PL1879624T3 - Terapia genowa dla zaburzeń neurometabolicznych - Google Patents
Terapia genowa dla zaburzeń neurometabolicznychInfo
- Publication number
- PL1879624T3 PL1879624T3 PL06759081T PL06759081T PL1879624T3 PL 1879624 T3 PL1879624 T3 PL 1879624T3 PL 06759081 T PL06759081 T PL 06759081T PL 06759081 T PL06759081 T PL 06759081T PL 1879624 T3 PL1879624 T3 PL 1879624T3
- Authority
- PL
- Poland
- Prior art keywords
- disorders
- disease
- nervous system
- central nervous
- gene therapy
- Prior art date
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
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- 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
- 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
-
- 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)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Epidemiology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Obesity (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Physical Education & Sports Medicine (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US67705705P | 2005-05-02 | 2005-05-02 | |
| US68580805P | 2005-05-31 | 2005-05-31 | |
| EP06759081A EP1879624B1 (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 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PL1879624T3 true PL1879624T3 (pl) | 2012-02-29 |
Family
ID=37308324
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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 |
| 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 |
Family Applications Before (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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 After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL19153520T PL3520823T3 (pl) | 2005-05-02 | 2006-05-02 | Terapia genowa zaburzeń neurometabolicznych |
Country Status (13)
| Country | Link |
|---|---|
| US (2) | US10632213B2 (pl) |
| EP (4) | EP2420256B1 (pl) |
| JP (3) | JP5829372B2 (pl) |
| CN (4) | CN104306986A (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) | PL2420256T3 (pl) |
| PT (4) | PT2420256T (pl) |
| WO (1) | WO2006119458A1 (pl) |
Families Citing this family (39)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP3302529B2 (ja) | 1995-04-28 | 2002-07-15 | 株式会社クボタ | ポンプ吸水槽の整流装置 |
| PT2420256T (pt) | 2005-05-02 | 2016-09-13 | Genzyme Corp | Terapia genética para distúrbios neurometabólicos |
| JP5766389B2 (ja) * | 2005-05-02 | 2015-08-19 | ジェンザイム・コーポレーション | 脊髄疾患の遺伝子療法 |
| HUE031156T2 (en) | 2006-06-07 | 2017-06-28 | Genzyme Corp | Gene therapy for amyotrophic lateral sclerosis and other spinal cord diseases |
| PL2158322T3 (pl) | 2007-06-06 | 2017-10-31 | Genzyme Corp | Terapia genowa dla lizosomalnych chorób spichrzeniowych |
| WO2010054379A2 (en) | 2008-11-10 | 2010-05-14 | The United States Of America, As Represensted By The Secretary, Department Of Health And Human Services | Gene signature for predicting prognosis of patients with solid tumors |
| SG10202109219SA (en) | 2009-05-02 | 2021-10-28 | Genzyme Corp | Gene therapy for neurodegenerative disorders |
| US9150926B2 (en) | 2010-12-06 | 2015-10-06 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Diagnosis and treatment of adrenocortical tumors using human microRNA-483 |
| US20130090374A1 (en) * | 2011-04-20 | 2013-04-11 | Miguel Sena-Esteves | Methods for the treatment of tay-sachs disease, sandhoff disease, and gm1-gangliosidosis |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| US10335466B2 (en) | 2014-11-05 | 2019-07-02 | Voyager Therapeutics, Inc. | AADC polynucleotides for the treatment of parkinson's disease |
