CR20170407A - Mejora del suministro de partículas virales al cuerpo estriado y al córtex - Google Patents

Mejora del suministro de partículas virales al cuerpo estriado y al córtex

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Publication number
CR20170407A
CR20170407A CR20170407A CR20170407A CR20170407A CR 20170407 A CR20170407 A CR 20170407A CR 20170407 A CR20170407 A CR 20170407A CR 20170407 A CR20170407 A CR 20170407A CR 20170407 A CR20170407 A CR 20170407A
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CR
Costa Rica
Prior art keywords
viral particles
cortex
improvement
supply
corrected body
Prior art date
Application number
CR20170407A
Other languages
English (en)
Inventor
Lamya Shihabuddin
Lisa M Stanek
Original Assignee
Genzyme Corp
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.)
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Publication date
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of CR20170407A publication Critical patent/CR20170407A/es

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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
    • 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
    • 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
    • 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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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
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  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Biotechnology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
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  • Physics & Mathematics (AREA)
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  • Psychiatry (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Immunology (AREA)
  • Pain & Pain Management (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

La invención proporciona nuevos métodos para suministrar partículas virales adeno-asociadas recombinantes (rAAV) al sistema nervioso central de un mamífero (p. ej. un ser humano). En aspectos, los métodos comprenden administrar partículas de rAAV que contienen un ácido nucleico heterólogo al cuerpo estriado y causar la expresión del ácido nucleico heterólogo en al menos el córtex cerebral y el cuerpo estriado del mamífero.
CR20170407A 2015-02-10 2016-02-09 Mejora del suministro de partículas virales al cuerpo estriado y al córtex CR20170407A (es)

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
CR20170407A true CR20170407A (es) 2017-11-14

Family

ID=56614902

Family Applications (1)

Application Number Title Priority Date Filing Date
CR20170407A CR20170407A (es) 2015-02-10 2016-02-09 Mejora del suministro de partículas virales al cuerpo estriado y al córtex

Country Status (27)

Country Link
US (2) US20190111157A1 (es)
EP (3) EP3256594B1 (es)
JP (3) JP6928558B2 (es)
KR (2) KR102625784B1 (es)
CN (2) CN107530447A (es)
AU (2) AU2016219398A1 (es)
BR (1) BR112017017037A2 (es)
CA (1) CA2976082A1 (es)
CL (1) CL2017002028A1 (es)
CR (1) CR20170407A (es)
DK (1) DK3256594T3 (es)
EA (1) EA201791803A1 (es)
ES (1) ES2904504T3 (es)
HK (1) HK1246340A1 (es)
HU (1) HUE057272T2 (es)
IL (4) IL296466B1 (es)
LT (1) LT3256594T (es)
MX (2) MX2017010370A (es)
PH (1) PH12017501436A1 (es)
PL (1) PL3256594T3 (es)
PT (1) PT3256594T (es)
RS (1) RS62811B1 (es)
SG (3) SG10201907399RA (es)
SI (1) SI3256594T1 (es)
TN (1) TN2017000353A1 (es)
TW (1) TWI730952B (es)
WO (1) WO2016130591A2 (es)

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AU2022204583A1 (en) 2022-07-21
PH12017501436A1 (en) 2018-01-15
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IL253894A0 (en) 2017-10-31
IL313840A (en) 2024-08-01
CL2017002028A1 (es) 2018-04-13
US20220395586A1 (en) 2022-12-15
PL3256594T3 (pl) 2022-02-14
JP2021178861A (ja) 2021-11-18
SG10201912926VA (en) 2020-02-27
EA201791803A1 (ru) 2017-11-30
LT3256594T (lt) 2022-02-10
CN107530447A (zh) 2018-01-02
JP2023058630A (ja) 2023-04-25
EP4023758A1 (en) 2022-07-06
BR112017017037A2 (pt) 2018-04-10
MX2017010370A (es) 2017-12-14
AU2016219398A1 (en) 2017-09-28
SG10201907399RA (en) 2019-09-27
SG11201706445SA (en) 2017-09-28
TN2017000353A1 (en) 2019-01-16
EP4219728A2 (en) 2023-08-02
HK1246340A1 (zh) 2018-09-07
KR102625784B1 (ko) 2024-01-15
EP3256594A2 (en) 2017-12-20
KR20240010539A (ko) 2024-01-23
CA2976082A1 (en) 2016-08-18
EP3256594B1 (en) 2021-10-20
JP2018506585A (ja) 2018-03-08
KR20170110718A (ko) 2017-10-11
IL296466A (en) 2022-11-01
SI3256594T1 (sl) 2022-07-29
IL285060B2 (en) 2023-02-01
IL285060A (en) 2021-08-31
IL296466B1 (en) 2024-07-01
NZ735290A (en) 2023-10-27
HUE057272T2 (hu) 2022-04-28
CN116019934A (zh) 2023-04-28
ES2904504T3 (es) 2022-04-05
TWI730952B (zh) 2021-06-21
MX2021004294A (es) 2021-06-04
DK3256594T3 (da) 2022-01-24
RS62811B1 (sr) 2022-02-28
WO2016130591A2 (en) 2016-08-18
TW201642909A (zh) 2016-12-16
EP4219728A3 (en) 2023-08-23
JP6928558B2 (ja) 2021-09-01
WO2016130591A3 (en) 2016-11-10
PT3256594T (pt) 2022-01-18
US20190111157A1 (en) 2019-04-18

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