 Research Institute At Nationwide Children's Hospital ADENO-ASSOCIATED VIRUS VECTOR ADMINISTRATION OF MUSCLE-SPECIFIC MICRO-DYSTROPHINE TO TREAT MUSCLE DYSTROPHY
AU2018351528B2 (en) * 2017-10-20 2025-02-27 Research Institute At Nationwide Children's Hospital Methods and materials for NT-3 gene therapy
JP7361701B2 (ja) 2018-01-31 2023-10-16 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 肢帯型筋ジストロフィー２ｃ型のための遺伝子治療
BR112020018354A2 (pt) * 2018-03-16 2020-12-29 Research Institute At Nationwide Children's Hospital Aumento de fornecimento gênico específico a tecido através de modificação de capsídeo
WO2019220435A1 (en) * 2018-05-15 2019-11-21 Ramot At Tel-Aviv University Ltd. Mir126-5p for treating motor neuron diseases
JP7646362B2 (ja) 2018-06-18 2025-03-17 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 筋ジストロフィーを治療するための筋肉特異的マイクロジストロフィンのアデノ随伴ウイルスベクターデリバリー
JP7534961B2 (ja) * 2018-06-18 2024-08-15 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 ジストログリカノパチーおよびラミニン欠損筋ジストロフィーを治療するための組換えアデノ随伴ウイルス生成物および方法
WO2020006458A1 (en) 2018-06-29 2020-01-02 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a
EP3598978B1 (en) 2018-07-26 2024-05-29 EXOFIX S.r.l. Fibroadipogenic progenitor-derived exosomes for regeneration of dystrophic muscles
JP7788855B2 (ja) * 2018-12-12 2025-12-19 ソリッド・バイオサイエンシーズ・インコーポレーテッド 筋ジストロフィーの治療のための併用療法
EP3930765A1 (en) 2019-02-26 2022-01-05 Research Institute at Nationwide Children's Hospital Adeno-associated virus vector delivery of b-sarcoglycan and the treatment of muscular dystrophy
CN113677697B (zh) * 2019-03-25 2024-06-04 吉尼松公司 使用重叠的aav载体生产大型拟抗肌萎缩蛋白
SI4017871T1 (sl) 2019-08-21 2024-06-28 Research Institute At Nationwide Children's Hospital Dostava alfa-sarkoglikana z adeno povezanim virusnim vektorjem in zdravljenje mišične distrofije
WO2021142435A1 (en) * 2020-01-10 2021-07-15 Solid Biosciences Inc. Combination therapy for treating muscular dystrophy
EP4087617A4 (en) * 2020-01-10 2024-05-01 Solid Biosciences Inc. VIRAL VECTOR FOR COMBINATION THERAPY
IL297753A (en) 2020-04-29 2022-12-01 Bristol Myers Squibb Co Minimized dystrophins with spectrin fusion domains and uses thereof
WO2021257595A1 (en) * 2020-06-15 2021-12-23 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery for muscular dystrophies
CN111733185A (zh) * 2020-06-30 2020-10-02 华南农业大学 通过Mdfi基因编辑来实现动物肉品质改良的方法和应用
EP3936616A1 (en) * 2020-07-10 2022-01-12 Charité - Universitätsmedizin Berlin Microrna-targeted therapy for cardiac repair
EP4384628A2 (en) 2021-08-11 2024-06-19 Solid Biosciences Inc. Treatment of muscular dystrophy
EP4401794A4 (en) * 2021-09-16 2025-09-17 Univ Florida AAV particles with modified inverted terminal repeats for enhanced gene expression in muscle
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
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
CN115819546B (zh) * 2022-08-19 2025-12-05 成都金唯科生物科技有限公司 表达微缩抗肌营养不良蛋白基因的腺相关病毒载体及其应用
EP4577663A2 (en) 2022-08-24 2025-07-02 RegenxBio Inc. Recombinant adeno-associated viruses and uses thereof
WO2025188755A1 (en) * 2024-03-04 2025-09-12 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of duchenne muscular dystrophy

Family Cites Families (54)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
DE69433922T2 (de) 1993-11-09 2005-07-28 Medical College Of Ohio, Toledo Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren
DK0733103T3 (da) 1993-11-09 2004-07-12 Univ Johns Hopkins Fremstilling af höje titre af rekombinante AAV vektorer
