PT3442602T - Entrega de micro-distrofina por vetor de vírus adenoassociado para tratar distrofia muscular - Google Patents

Entrega de micro-distrofina por vetor de vírus adenoassociado para tratar distrofia muscular

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Publication number
PT3442602T
PT3442602T PT177832367T PT17783236T PT3442602T PT 3442602 T PT3442602 T PT 3442602T PT 177832367 T PT177832367 T PT 177832367T PT 17783236 T PT17783236 T PT 17783236T PT 3442602 T PT3442602 T PT 3442602T
Authority
PT
Portugal
Prior art keywords
dystrophin
adeno
micro
associated virus
muscular dystrophy
Prior art date
Application number
PT177832367T
Other languages
English (en)
Portuguese (pt)
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
Publication of PT3442602T publication Critical patent/PT3442602T/pt

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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
    • 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
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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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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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
    • C—CHEMISTRY; METALLURGY
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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
    • C12N15/65—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression using markers
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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
    • C—CHEMISTRY; METALLURGY
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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
    • 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
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    • 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
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
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    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors
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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
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    • 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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    • 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

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  • Health & Medical Sciences (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Immunology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Toxicology (AREA)
  • Dermatology (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
PT177832367T 2016-04-15 2017-04-14 Entrega de micro-distrofina por vetor de vírus adenoassociado para tratar distrofia muscular PT3442602T (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662323163P 2016-04-15 2016-04-15
US201762473253P 2017-03-17 2017-03-17

Publications (1)

Publication Number Publication Date
PT3442602T true PT3442602T (pt) 2025-05-07

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PT177832367T PT3442602T (pt) 2016-04-15 2017-04-14 Entrega de micro-distrofina por vetor de vírus adenoassociado para tratar distrofia muscular

Country Status (28)

Country Link
US (6) US11298429B2 (cg-RX-API-DMAC7.html)
EP (5) EP4410979A3 (cg-RX-API-DMAC7.html)
JP (6) JP7189771B2 (cg-RX-API-DMAC7.html)
KR (3) KR20250061772A (cg-RX-API-DMAC7.html)
CN (2) CN109069672B (cg-RX-API-DMAC7.html)
AU (4) AU2017250791B2 (cg-RX-API-DMAC7.html)
BR (1) BR112018071182A2 (cg-RX-API-DMAC7.html)
CA (2) CA3020999A1 (cg-RX-API-DMAC7.html)
CO (1) CO2018012084A2 (cg-RX-API-DMAC7.html)
DK (1) DK3442602T3 (cg-RX-API-DMAC7.html)
ES (3) ES2977730T3 (cg-RX-API-DMAC7.html)
FI (1) FI3442602T3 (cg-RX-API-DMAC7.html)
HR (1) HRP20250541T1 (cg-RX-API-DMAC7.html)
HU (1) HUE071243T2 (cg-RX-API-DMAC7.html)
IL (3) IL319636A (cg-RX-API-DMAC7.html)
LT (1) LT3442602T (cg-RX-API-DMAC7.html)
MA (2) MA44683B1 (cg-RX-API-DMAC7.html)
MD (1) MD3442602T2 (cg-RX-API-DMAC7.html)
MX (2) MX2018012604A (cg-RX-API-DMAC7.html)
MY (2) MY195438A (cg-RX-API-DMAC7.html)
NZ (2) NZ747049A (cg-RX-API-DMAC7.html)
PL (1) PL3442602T3 (cg-RX-API-DMAC7.html)
PT (1) PT3442602T (cg-RX-API-DMAC7.html)
RS (1) RS66847B1 (cg-RX-API-DMAC7.html)
SG (1) SG11201808941PA (cg-RX-API-DMAC7.html)
SI (1) SI3442602T1 (cg-RX-API-DMAC7.html)
WO (3) WO2017181011A1 (cg-RX-API-DMAC7.html)
ZA (2) ZA201806746B (cg-RX-API-DMAC7.html)

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EP3377042A4 (en) 2015-11-16 2019-05-29 The Research Institute at Nationwide Children's Hospital MATERIALS AND METHOD FOR THE TREATMENT OF TITIN-BASED MYOPATHIES AND OTHER TITINOPATHIES
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
ES2948233T3 (es) * 2017-03-17 2023-09-06 Res Inst Nationwide Childrens Hospital Administración mediante vector de virus adenoasociado de microdistrofina específica de músculo para tratar la distrofia muscular
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
MA50836A (fr) 2017-10-18 2020-08-26 Res Inst Nationwide Childrens Hospital Administration par vecteur à virus adéno-associé de micro-dystrophine spécifique de muscles pour traiter la dystrophie musculaire
MY207563A (en) * 2017-10-20 2025-03-04 Res Inst Nationwide Childrens Hospital Methods and materials for nt-3 gene therapy
TWI815856B (zh) 2018-01-31 2023-09-21 美國全美兒童醫院之研究學會 用於肢帶型肌肉失養症2c型之基因療法
IL277270B2 (en) 2018-03-16 2025-05-01 Res Inst Nationwide Childrens Hospital Increasing tissue-specific gene delivery through capsid modification
US20210244828A1 (en) * 2018-05-15 2021-08-12 Ramot At Tel-Aviv University Ltd. Miri26-5p for treating motor neuron diseases
PT3807413T (pt) * 2018-06-18 2025-08-19 Res Inst Nationwide Childrens Hospital Administração por vetor de vírus adenoassociado de microdistrofina específica do músculo para tratar a distrofia muscular
CA3104471A1 (en) * 2018-06-18 2019-12-26 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating dystroglycanopathies and laminin-deficient muscular dystrophies
CN112543810A (zh) 2018-06-29 2021-03-23 全国儿童医院研究所 治疗肢带型肌营养不良2a型的重组腺相关病毒产品和方法
EP3598978B1 (en) 2018-07-26 2024-05-29 EXOFIX S.r.l. Fibroadipogenic progenitor-derived exosomes for regeneration of dystrophic muscles
CN113646004A (zh) * 2018-12-12 2021-11-12 坚固生物科技公司 用于治疗肌营养不良的组合疗法
BR112021016570A2 (pt) 2019-02-26 2021-11-03 Res Inst Nationwide Childrens Hospital Entrega de vetor de vírus adenoassociado de beta-sarcoglicano e tratamento de distrofia muscular
WO2020193636A1 (en) * 2019-03-25 2020-10-01 Genethon Production of large-sized quasidystrophins using overlapping aav vectors
EP4374880A3 (en) 2019-08-21 2024-08-14 Research Institute at Nationwide Children's Hospital Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy
WO2021142435A1 (en) * 2020-01-10 2021-07-15 Solid Biosciences Inc. Combination therapy for treating muscular dystrophy
WO2021142447A1 (en) * 2020-01-10 2021-07-15 Solid Biosciences Inc. Viral vector for combination therapy
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