ATE367446T1 - Zusammensetzungen zur verabreichung von enzymen involviert im aminosäuremetabolismus durch verwendung rekombinanter adeno-assoziierter virus vironen (raav vironen), und methode und verwendung zur behandlung von aminosäuremetabolischen störungen unter verwendung solcher raav vironen - Google Patents
Zusammensetzungen zur verabreichung von enzymen involviert im aminosäuremetabolismus durch verwendung rekombinanter adeno-assoziierter virus vironen (raav vironen), und methode und verwendung zur behandlung von aminosäuremetabolischen störungen unter verwendung solcher raav vironenInfo
- Publication number
- ATE367446T1 ATE367446T1 AT03717600T AT03717600T ATE367446T1 AT E367446 T1 ATE367446 T1 AT E367446T1 AT 03717600 T AT03717600 T AT 03717600T AT 03717600 T AT03717600 T AT 03717600T AT E367446 T1 ATE367446 T1 AT E367446T1
- Authority
- AT
- Austria
- Prior art keywords
- virons
- amino acid
- raav
- associated virus
- enzymes involved
- Prior art date
Links
- 208000030159 metabolic disease Diseases 0.000 title abstract 4
- 241000702421 Dependoparvovirus Species 0.000 title abstract 3
- 102000004190 Enzymes Human genes 0.000 title abstract 2
- 108090000790 Enzymes Proteins 0.000 title abstract 2
- 230000037354 amino acid metabolism Effects 0.000 title abstract 2
- 238000000034 method Methods 0.000 title abstract 2
- 150000001413 amino acids Chemical class 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
- 108090000623 proteins and genes Proteins 0.000 abstract 5
- 210000002845 virion Anatomy 0.000 abstract 2
- 102000000632 Aromatic amino acid hydroxylases Human genes 0.000 abstract 1
- 108050008079 Aromatic amino acid hydroxylases Proteins 0.000 abstract 1
- 102000003823 Aromatic-L-amino-acid decarboxylases Human genes 0.000 abstract 1
- 108090000121 Aromatic-L-amino-acid decarboxylases Proteins 0.000 abstract 1
- 125000003118 aryl group Chemical group 0.000 abstract 1
- 230000015572 biosynthetic process Effects 0.000 abstract 1
- 230000007547 defect Effects 0.000 abstract 1
- 208000016097 disease of metabolism Diseases 0.000 abstract 1
- 230000002503 metabolic effect Effects 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
- FNKQXYHWGSIFBK-RPDRRWSUSA-N sapropterin Chemical compound N1=C(N)NC(=O)C2=C1NC[C@H]([C@@H](O)[C@@H](O)C)N2 FNKQXYHWGSIFBK-RPDRRWSUSA-N 0.000 abstract 1
- 229960004617 sapropterin Drugs 0.000 abstract 1
- 208000024891 symptom Diseases 0.000 abstract 1
- 238000003786 synthesis reaction Methods 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
- A61K38/44—Oxidoreductases (1)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/69—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
- A61K47/6901—Conjugates being cells, cell fragments, viruses, ghosts, red blood cells or viral vectors
-
- 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
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
- C12Y114/16—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with reduced pteridine as one donor, and incorporation of one atom of oxygen (1.14.16)
- C12Y114/16001—Phenylalanine 4-monooxygenase (1.14.16.1)
-
- 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)
- Genetics & Genomics (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Biomedical Technology (AREA)
- Virology (AREA)
- Biochemistry (AREA)
- Cardiology (AREA)
- Hematology (AREA)
- Microbiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Cell Biology (AREA)
- Heart & Thoracic Surgery (AREA)
- Plant Pathology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Physics & Mathematics (AREA)
- Immunology (AREA)
- Biophysics (AREA)
- Obesity (AREA)
- Hospice & Palliative Care (AREA)
- Diabetes (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US10/124,749 US20030198620A1 (en) | 2002-04-16 | 2002-04-16 | Method of treating amino acid metabolic disorders using recombinant adeno-associated virus virions |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ATE367446T1 true ATE367446T1 (de) | 2007-08-15 |
