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)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Neurosurgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (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
EP06752135A EP1879623B1 (en) 2005-05-02 2006-05-02 Gene therapy for spinal cord disorders
PCT/US2006/016943 WO2006119341A2 (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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EP1879623B1 (en) 2005-05-02 2012-10-03 Genzyme Corporation Gene therapy for spinal cord disorders
BRPI0611379A2 (pt) 2005-05-02 2010-08-31 Genzyme Corp terapia genética para distúrbios neurometabólicos
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
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
EP2465925A1 (en) * 2005-07-22 2012-06-20 The Board Of Trustees Of The Leland Light-activated cation channel and uses thereof
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
MX364444B (es) * 2006-06-07 2019-04-26 Genzyme Corp Terapia génica para esclerosis lateral amiotrófica y otros trastornos de la médula espinal.
WO2008086470A1 (en) 2007-01-10 2008-07-17 The Board Of Trustees Of The Leland Stanford Junior University System for optical stimulation of target cells
US8401609B2 (en) 2007-02-14 2013-03-19 The Board Of Trustees Of The Leland Stanford Junior University System, method and applications involving identification of biological circuits such as neurological characteristics
WO2008106694A2 (en) 2007-03-01 2008-09-04 The Board Of Trustees Of The Leland Stanford Junior University Systems, methods and compositions for optical stimulation of target cells
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
EP3165534B1 (en) 2008-04-23 2018-09-26 The Board of Trustees of The Leland Stanford Junior University Systems, methods and compositions for optical stimulation of target cells
EP2610350B1 (en) 2008-05-29 2014-12-17 The Board Of Trustees Of The Leland Stanford Junior University Cell line, system and method for optical control of secondary messengers
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
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
US9101759B2 (en) * 2008-07-08 2015-08-11 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
EP3988660B1 (en) * 2009-05-02 2025-05-14 Genzyme Corporation Gene therapy for neurodegenerative disorders
CN106011073A (zh) 2010-03-17 2016-10-12 小利兰·斯坦福大学托管委员会 光敏离子透过性分子
CN103384469B (zh) 2010-11-05 2016-06-15 斯坦福大学托管董事会 光控cns功能障碍
CN110215614A (zh) 2010-11-05 2019-09-10 斯坦福大学托管董事会 用于光遗传学方法的光的上转换
EP3486253A1 (en) 2010-11-05 2019-05-22 The Board of Trustees of The Leland Stanford Junior University Light-activated chimeric opsins and methods of using the same
US9992981B2 (en) 2010-11-05 2018-06-12 The Board Of Trustees Of The Leland Stanford Junior University Optogenetic control of reward-related behaviors
CA2816972C (en) 2010-11-05 2019-12-03 The Board Of Trustees Of The Leland Stanford Junior University Control and characterization of memory function
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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
CA3036859A1 (en) 2011-12-16 2013-06-20 The Board Of Trustees Of The Leland Stanford Junior University Opsin polypeptides and methods of use thereof
AU2013222443B2 (en) 2012-02-21 2017-12-14 Circuit Therapeutics, Inc. Compositions and methods for treating neurogenic disorders of the pelvic floor
IN2014KN02672A (pl) * 2012-05-18 2015-05-08 Univ Iowa Res Found
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
WO2014144409A1 (en) 2013-03-15 2014-09-18 The Board Of Trustees Of The Leland Stanford Junior University Optogenetic control of behavioral state
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
AU2013388083B2 (en) * 2013-05-01 2019-08-22 Genzyme Corporation Compositions and methods for treating spinal muscular atrophy
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EP3033427A4 (en) 2013-08-14 2017-05-31 The Board Of Trustees Of The University Of the Leland Stanford Junior University Compositions and methods for controlling pain
US10568516B2 (en) 2015-06-22 2020-02-25 The Board Of Trustees Of The Leland Stanford Junior University Methods and devices for imaging and/or optogenetic control of light-responsive neurons
US11294165B2 (en) 2017-03-30 2022-04-05 The Board Of Trustees Of The Leland Stanford Junior University Modular, electro-optical device for increasing the imaging field of view using time-sequential capture
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Also Published As

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

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