ES2562490T3 - Terapia génica para trastornos neurometabólicos - Google Patents
Terapia génica para trastornos neurometabólicos Download PDFInfo
- Publication number
- ES2562490T3 ES2562490T3 ES04760694.2T ES04760694T ES2562490T3 ES 2562490 T3 ES2562490 T3 ES 2562490T3 ES 04760694 T ES04760694 T ES 04760694T ES 2562490 T3 ES2562490 T3 ES 2562490T3
- Authority
- ES
- Spain
- Prior art keywords
- composition
- site
- use according
- aav
- disease
- Prior art date
- Legal status (The legal status 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 status listed.)
- Expired - Lifetime
Links
- 238000001415 gene therapy Methods 0.000 title 1
- 239000000203 mixture Substances 0.000 claims abstract 23
- 230000002132 lysosomal effect Effects 0.000 claims abstract 15
- 102000004157 Hydrolases Human genes 0.000 claims abstract 9
- 108090000604 Hydrolases Proteins 0.000 claims abstract 9
- 201000010099 disease Diseases 0.000 claims abstract 8
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims abstract 8
- 210000002569 neuron Anatomy 0.000 claims abstract 6
- 239000013607 AAV vector Substances 0.000 claims abstract 4
- 108700019146 Transgenes Proteins 0.000 claims abstract 3
- 230000008021 deposition Effects 0.000 claims abstract 3
- 239000013598 vector Substances 0.000 claims abstract 2
- 239000002253 acid Substances 0.000 claims 7
- 241000702421 Dependoparvovirus Species 0.000 claims 4
- 210000004556 brain Anatomy 0.000 claims 4
- 108010061312 Sphingomyelin Phosphodiesterase Proteins 0.000 claims 3
- 102000010126 acid sphingomyelin phosphodiesterase activity proteins Human genes 0.000 claims 3
- 210000004027 cell Anatomy 0.000 claims 3
- 210000003169 central nervous system Anatomy 0.000 claims 3
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 2
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 2
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 2
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 2
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 2
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 2
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 2
- ZSLZBFCDCINBPY-ZSJPKINUSA-N acetyl-CoA Chemical compound O[C@@H]1[C@H](OP(O)(O)=O)[C@@H](COP(O)(=O)OP(O)(=O)OCC(C)(C)[C@@H](O)C(=O)NCCC(=O)NCCSC(=O)C)O[C@H]1N1C2=NC=NC(N)=C2N=C1 ZSLZBFCDCINBPY-ZSJPKINUSA-N 0.000 claims 2
- 230000003376 axonal effect Effects 0.000 claims 2
- 210000001638 cerebellum Anatomy 0.000 claims 2
- 210000001320 hippocampus Anatomy 0.000 claims 2
- 210000001577 neostriatum Anatomy 0.000 claims 2
- 102100022146 Arylsulfatase A Human genes 0.000 claims 1
- 102100031491 Arylsulfatase B Human genes 0.000 claims 1
- 108010023546 Aspartylglucosylaminase Proteins 0.000 claims 1
- 101100080277 Caenorhabditis elegans ncr-1 gene Proteins 0.000 claims 1
- 102000005572 Cathepsin A Human genes 0.000 claims 1
- 108010059081 Cathepsin A Proteins 0.000 claims 1
- 108090000751 Ceramidases Proteins 0.000 claims 1
- 102000004201 Ceramidases Human genes 0.000 claims 1
- 108010036867 Cerebroside-Sulfatase Proteins 0.000 claims 1
- 102000003779 Dipeptidyl-peptidases and tripeptidyl-peptidases Human genes 0.000 claims 1
- 108090000194 Dipeptidyl-peptidases and tripeptidyl-peptidases Proteins 0.000 claims 1
