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RS64299B1 - Isporuka adeno asociranog virusnog vektora mikrodistrofina specifičnog za mišiće za lečenje mišićne distrofije - Google Patents

Isporuka adeno asociranog virusnog vektora mikrodistrofina specifičnog za mišiće za lečenje mišićne distrofije

Info

Publication number
RS64299B1
RS64299B1 RS20230434A RSP20230434A RS64299B1 RS 64299 B1 RS64299 B1 RS 64299B1 RS 20230434 A RS20230434 A RS 20230434A RS P20230434 A RSP20230434 A RS P20230434A RS 64299 B1 RS64299 B1 RS 64299B1
Authority
RS
Serbia
Prior art keywords
dystrophin
adeno
associated virus
muscular dystrophy
virus vector
Prior art date
Application number
RS20230434A
Other languages
English (en)
Inventor
Louise Rodino-Klapac
Jerry R Mendell
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 RS64299B1 publication Critical patent/RS64299B1/sr

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A61K9/0029Parenteral nutrition; Parenteral nutrition compositions as drug carriers
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707Muscular dystrophy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • A01K2267/0306Animal model for genetic diseases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151Methods of production or purification of viral material
    • C12N2750/14152Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Toxicology (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Nutrition Science (AREA)
  • Dermatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
RS20230434A 2017-03-17 2018-03-16 Isporuka adeno asociranog virusnog vektora mikrodistrofina specifičnog za mišiće za lečenje mišićne distrofije RS64299B1 (sr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762473148P 2017-03-17 2017-03-17
PCT/US2018/022881 WO2018170408A1 (en) 2017-03-17 2018-03-16 Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
EP18768395.8A EP3596222B1 (en) 2017-03-17 2018-03-16 Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy

Publications (1)

Publication Number Publication Date
RS64299B1 true RS64299B1 (sr) 2023-07-31

Family

ID=63523321

Family Applications (1)

Application Number Title Priority Date Filing Date
RS20230434A RS64299B1 (sr) 2017-03-17 2018-03-16 Isporuka adeno asociranog virusnog vektora mikrodistrofina specifičnog za mišiće za lečenje mišićne distrofije

Country Status (30)

Country Link
US (2) US20200199621A1 (sr)
EP (2) EP3596222B1 (sr)
JP (2) JP2020513811A (sr)
KR (2) KR102683682B1 (sr)
CN (2) CN118497275A (sr)
AR (1) AR111292A1 (sr)
AU (1) AU2018233732B2 (sr)
BR (1) BR112019019248A2 (sr)
CA (1) CA3056638A1 (sr)
CO (1) CO2019011250A2 (sr)
CY (1) CY1126080T1 (sr)
DK (1) DK3596222T5 (sr)
EA (1) EA201992201A1 (sr)
ES (1) ES2948233T3 (sr)
FI (1) FI3596222T3 (sr)
HR (1) HRP20230522T1 (sr)
HU (1) HUE062476T2 (sr)
IL (2) IL311713A (sr)
LT (1) LT3596222T (sr)
MA (1) MA52112B1 (sr)
MD (1) MD3596222T2 (sr)
MX (1) MX2019011046A (sr)
PL (1) PL3596222T3 (sr)
PT (1) PT3596222T (sr)
RS (1) RS64299B1 (sr)
SG (1) SG11201908575SA (sr)
SI (1) SI3596222T1 (sr)
TW (1) TW201840850A (sr)
WO (1) WO2018170408A1 (sr)
ZA (1) ZA201906251B (sr)

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CN113710809A (zh) * 2019-02-02 2021-11-26 联邦高等教育系统-匹兹堡大学 通过基因置换和抗炎的针对杜氏肌营养不良的一步基因疗法
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WO2022029543A1 (en) * 2020-08-06 2022-02-10 Intas Pharmaceuticals Ltd. Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy
CN116783293A (zh) * 2020-11-12 2023-09-19 精密生物科学公司 对肌营养不良蛋白基因中的识别序列具有特异性的工程化大范围核酸酶
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WO2023019168A1 (en) * 2021-08-11 2023-02-16 Ultragenyx Pharmaceutical Inc. Compositions and methods for treating a muscular dystrophy
CN114316070B (zh) * 2021-12-29 2022-11-15 上海勉亦生物科技有限公司 用于治疗肌营养不良症的转基因表达盒
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
WO2023202469A1 (zh) * 2022-04-19 2023-10-26 康霖生物科技(杭州)有限公司 一种用于遗传性凝血因子缺乏病治疗的核酸构建体及其用途
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JP2023053254A (ja) 2023-04-12
JP2020513811A (ja) 2020-05-21
EP4245852A3 (en) 2023-11-22
AU2018233732A1 (en) 2019-10-03
TW201840850A (zh) 2018-11-16
EP3596222A1 (en) 2020-01-22
CN118497275A (zh) 2024-08-16
IL269391B2 (en) 2024-09-01
MD3596222T2 (ro) 2023-08-31
SG11201908575SA (en) 2019-10-30
HRP20230522T1 (hr) 2023-08-04
CN110997923B (zh) 2024-01-02
DK3596222T3 (da) 2023-06-19
HUE062476T2 (hu) 2023-11-28
AU2018233732B2 (en) 2024-10-24
KR20240112964A (ko) 2024-07-19
MA52112A (fr) 2020-01-22
BR112019019248A2 (pt) 2020-04-28
MX2019011046A (es) 2019-10-17
EP4245852A2 (en) 2023-09-20
KR20190130591A (ko) 2019-11-22
CY1126080T1 (el) 2023-11-15
LT3596222T (lt) 2023-07-25
US20200199621A1 (en) 2020-06-25
EP3596222A4 (en) 2021-01-06
IL269391A (en) 2019-11-28
FI3596222T3 (fi) 2023-06-08
IL269391B1 (en) 2024-05-01
ES2948233T3 (es) 2023-09-06
PT3596222T (pt) 2023-06-20
EP3596222B1 (en) 2023-04-12
DK3596222T5 (da) 2024-09-02
IL311713A (en) 2024-05-01
EA201992201A1 (ru) 2020-03-19
US20220364117A1 (en) 2022-11-17
CO2019011250A2 (es) 2020-02-28
CN110997923A (zh) 2020-04-10
KR102683682B1 (ko) 2024-07-09
SI3596222T1 (sl) 2024-05-31
ZA201906251B (en) 2024-01-31
AR111292A1 (es) 2019-06-26
PL3596222T3 (pl) 2023-10-09
CA3056638A1 (en) 2018-09-20
WO2018170408A1 (en) 2018-09-20
MA52112B1 (fr) 2023-08-31

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