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
CA2207927A1 (en) 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
FR2737730B1 (fr) 1995-08-10 1997-09-05 Pasteur Merieux Serums Vacc Procede de purification de virus par chromatographie
WO1997008298A1 (en) 1995-08-30 1997-03-06 Genzyme Corporation Chromatographic purification of adenovirus and aav
CA2230758A1 (en) 1995-09-08 1997-03-13 Genzyme Corporation Improved aav vectors for gene therapy
US5795872A (en) 1995-09-19 1998-08-18 Pharmadigm, Inc. DNA construct for immunization
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
CA2264483C (en) 1996-09-06 2011-03-22 James M. Wilson Method for recombinant adeno-associated virus-directed gene therapy
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
EP1944362B1 (en) 1997-09-05 2015-10-21 Genzyme Corporation Methods for generating high titer helper-free preparations of recombinant AAV vectors
JPH11318467A (ja) 1998-05-08 1999-11-24 Japan Science & Technology Corp 短縮型ジストロフィン
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
JP2004514407A (ja) 2000-04-28 2004-05-20 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 異種キャプシド中にシュードタイピングされたａａｖ５キャプシドおよびａａｖ５ベクターを含む組換えａａｖベクター
CN100422320C (zh) 2000-04-28 2008-10-01 肖啸 编码抗肌萎缩蛋白小基因的dna序列及其使用方法
AU2294102A (en) 2000-07-14 2002-01-30 Univ Michigan Mutant muscle-specific enhancers
ATE400656T1 (de) 2000-10-06 2008-07-15 Univ Michigan Mini-dystrophin nukleinsäure- und peptidsequenzen
US6962815B2 (en) 2001-01-05 2005-11-08 Children's Hopital Inc. AAV2 vectors and methods
EP1453547B1 (en) 2001-12-17 2016-09-21 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor
CA2554818A1 (en) 2004-02-09 2005-08-25 Thomas Jefferson University Diagnosis and treatment of cancers with microrna located in or near cancer-associated chromosomal features
US20080044393A1 (en) 2004-07-16 2008-02-21 White Robert L Retinal dystrophin transgene and methods of use thereof
US20100184209A1 (en) 2006-02-17 2010-07-22 Dharmacon, Inc. Compositions and methods for inhibiting gene silencing by rna interference
CN101711164B (zh) 2007-01-18 2014-06-04 密苏里-哥伦比亚大学 可修复肌纤维膜中的神经元型一氧化氮合酶的合成小/微-抗肌萎缩蛋白基因
ES2621161T3 (es) 2007-07-31 2017-07-03 The Board Of Regents Of The University Of Texas System Familia de micro-ARN que modula la fibrosis y usos de la misma
US8236557B2 (en) * 2008-05-28 2012-08-07 University Of Missouri-Columbia Hybrid-AAV vectors to deliver large gene expression cassette
WO2010071454A1 (en) 2008-12-17 2010-06-24 Auckland Uniservices Limited Adeno-associated viral vectors and uses thereof
EP4488370A2 (en) 2011-07-25 2025-01-08 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expresssion of dux4
WO2013075008A1 (en) * 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy
WO2013078316A1 (en) 2011-11-23 2013-05-30 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
US20150045416A1 (en) * 2012-01-05 2015-02-12 Hadasit Medical Research Services & Development Ltd. Methods and Compositions for Gene Delivery
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
US9624282B2 (en) 2012-11-26 2017-04-18 The Curators Of The University Of Missouri Microdystrophin peptides and methods for treating muscular dystrophy using the same
WO2014172669A1 (en) * 2013-04-20 2014-10-23 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus delivery of exon 2-targeted u7snrna polynucleotide constructs
