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
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)

Families Citing this family (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2006242129A1 (en) 2005-05-02 2006-11-09 Genzyme Corporation Gene therapy for spinal cord disorders
PT3520823T (pt) 2005-05-02 2021-09-16 Genzyme Corp Terapia genética para distúrbios neurometabólicos
US20090093403A1 (en) 2007-03-01 2009-04-09 Feng Zhang Systems, methods and compositions for optical stimulation of target 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
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
WO2007024391A2 (en) 2005-07-22 2007-03-01 The Board Of Trustees Of The Leland Stanford Junior University 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
PT2029742T (pt) 2006-06-07 2016-09-09 Genzyme Corp Terapia genética para esclerose lateral amiotrófica e outros transtornos da medula 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
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
KR20110005280A (ko) 2008-04-23 2011-01-17 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 표적 세포의 광학적 자극을 위한 시스템, 방법 및 조성물
EP2294208B1 (en) 2008-05-29 2013-05-08 The Board of Trustees of The Leland Stanford Junior University Cell line, system and method for optical control of secondary messengers
JP5887136B2 (ja) 2008-06-17 2016-03-16 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 細胞発達を制御するための装置および方法
MY156389A (en) 2008-06-17 2016-02-15 Univ Leland Stanford Junior Methods, systems and devices for optical stimulation of target cells using an optical transmission element
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
EP3421603B1 (en) * 2009-05-02 2021-10-06 Genzyme Corporation Gene therapy for neurodegenerative disorders
AU2011227131B2 (en) 2010-03-17 2014-11-13 The Board Of Trustees Of The Leland Stanford Junior University Light-sensitive ion-passing molecules
EP2635109A4 (en) 2010-11-05 2014-03-19 Univ Leland Stanford Junior OPTICALLY CONTROLLED CNS DYSFUNCTION
WO2012061684A1 (en) 2010-11-05 2012-05-10 The Board Of Trustees Of The Leland Stanford Junior University Upconversion of light for use in optogenetic methods
CA2816990A1 (en) 2010-11-05 2012-05-10 The Board Of Trustees Of The Leland Stanford Junior University Stabilized step function opsin proteins and methods of using the same
ES2716819T3 (es) 2010-11-05 2019-06-17 Univ Leland Stanford Junior Opsinas quiméricas activadas por luz y métodos de uso de las mismas
AU2011323228B2 (en) 2010-11-05 2016-11-10 The Board Of Trustees Of The Leland Stanford Junior University Control and characterization of memory function
CN103476456B (zh) 2010-11-05 2017-10-03 斯坦福大学托管董事会 奖赏相关行为的光遗传学控制
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
US9365628B2 (en) 2011-12-16 2016-06-14 The Board Of Trustees Of The Leland Stanford Junior University Opsin polypeptides and methods of use thereof
WO2013126521A1 (en) 2012-02-21 2013-08-29 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods for treating neurogenic disorders of the pelvic floor
US20150183850A1 (en) * 2012-05-18 2015-07-02 University Of Iowa Research Foundation Methods and compositions for treating amyloid deposits
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
EP2968997B1 (en) 2013-03-15 2019-06-26 The Board of Trustees of the Leland Stanford Junior University Optogenetic control of behavioral state
EP2991491B1 (en) 2013-04-29 2019-12-25 The Board of Trustees of the Leland Stanford Junior University Devices, systems and methods for optogenetic modulation of action potentials in target cells
AU2013388083B2 (en) * 2013-05-01 2019-08-22 Genzyme Corporation Compositions and methods for treating spinal muscular atrophy
GB201308917D0 (en) 2013-05-17 2013-07-03 Renishaw Plc Delivery
CN105829538A (zh) 2013-08-14 2016-08-03 小利兰·斯坦福大学托管委员会 用于控制疼痛的组合物和方法
WO2016209654A1 (en) 2015-06-22 2016-12-29 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
WO2019034723A1 (en) * 2017-08-16 2019-02-21 Lgv1 S.R.L. VTFT ISOFORM OF BPIFB4 PROTEIN INTENDED FOR USE IN NEURONAL DISEASES AND INJURIES
CN113755524B (zh) * 2020-06-02 2023-11-03 舒泰神（北京）生物制药股份有限公司 用于治疗脊髓性肌萎缩的腺相关病毒载体及其用途

Family Cites Families (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4025A (en) * 1845-05-01 Feinting press
SE8303626D0 (sv) 1983-06-23 1983-06-23 Kabigen Ab A recombinant plasmid a transformant microorganism, a polydoxyrebonucleotide segment, a process for producing a biologically active protein, and the protein thus produced
US5672344A (en) * 1987-12-30 1997-09-30 The Regents Of The University Of Michigan Viral-mediated gene transfer system
US5773278A (en) * 1991-05-03 1998-06-30 Mount Sinai Medical Center Acid sphingomyelinase gene
ES2249761T3 (es) 1993-06-24 2006-04-01 Advec Inc. Vectores de adenovirus para terapia genica.
DK0797676T3 (da) 1993-10-25 2006-04-18 Canji Inc Rekombinant adenoviral vektor og fremgangsmåder til anvendelse deraf
AU688428B2 (en) * 1993-11-09 1998-03-12 Johns Hopkins University, The Generation of high titers of recombinant AAV vectors
US7252989B1 (en) 1994-04-04 2007-08-07 Board Of Regents, The University Of Texas System Adenovirus supervector system
CA2187626C (en) * 1994-04-13 2009-11-03 Michael G. Kaplitt Aav-mediated delivery of dna to cells of the nervous system
WO1997039789A1 (en) * 1996-04-22 1997-10-30 Medtronic, Inc. Two-stage angled venous cannula
US6544785B1 (en) * 1998-09-14 2003-04-08 Mount Sinai School Of Medicine Of New York University Helper-free rescue of recombinant negative strand RNA viruses
WO2001036603A2 (en) 1999-11-17 2001-05-25 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
US20030165481A1 (en) 2000-02-24 2003-09-04 Hersh Louis B. Amyloid peptide inactivating enzyme to treat Alzheimer's disease
JP2004516016A (ja) * 2000-09-18 2004-06-03 ジェンザイム・コーポレイション ハイブリッドユビキチンプロモーターを含む発現ベクター
US20030050273A1 (en) 2001-08-29 2003-03-13 Keiya Ozawa Compositions and methods for treating neurodegenerative diseases
EP3517134B1 (en) * 2001-12-17 2024-01-17 The Trustees of the University of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences, vectors containing same and uses therefor
US6998118B2 (en) * 2001-12-21 2006-02-14 The Salk Institute For Biological Studies Targeted retrograde gene delivery for neuronal protection
CA2471812C (en) 2001-12-21 2012-09-04 The Salk Institute For Biological Studies Targeted retrograde gene delivery to motor neurons
WO2004098648A1 (en) 2003-05-01 2004-11-18 Genzyme Corporation Gene therapy for neurometabolic disorders
US20040258666A1 (en) * 2003-05-01 2004-12-23 Passini Marco A. Gene therapy for neurometabolic disorders
EP1716870A1 (en) 2005-04-29 2006-11-02 Bracco Imaging S.p.A. MRI contrast agents endowed with concentration independent responsiveness
PT3520823T (pt) * 2005-05-02 2021-09-16 Genzyme Corp Terapia genética para distúrbios neurometabólicos
AU2006242129A1 (en) 2005-05-02 2006-11-09 Genzyme Corporation Gene therapy for spinal cord disorders
PL1986661T3 (pl) 2006-02-08 2019-02-28 Genzyme Corporation Terapia genowa choroby Niemanna-Picka typu A

Also Published As

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

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