HUE044182T2 - Génterápia amiotrófiás laterális szklerózis és más gerincvelõ-rendellenességek kezelésére - Google Patents

Génterápia amiotrófiás laterális szklerózis és más gerincvelõ-rendellenességek kezelésére

Info

Publication number
HUE044182T2
HUE044182T2 HUE12152550A HUE044182T2 HU E044182 T2 HUE044182 T2 HU E044182T2 HU E12152550 A HUE12152550 A HU E12152550A HU E044182 T2 HUE044182 T2 HU E044182T2
Authority
HU
Hungary
Prior art keywords
gene therapy
spinal cord
lateral sclerosis
amyotrophic lateral
cord disorders
Prior art date
Application number
Other languages
English (en)
Inventor
James Dodge
Lamya Shihabuddin
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 HUE044182T2 publication Critical patent/HUE044182T2/hu

Links

Classifications

    • 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 (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
HUE12152550 2006-06-07 2007-06-07 Génterápia amiotrófiás laterális szklerózis és más gerincvelõ-rendellenességek kezelésére HUE044182T2 (hu)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US81141906P 2006-06-07 2006-06-07

Publications (1)

Publication Number Publication Date
HUE044182T2 true HUE044182T2 (hu) 2019-10-28

Family

ID=38832369

Family Applications (2)

Application Number Title Priority Date Filing Date
HUE12152550 HUE044182T2 (hu) 2006-06-07 2007-06-07 Génterápia amiotrófiás laterális szklerózis és más gerincvelõ-rendellenességek kezelésére
HUE07795835A HUE031156T2 (hu) 2006-06-07 2007-06-07 Génterápia amiotróf laterális szklerózis és egyéb gerincvelõ-betegségek ellen

Family Applications After (1)

Application Number Title Priority Date Filing Date
HUE07795835A HUE031156T2 (hu) 2006-06-07 2007-06-07 Génterápia amiotróf laterális szklerózis és egyéb gerincvelõ-betegségek ellen

Country Status (19)

Country Link
US (4) US9034836B2 (hu)
EP (3) EP2489733B1 (hu)
JP (6) JP2009539847A (hu)
CN (1) CN101460621B (hu)
AR (2) AR061282A1 (hu)
BR (1) BRPI0711965A2 (hu)
CA (2) CA3139726A1 (hu)
CY (1) CY1118555T1 (hu)
ES (2) ES2725552T3 (hu)
HU (2) HUE044182T2 (hu)
IL (2) IL195647A (hu)
LT (2) LT2029742T (hu)
MX (4) MX364444B (hu)
PL (2) PL2489733T3 (hu)
PT (2) PT2029742T (hu)
RU (1) RU2008152821A (hu)
SI (2) SI2489733T1 (hu)
TR (1) TR201906106T4 (hu)
WO (1) WO2007146046A2 (hu)