Family
ID=29214641
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AT03717600T ATE367446T1 (de) | 2002-04-16 | 2003-04-16 | Zusammensetzungen zur verabreichung von enzymen involviert im aminosäuremetabolismus durch verwendung rekombinanter adeno-assoziierter virus vironen (raav vironen), und methode und verwendung zur behandlung von aminosäuremetabolischen störungen unter verwendung solcher raav vironen |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US20030198620A1 (de) |
| EP (2) | EP1847614A1 (de) |
| JP (1) | JP2005522517A (de) |
| AT (1) | ATE367446T1 (de) |
| AU (1) | AU2003222451A1 (de) |
| CA (1) | CA2479996A1 (de) |
| DE (1) | DE60315009T8 (de) |
| ES (1) | ES2290447T3 (de) |
| WO (1) | WO2003087383A1 (de) |
Families Citing this family (39)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20040142416A1 (en) * | 2002-04-30 | 2004-07-22 | Laipis Philip J. | Treatment for phenylketonuria |
| US7442372B2 (en) | 2003-08-29 | 2008-10-28 | Biomarin Pharmaceutical Inc. | Delivery of therapeutic compounds to the brain and other tissues |
| US20100009997A1 (en) * | 2007-01-12 | 2010-01-14 | Biomarin Pharmaceutical Inc. | Method of treating a metabolic or neuropsychiarty disorder with a bh4 derivative prodrug |
| WO2012135389A2 (en) * | 2011-03-28 | 2012-10-04 | The Regents Of The University Of California | Host cells and methods for oxidizing aromatic amino acids |
| US11077238B2 (en) | 2013-06-07 | 2021-08-03 | Allena Pharmaceuticals, Inc. | Compositions, methods, and devices for dialysis |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| CN112553229A (zh) | 2014-11-05 | 2021-03-26 | 沃雅戈治疗公司 | 用于治疗帕金森病的aadc多核苷酸 |
| CN112410339A (zh) | 2014-11-14 | 2021-02-26 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| SG11201703281RA (en) | 2014-11-14 | 2017-05-30 | Voyager Therapeutics Inc | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| EP3230441A4 (de) | 2014-12-12 | 2018-10-03 | Voyager Therapeutics, Inc. | Zusammensetzungen und verfahren zur herstellung von scaav |
| US10983110B2 (en) | 2015-12-02 | 2021-04-20 | Voyager Therapeutics, Inc. | Assays for the detection of AAV neutralizing antibodies |
| US11326182B2 (en) | 2016-04-29 | 2022-05-10 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| WO2017189964A2 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| RU2758488C2 (ru) | 2016-05-18 | 2021-10-28 | Вояджер Терапьютикс, Инк. | Модулирующие полинуклеотиды |
| CA3024449A1 (en) | 2016-05-18 | 2017-11-23 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
| BR112019001532A2 (pt) | 2016-07-26 | 2019-09-10 | Biomarin Pharm Inc | proteínas de capsídeo de vírus adenoassociado inovadoras |
| 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 |
| CN110325199A (zh) * | 2016-12-30 | 2019-10-11 | 宾夕法尼亚州立大学托管会 | 用于治疗苯丙酮尿症的基因疗法 |
| CA3057142A1 (en) | 2017-04-03 | 2018-10-11 | American Gene Technologies International Inc. | Compositions and methods for treating phenylketonuria |
| SG11201909868YA (en) | 2017-05-05 | 2019-11-28 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
| CN110913866A (zh) | 2017-05-05 | 2020-03-24 | 沃雅戈治疗公司 | 治疗肌萎缩性侧索硬化(als)的组合物和方法 |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| JP7229989B2 (ja) | 2017-07-17 | 2023-02-28 | ボイジャー セラピューティクス インコーポレイテッド | 軌道アレイガイドシステム |
| KR20200044793A (ko) | 2017-08-03 | 2020-04-29 | 보이저 테라퓨틱스, 인크. | Aav의 전달을 위한 조성물 및 방법 |
| WO2019036484A1 (en) * | 2017-08-15 | 2019-02-21 | The Trustees Of The University Of Pennsylvania | COMPOSITIONS AND METHODS FOR THE TREATMENT OF ARGININOSUCCINIC ACIDURIA |
| US20200318081A1 (en) * | 2017-10-02 | 2020-10-08 | American Gene Technologies International Inc. | Vectors with promoter and enhancer combinations for treating phenylketonuria |