- 102100028496 Galactocerebrosidase Human genes 0.000 claims 1
- 108010042681 Galactosylceramidase Proteins 0.000 claims 1
- 102000004547 Glucosylceramidase Human genes 0.000 claims 1
- 108010017544 Glucosylceramidase Proteins 0.000 claims 1
- 102100029199 Iduronate 2-sulfatase Human genes 0.000 claims 1
- 101710096421 Iduronate 2-sulfatase Proteins 0.000 claims 1
- 102000004882 Lipase Human genes 0.000 claims 1
- 108090001060 Lipase Proteins 0.000 claims 1
- 239000004367 Lipase Substances 0.000 claims 1
- 102100021003 N(4)-(beta-N-acetylglucosaminyl)-L-asparaginase Human genes 0.000 claims 1
- 108010027520 N-Acetylgalactosamine-4-Sulfatase Proteins 0.000 claims 1
- 108010023320 N-acetylglucosamine-6-sulfatase Proteins 0.000 claims 1
- 101710202061 N-acetyltransferase Proteins 0.000 claims 1
- 101100459404 Neurospora crassa (strain ATCC 24698 / 74-OR23-1A / CBS 708.71 / DSM 1257 / FGSC 987) npc-1 gene Proteins 0.000 claims 1
- 102000005262 Sulfatase Human genes 0.000 claims 1
- 102000005488 Thioesterase Human genes 0.000 claims 1
- 108010044965 UDP-N-acetylglucosamine-lysosomal-enzyme N-acetylglucosaminephosphotransferase Proteins 0.000 claims 1
- 210000003050 axon Anatomy 0.000 claims 1
- 210000003710 cerebral cortex Anatomy 0.000 claims 1
- 230000001010 compromised effect Effects 0.000 claims 1
- 210000001947 dentate gyrus Anatomy 0.000 claims 1
- 210000001353 entorhinal cortex Anatomy 0.000 claims 1
- 210000001652 frontal lobe Anatomy 0.000 claims 1
- 108010089932 heparan sulfate sulfatase Proteins 0.000 claims 1
- 210000003016 hypothalamus Anatomy 0.000 claims 1
- 238000002347 injection Methods 0.000 claims 1
- 239000007924 injection Substances 0.000 claims 1
- 235000019421 lipase Nutrition 0.000 claims 1
- 210000001767 medulla oblongata Anatomy 0.000 claims 1
- 210000004498 neuroglial cell Anatomy 0.000 claims 1
- 210000000869 occipital lobe Anatomy 0.000 claims 1
- 125000001312 palmitoyl group Chemical group O=C([*])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])[H] 0.000 claims 1
- 210000001152 parietal lobe Anatomy 0.000 claims 1
- 230000007170 pathology Effects 0.000 claims 1
- 210000001176 projection neuron Anatomy 0.000 claims 1
- 230000001681 protective effect Effects 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 210000003900 secondary neuron Anatomy 0.000 claims 1
- 210000000278 spinal cord Anatomy 0.000 claims 1
- 239000000126 substance Substances 0.000 claims 1
- 108060007951 sulfatase Proteins 0.000 claims 1
- 210000003478 temporal lobe Anatomy 0.000 claims 1
- 210000001103 thalamus Anatomy 0.000 claims 1
- 108020002982 thioesterase Proteins 0.000 claims 1
- 241000124008 Mammalia Species 0.000 abstract 2
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)
- Neurology (AREA)
- Neurosurgery (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Epidemiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Diabetes (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US46719503P | 2003-05-01 | 2003-05-01 | |
| US467195P | 2003-05-01 | ||
| US47572603P | 2003-06-04 | 2003-06-04 | |
| US475726P | 2003-06-04 | ||
| PCT/US2004/013685 WO2004098648A1 (en) | 2003-05-01 | 2004-04-30 | Gene therapy for neurometabolic disorders |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ES2562490T3 true ES2562490T3 (es) | 2016-03-04 |