Families Citing this family (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2725552T3 (es) 2006-06-07 2019-09-24 Genzyme Corp Terapia génica para esclerosis lateral amiotrófica y otros trastornos de la médula espinal
CN102006882B (zh) * 2008-01-16 2017-06-06 神经动力公司 利用颗粒体蛋白前体(pgrn)治疗神经变性疾病
CN107349209A (zh) 2008-10-31 2017-11-17 神经机能生命科学公司 神经毒性甾醇糖苷
ES2686504T3 (es) 2009-05-02 2018-10-18 Genzyme Corporation Terapia génica para trastornos neurodegenerativos
CA2818253A1 (en) * 2010-11-16 2012-05-24 Denis G. Kay Method for increasing neprilysin expression and activity
CN103083667A (zh) * 2011-11-01 2013-05-08 北京清美联创干细胞科技有限公司 嗅鞘细胞增殖试剂的连续给药方案
US9663564B2 (en) 2013-03-15 2017-05-30 The Regents Of The University Of California Vectors and methods to treat ischemia
US20140065110A1 (en) * 2012-08-31 2014-03-06 The Regents Of The University Of California Genetically modified msc and therapeutic methods
WO2014065341A1 (ja) 2012-10-24 2014-05-01 第一三共株式会社 筋萎縮性側索硬化症治療剤
JP6232547B2 (ja) * 2012-11-28 2017-11-22 国立大学法人名古屋大学 聴覚障害又は小脳性運動失調症の予防・治療剤
WO2014178863A1 (en) * 2013-05-01 2014-11-06 Genzyme Corporation Compositions and methods for treating spinal muscular atrophy
ES2859605T3 (es) * 2013-07-26 2021-10-04 Univ Iowa Res Found Procedimientos y composiciones para tratar enfermedades del cerebro
KR101548989B1 (ko) 2013-07-30 2015-09-01 광주과학기술원 ＳｉｒＴ１을 이용한 관절 질환의 예방 또는 치료용 물질의 스크리닝 방법
US9119832B2 (en) 2014-02-05 2015-09-01 The Regents Of The University Of California Methods of treating mild brain injury
DE102014207498A1 (de) * 2014-04-17 2015-10-22 Universitätsklinikum Hamburg-Eppendorf Viraler Vektor für den zielgerichteten Gentransfer in Gehirn und Rückenmark
CN112553229A (zh) 2014-11-05 2021-03-26 沃雅戈治疗公司 用于治疗帕金森病的aadc多核苷酸
KR20180014051A (ko) * 2015-05-28 2018-02-07 코넬 유니버시티 혈관 부종 치료제로서의 아데노-관련 바이러스 매개 c1e1의 전달
CL2015003024A1 (es) 2015-10-09 2016-05-13 Univ Chile Método de tratamiento genético utilizando el virus aav-xbp1s/gfp, y su uso en la prevención y tratamiento de la esclerosis lateral amiotrofica.
US10357543B2 (en) 2015-11-16 2019-07-23 Ohio State Innovation Foundation Methods and compositions for treating disorders and diseases using Survival Motor Neuron (SMN) protein
AU2017227803A1 (en) * 2016-03-02 2018-10-25 Defne AMADO Therapy for frontotemporal dementia
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
SG10201912976WA (en) 2017-02-28 2020-02-27 Univ Pennsylvania Adeno-associated virus (aav) clade f vector and uses therefor
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CN112553210A (zh) 2017-10-03 2021-03-26 普利维尔治疗公司 用于溶酶体障碍的基因疗法
US11802294B2 (en) 2017-10-03 2023-10-31 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
WO2019073299A1 (en) * 2017-10-10 2019-04-18 Medicenna Therapeutics, Inc. IL-4 FUSION FORMULATIONS FOR THE TREATMENT OF CENTRAL NERVOUS SYSTEM (CNS) TUMORS
RU2731514C2 (ru) * 2018-12-21 2020-09-03 Селл энд Джин Терапи Лтд Генотерапевтический ДНК-вектор на основе генотерапевтического ДНК-вектора VTvaf17, несущий целевой ген, выбранный из группы генов SHH, CTNNB1, NOG, WNT7A для повышения уровня экспрессии этих целевых генов, способ его получения и применения, штамм Escherichia coli SCS110-AF/VTvaf17-SHH, или Escherichia coli SCS110-AF/VTvaf17-CTNNB1, или Escherichia coli SCS110-AF/VTvaf17-NOG, или Escherichia coli SCS110-AF/VTvaf17-WNT7A, несущий генотерапевтический ДНК-вектор, способ его получения, способ производства в промышленных масштабах генотерапевтического ДНК-вектора
TW202045730A (zh) * 2019-02-22 2020-12-16 賓州大學委員會 用於治療grn相關的成年發病型神經系統退化症之重組腺相關病毒
SG11202111195VA (en) * 2019-04-12 2021-11-29 Encoded Therapeutics Inc Compositions and methods for administration of therapeutics
US20220235373A1 (en) * 2019-10-12 2022-07-28 Shenzhen University Agent that enables sirt7 gene expression and the use thereof
CN114981297A (zh) 2019-12-23 2022-08-30 戴纳立制药公司 颗粒蛋白前体变体
CN115323002A (zh) * 2022-07-28 2022-11-11 深圳先进技术研究院 一种基因递送系统在从脑部逆行递送基因到脊髓神经元中的应用

