PL3058959T3 - Terapia genowa dla zaburzeń neurometabolicznych - Google Patents

Terapia genowa dla zaburzeń neurometabolicznych

Info

Publication number: PL3058959T3
Authority: PL; Poland
Prior art keywords: disorders; disease; nervous system; central nervous; gene therapy
Prior art date: 2005-05-02

Application number

PL16162716T

Other languages

English (en)

Inventor

Marco A. Passini

James Dodge

Gregory R Stewart

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.)

2005-05-02

Filing date

2006-05-02

Publication date

2019-07-31

2006-05-02 Application filed by Genzyme Corporation filed Critical Genzyme Corporation

2019-07-31 Publication of PL3058959T3 publication Critical patent/PL3058959T3/pl

Links

238000001415 gene therapy Methods 0.000 title 1
208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 5
210000003169 central nervous system Anatomy 0.000 abstract 3
108700019146 Transgenes Proteins 0.000 abstract 2
239000000203 mixture Substances 0.000 abstract 2
239000013607 AAV vector Substances 0.000 abstract 1
208000024827 Alzheimer disease Diseases 0.000 abstract 1
208000015439 Lysosomal storage disease Diseases 0.000 abstract 1
230000003376 axonal effect Effects 0.000 abstract 1
201000010099 disease Diseases 0.000 abstract 1
210000002569 neuron Anatomy 0.000 abstract 1
230000001225 therapeutic effect Effects 0.000 abstract 1

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
- A61K48/005—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
- 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
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
- 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
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/06—Antihyperlipidemics
- 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
- 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
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- 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
- 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
- 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

Landscapes

Health & Medical Sciences (AREA)
Life Sciences & Earth Sciences (AREA)
Engineering & Computer Science (AREA)
Genetics & Genomics (AREA)
Chemical & Material Sciences (AREA)
Organic Chemistry (AREA)
Bioinformatics & Cheminformatics (AREA)
General Health & Medical Sciences (AREA)
Biotechnology (AREA)
Biomedical Technology (AREA)
Medicinal Chemistry (AREA)
Zoology (AREA)
Public Health (AREA)
Animal Behavior & Ethology (AREA)
Veterinary Medicine (AREA)
Pharmacology & Pharmacy (AREA)
Molecular Biology (AREA)
Wood Science & Technology (AREA)
General Engineering & Computer Science (AREA)
General Chemical & Material Sciences (AREA)
Chemical Kinetics & Catalysis (AREA)
Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
Biophysics (AREA)
Biochemistry (AREA)
Microbiology (AREA)
Virology (AREA)
Physics & Mathematics (AREA)
Plant Pathology (AREA)
Epidemiology (AREA)
Neurosurgery (AREA)
Neurology (AREA)
Obesity (AREA)
Diabetes (AREA)
Hematology (AREA)
Toxicology (AREA)
Gastroenterology & Hepatology (AREA)
Proteomics, Peptides & Aminoacids (AREA)
Hospice & Palliative Care (AREA)
Psychiatry (AREA)
Physical Education & Sports Medicine (AREA)

PL16162716T 2005-05-02 2006-05-02 Terapia genowa dla zaburzeń neurometabolicznych PL3058959T3 (pl)

Applications Claiming Priority (6)

Application Number	Priority Date	Filing Date	Title
US67705705P	2005-05-02	2005-05-02
US68580805P	2005-05-31	2005-05-31
EP11169633.2A EP2420256B1 (en)	2005-05-02	2006-05-02	Gene therapy for neurometabolic disorders
PCT/US2006/017242 WO2006119458A1 (en)	2005-05-02	2006-05-02	Gene therapy for neurometabolic disorders
EP16162716.1A EP3058959B1 (en)	2005-05-02	2006-05-02	Gene therapy for neurometabolic disorders
EP06759081A EP1879624B1 (en)	2005-05-02	2006-05-02	Gene therapy for neurometabolic disorders

Publications (1)

Publication Number	Publication Date
PL3058959T3 true PL3058959T3 (pl)	2019-07-31

Family

ID=37308324

Family Applications (4)

Application Number	Title	Priority Date	Filing Date
PL06759081T PL1879624T3 (pl)	2005-05-02	2006-05-02	Terapia genowa dla zaburzeń neurometabolicznych
PL19153520T PL3520823T3 (pl)	2005-05-02	2006-05-02	Terapia genowa zaburzeń neurometabolicznych
PL11169633.2T PL2420256T3 (pl)	2005-05-02	2006-05-02	Terapia genowa dla zaburzeń neurometabolicznych
PL16162716T PL3058959T3 (pl)	2005-05-02	2006-05-02	Terapia genowa dla zaburzeń neurometabolicznych