JP2015092462A (ja) 2013-09-30 2015-05-14 Tdk株式会社 正極及びそれを用いたリチウムイオン二次電池
GB201400598D0 (en) 2014-01-14 2014-03-05 Univ Glasgow Materials and methods for modulation of tendon healing
SG11201605906UA (en) 2014-01-21 2016-08-30 Univ Bruxelles Muscle-specific nucleic acid regulatory elements and methods and use thereof
JP6202701B2 (ja) 2014-03-21 2017-09-27 株式会社日立国際電気 基板処理装置、半導体装置の製造方法及びプログラム
WO2015157306A1 (en) 2014-04-09 2015-10-15 University Of Houston Therapeutic mirnas for treating heart and skeletal muscle diseases
US10119140B2 (en) 2014-04-18 2018-11-06 Board Of Regents Of The Nevada System Of Higher Education On Behalf Of The University Of Nevada, Reno Methods for enhancing or decreasing the levels of MIR124 and MIR29 in subjects with muscular dystrophy
EP2960336A1 (en) 2014-06-27 2015-12-30 Genethon Efficient systemic treatment of dystrophic muscle pathologies
EP3180435A4 (en) * 2014-08-09 2018-01-17 The Research Institute at Nationwide Children's Hospital Methods and materials for activating an internal ribosomal entry site in exon 5 of the dmd gene
JP6197169B2 (ja) 2014-09-29 2017-09-20 東芝メモリ株式会社 半導体装置の製造方法
US10842886B2 (en) * 2014-10-10 2020-11-24 Research Institute At Nationwide Children's Hospital Guided injections for AAV gene transfer to muscle
EP4599851A3 (en) 2015-01-16 2025-12-31 University of Washington NEW MICRO-DYSTROPHINS AND ASSOCIATED METHODS OF USE
GB201507842D0 (en) 2015-05-07 2015-06-17 New Royal Holloway & Bedford Production of large-sized microdystrophins in an AAV-based vector configuration
KR102799163B1 (ko) * 2015-09-17 2025-04-21 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 Galgt2 유전자 치료 방법 및 물질
WO2017165859A1 (en) 2016-03-24 2017-09-28 Research Institute At Nationwide Children's Hospital Modified viral capsid proteins
MA45477A (fr) 2016-04-15 2019-02-20 Res Inst Nationwide Childrens Hospital Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire
JP7079733B2 (ja) 2016-04-15 2022-06-02 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 β－サルコグリカン及びマイクロＲＮＡ－２９のアデノ随伴ウイルスベクター送達ならびに筋ジストロフィーの治療
CA2971303A1 (en) 2016-06-21 2017-12-21 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
JP7646362B2 (ja) 2018-06-18 2025-03-17 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 筋ジストロフィーを治療するための筋肉特異的マイクロジストロフィンのアデノ随伴ウイルスベクターデリバリー

Also Published As

Publication number Publication date
MX2024005644A (es) 2024-05-24
NZ787316A (en) 2025-10-31
KR20240042136A (ko) 2024-04-01
CN109069672B (zh) 2024-06-21
US20190117795A1 (en) 2019-04-25
AU2025208472A1 (en) 2025-08-14
JP7078546B2 (ja) 2022-05-31
JP7189771B2 (ja) 2022-12-14
PT3442602T (pt) 2025-05-07
US20250161490A1 (en) 2025-05-22
EP3442601A4 (en) 2019-11-20
US20190055581A1 (en) 2019-02-21
ES3026989T3 (en) 2025-06-12
JP2019513393A (ja) 2019-05-30
KR20250061772A (ko) 2025-05-08
CO2018012084A2 (es) 2019-02-08
NZ747049A (en) 2025-10-31
EP4541894A3 (en) 2025-06-11
BR112018071182A2 (pt) 2019-04-09
EP3442602A1 (en) 2019-02-20
ES2977730T3 (es) 2024-08-29
CN118895311A (zh) 2024-11-05
US20230173101A1 (en) 2023-06-08
IL262194A (en) 2018-11-29
LT3442602T (lt) 2025-06-10
JP2024023439A (ja) 2024-02-21
JP7431789B2 (ja) 2024-02-15
IL302200A (en) 2023-06-01
AU2017250791A1 (en) 2018-10-25
EP3442602B1 (en) 2025-02-19
ZA201906608B (en) 2025-03-26
MD3442602T2 (ro) 2025-07-31
EP4410979A3 (en) 2024-11-06
EP3442561A1 (en) 2019-02-20
IL302200B2 (en) 2025-08-01
