PL1879623T3 - Terapia genowa zaburzeń rdzenia kręgowego - Google Patents

Terapia genowa zaburzeń rdzenia kręgowego

Info

Publication number
PL1879623T3
PL1879623T3 PL06752135T PL06752135T PL1879623T3 PL 1879623 T3 PL1879623 T3 PL 1879623T3 PL 06752135 T PL06752135 T PL 06752135T PL 06752135 T PL06752135 T PL 06752135T PL 1879623 T3 PL1879623 T3 PL 1879623T3
Authority
PL
Poland
Prior art keywords
gene therapy
spinal cord
cord disorders
disorders
spinal
Prior art date
Application number
PL06752135T
Other languages
English (en)
Inventor
James Dodge
Lamya Shihabuddin
Marco Passini
Seng Cheng
Catherine R O'riordan
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.)
Filing date
Publication date
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of PL1879623T3 publication Critical patent/PL1879623T3/pl

Links

Classifications

    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475—Growth factors; Growth regulators
    • 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
    • C12N2750/14041—Use of virus, viral particle or viral elements as a 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Toxicology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
PL06752135T 2005-05-02 2006-05-02 Terapia genowa zaburzeń rdzenia kręgowego PL1879623T3 (pl)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US67721305P 2005-05-02 2005-05-02
US79021706P 2006-04-08 2006-04-08
PCT/US2006/016943 WO2006119341A2 (en) 2005-05-02 2006-05-02 Gene therapy for spinal cord disorders
EP06752135A EP1879623B1 (en) 2005-05-02 2006-05-02 Gene therapy for spinal cord disorders

Publications (1)

Publication Number Publication Date
PL1879623T3 true PL1879623T3 (pl) 2013-03-29

Family

ID=37308661

Family Applications (1)

Application Number Title Priority Date Filing Date
PL06752135T PL1879623T3 (pl) 2005-05-02 2006-05-02 Terapia genowa zaburzeń rdzenia kręgowego

Country Status (14)

Country Link
US (2) US10744210B2 (pl)
EP (1) EP1879623B1 (pl)
JP (5) JP5766389B2 (pl)
CN (1) CN105770909A (pl)
AU (1) AU2006242129A1 (pl)
BR (1) BRPI0611540A2 (pl)
CA (1) CA2606490C (pl)
DK (1) DK1879623T3 (pl)
ES (1) ES2394482T3 (pl)
IL (2) IL187023A (pl)
MX (1) MX2007013734A (pl)
PL (1) PL1879623T3 (pl)
PT (1) PT1879623E (pl)
WO (1) WO2006119341A2 (pl)