| SG11201703281RA (en) | 2014-11-14 | 2017-05-30 | Voyager Therapeutics Inc | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| JP6863891B2 (ja) | 2014-11-14 | 2021-04-21 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | 調節性ポリヌクレオチド |
| WO2016081924A1 (en) * | 2014-11-20 | 2016-05-26 | Duke University | Compositions, systems and methods for cell therapy |
| HK1245326A1 (zh) | 2014-12-12 | 2018-08-24 | Voyager Therapeutics, Inc. | 用於生产scaav的组合物和方法 |
| KR102863734B1 (ko) | 2015-02-10 | 2025-09-25 | 젠자임 코포레이션 | 선조체 및 피질로의 바이러스 입자의 향상된 전달 |
| AU2016235685B2 (en) * | 2015-03-26 | 2019-04-18 | Shanghai Auzone Biological Technology Co., Ltd. | Method of diagnosis or treatment of neurological disorders with p75ECD and/or p75 |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| WO2016196507A1 (en) * | 2015-05-29 | 2016-12-08 | University Of Iowa Research Foundation | Methods of delivery of transgenes for treating brain diseases |
| EP3448987A4 (en) | 2016-04-29 | 2020-05-27 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| WO2017189959A1 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| JP7220080B2 (ja) | 2016-05-18 | 2023-02-09 | ボイジャー セラピューティクス インコーポレイテッド | ハンチントン病治療組成物及び方法 |
| EP3458588B1 (en) | 2016-05-18 | 2025-10-29 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| JP2019531787A (ja) | 2016-08-30 | 2019-11-07 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 生物医学的ターゲティング及びデリバリーの方法並びにそれを実行するための装置及びシステム |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| CA3061652A1 (en) | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| AU2018261003A1 (en) | 2017-05-05 | 2019-11-14 | Voyager Therapeutics, Inc. | Compositions and methods of treating Huntington's Disease |
| BR112019025888A2 (pt) | 2017-06-07 | 2020-06-30 | Regeneron Pharmaceuticals, Inc. | polinucleotídeo que codifica uma proteína terapêutica multidomínio, vetor de terapia gênica, proteína terapêutica multidomínio recombinante, método de expressão, métodos para reduzir o acúmulo de glicogênio em um tecido em um paciente em necessidade, para reduzir o acúmulo de glicogênio em um tecido em um paciente em necessidade epara tratar a deficiência enzimática em um paciente em necessidade e/ou tolerar o paciente à enzima para a qual é deficiente, anticorpo anti-cd63 ou fragmento de ligação a antígeno, e, composição farmacêutica |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| EP3654860A1 (en) | 2017-07-17 | 2020-05-27 | Voyager Therapeutics, Inc. | Trajectory array guide system |
| MX2020001187A (es) | 2017-08-03 | 2020-10-05 | Voyager Therapeutics Inc | Composiciones y métodos para la administración de virus adenoasociados. |
| AU2018338728B2 (en) | 2017-09-29 | 2025-01-02 | Centre National De La Recherche Scientifique (Cnrs) | Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery |
| EP4454654A3 (en) | 2017-10-16 | 2025-02-19 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| TW202413649A (zh) | 2017-10-16 | 2024-04-01 | 美商航海家醫療公司 | 肌萎縮性脊髓側索硬化症(als)之治療 |
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| TW202012628A (zh) | 2018-05-15 | 2020-04-01 | 美商航海家醫療公司 | 用於帕金森氏症治療的組成物及方法 |
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| AU2019346655B2 (en) | 2018-09-28 | 2025-03-13 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| KR20240126916A (ko) * | 2023-02-14 | 2024-08-22 | 경북대학교 산학협력단 | Asm 단백질 또는 이의 단편을 포함하는 퇴행성 신경질환 또는 우울증 예방 또는 치료용 조성물 |
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| WO2007092563A2 (en) | 2006-02-08 | 2007-08-16 | Genzyme Corporation | Gene therapy for niemann-pick disease type a |
| JP2014513727A (ja) | 2011-05-16 | 2014-06-05 | ジェンザイム・コーポレーション | Ｃｘｃｒ４拮抗薬の使用 |
| TWI821227B (zh) | 2017-12-22 | 2023-11-11 | 美商健臻公司 | 胺甲喋呤誘發的免疫耐受性之生物標記 |
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