KR102649707B1 (ko) 2024-03-22
HRP20250541T1 (hr) 2025-07-18
EP3442602A4 (en) 2019-11-27
US11406717B2 (en) 2022-08-09
JP2019513389A (ja) 2019-05-30
SG11201808941PA (en) 2018-11-29
EP3442601B1 (en) 2024-07-03
KR20180133486A (ko) 2018-12-14
JP2025118741A (ja) 2025-08-13
KR102801562B1 (ko) 2025-04-30
EP4410979A2 (en) 2024-08-07
JP2019513779A (ja) 2019-05-30
MX2018012604A (es) 2019-07-04
AU2017250791B2 (en) 2022-11-24
AU2017250790B2 (en) 2024-06-20
MA45477A (fr) 2019-02-20
PL3442602T3 (pl) 2025-07-07
WO2017181014A1 (en) 2017-10-19
WO2017181011A1 (en) 2017-10-19
FI3442602T3 (fi) 2025-05-16
IL262194B2 (en) 2023-09-01
SI3442602T1 (sl) 2025-07-31
MY195438A (en) 2023-01-21
ZA201806746B (en) 2023-12-20
WO2017181015A1 (en) 2017-10-19
AU2017250790A1 (en) 2018-11-15
CA3020996A1 (en) 2017-10-19
CN109069672A (zh) 2018-12-21
CA3020999A1 (en) 2017-10-19
MY209041A (en) 2025-06-17
EP3442561A4 (en) 2019-12-18
EP3442561B1 (en) 2024-02-14
JP2022020839A (ja) 2022-02-01
JP7153562B2 (ja) 2022-10-14
US11723986B2 (en) 2023-08-15
US20190060489A1 (en) 2019-02-28
US11298429B2 (en) 2022-04-12
RS66847B1 (sr) 2025-06-30
HUE071243T2 (hu) 2025-08-28
ES2985822T3 (es) 2024-11-07
US20230001015A1 (en) 2023-01-05
AU2023201093A1 (en) 2023-05-18
IL302200B1 (en) 2025-04-01
EP4541894A2 (en) 2025-04-23
MA44683B1 (fr) 2025-07-31
AU2023201093B2 (en) 2025-05-15
MA44683A (fr) 2019-02-20
EP3442601A1 (en) 2019-02-20
IL262194B1 (en) 2023-05-01
IL319636A (en) 2025-05-01

Similar Documents

Publication Publication Date Title
DK3442602T3 (da) Adeno-associeret virusvektors frigørelse af mikrodystrofin til behandling af muskeldystrofi
DK3596222T5 (da) Adenoassocieret virusvektorfremføring af muskelspecifik mikro-dystrophin til behandling af muskeldystrofi
IL280459A (en) Phosphoramidates for the treatment of hepatitis b virus
DK3349758T3 (da) Fremgangsmåder til behandling af arenaviridae-virusinfektioner
IL266264A (en) Adeno-associated virus formulations
DK3442600T5 (da) ADENOASSOCIERET VIRUSVEKTORFREMFØRING AF ß-SARCOGLYCAN OG MIKRORNA-29 OG BEHANDLING AF MUSKELDYSTROFI
DK3807413T3 (da) Adenoassocieret virusvektorfremføring af muskelspecifik mikrodystrofin til behandling af muskeldystrofi
DK3108000T3 (da) Adeno-associeret virusvektor
DK3400291T3 (da) Manipuleret virus
DK3096741T3 (da) Fremgangsmåde til fremstillingen af hybridosomer
DK3245206T3 (da) Pyrazinforbindelser til behandling af infektionssygdomme
DK3439635T3 (da) Formuleringer til behandling af fast tumor
DK3492632T3 (da) Metoder til behandling af lithiumholdige materialer
DK3556775T3 (da) Anti-lag-3 antistoffer til behandling af hæmatologiske maligniteter
DK3143025T3 (da) Forbindelser til behandling af spinal muskelatrofi
DK3125875T3 (da) Inhalerbar rapamycinformulering til behandling af aldersrelaterede tilstande
IL264143B (en) Processes for preparing olaparib
DK3139925T3 (da) Valbenazin-doseringssystem til behandling af hyperkinetiske bevægelsesforstyrrelser
DK3174858T3 (da) Fremgangsmåde til fremstilling af pyrazoler
DK3504187T3 (da) Anvendelse af pridopidin til behandling af funktionel tilbagegang
DK3362066T3 (da) Kombinationsterapi til behandling af maligniteter
DK3503890T3 (da) Anvendelse af pridopidin til behandling af dystonier
DK3436074T3 (da) Thiazolidforbindelser til behandling af virale infektioner
DK3258919T3 (da) Næsepulverformulering til behandling af hypoglykæmi
DK3324824T3 (da) Fremgangsmåde til behandling af binokulare dysfunktioner