CY1117457T1 - Γονιδιακη θεραπεια για τις νευρομεταβολικες διαταραχες - Google Patents
Γονιδιακη θεραπεια για τις νευρομεταβολικες διαταραχεςInfo
- Publication number
- CY1117457T1 CY1117457T1 CY20161100204T CY161100204T CY1117457T1 CY 1117457 T1 CY1117457 T1 CY 1117457T1 CY 20161100204 T CY20161100204 T CY 20161100204T CY 161100204 T CY161100204 T CY 161100204T CY 1117457 T1 CY1117457 T1 CY 1117457T1
- Authority
- CY
- Cyprus
- Prior art keywords
- neuro
- metabolic disorders
- treatment
- aav
- gender treatment
- Prior art date
Links
- 208000030159 metabolic disease Diseases 0.000 title 1
- 108700019146 Transgenes Proteins 0.000 abstract 2
- 238000000034 method Methods 0.000 abstract 2
- 239000013607 AAV vector Substances 0.000 abstract 1
- 208000015439 Lysosomal storage disease Diseases 0.000 abstract 1
- 208000029726 Neurodevelopmental disease Diseases 0.000 abstract 1
- 208000014060 Niemann-Pick disease Diseases 0.000 abstract 1
- 230000003376 axonal effect Effects 0.000 abstract 1
- 210000003169 central nervous system Anatomy 0.000 abstract 1
- 210000002569 neuron Anatomy 0.000 abstract 1
- 230000001225 therapeutic effect Effects 0.000 abstract 1
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 (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US46719503P | 2003-05-01 | 2003-05-01 | |
| US47572603P | 2003-06-04 | 2003-06-04 | |
| EP04760694.2A EP1620133B1 (en) | 2003-05-01 | 2004-04-30 | Gene therapy for neurometabolic disorders |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CY1117457T1 true CY1117457T1 (el) | 2017-04-26 |
Family
ID=33436721
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CY20161100204T CY1117457T1 (el) | 2003-05-01 | 2016-03-09 | Γονιδιακη θεραπεια για τις νευρομεταβολικες διαταραχες |
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 |
| ES2562490T3 (es) | 2016-03-04 |
| 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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