PL1620133T3 - Terapia genowa dla zaburzeń neurometabolicznych - Google Patents

Terapia genowa dla zaburzeń neurometabolicznych

Info

Publication number
PL1620133T3
PL1620133T3 PL04760694T PL04760694T PL1620133T3 PL 1620133 T3 PL1620133 T3 PL 1620133T3 PL 04760694 T PL04760694 T PL 04760694T PL 04760694 T PL04760694 T PL 04760694T PL 1620133 T3 PL1620133 T3 PL 1620133T3
Authority
PL
Poland
Prior art keywords
gene therapy
neurometabolic disorders
neurometabolic
disorders
therapy
Prior art date
Application number
PL04760694T
Other languages
English (en)
Inventor
Marco A Passini
Gregory R Stewart
James Dodge
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 PL1620133T3 publication Critical patent/PL1620133T3/pl

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
    • 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
    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/4886—Metalloendopeptidases (3.4.24), e.g. collagenase
    • 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
    • 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
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/04—Phosphoric diester hydrolases (3.1.4)
    • C12Y301/04003—Phospholipase C (3.1.4.3)
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Biochemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
PL04760694T 2003-05-01 2004-04-30 Terapia genowa dla zaburzeń neurometabolicznych PL1620133T3 (pl)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US46719503P 2003-05-01 2003-05-01
US47572603P 2003-06-04 2003-06-04
PCT/US2004/013685 WO2004098648A1 (en) 2003-05-01 2004-04-30 Gene therapy for neurometabolic disorders
EP04760694.2A EP1620133B1 (en) 2003-05-01 2004-04-30 Gene therapy for neurometabolic disorders

Publications (1)

Publication Number Publication Date
PL1620133T3 true PL1620133T3 (pl) 2016-05-31

Family

ID=33436721

Family Applications (1)

Application Number Title Priority Date Filing Date
PL04760694T PL1620133T3 (pl) 2003-05-01 2004-04-30 Terapia genowa dla zaburzeń neurometabolicznych

Country Status (11)

Country Link
EP (3) EP3513814A1 (pl)
JP (6) JP4979067B2 (pl)
CY (1) CY1117457T1 (pl)
DK (1) DK1620133T3 (pl)
ES (1) ES2562490T3 (pl)
HU (1) HUE026861T2 (pl)
IL (2) IL171635A (pl)
PL (1) PL1620133T3 (pl)
PT (1) PT1620133E (pl)
SI (1) SI1620133T1 (pl)
WO (1) WO2004098648A1 (pl)