Family Cites Families (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
AU687829B2 (en) 1993-06-24 1998-03-05 Advec, Inc. Adenovirus vectors for gene therapy
SK283703B6 (sk) 1993-10-25 2003-12-02 Canji, Inc. Rekombinantný adenovírusový vektor a jeho použitie
DK0733103T3 (da) 1993-11-09 2004-07-12 Univ Johns Hopkins Fremstilling af höje titre af rekombinante AAV vektorer
US7252989B1 (en) 1994-04-04 2007-08-07 Board Of Regents, The University Of Texas System Adenovirus supervector system
ATE386131T1 (de) 1994-04-13 2008-03-15 Univ Rockefeller Aav-vermittelte überbringung von dna in zellen des nervensystems
US5882914A (en) 1995-06-06 1999-03-16 The Johns Hopkins University School Of Medicine Nucleic acids encoding the hypoxia inducible factor-1
WO1997039789A1 (en) * 1996-04-22 1997-10-30 Medtronic, Inc. Two-stage angled venous cannula
US20040076613A1 (en) * 2000-11-03 2004-04-22 Nicholas Mazarakis Vector system
CA2311643C (en) 1997-12-04 2009-04-07 Genzyme Corporation Compositions and methods for inducing gene expression
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
US6468524B1 (en) * 2000-03-22 2002-10-22 The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services AAV4 vector and uses thereof
US7037493B2 (en) * 2000-05-01 2006-05-02 Cornell Research Foundation, Inc. Method of inducing neuronal production in the brain and spinal cord
EP1286703B1 (en) * 2000-06-01 2009-08-05 University Of North Carolina At Chapel Hill Methods and compounds for controlled release of recombinant parvovirus vectors
DE60115070T2 (de) 2000-09-18 2006-07-27 Genzyme Corp., Cambridge Expressionsvektoren mit hybriden ubiquitin-promotoren
GB0103998D0 (en) * 2001-02-19 2001-04-04 King S College London Method
JP2002322068A (ja) 2001-02-26 2002-11-08 Japan Science & Technology Corp 血管再生促進剤
CA2452980A1 (en) * 2001-07-03 2003-01-16 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods of administering vectors to synaptically connected neurons
IL162630A0 (en) 2001-12-21 2005-11-20 Salk Inst For Biological Studi A composition containing a viral vector containinga heterologous gene to be transduced into a neuron
US7241444B2 (en) * 2002-01-18 2007-07-10 Pierre Fabre Medicament Anti-IGF-IR antibodies and uses thereof
WO2003070189A2 (en) * 2002-02-15 2003-08-28 Cornell Research Foundation, Inc. Enhancing neurotrophin-induced neurogenesis by endogenous neural progenitor cells by concurrent overexpression of brain derived neurotrophic factor and an inhibitor of a pro-gliogenic bone morphogenetic protein
AU2003268484A1 (en) * 2002-09-06 2004-03-29 The General Hospital Corporation Delivery of therapeutics to the brain and spinal cord
JPWO2006035741A1 (ja) * 2004-09-29 2008-05-15 伸弥 山中 Ｅｓ細胞特異的発現遺伝子及びその利用
JP5103615B2 (ja) 2004-11-18 2012-12-19 国立大学法人 東京医科歯科大学 新規タンパク質及びそれを利用したポリグルタミン病等の神経変性疾患の予防・治療薬
AU2006243776A1 (en) 2005-05-02 2006-11-09 Genzyme Corporation Gene therapy for neurometabolic disorders
PL1879623T3 (pl) * 2005-05-02 2013-03-29 Genzyme Corp Terapia genowa zaburzeń rdzenia kręgowego
ES2725552T3 (es) 2006-06-07 2019-09-24 Genzyme Corp Terapia génica para esclerosis lateral amiotrófica y otros trastornos de la médula espinal
JP5992049B2 (ja) 2011-11-01 2016-09-14 レスバーロジックス コーポレイション 置換されたキナゾリノンのための経口速放性製剤
EP3536795A3 (en) 2012-06-21 2019-12-11 Association Institut de Myologie Widespread gene expression

Also Published As

Publication number Publication date
ES2725552T3 (es) 2019-09-24
JP2020079234A (ja) 2020-05-28
JP6606215B2 (ja) 2019-11-13
EP2029742A4 (en) 2009-09-30
LT2029742T (lt) 2016-11-10
US11554161B2 (en) 2023-01-17
CA2654292A1 (en) 2007-12-21
EP2489733B1 (en) 2019-02-13
US20230364200A1 (en) 2023-11-16
IL241890A0 (en) 2015-11-30
CA2654292C (en) 2022-01-11
WO2007146046A3 (en) 2008-05-22
TR201906106T4 (tr) 2019-05-21
SI2029742T1 (sl) 2016-11-30
EP2029742B1 (en) 2016-07-20
EP2489733A3 (en) 2012-11-14
JP2018118995A (ja) 2018-08-02
JP2017078071A (ja) 2017-04-27
PT2489733T (pt) 2019-05-27
JP2014012697A (ja) 2014-01-23
WO2007146046A2 (en) 2007-12-21
AR061282A1 (es) 2008-08-20
MX2019004843A (es) 2021-06-15
PT2029742T (pt) 2016-09-09
MX2023001965A (es) 2023-02-23
US20160067312A1 (en) 2016-03-10
ES2596885T3 (es) 2017-01-12
EP3540054A2 (en) 2019-09-18
CA3139726A1 (en) 2007-12-21
HUE031156T2 (hu) 2017-06-28
MX364444B (es) 2019-04-26
IL195647A0 (en) 2009-09-01
US9034836B2 (en) 2015-05-19
AR109171A2 (es) 2018-11-07
LT2489733T (lt) 2019-05-27
EP3540054A3 (en) 2019-10-09
SI2489733T1 (sl) 2019-06-28
CN101460621A (zh) 2009-06-17
IL195647A (en) 2015-10-29
EP2489733A2 (en) 2012-08-22
PL2029742T3 (pl) 2017-08-31
EP2029742A2 (en) 2009-03-04
US20100267812A1 (en) 2010-10-21
MX2008015530A (es) 2009-02-06
PL2489733T3 (pl) 2019-08-30
RU2008152821A (ru) 2010-07-20
BRPI0711965A2 (pt) 2012-01-24
JP6320489B2 (ja) 2018-05-09
JP2015120719A (ja) 2015-07-02
CN101460621B (zh) 2016-01-27
JP2009539847A (ja) 2009-11-19
CY1118555T1 (el) 2017-07-12
US20200101138A1 (en) 2020-04-02

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