Family
ID=33436721
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ES04760694.2T Expired - Lifetime ES2562490T3 (es) | 2003-05-01 | 2004-04-30 | Terapia génica para trastornos neurometabólicos |
Country Status (11)
| Country | Link |
|---|---|
| EP (3) | EP1620133B1 (https=) |
| JP (6) | JP4979067B2 (https=) |
| CY (1) | CY1117457T1 (https=) |
| DK (1) | DK1620133T3 (https=) |
| ES (1) | ES2562490T3 (https=) |
| HU (1) | HUE026861T2 (https=) |
| IL (2) | IL171635A (https=) |
| PL (1) | PL1620133T3 (https=) |
| PT (1) | PT1620133E (https=) |
| SI (1) | SI1620133T1 (https=) |
| WO (1) | WO2004098648A1 (https=) |
Families Citing this family (40)
| 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 |
| US7994149B2 (en) | 2003-02-03 | 2011-08-09 | Medtronic, Inc. | Method for treatment of Huntington's disease through intracranial 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 |
| US20040258666A1 (en) | 2003-05-01 | 2004-12-23 | Passini Marco A. | Gene therapy for neurometabolic disorders |
| EP1620133B1 (en) * | 2003-05-01 | 2015-12-09 | Genzyme Corporation | Gene therapy for neurometabolic disorders |
| CA2606490C (en) | 2005-05-02 | 2018-02-06 | Genzyme Corporation | Gene therapy for spinal cord disorders |
| JP5829372B2 (ja) * | 2005-05-02 | 2015-12-09 | ジェンザイム・コーポレーション | 神経代謝性疾患のための遺伝子治療 |
| US7902352B2 (en) | 2005-05-06 | 2011-03-08 | Medtronic, Inc. | Isolated nucleic acid duplex for reducing huntington gene expression |
| WO2006121960A2 (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 |
| 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 |
| HRP20250897T1 (hr) | 2009-05-02 | 2025-09-26 | Genzyme Corporation | Genska terapija za neurodegenerativne poremećaje |
| 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 |
| AU2013296425B2 (en) | 2012-08-01 | 2018-06-07 | Nationwide Children's Hospital | Intrathecal delivery of recombinant adeno-associated virus 9 |
| EP4344741A3 (en) | 2014-11-21 | 2024-08-28 | The University of North Carolina at Chapel Hill | Aav vectors targeted to the central nervous system |
| WO2016100575A1 (en) | 2014-12-16 | 2016-06-23 | Board Of Regents Of The University Of Nebraska | Gene therapy for juvenile batten disease |
| EP3285788B1 (en) | 2015-04-23 | 2024-12-18 | University of Massachusetts | Modulation of aav vector transgene expression |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| CN109415730A (zh) | 2016-06-13 | 2019-03-01 | 北卡罗来纳大学教堂山分校 | 优化的cln1基因和表达盒以及它们的应用 |
| DK3635009T3 (da) | 2017-06-07 | 2026-03-30 | Regeneron Pharma | Sammensætninger og fremgangsmåder til internalisering af enzymer |
| AU2018346104B2 (en) | 2017-10-03 | 2023-07-27 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| BR112020006661A2 (pt) | 2017-10-03 | 2020-10-13 | Prevail Therapeutics, Inc. | terapias de genes para distúrbios lipossomais |
| CN112501208A (zh) | 2017-10-03 | 2021-03-16 | 普利维尔治疗公司 | 用于溶酶体障碍的基因疗法 |
| US12258597B2 (en) | 2018-02-07 | 2025-03-25 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for therapeutic protein delivery |
| CN112203697A (zh) | 2018-04-13 | 2021-01-08 | 马萨诸塞大学 | 编码氨基己糖苷酶alpha和beta亚基的双顺反子AAV载体及其用途 |
| BR112020021962A2 (pt) * | 2018-04-30 | 2021-01-26 | Amicus Therapeutics, Inc. | construtos para terapia gênica e métodos de uso |
