BR112017017037A2 - liberação realçada de partículas virais para o estriado e córtex - Google Patents

liberação realçada de partículas virais para o estriado e córtex

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Publication number
BR112017017037A2
BR112017017037A2 BR112017017037A BR112017017037A BR112017017037A2 BR 112017017037 A2 BR112017017037 A2 BR 112017017037A2 BR 112017017037 A BR112017017037 A BR 112017017037A BR 112017017037 A BR112017017037 A BR 112017017037A BR 112017017037 A2 BR112017017037 A2 BR 112017017037A2
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BR
Brazil
Prior art keywords
striatum
viral particles
cortex
enhanced release
mammal
Prior art date
Application number
BR112017017037A
Other languages
English (en)
Inventor
Shihabuddin Lamya
M Stanek Lisa
Original Assignee
Genzyme Corp
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Publication date
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of BR112017017037A2 publication Critical patent/BR112017017037A2/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
    • A61K48/0075—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
    • 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
    • A61K48/005—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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/08—Antiepileptics; Anticonvulsants
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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    • 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
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    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • Pharmacology & Pharmacy (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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Abstract

a presente invenção refere-se a novos métodos para liberar partículas virais adenoassociadas recombinantes (raav) para o sistema nervoso central de um mamífero (por exemplo, um humano). em aspectos, os métodos envolvem administrar partículas de raav contendo um ácido nucleico heterólogo ao estriado e causar a expressão do ácido nucleico heterólogo pelo menos no córtex cerebral e no estriado do mamífero.
BR112017017037A 2015-02-10 2016-02-09 liberação realçada de partículas virais para o estriado e córtex BR112017017037A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562114544P 2015-02-10 2015-02-10
US201562220997P 2015-09-19 2015-09-19
PCT/US2016/017210 WO2016130591A2 (en) 2015-02-10 2016-02-09 Enhanced delivery of viral particles to the striatum and cortex

Publications (1)

Publication Number Publication Date
BR112017017037A2 true BR112017017037A2 (pt) 2018-04-10

Family

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Family Applications (1)

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BR112017017037A BR112017017037A2 (pt) 2015-02-10 2016-02-09 liberação realçada de partículas virais para o estriado e córtex

Country Status (27)

Country Link
US (2) US20190111157A1 (pt)
EP (3) EP4023758A1 (pt)
JP (3) JP6928558B2 (pt)
KR (2) KR20240010539A (pt)
CN (2) CN107530447A (pt)
AU (2) AU2016219398A1 (pt)
BR (1) BR112017017037A2 (pt)
CA (1) CA2976082A1 (pt)
CL (1) CL2017002028A1 (pt)
CR (1) CR20170407A (pt)
DK (1) DK3256594T3 (pt)
EA (1) EA201791803A1 (pt)
ES (1) ES2904504T3 (pt)
HK (1) HK1246340A1 (pt)
HU (1) HUE057272T2 (pt)
IL (3) IL296466A (pt)
LT (1) LT3256594T (pt)
MX (2) MX2017010370A (pt)
PH (1) PH12017501436A1 (pt)
PL (1) PL3256594T3 (pt)
PT (1) PT3256594T (pt)
RS (1) RS62811B1 (pt)
SG (3) SG10201907399RA (pt)
SI (1) SI3256594T1 (pt)
TN (1) TN2017000353A1 (pt)
TW (1) TWI730952B (pt)
WO (1) WO2016130591A2 (pt)

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EP4219728A3 (en) 2023-08-23
RS62811B1 (sr) 2022-02-28
CN116019934A (zh) 2023-04-28
TN2017000353A1 (en) 2019-01-16
SG11201706445SA (en) 2017-09-28
CR20170407A (es) 2017-11-14
KR20240010539A (ko) 2024-01-23
JP2021178861A (ja) 2021-11-18
PT3256594T (pt) 2022-01-18
SG10201907399RA (en) 2019-09-27
NZ735290A (en) 2023-10-27
TW201642909A (zh) 2016-12-16
KR102625784B1 (ko) 2024-01-15
HUE057272T2 (hu) 2022-04-28
US20190111157A1 (en) 2019-04-18
WO2016130591A2 (en) 2016-08-18
LT3256594T (lt) 2022-02-10
CN107530447A (zh) 2018-01-02
ES2904504T3 (es) 2022-04-05
SG10201912926VA (en) 2020-02-27
WO2016130591A3 (en) 2016-11-10
AU2016219398A1 (en) 2017-09-28
US20220395586A1 (en) 2022-12-15
PL3256594T3 (pl) 2022-02-14
EP4219728A2 (en) 2023-08-02
TWI730952B (zh) 2021-06-21
DK3256594T3 (da) 2022-01-24
JP2023058630A (ja) 2023-04-25
IL296466A (en) 2022-11-01
IL253894B (en) 2021-08-31
AU2022204583A1 (en) 2022-07-21
PH12017501436A1 (en) 2018-01-15
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