Family Applications Before (3)

Application Number	Title	Priority Date	Filing Date
PL06759081T PL1879624T3 (pl)	2005-05-02	2006-05-02	Terapia genowa dla zaburzeń neurometabolicznych
PL19153520T PL3520823T3 (pl)	2005-05-02	2006-05-02	Terapia genowa zaburzeń neurometabolicznych
PL11169633.2T PL2420256T3 (pl)	2005-05-02	2006-05-02	Terapia genowa dla zaburzeń neurometabolicznych

Country Status (13)

Country	Link
US (2)	US10632213B2 (pl)
EP (4)	EP3520823B1 (pl)
JP (3)	JP5829372B2 (pl)
CN (4)	CN101212988A (pl)
AT (1)	ATE525092T1 (pl)
AU (1)	AU2006243776A1 (pl)
BR (1)	BRPI0611379A2 (pl)
CA (2)	CA2607173C (pl)
ES (1)	ES2887076T3 (pl)
IL (2)	IL187078A (pl)
PL (4)	PL1879624T3 (pl)
PT (4)	PT3058959T (pl)
WO (1)	WO2006119458A1 (pl)

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JOP20190200A1 (ar)	2017-02-28	2019-08-27	Univ Pennsylvania	تركيبات نافعة في معالجة ضمور العضل النخاعي
CN119491003A (zh)	2017-05-05	2025-02-21	沃雅戈治疗公司	治疗亨廷顿病的组合物和方法
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JP7502991B2 (ja)	2017-10-16	2024-06-19	ボイジャーセラピューティクスインコーポレイテッド	筋萎縮性側索硬化症（ａｌｓ）の治療
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2006
- 2006-05-02 AU AU2006243776A patent/AU2006243776A1/en not_active Abandoned
- 2006-05-02 BR BRPI0611379-6A patent/BRPI0611379A2/pt not_active Application Discontinuation
- 2006-05-02 ES ES19153520T patent/ES2887076T3/es active Active
- 2006-05-02 CN CNA2006800237754A patent/CN101212988A/zh active Pending
- 2006-05-02 EP EP19153520.2A patent/EP3520823B1/en active Active
- 2006-05-02 PT PT16162716T patent/PT3058959T/pt unknown
- 2006-05-02 EP EP11169633.2A patent/EP2420256B1/en active Active
- 2006-05-02 JP JP2008510224A patent/JP5829372B2/ja active Active
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Publication number	Publication date
IL239167A0 (en)	2015-07-30
US20200306387A1 (en)	2020-10-01
CN107007842A (zh)	2017-08-04
BRPI0611379A2 (pt)	2010-08-31
CA2607173C (en)	2018-04-24
CA2998603C (en)	2019-07-23
EP2420256B1 (en)	2016-06-22
PT1879624E (pt)	2012-02-02
PT2420256T (pt)	2016-09-13
PT3520823T (pt)	2021-09-16
JP6338560B2 (ja)	2018-06-06
CA2998603A1 (en)	2006-11-09
EP3058959B1 (en)	2019-01-30
US20140356327A9 (en)	2014-12-04
CN101212988A (zh)	2008-07-02
IL187078A (en)	2015-06-30
CN104306986A (zh)	2015-01-28
IL187078A0 (en)	2008-02-09
PL1879624T3 (pl)	2012-02-29
ATE525092T1 (de)	2011-10-15
US10632213B2 (en)	2020-04-28
EP3520823B1 (en)	2021-06-30
EP1879624A1 (en)	2008-01-23
ES2887076T3 (es)	2021-12-21
CN107362371A (zh)	2017-11-21
JP5829372B2 (ja)	2015-12-09
US11957765B2 (en)	2024-04-16
CA2607173A1 (en)	2006-11-09
JP2016041706A (ja)	2016-03-31
EP3058959A1 (en)	2016-08-24
EP1879624B1 (en)	2011-09-21
EP2420256A1 (en)	2012-02-22
EP1879624A4 (en)	2009-03-25
JP2014037418A (ja)	2014-02-27
WO2006119458A1 (en)	2006-11-09
US20080292593A1 (en)	2008-11-27
AU2006243776A1 (en)	2006-11-09
PT3058959T (pt)	2019-05-24
EP3520823A1 (en)	2019-08-07
PL3520823T3 (pl)	2021-11-22
WO2006119458A9 (en)	2007-02-15
JP2008540445A (ja)	2008-11-20
PL2420256T3 (pl)	2016-12-30

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