PT3596222T - Administração por vetor de vírus adenoassociado de microdistrofina específica do músculo para tratar a distrofia muscular - Google Patents

Administração por vetor de vírus adenoassociado de microdistrofina específica do músculo para tratar a distrofia muscular

Info

Publication number
PT3596222T
PT3596222T PT187683958T PT18768395T PT3596222T PT 3596222 T PT3596222 T PT 3596222T PT 187683958 T PT187683958 T PT 187683958T PT 18768395 T PT18768395 T PT 18768395T PT 3596222 T PT3596222 T PT 3596222T
Authority
PT
Portugal
Prior art keywords
dystrophin
adeno
associated virus
muscular dystrophy
virus vector
Prior art date
Application number
PT187683958T
Other languages
English (en)
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 PT3596222T publication Critical patent/PT3596222T/pt

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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
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • 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
    • 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
    • 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
    • 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
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • 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

Landscapes

  • 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)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (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)
PT187683958T 2017-03-17 2018-03-16 Administração por vetor de vírus adenoassociado de microdistrofina específica do músculo para tratar a distrofia muscular PT3596222T (pt)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201762473148P 2017-03-17 2017-03-17

Publications (1)

Publication Number Publication Date
PT3596222T true PT3596222T (pt) 2023-06-20

Family

ID=63523321

Family Applications (1)

Application Number Title Priority Date Filing Date
PT187683958T PT3596222T (pt) 2017-03-17 2018-03-16 Administração por vetor de vírus adenoassociado de microdistrofina específica do músculo para tratar a distrofia muscular

Country Status (30)

Country Link
US (2) US20200199621A1 (pt)
EP (2) EP3596222B1 (pt)
JP (2) JP2020513811A (pt)
KR (2) KR20240112964A (pt)
CN (2) CN110997923B (pt)
AR (1) AR111292A1 (pt)
AU (1) AU2018233732B2 (pt)
BR (1) BR112019019248A2 (pt)
CA (1) CA3056638A1 (pt)
CO (1) CO2019011250A2 (pt)
CY (1) CY1126080T1 (pt)
DK (1) DK3596222T5 (pt)
EA (1) EA201992201A1 (pt)
ES (1) ES2948233T3 (pt)
FI (1) FI3596222T3 (pt)
HR (1) HRP20230522T1 (pt)
HU (1) HUE062476T2 (pt)
IL (2) IL311713A (pt)
LT (1) LT3596222T (pt)
MA (1) MA52112B1 (pt)
MD (1) MD3596222T2 (pt)
MX (1) MX2019011046A (pt)
PL (1) PL3596222T3 (pt)
PT (1) PT3596222T (pt)
RS (1) RS64299B1 (pt)
SG (1) SG11201908575SA (pt)
SI (1) SI3596222T1 (pt)
TW (1) TW201840850A (pt)
WO (1) WO2018170408A1 (pt)
ZA (1) ZA201906251B (pt)

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SG11202003285PA (en) 2017-10-20 2020-05-28 Res Inst Nationwide Childrens Hospital Methods and materials for nt-3 gene therapy
EP3897689A1 (en) * 2018-12-21 2021-10-27 Northwestern University Use of annexins in preventing and treating muscle membrane injury
EP3918081A4 (en) * 2019-02-02 2022-11-02 University of Pittsburgh - of the Commonwealth System of Higher Education ONE-STAGE GENE THERAPY FOR DUCHENNE MUSCULAR DYSTROPHY THROUGH GENER REPLACEMENT AND ANTI-INFLAMMATION
AU2020284255A1 (en) * 2019-05-30 2022-01-06 Solid Biosciences Inc. Recombinant herpesvirales vector
US20220349017A1 (en) * 2019-08-29 2022-11-03 Siemens Healthcare Diagnostics Inc. Reagents and methods for detecting aav shedding
MX2022006427A (es) * 2019-11-28 2022-09-07 Regenxbio Inc "construcciones para terapia génica con microdistrofina y uso de las mismas.
EP4117783A4 (en) * 2019-12-16 2024-08-21 Res Inst Nationwide Childrens Hospital COMPOSITIONS AND METHODS FOR RESTORING AND MAINTAINING DYSTROPHIN-ASSOCIATED PROTEIN COMPLEX (DAPC)
KR20230003557A (ko) 2020-04-29 2023-01-06 브리스톨-마이어스 스큅 컴퍼니 스펙트린 융합 도메인을 갖는 소형화 디스트로핀 및 그의 용도
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
EP4086276A1 (en) 2021-05-03 2022-11-09 Université d'Aix-Marseille Composition for treating dysferlinopathy
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
AU2022325165A1 (en) * 2021-08-05 2024-01-18 Insmed Incorporated Adeno-associated virus particles and methods of use thereof
JP2024531138A (ja) * 2021-08-11 2024-08-29 ウルトラジェニックス ファーマシューティカル インコーポレイテッド 筋ジストロフィーを処置するための組成物および方法
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
CN117247973B (zh) * 2022-04-19 2024-05-10 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
WO2023248251A1 (en) * 2022-06-24 2023-12-28 Indian Institute Of Technology Kanpur An optimized aav vector for gene therapy of muscular dystrophy

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Also Published As

Publication number Publication date
EP4245852A2 (en) 2023-09-20
ES2948233T3 (es) 2023-09-06
SI3596222T1 (sl) 2024-05-31
CN110997923A (zh) 2020-04-10
DK3596222T3 (da) 2023-06-19
IL269391A (en) 2019-11-28
LT3596222T (lt) 2023-07-25
US20200199621A1 (en) 2020-06-25
EP3596222B1 (en) 2023-04-12
US20220364117A1 (en) 2022-11-17
TW201840850A (zh) 2018-11-16
EP3596222A1 (en) 2020-01-22
CN118497275A (zh) 2024-08-16
IL269391B2 (en) 2024-09-01
AR111292A1 (es) 2019-06-26
CN110997923B (zh) 2024-01-02
FI3596222T3 (fi) 2023-06-08
MA52112B1 (fr) 2023-08-31
KR20190130591A (ko) 2019-11-22
CY1126080T1 (el) 2023-11-15
CA3056638A1 (en) 2018-09-20
MA52112A (fr) 2020-01-22
AU2018233732B2 (en) 2024-10-24
CO2019011250A2 (es) 2020-02-28
EA201992201A1 (ru) 2020-03-19
MD3596222T2 (ro) 2023-08-31
HUE062476T2 (hu) 2023-11-28
WO2018170408A1 (en) 2018-09-20
PL3596222T3 (pl) 2023-10-09
ZA201906251B (en) 2024-01-31
JP2020513811A (ja) 2020-05-21
BR112019019248A2 (pt) 2020-04-28
IL269391B1 (en) 2024-05-01
DK3596222T5 (da) 2024-09-02
JP2023053254A (ja) 2023-04-12
EP3596222A4 (en) 2021-01-06
HRP20230522T1 (hr) 2023-08-04
MX2019011046A (es) 2019-10-17
IL311713A (en) 2024-05-01
RS64299B1 (sr) 2023-07-31
KR102683682B1 (ko) 2024-07-09
EP4245852A3 (en) 2023-11-22
SG11201908575SA (en) 2019-10-30
KR20240112964A (ko) 2024-07-19
AU2018233732A1 (en) 2019-10-03

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