PL1620133T3 - Terapia genowa dla zaburzeń neurometabolicznych - Google Patents

Terapia genowa dla zaburzeń neurometabolicznych

Info

Publication number: PL1620133T3
Authority: PL; Poland
Prior art keywords: gene therapy; neurometabolic disorders; neurometabolic; disorders; therapy
Prior art date: 2003-05-01

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.)

2003-05-01

Filing date

2004-04-30

Publication date

2016-05-31

2004-04-30 Application filed by Genzyme Corp filed Critical Genzyme Corp

2016-05-31 Publication of PL1620133T3 publication Critical patent/PL1620133T3/pl

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

Families Citing this family (35)

* Cited by examiner, † Cited by third party
Publication number	Priority date	Publication date	Assignee	Title
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
US20040258666A1 (en)	2003-05-01	2004-12-23	Passini Marco A.	Gene therapy for neurometabolic disorders
PL1620133T3 (pl) *	2003-05-01	2016-05-31	Genzyme Corp	Terapia genowa dla zaburzeń neurometabolicznych
WO2006119341A2 (en)	2005-05-02	2006-11-09	Genzyme Corporation	Gene therapy for spinal cord disorders
ES2887076T3 (es) *	2005-05-02	2021-12-21	Genzyme Corp	Terapia génica para trastornos neurometabólicos
WO2006121960A2 (en)	2005-05-06	2006-11-16	Medtronic, Inc.	Methods and sequences to suppress primate huntington gene expression
US7902352B2 (en)	2005-05-06	2011-03-08	Medtronic, Inc.	Isolated nucleic acid duplex for reducing huntington gene expression
US9133517B2 (en)	2005-06-28	2015-09-15	Medtronics, Inc.	Methods and sequences to preferentially suppress expression of mutated huntingtin
CN106729752A (zh) *	2006-02-08	2017-05-31	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
CN107083400A (zh)	2009-05-02	2017-08-22	建新公司	神经退行性疾病的基因治疗
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
EP3769789A1 (en)	2012-08-01	2021-01-27	Nationwide Children's Hospital	Intrathecal delivery of recombinant adeno-associated virus 9
WO2016081811A1 (en)	2014-11-21	2016-05-26	The University Of North Carolina At Chapel Hill	Aav vectors targeted to the central nervous system
EP3233131A1 (en)	2014-12-16	2017-10-25	Board of Regents of the University of Nebraska	Gene therapy for juvenile batten disease
CA3019315A1 (en)	2015-04-23	2016-10-27	University Of Massachusetts	Modulation of aav vector transgene expression
GB201508025D0 (en)	2015-05-11	2015-06-24	Ucl Business Plc	Fabry disease gene therapy
MX2018015461A (es)	2016-06-13	2019-05-27	Univ North Carolina Chapel Hill	Genes cln1 optimizados y cassettes de expresion y su uso.
IL271193B2 (en)	2017-06-07	2025-01-01	Regeneron Pharma	Preparations and methods for internalizing enzymes
KR102709597B1 (ko)	2017-10-03	2024-09-26	프리베일 테라퓨틱스, 인크.	리소좀 장애를 위한 유전자 요법
KR102693318B1 (ko)	2017-10-03	2024-08-07	프리베일 테라퓨틱스, 인크.	리소좀 장애를 위한 유전자 요법
EP3692151A4 (en)	2017-10-03	2021-07-14	Prevail Therapeutics, Inc.	GENE THERAPIES FOR LYSOSOMAL DISORDERS
US10874750B2 (en) *	2018-04-30	2020-12-29	Amicus Therapeutics, Inc.	Gene therapy constructs and methods of use
WO2019222663A1 (en)	2018-05-17	2019-11-21	Regeneron Pharmaceuticals, Inc.	Anti-cd63 antibodies, conjugates, and uses thereof
SG11202103640VA (en)	2018-10-10	2021-05-28	Amicus Therapeutics Inc	Disulfide bond stabilized polypeptide compositions and methods of use
WO2020210713A1 (en)	2019-04-10	2020-10-15	Prevail Therapeutics, Inc.	Gene therapies for lysosomal disorders

