CY1118555T1 - Γονιδιακη θεραπεια για την αμυοτροφικη πλευρικη σκληρυνση και αλλες διαταραχες της σπονδυλικης στηλης - Google Patents

Γονιδιακη θεραπεια για την αμυοτροφικη πλευρικη σκληρυνση και αλλες διαταραχες της σπονδυλικης στηλης

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Publication number
CY1118555T1
CY1118555T1 CY20161101055T CY161101055T CY1118555T1 CY 1118555 T1 CY1118555 T1 CY 1118555T1 CY 20161101055 T CY20161101055 T CY 20161101055T CY 161101055 T CY161101055 T CY 161101055T CY 1118555 T1 CY1118555 T1 CY 1118555T1
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CY
Cyprus
Prior art keywords
subject
transgene
brain
subjective
viral vector
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CY20161101055T
Other languages
English (en)
Inventor
James Dodge
Lamya Shihabuddin
Catherine R O'riordan
Original Assignee
Genzyme Corporation
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Publication date
Application filed by Genzyme Corporation filed Critical Genzyme Corporation
Publication of CY1118555T1 publication Critical patent/CY1118555T1/el

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    • 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
    • A61K38/22—Hormones
    • A61K38/30—Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
    • 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
    • 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
    • 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
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • 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
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • 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
    • 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
    • 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
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • 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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Diabetes (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Endocrinology (AREA)
  • Zoology (AREA)
  • Hospice & Palliative Care (AREA)
  • Pain & Pain Management (AREA)
  • Psychiatry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)

Abstract

Αυτή η δημοσιοποίηση παρέχει μεθόδους και σκευάσματα για την θεραπεία διαταραχών ή τραυματισμών που επηρεάζουν την κινητική λειτουργία και τον έλεγχο σε ένα υποκείμενο. Κατά μία άποψη, η εφεύρεση ενός διαγονιδιακού προϊόντος χορηγείται στο νωτιαίο μυελό ενός υποκειμένου με τη χορήγηση στον εγκέφαλο ενός ανασυνδυασμένου νευροτροφικού ιικού φορέα που περιέχει το διαγονίδιο. Ο ιικός φορέας παραδίδει το διαγονίδιο σε μια περιοχή του εγκεφάλου η οποία είναι ευαίσθητη σε μόλυνση από τον ιό και η οποία αποκωδικοποιεί το κωδικοποιημένο προϊόν ανασυνδυασμένου ιικού γονιδίου. Επίσης παρέχονται σκευάσματα για την παροχή ενός διαγονιδιακού προϊόντος στο νωτιαίο μυελό ενός υποκειμένου με τη χορήγηση στον εγκέφαλο του υποκειμένου ενός ανασυνδυασμένου νευροτροφικού ιικού φορέα που περιέχει το διαγονίδιο.
CY20161101055T 2006-06-07 2016-10-19 Γονιδιακη θεραπεια για την αμυοτροφικη πλευρικη σκληρυνση και αλλες διαταραχες της σπονδυλικης στηλης CY1118555T1 (el)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US81141906P 2006-06-07 2006-06-07
PCT/US2007/013391 WO2007146046A2 (en) 2006-06-07 2007-06-07 Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders

Publications (1)

Publication Number Publication Date
CY1118555T1 true CY1118555T1 (el) 2017-07-12

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Country Status (19)

Country Link
US (4) US9034836B2 (el)
EP (3) EP2029742B1 (el)
JP (6) JP2009539847A (el)
CN (1) CN101460621B (el)
AR (2) AR061282A1 (el)
BR (1) BRPI0711965A2 (el)
CA (2) CA3139726A1 (el)
CY (1) CY1118555T1 (el)
ES (2) ES2725552T3 (el)
HU (2) HUE031156T2 (el)
IL (2) IL195647A (el)
LT (2) LT2489733T (el)
MX (4) MX2008015530A (el)
PL (2) PL2029742T3 (el)
PT (2) PT2029742T (el)
RU (1) RU2008152821A (el)
SI (2) SI2489733T1 (el)
TR (1) TR201906106T4 (el)
WO (1) WO2007146046A2 (el)

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EP3540054A3 (en) 2019-10-09
LT2489733T (lt) 2019-05-27
US20200101138A1 (en) 2020-04-02
PL2489733T3 (pl) 2019-08-30
PL2029742T3 (pl) 2017-08-31
JP2015120719A (ja) 2015-07-02
EP2489733A2 (en) 2012-08-22
MX364444B (es) 2019-04-26
EP2029742A4 (en) 2009-09-30
US20230364200A1 (en) 2023-11-16
CN101460621A (zh) 2009-06-17
SI2029742T1 (sl) 2016-11-30
WO2007146046A2 (en) 2007-12-21
MX2019004843A (es) 2021-06-15
JP2009539847A (ja) 2009-11-19
CA3139726A1 (en) 2007-12-21
US20160067312A1 (en) 2016-03-10
JP2017078071A (ja) 2017-04-27
US9034836B2 (en) 2015-05-19
CA2654292A1 (en) 2007-12-21
ES2596885T3 (es) 2017-01-12
AR061282A1 (es) 2008-08-20
SI2489733T1 (sl) 2019-06-28
IL241890A0 (en) 2015-11-30
CN101460621B (zh) 2016-01-27
HUE031156T2 (en) 2017-06-28
MX2008015530A (es) 2009-02-06
JP2020079234A (ja) 2020-05-28
US11554161B2 (en) 2023-01-17
EP3540054A2 (en) 2019-09-18
JP6320489B2 (ja) 2018-05-09
US20100267812A1 (en) 2010-10-21
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PT2029742T (pt) 2016-09-09
TR201906106T4 (tr) 2019-05-21
RU2008152821A (ru) 2010-07-20
PT2489733T (pt) 2019-05-27
JP2018118995A (ja) 2018-08-02
WO2007146046A3 (en) 2008-05-22
MX2023001965A (es) 2023-02-23
EP2489733A3 (en) 2012-11-14
AR109171A2 (es) 2018-11-07
JP6606215B2 (ja) 2019-11-13
EP2029742B1 (en) 2016-07-20
JP2014012697A (ja) 2014-01-23
ES2725552T3 (es) 2019-09-24
BRPI0711965A2 (pt) 2012-01-24
EP2489733B1 (en) 2019-02-13
IL195647A0 (en) 2009-09-01
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