| CA3077651A1 (en) * | 2017-10-03 | 2019-04-11 | Joseph Lillegard | Vectors and methods of use |
| CN111479924B (zh) | 2017-10-16 | 2024-06-14 | 沃雅戈治疗公司 | 肌萎缩性侧索硬化症(als)的治疗 |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| WO2019152841A1 (en) * | 2018-02-01 | 2019-08-08 | Homology Medicines, Inc. | Adeno-associated virus compositions for pah gene transfer and methods of use thereof |
| PE20212076A1 (es) * | 2018-02-01 | 2021-10-26 | Homology Medicines Inc | Composiciones de virus adeno-asociado para restaurar la funcion del gen pah y metodos de uso de las mismas |
| PE20210112A1 (es) * | 2018-05-09 | 2021-01-19 | Biomarin Pharm Inc | Metodos de tratamiento de fenilcetonuria |
| TW202005978A (zh) | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | 新穎肝靶向腺相關病毒載體 |
| JP2020068734A (ja) * | 2018-11-01 | 2020-05-07 | 国立大学法人東京工業大学 | Ｓｐｒをコードするポリヌクレオチドを含む組換えベクター、及びそれを含む組成物 |
| US20220226502A1 (en) * | 2019-06-03 | 2022-07-21 | Institute For Cancer Research d/b/aThe Research Institute of Fox Chase Cancer Center | Adeno-associated virus vector delivery of cystathionine beta-synthase (cbs) enzyme for treating cbs deficiency |
| TW202140791A (zh) | 2020-01-13 | 2021-11-01 | 美商霍蒙拉奇醫藥公司 | 治療苯酮尿症之方法 |
| EP4166157A4 (de) * | 2020-06-16 | 2024-03-06 | Novel Pharma Inc. | Zusammensetzung zur behandlung von glutarazidurie und verabreichungsverfahren dafür |
| KR102226574B1 (ko) * | 2020-06-16 | 2021-03-11 | 노바자임 주식회사 | 글루타르산뇨증 치료용 조성물 및 이의 투여방법 |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5872005A (en) * | 1994-11-03 | 1999-02-16 | Cell Genesys Inc. | Packaging cell lines for adeno-associated viral vectors |
| US5885971A (en) * | 1995-03-24 | 1999-03-23 | The Regents Of The University Of California | Gene therapy by secretory gland expression |
| US6521225B1 (en) * | 1996-09-06 | 2003-02-18 | Chiron Corporation | AAV vectors |
| ATE486614T1 (de) * | 1997-03-14 | 2010-11-15 | Philadelphia Children Hospital | Zusammensetzungen zum einsatz in einer gentherapie zur behandlung von hämophilie |
| AU1775901A (en) * | 1999-11-17 | 2001-05-30 | Avigen, Inc. | Recombinant adeno-associated virus virions for the treatment of lysosomal disorders |
| WO2002014487A2 (en) * | 2000-08-17 | 2002-02-21 | Keiya Ozawa | Adeno-associated virus-mediated delivery of angiogenic factors |
-
2002
- 2002-04-16 US US10/124,749 patent/US20030198620A1/en not_active Abandoned
-
2003
- 2003-04-16 DE DE60315009T patent/DE60315009T8/de not_active Expired - Fee Related
- 2003-04-16 ES ES03717600T patent/ES2290447T3/es not_active Expired - Lifetime
- 2003-04-16 EP EP07013616A patent/EP1847614A1/de not_active Withdrawn
- 2003-04-16 JP JP2003584322A patent/JP2005522517A/ja active Pending
- 2003-04-16 EP EP03717600A patent/EP1497436B1/de not_active Expired - Lifetime
- 2003-04-16 AT AT03717600T patent/ATE367446T1/de not_active IP Right Cessation
- 2003-04-16 WO PCT/JP2003/004856 patent/WO2003087383A1/en active IP Right Grant
- 2003-04-16 CA CA002479996A patent/CA2479996A1/en not_active Abandoned
- 2003-04-16 AU AU2003222451A patent/AU2003222451A1/en not_active Abandoned
-
2004
- 2004-11-18 US US10/992,346 patent/US20050147592A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| AU2003222451A1 (en) | 2003-10-27 |
| EP1497436A1 (de) | 2005-01-19 |
| DE60315009T8 (de) | 2009-02-05 |
| DE60315009D1 (de) | 2007-08-30 |
| EP1847614A1 (de) | 2007-10-24 |
| US20030198620A1 (en) | 2003-10-23 |
| EP1497436B1 (de) | 2007-07-18 |
| ES2290447T3 (es) | 2008-02-16 |
| US20050147592A1 (en) | 2005-07-07 |
| WO2003087383A1 (en) | 2003-10-23 |
| CA2479996A1 (en) | 2003-10-23 |
| DE60315009T2 (de) | 2008-04-03 |
| JP2005522517A (ja) | 2005-07-28 |
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| Date | Code | Title | Description |
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| RER | Ceased as to paragraph 5 lit. 3 law introducing patent treaties |