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number	Priority date	Publication date	Assignee	Title
US5773278A (en)	1991-05-03	1998-06-30	Mount Sinai Medical Center	Acid sphingomyelinase gene
JPH09509564A (ja)	1993-11-09	1997-09-30	ターゲテッドジェネティックスコーポレイション	高力価組換えａａｖベクターの生成
ATE386131T1 (de)	1994-04-13	2008-03-15	Univ Rockefeller	Aav-vermittelte überbringung von dna in zellen des nervensystems
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

2004
- 2004-04-30 PL PL04760694T patent/PL1620133T3/pl unknown
- 2004-04-30 EP EP18212196.2A patent/EP3513814A1/en not_active Withdrawn
- 2004-04-30 HU HUE04760694A patent/HUE026861T2/en unknown
- 2004-04-30 DK DK04760694.2T patent/DK1620133T3/en active
- 2004-04-30 EP EP10179332.1A patent/EP2269658B1/en not_active Expired - Lifetime
- 2004-04-30 ES ES04760694.2T patent/ES2562490T3/es not_active Expired - Lifetime
- 2004-04-30 JP JP2006514239A patent/JP4979067B2/ja not_active Expired - Fee Related
- 2004-04-30 WO PCT/US2004/013685 patent/WO2004098648A1/en active Application Filing
- 2004-04-30 SI SI200432298T patent/SI1620133T1/sl unknown
- 2004-04-30 EP EP04760694.2A patent/EP1620133B1/en not_active Expired - Lifetime
- 2004-04-30 PT PT47606942T patent/PT1620133E/pt unknown
2005
- 2005-10-27 IL IL171635A patent/IL171635A/en active IP Right Grant
2011
- 2011-08-01 JP JP2011168292A patent/JP2011251992A/ja active Pending
2013
- 2013-09-24 JP JP2013196398A patent/JP5841574B2/ja not_active Expired - Fee Related
2015
- 2015-05-26 JP JP2015106021A patent/JP6170519B2/ja not_active Expired - Fee Related
2016
- 2016-03-09 CY CY20161100204T patent/CY1117457T1/el unknown
- 2016-10-20 JP JP2016205651A patent/JP6312771B2/ja not_active Expired - Lifetime
2017
- 2017-06-11 IL IL252829A patent/IL252829A0/en unknown
2018
- 2018-01-05 JP JP2018000518A patent/JP2018058901A/ja active Pending

Also Published As

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

Publication	Publication Date	Title
PT1620133E (pt)	2016-03-31	Terapia genética para distúrbios neurometabólicos
HK1152952A1 (en)	2012-03-16	Combination therapy for cell disorders
IL237658A0 (en)	2015-04-30	Gene therapy for spinal cord disorders
EP1648560A4 (en)	2015-10-28	VAGAL STIMULATION FOR ANTI-EMBOLIC THERAPY
GB0318428D0 (en)	2003-09-10	Gene drivers
IL169332A0 (en)	2007-07-04	Compounds for the treatment of metabolic disorders
EP1490074A4 (en)	2006-05-31	MIXED CELL GENE THERAPY
AU2003293099A8 (en)	2004-06-23	Treatment of dna damage related disorders
GB0406728D0 (en)	2004-04-28	Gene therapy
GB0327384D0 (en)	2003-12-31	Gene therapy
ZA200707236B (en)	2008-11-26	Microorganisms for therapy
EP1532987A4 (en)	2005-12-28	AGENTS FOR GENE THERAPY IN CEREBROVASCULAR DISEASES
SI1879623T1 (sl)	2013-01-31	Genska terapija za motnje hrbtenjače
SI1631313T1 (sl)	2015-06-30	Kombinirana terapija za B-celične motnje
EP1786917A4 (en)	2008-05-28	OPTIMIZED INTERFERON BETA-GEN
IL164850A0 (en)	2005-12-18	Gene therapy for kidney diseases
EP1693069A4 (en)	2008-01-30	MEANS FOR THE TREATMENT OF SOLID TUMORS
AU2003294705A8 (en)	2004-06-07	Ntsm gene
GB0223424D0 (en)	2002-11-13	Disease-associated gene
AU2003260820A8 (en)	2004-02-25	Nephritis treatment by gene therapy
EP1646282A4 (en)	2006-10-11	ANTICANCER GENETIC VACCINES
GB0326598D0 (en)	2003-12-17	Gene
GB0313773D0 (en)	2003-07-23	Gene
EP1370138A4 (en)	2004-08-18	Gene therapy for nerve tissue
GB0325021D0 (en)	2003-12-03	Therapeutic combinations