LT1986661T - Nimano-piko a tipo ligos genų terapija - Google Patents

Nimano-piko a tipo ligos genų terapija

Info

Publication number
LT1986661T
LT1986661T LTEP07763560.5T LT07763560T LT1986661T LT 1986661 T LT1986661 T LT 1986661T LT 07763560 T LT07763560 T LT 07763560T LT 1986661 T LT1986661 T LT 1986661T
Authority
LT
Lithuania
Prior art keywords
niemann
gene therapy
disease type
pick disease
pick
Prior art date
Application number
LTEP07763560.5T
Other languages
English (en)
Lithuanian (lt)
Inventor
Marco Passini
Robin Ziegler
James Dodge
Lamya Shihabuddin
Seng Cheng
Original Assignee
Genzyme Corporation
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 Corporation filed Critical Genzyme Corporation
Publication of LT1986661T publication Critical patent/LT1986661T/lt

Links

Classifications

    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • 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
    • A61K48/0083—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 administration regime
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive 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
    • A61P25/28—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
    • A61P3/00—Drugs for disorders of the metabolism
    • 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
    • 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
    • C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • 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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2207/00—Modified animals
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2500/00—Screening for compounds of potential therapeutic value

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
LTEP07763560.5T 2006-02-08 2007-02-08 Nimano-piko a tipo ligos genų terapija LT1986661T (lt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US77162806P 2006-02-08 2006-02-08
US77236006P 2006-02-09 2006-02-09
PCT/US2007/003388 WO2007092563A2 (en) 2006-02-08 2007-02-08 Gene therapy for niemann-pick disease type a

Publications (1)

Publication Number Publication Date
LT1986661T true LT1986661T (lt) 2018-12-10

Family

ID=38345806

Family Applications (1)

Application Number Title Priority Date Filing Date
LTEP07763560.5T LT1986661T (lt) 2006-02-08 2007-02-08 Nimano-piko a tipo ligos genų terapija

Country Status (16)

Country Link
US (3) US20090117156A1 (OSRAM)
EP (2) EP1986661B1 (OSRAM)
JP (2) JP5706602B2 (OSRAM)
AR (1) AR059371A1 (OSRAM)
BR (1) BRPI0707590A2 (OSRAM)
CY (1) CY1121380T1 (OSRAM)
DK (1) DK1986661T3 (OSRAM)
ES (1) ES2700048T3 (OSRAM)
HU (1) HUE041928T2 (OSRAM)
IL (2) IL193165B (OSRAM)
LT (1) LT1986661T (OSRAM)
MX (3) MX360595B (OSRAM)
PL (1) PL1986661T3 (OSRAM)
PT (1) PT1986661T (OSRAM)
SI (1) SI1986661T1 (OSRAM)
WO (1) WO2007092563A2 (OSRAM)

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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
PT3252161T (pt) * 2007-06-06 2022-02-01 Genzyme Corp Terapia genética para doenças do armazenamento lisossomal
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
EP3540055A1 (en) * 2010-04-23 2019-09-18 University of Massachusetts Cns targeting aav vectors and methods of use thereof
WO2012145646A1 (en) * 2011-04-20 2012-10-26 Miguel Sena-Esteves Methods for the treatment of tay-sachs disease, sandhoff disease, and gmi-gangliosidosis
CA2912678C (en) * 2013-05-15 2023-10-10 Regents Of The University Of Minnesota Adeno-associated virus mediated gene transfer to the central nervous system
RU2738421C2 (ru) 2014-10-21 2020-12-14 Юниверсити Оф Массачусетс Варианты рекомбинантных aav и их применения
ES2764453T3 (es) * 2015-03-17 2020-06-03 Univ Brussel Vrije Sistemas de expresión específica de hígado optimizados para FVIII y FIX
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
IL305449B1 (en) 2016-04-15 2025-09-01 Univ Pennsylvania Gene therapy for the treatment of mucocutaneous type ii diabetes
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US11578340B2 (en) 2016-10-13 2023-02-14 University Of Massachusetts AAV capsid designs
EP3534892A4 (en) * 2016-11-04 2020-05-27 The Children's Hospital of Philadelphia GENE TRANSFER COMPOSITIONS, METHODS AND USE FOR TREATING NEURODEGENERATIVE DISEASES
WO2018156892A1 (en) 2017-02-23 2018-08-30 Adrx, Inc. Peptide inhibitors of transcription factor aggregation
CN110997693A (zh) 2017-06-07 2020-04-10 阿德克斯公司 τ聚集抑制剂
EP3642345A1 (en) 2017-06-20 2020-04-29 The U.S.A. As Represented By The Secretary, Department Of Health And Human Services Codon-optimized human npc1 genes for the treatment of niemann-pick type c1 deficiency and related conditions
SG11202002457RA (en) 2017-09-22 2020-04-29 Univ Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
SG11202009452WA (en) 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
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JP2021519581A (ja) 2018-04-03 2021-08-12 ストライドバイオ，インコーポレイテッド 抗体を回避するウイルスベクター
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BR112021020311A2 (pt) * 2019-04-24 2021-12-14 Univ Oregon Health & Science Promotor, vetor de expressão, capsídeo, aav de fita simples, vírus adenoassociado, métodos para preparar o aav e para intensificar a expressão de gene e célula modificada
KR20220083714A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터
JP2023532923A (ja) * 2020-06-30 2023-08-01 チルドレンズ ナショナル メディカル センター 骨格筋線維の修復を改善するためのリコンビナントヒト酸性スフィンゴミエリナーゼの使用
AR123288A1 (es) 2020-08-19 2022-11-16 Stridebio Inc Vectores de virus adenoasociados para el tratamiento del síndrome de rett
JP2024506860A (ja) * 2021-02-01 2024-02-15 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ニーマンピック病ａ型を治療するための組成物及び方法

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Also Published As

Publication number Publication date
ES2700048T3 (es) 2019-02-13
WO2007092563A2 (en) 2007-08-16
AR059371A1 (es) 2008-03-26
EP3456331B1 (en) 2021-05-26
EP3456331A1 (en) 2019-03-20
JP2013166777A (ja) 2013-08-29
MX360595B (es) 2018-11-09
DK1986661T3 (da) 2019-01-02
PT1986661T (pt) 2018-12-05
IL258587A (en) 2018-06-28
SI1986661T1 (sl) 2018-12-31
PL1986661T3 (pl) 2019-02-28
EP1986661A2 (en) 2008-11-05
IL193165B (en) 2018-04-30
US20230287366A1 (en) 2023-09-14
MX2018013684A (es) 2021-06-15
JP5706602B2 (ja) 2015-04-22
EP1986661B1 (en) 2018-08-29
US20090117156A1 (en) 2009-05-07
CY1121380T1 (el) 2020-05-29
WO2007092563A3 (en) 2007-11-22
BRPI0707590A2 (pt) 2011-05-10
US20200080066A1 (en) 2020-03-12
EP1986661A4 (en) 2012-04-25
JP2009526067A (ja) 2009-07-16
JP5881641B2 (ja) 2016-03-09
MX2018008355A (es) 2021-06-15
HUE041928T2 (hu) 2019-06-28

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