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US7829694B2 (en) 2002-11-26 2010-11-09 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
US7605249B2 (en) 2002-11-26 2009-10-20 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
US7618948B2 (en) 2002-11-26 2009-11-17 Medtronic, Inc. Devices, systems and methods for improving and/or cognitive function through brain delivery of siRNA
US7732591B2 (en) 2003-11-25 2010-06-08 Medtronic, Inc. Compositions, devices and methods for treatment of huntington's disease through intracranial delivery of sirna
US7994149B2 (en) 2003-02-03 2011-08-09 Medtronic, Inc. Method for treatment of Huntington's disease through intracranial delivery of sirna
PL1620133T3 (pl) * 2003-05-01 2016-05-31 Genzyme Corp Terapia genowa dla zaburzeń neurometabolicznych
US20040258666A1 (en) 2003-05-01 2004-12-23 Passini Marco A. Gene therapy for neurometabolic disorders
PL1879623T3 (pl) 2005-05-02 2013-03-29 Genzyme Corp Terapia genowa zaburzeń rdzenia kręgowego
CN101212988A (zh) 2005-05-02 2008-07-02 建新公司 神经代谢疾病的基因治疗
US7902352B2 (en) 2005-05-06 2011-03-08 Medtronic, Inc. Isolated nucleic acid duplex for reducing huntington gene expression
US20060257912A1 (en) 2005-05-06 2006-11-16 Medtronic, Inc. Methods and sequences to suppress primate huntington gene expression
US9133517B2 (en) 2005-06-28 2015-09-15 Medtronics, Inc. Methods and sequences to preferentially suppress expression of mutated huntingtin
CN101405008A (zh) * 2006-02-08 2009-04-08 建新公司 A型尼曼-皮克病的基因治疗
US9273356B2 (en) 2006-05-24 2016-03-01 Medtronic, Inc. Methods and kits for linking polymorphic sequences to expanded repeat mutations
US8324367B2 (en) 2006-11-03 2012-12-04 Medtronic, Inc. Compositions and methods for making therapies delivered by viral vectors reversible for safety and allele-specificity
US9375440B2 (en) 2006-11-03 2016-06-28 Medtronic, Inc. Compositions and methods for making therapies delivered by viral vectors reversible for safety and allele-specificity
US7988668B2 (en) 2006-11-21 2011-08-02 Medtronic, Inc. Microsyringe for pre-packaged delivery of pharmaceuticals
US7819842B2 (en) 2006-11-21 2010-10-26 Medtronic, Inc. Chronically implantable guide tube for repeated intermittent delivery of materials or fluids to targeted tissue sites
US11219696B2 (en) 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides using recombinant AAV9
DK3421603T3 (da) 2009-05-02 2022-01-10 Genzyme Corp Genterapi for neurodegenerative forstyrrelser
US20130039888A1 (en) 2011-06-08 2013-02-14 Nationwide Children's Hospital Inc. Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders
PT2879719T (pt) 2012-08-01 2018-10-08 Ohio State Innovation Foundation Administração intratecal do vírus adeno-associado 9 recombinante
KR20240063169A (ko) 2014-11-21 2024-05-10 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 중추 신경계에 표적화된 aav 벡터
CA2967468A1 (en) 2014-12-16 2016-06-23 Board Of Regents Of The University Of Nebraska Gene therapy for juvenile batten disease
US11020443B2 (en) 2015-04-23 2021-06-01 University Of Massachusetts Modulation of AAV vector transgene expression
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
US11504435B2 (en) 2016-06-13 2022-11-22 The University Of North Carolina At Chapel Hill Optimized CLN1 genes and expression cassettes and their use
EP3635009A1 (en) 2017-06-07 2020-04-15 Regeneron Pharmaceuticals, Inc. Compositions and methods for internalizing enzymes
CN111465691A (zh) 2017-10-03 2020-07-28 普利维尔治疗公司 用于溶酶体障碍的基因疗法
JP7254815B2 (ja) 2017-10-03 2023-04-10 プリベイル セラピューティクス，インコーポレーテッド ライソゾーム病の遺伝子治療
KR102709597B1 (ko) 2017-10-03 2024-09-26 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
IL322464A (en) 2018-02-07 2025-09-01 Regeneron Pharma Methods and compositions for administering therapeutic protein
BR112020020836A2 (pt) 2018-04-13 2021-01-19 University Of Massachusetts Vetores de aav bicistrônicos codificando subunidades alfa e beta de hexosaminidase e usos dos mesmos
BR112020021962A2 (pt) 2018-04-30 2021-01-26 Amicus Therapeutics, Inc. construtos para terapia gênica e métodos de uso
EP3793591A1 (en) 2018-05-17 2021-03-24 Regeneron Pharmaceuticals, Inc. Anti-cd63 antibodies, conjugates, and uses thereof
CN113166780A (zh) 2018-10-05 2021-07-23 马萨诸塞大学 用于治疗gm1和gm2神经节苷脂贮积病的raav载体
US11097015B2 (en) 2018-10-10 2021-08-24 Amicus Therapeutics, Inc. Disulfide bond stabilized polypeptide compositions and methods of use
WO2020117898A1 (en) 2018-12-05 2020-06-11 Abeona Therapeutics Inc. Recombinant adeno-associated viral vector for gene delivery
CN114026115A (zh) 2019-04-10 2022-02-08 普利维尔治疗公司 用于溶酶体病症的基因疗法
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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US5773278A (en) 1991-05-03 1998-06-30 Mount Sinai Medical Center Acid sphingomyelinase gene
ES2216005T3 (es) 1993-11-09 2004-10-16 Targeted Genetics Corporation Produccion de titulos elevados de vectores de aav recombinantes.
EP0755454B1 (en) 1994-04-13 2008-02-13 The Rockefeller University Aav-mediated delivery of dna to cells of the nervous system
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
PL1620133T3 (pl) * 2003-05-01 2016-05-31 Genzyme Corp Terapia genowa dla zaburzeń neurometabolicznych

Also Published As

Publication number Publication date
JP2015178519A (ja) 2015-10-08
ES2562490T3 (es) 2016-03-04
JP2011251992A (ja) 2011-12-15
JP2017008118A (ja) 2017-01-12
JP6312771B2 (ja) 2018-04-18
EP2269658A1 (en) 2011-01-05
SI1620133T1 (sl) 2016-03-31
JP2014040433A (ja) 2014-03-06
IL252829A0 (en) 2017-08-31
JP2006525363A (ja) 2006-11-09
IL171635A (en) 2017-06-29
JP4979067B2 (ja) 2012-07-18
WO2004098648A1 (en) 2004-11-18
DK1620133T3 (en) 2016-02-29
JP5841574B2 (ja) 2016-01-13
JP2018058901A (ja) 2018-04-12
EP2269658B1 (en) 2018-12-19
EP3513814A1 (en) 2019-07-24
EP1620133A1 (en) 2006-02-01
EP1620133B1 (en) 2015-12-09
PT1620133E (pt) 2016-03-31
JP6170519B2 (ja) 2017-07-26
CY1117457T1 (el) 2017-04-26
HUE026861T2 (en) 2016-07-28

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