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PL1879623T3 (pl) 2005-05-02 2013-03-29 Genzyme Corp Terapia genowa zaburzeń rdzenia kręgowego
PL1879624T3 (pl) 2005-05-02 2012-02-29 Genzyme Corp Terapia genowa dla zaburzeń neurometabolicznych
US8926959B2 (en) 2005-07-22 2015-01-06 The Board Of Trustees Of The Leland Stanford Junior University System for optical stimulation of target cells
US20090093403A1 (en) 2007-03-01 2009-04-09 Feng Zhang Systems, methods and compositions for optical stimulation of target cells
EP2465925A1 (en) 2005-07-22 2012-06-20 The Board Of Trustees Of The Leland Light-activated cation channel and uses thereof
US9238150B2 (en) 2005-07-22 2016-01-19 The Board Of Trustees Of The Leland Stanford Junior University Optical tissue interface method and apparatus for stimulating cells
US9274099B2 (en) * 2005-07-22 2016-03-01 The Board Of Trustees Of The Leland Stanford Junior University Screening test drugs to identify their effects on cell membrane voltage-gated ion channel
US10052497B2 (en) * 2005-07-22 2018-08-21 The Board Of Trustees Of The Leland Stanford Junior University System for optical stimulation of target cells
PL2029742T3 (pl) * 2006-06-07 2017-08-31 Genzyme Corporation Terapia genowa w stwardnieniu zanikowym bocznym i innych zaburzeniach rdzenia kręgowego
US8398692B2 (en) 2007-01-10 2013-03-19 The Board Of Trustees Of The Leland Stanford Junior University System for optical stimulation of target cells
WO2008101128A1 (en) 2007-02-14 2008-08-21 The Board Of Trustees Of The Leland Stanford Junior University System, method and applications involving identification of biological circuits such as neurological characteristics
EP2019143A1 (en) * 2007-07-23 2009-01-28 Genethon CNS gene delivery using peripheral administration of AAV vectors
EP2058401A1 (en) 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
US10035027B2 (en) * 2007-10-31 2018-07-31 The Board Of Trustees Of The Leland Stanford Junior University Device and method for ultrasonic neuromodulation via stereotactic frame based technique
US10434327B2 (en) * 2007-10-31 2019-10-08 The Board Of Trustees Of The Leland Stanford Junior University Implantable optical stimulators
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CA2726128C (en) 2008-05-29 2016-10-18 The Board Of Trustees Of The Leland Stanford Junior University Cell line, system and method for optical control of secondary messengers
US10711242B2 (en) 2008-06-17 2020-07-14 The Board Of Trustees Of The Leland Stanford Junior University Apparatus and methods for controlling cellular development
ES2612052T3 (es) 2008-06-17 2017-05-11 The Board Of Trustees Of The Leland Stanford Junior University Dispositivos para la estimulación óptica de células diana, utilizando un elemento de transmisión óptica
WO2010006049A1 (en) 2008-07-08 2010-01-14 The Board Of Trustees Of The Leland Stanford Junior University Materials and approaches for optical stimulation of the peripheral nervous system
NZ602416A (en) 2008-11-14 2014-08-29 Univ Leland Stanford Junior Optically-based stimulation of target cells and modifications thereto
MX356669B (es) * 2009-05-02 2018-06-08 Genzyme Corp Terapia genica para trastornos neurodegenerativos.
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AU2011323235B2 (en) 2010-11-05 2015-10-29 The Board Of Trustees Of The Leland Stanford Junior University Optogenetic control of reward-related behaviors
ES2716819T3 (es) 2010-11-05 2019-06-17 Univ Leland Stanford Junior Opsinas quiméricas activadas por luz y métodos de uso de las mismas
WO2012061690A2 (en) 2010-11-05 2012-05-10 The Board Of Trustees Of The Leland Stanford Junior University Optically-controlled cns dysfunction
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JP6002140B2 (ja) 2010-11-05 2016-10-05 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー 安定化階段関数オプシンタンパク質及びその使用方法
US8696722B2 (en) 2010-11-22 2014-04-15 The Board Of Trustees Of The Leland Stanford Junior University Optogenetic magnetic resonance imaging
US20130164367A1 (en) * 2011-12-08 2013-06-27 The Board Of Regents Of The University Of Texas System Treatment of neurodegenerative disease with creb-binding protein
WO2013090356A2 (en) 2011-12-16 2013-06-20 The Board Of Trustees Of The Leland Stanford Junior University Opsin polypeptides and methods of use thereof
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US9636380B2 (en) 2013-03-15 2017-05-02 The Board Of Trustees Of The Leland Stanford Junior University Optogenetic control of inputs to the ventral tegmental area
JP6549559B2 (ja) 2013-04-29 2019-07-24 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー 標的細胞における活動電位の光遺伝学的調節のための装置、システム及び方法
WO2014178863A1 (en) * 2013-05-01 2014-11-06 Genzyme Corporation Compositions and methods for treating spinal muscular atrophy
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PL1879623T3 (pl) 2005-05-02 2013-03-29 Genzyme Corp Terapia genowa zaburzeń rdzenia kręgowego
HUE041928T2 (hu) 2006-02-08 2019-06-28 Genzyme Corp Génterápia A-típusú Niemann-Pick-betegségre

Also Published As

Publication number Publication date
JP2013082731A (ja) 2013-05-09
JP6887008B2 (ja) 2021-06-16
US10744210B2 (en) 2020-08-18
CA2606490C (en) 2018-02-06
PT1879623E (pt) 2012-12-20
DK1879623T3 (da) 2012-12-17
EP1879623A4 (en) 2009-03-25
JP2020050660A (ja) 2020-04-02
WO2006119341A2 (en) 2006-11-09
JP2017132774A (ja) 2017-08-03
MX2007013734A (es) 2008-03-14
JP2008540435A (ja) 2008-11-20
ES2394482T3 (es) 2013-02-01
IL237658A0 (en) 2015-04-30
US20210008227A1 (en) 2021-01-14
BRPI0611540A2 (pt) 2010-09-21
WO2006119341A3 (en) 2007-06-28
EP1879623B1 (en) 2012-10-03
CA2606490A1 (en) 2006-11-09
US20090069261A1 (en) 2009-03-12
JP2015163613A (ja) 2015-09-10
IL187023A0 (en) 2008-02-09
JP5766389B2 (ja) 2015-08-19
CN105770909A (zh) 2016-07-20
EP1879623A2 (en) 2008-01-23
AU2006242129A1 (en) 2006-11-09
IL187023A (en) 2015-10-29

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