| BR112020023145A2 (pt) | 2018-05-17 | 2021-02-02 | Regeneron Pharmaceuticals, Inc. | anticorpo anti-cd63 ou fragmento de ligação ao antígeno do mesmo, molécula de ligação ao antígeno biespecífica, proteína terapêutica de múltiplos domínios, polinucleotídeo composição farmacêutica, e, composto |
| US12448629B2 (en) | 2018-10-05 | 2025-10-21 | University Of Massachusetts | rAAV vectors for the treatment of GM1 and GM2 gangliosidosis |
| CN113631182B (zh) | 2018-10-10 | 2025-03-21 | 阿米库斯治疗学公司 | 二硫键稳定的多肽组合物和使用方法 |
| KR20220022107A (ko) | 2018-12-05 | 2022-02-24 | 아베오나 테라퓨틱스 인코퍼레이티드 | 유전자 전달을 위한 재조합 아데노-연관 바이러스 벡터 |
| JP7664848B2 (ja) | 2019-04-10 | 2025-04-18 | プリベイル セラピューティクス，インコーポレーテッド | リソソーム障害のための遺伝子療法 |
| EP4284442A4 (en) * | 2021-02-01 | 2025-04-23 | The Trustees of The University of Pennsylvania | COMPOSITIONS AND METHODS FOR TREATING NIEMANN PICK DISEASE TYPE A |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5773278A (en) | 1991-05-03 | 1998-06-30 | Mount Sinai Medical Center | Acid sphingomyelinase gene |
| WO1995013365A1 (en) | 1993-11-09 | 1995-05-18 | Targeted Genetics Corporation | Generation of high titers of recombinant aav vectors |
| 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 |
| US6582692B1 (en) * | 1999-11-17 | 2003-06-24 | Avigen, Inc. | Recombinant adeno-associated virus virions for the treatment of lysosomal disorders |
| EP1620133B1 (en) * | 2003-05-01 | 2015-12-09 | Genzyme Corporation | Gene therapy for neurometabolic disorders |
-
2004
- 2004-04-30 EP EP04760694.2A patent/EP1620133B1/en not_active Expired - Lifetime
- 2004-04-30 EP EP10179332.1A patent/EP2269658B1/en not_active Expired - Lifetime
- 2004-04-30 EP EP18212196.2A patent/EP3513814A1/en not_active Withdrawn
- 2004-04-30 SI SI200432298T patent/SI1620133T1/sl unknown
- 2004-04-30 DK DK04760694.2T patent/DK1620133T3/en active
- 2004-04-30 PT PT47606942T patent/PT1620133E/pt unknown
- 2004-04-30 JP JP2006514239A patent/JP4979067B2/ja not_active Expired - Fee Related
- 2004-04-30 HU HUE04760694A patent/HUE026861T2/en unknown
- 2004-04-30 WO PCT/US2004/013685 patent/WO2004098648A1/en not_active Ceased
- 2004-04-30 PL PL04760694T patent/PL1620133T3/pl unknown
- 2004-04-30 ES ES04760694.2T patent/ES2562490T3/es not_active Expired - Lifetime
-
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 |
|---|---|
| PT1620133E (pt) | 2016-03-31 |
| JP2017008118A (ja) | 2017-01-12 |
| JP6170519B2 (ja) | 2017-07-26 |
| EP2269658A1 (en) | 2011-01-05 |
| WO2004098648A1 (en) | 2004-11-18 |
| IL252829A0 (en) | 2017-08-31 |
| EP3513814A1 (en) | 2019-07-24 |
| EP1620133A1 (en) | 2006-02-01 |
| HUE026861T2 (en) | 2016-07-28 |
| PL1620133T3 (pl) | 2016-05-31 |
| IL171635A (en) | 2017-06-29 |
| EP2269658B1 (en) | 2018-12-19 |
| JP5841574B2 (ja) | 2016-01-13 |
| JP2015178519A (ja) | 2015-10-08 |
| JP2018058901A (ja) | 2018-04-12 |
| JP2014040433A (ja) | 2014-03-06 |
| JP4979067B2 (ja) | 2012-07-18 |
| JP2011251992A (ja) | 2011-12-15 |
| CY1117457T1 (el) | 2017-04-26 |
| EP1620133B1 (en) | 2015-12-09 |
| JP2006525363A (ja) | 2006-11-09 |
| JP6312771B2 (ja) | 2018-04-18 |
| SI1620133T1 (sl) | 2016-03-31 |
| DK1620133T3 (en) | 2016-02-29 |
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