PT2879719T - Administração intratecal do vírus adeno-associado 9 recombinante - Google Patents
Administração intratecal do vírus adeno-associado 9 recombinanteInfo
- Publication number
- PT2879719T PT2879719T PT13747755T PT13747755T PT2879719T PT 2879719 T PT2879719 T PT 2879719T PT 13747755 T PT13747755 T PT 13747755T PT 13747755 T PT13747755 T PT 13747755T PT 2879719 T PT2879719 T PT 2879719T
- Authority
- PT
- Portugal
- Prior art keywords
- associated virus
- recombinant adeno
- intrathecal delivery
- intrathecal
- delivery
- Prior art date
Links
Classifications
-
- 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
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
-
- 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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K49/00—Preparations for testing in vivo
- A61K49/04—X-ray contrast preparations
- A61K49/0433—X-ray contrast preparations containing an organic halogenated X-ray contrast-enhancing agent
- A61K49/0438—Organic X-ray contrast-enhancing agent comprising an iodinated group or an iodine atom, e.g. iopamidol
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
-
- 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
- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
-
- 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
- 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
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
-
- 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
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201261678458P | 2012-08-01 | 2012-08-01 |
Publications (1)
Publication Number | Publication Date |
---|---|
PT2879719T true PT2879719T (pt) | 2018-10-08 |
Family
ID=48951610
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PT13747755T PT2879719T (pt) | 2012-08-01 | 2013-07-31 | Administração intratecal do vírus adeno-associado 9 recombinante |
Country Status (10)
Country | Link |
---|---|
US (12) | US20150252384A1 (pt) |
EP (3) | EP3415167A1 (pt) |
JP (5) | JP6314138B2 (pt) |
AU (4) | AU2013296425B2 (pt) |
BR (1) | BR112015002168A2 (pt) |
CA (2) | CA3086754C (pt) |
DK (1) | DK2879719T3 (pt) |
ES (1) | ES2684222T3 (pt) |
PT (1) | PT2879719T (pt) |
WO (1) | WO2014022582A1 (pt) |
Families Citing this family (32)
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US11219696B2 (en) | 2008-12-19 | 2022-01-11 | Nationwide Children's Hospital | Delivery of polynucleotides using recombinant AAV9 |
EP3421603B1 (en) | 2009-05-02 | 2021-10-06 | Genzyme Corporation | Gene therapy for neurodegenerative disorders |
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 |
ES2684222T3 (es) * | 2012-08-01 | 2018-10-01 | Nationwide Children's Hospital | Administración intratecal de virus adenoasociado 9 recombinante |
SG11201509419QA (en) * | 2013-05-15 | 2015-12-30 | Univ Minnesota | Adeno-associated virus mediated gene transfer to the central nervous system |
WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
JP6401871B2 (ja) | 2014-11-05 | 2018-10-10 | 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. | パーキンソン病の治療のためのaadcポリヌクレオチド |
EP3218484A4 (en) | 2014-11-14 | 2018-05-30 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
SG11201703419UA (en) | 2014-11-14 | 2017-05-30 | Voyager Therapeutics Inc | Modulatory polynucleotides |
EP3230441A4 (en) | 2014-12-12 | 2018-10-03 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scaav |
PL3411484T3 (pl) * | 2016-02-05 | 2024-02-19 | Emory University | Wstrzykiwanie jednoniciowego lub samokomplementarnego wirusa adenosatelitarnego 9 do płynu mózgowego |
SG10201912761UA (en) | 2016-04-15 | 2020-02-27 | The Trustees Of The Univ Of Pennsyvania | Gene therapy for treating mucopolysaccharidosis type ii |
WO2017189964A2 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
EP3448874A4 (en) | 2016-04-29 | 2020-04-22 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
EP3458588A4 (en) | 2016-05-18 | 2020-01-15 | Voyager Therapeutics, Inc. | MODULATING POLYNUCLEOTIDES |
WO2017201258A1 (en) | 2016-05-18 | 2017-11-23 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
EP3831281A1 (en) | 2016-08-30 | 2021-06-09 | The Regents of The University of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
WO2018204803A1 (en) | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
JP2020518258A (ja) | 2017-05-05 | 2020-06-25 | 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. | 筋萎縮性側索硬化症(als)治療組成物および方法 |
JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
JP2020532284A (ja) * | 2017-07-08 | 2020-11-12 | ジェネトン | 脊髄性筋萎縮症の治療 |
US11497576B2 (en) | 2017-07-17 | 2022-11-15 | Voyager Therapeutics, Inc. | Trajectory array guide system |
EP3808849A1 (en) | 2017-08-03 | 2021-04-21 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
EP3684938A1 (en) | 2017-09-22 | 2020-07-29 | The Trustees of the University of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type ii |
WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
AU2018352236A1 (en) | 2017-10-16 | 2020-04-23 | The Curators Of The University Of Missouri | Treatment of amyotrophic lateral sclerosis (ALS) |
US20210317474A1 (en) * | 2017-11-08 | 2021-10-14 | Novartis Ag | Means and method for producing and purifying viral vectors |
KR20210018919A (ko) * | 2018-06-08 | 2021-02-18 | 노파르티스 아게 | 약물 산물 효능을 측정하기 위한 세포-기반 분석 |
JP2020095428A (ja) * | 2018-12-12 | 2020-06-18 | 株式会社東芝 | モデル学習システム、モデル学習方法、プログラム、及び記憶媒体 |
MX2021009401A (es) * | 2019-02-04 | 2021-11-12 | Res Inst Nationwide Childrens Hospital | Administracion de virus adenoasociado de polinucleotido de cln3. |
CA3137080A1 (en) * | 2019-04-15 | 2020-10-22 | Sanford Research | Gene therapy for treating or preventing visual effects in batten disease |
CA3233427A1 (en) * | 2021-09-30 | 2023-04-06 | The Board Of Regents Of The University Of Texas System | Slc13a5 gene therapy vectors and uses thereof |
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US11219696B2 (en) | 2008-12-19 | 2022-01-11 | Nationwide Children's Hospital | Delivery of polynucleotides using recombinant AAV9 |
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MX2021009401A (es) | 2019-02-04 | 2021-11-12 | Res Inst Nationwide Childrens Hospital | Administracion de virus adenoasociado de polinucleotido de cln3. |
KR20210124299A (ko) | 2019-02-04 | 2021-10-14 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | Cln6 폴리뉴클레오티드의 아데노-관련 바이러스 전달 |
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2013
- 2013-07-31 ES ES13747755.0T patent/ES2684222T3/es active Active
- 2013-07-31 EP EP18181263.7A patent/EP3415167A1/en active Pending
- 2013-07-31 BR BR112015002168A patent/BR112015002168A2/pt not_active Application Discontinuation
- 2013-07-31 JP JP2015525565A patent/JP6314138B2/ja active Active
- 2013-07-31 CA CA3086754A patent/CA3086754C/en active Active
- 2013-07-31 EP EP13747755.0A patent/EP2879719B1/en active Active
- 2013-07-31 AU AU2013296425A patent/AU2013296425B2/en active Active
- 2013-07-31 DK DK13747755.0T patent/DK2879719T3/en active
- 2013-07-31 EP EP20176640.9A patent/EP3769789A1/en active Pending
- 2013-07-31 CA CA2880653A patent/CA2880653C/en active Active
- 2013-07-31 PT PT13747755T patent/PT2879719T/pt unknown
- 2013-07-31 US US14/417,823 patent/US20150252384A1/en not_active Abandoned
- 2013-07-31 WO PCT/US2013/053065 patent/WO2014022582A1/en active Application Filing
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2017
- 2017-04-14 US US15/488,203 patent/US20170216458A1/en not_active Abandoned
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2018
- 2018-03-26 JP JP2018058524A patent/JP6708686B2/ja active Active
- 2018-06-04 US US15/997,433 patent/US11730829B2/en active Active
- 2018-09-07 AU AU2018226501A patent/AU2018226501A1/en not_active Abandoned
- 2018-09-12 US US16/129,096 patent/US20190099503A1/en not_active Abandoned
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2019
- 2019-01-14 US US16/247,380 patent/US11311634B2/en active Active
- 2019-01-14 US US16/247,420 patent/US20190134226A1/en active Pending
- 2019-03-07 JP JP2019041462A patent/JP2019116493A/ja active Pending
- 2019-05-08 US US16/406,904 patent/US11413357B2/en active Active
- 2019-05-08 US US16/406,895 patent/US11738094B2/en active Active
- 2019-07-03 US US16/502,944 patent/US11040116B2/en active Active
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2020
- 2020-06-10 AU AU2020203844A patent/AU2020203844B2/en active Active
- 2020-08-19 JP JP2020138445A patent/JP2021006034A/ja active Pending
- 2020-11-23 US US17/102,291 patent/US20210100918A1/en active Pending
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2021
- 2021-03-09 US US17/196,709 patent/US20210220487A1/en not_active Abandoned
- 2021-03-19 US US17/206,878 patent/US20210228743A1/en active Pending
- 2021-06-04 JP JP2021094213A patent/JP7230112B2/ja active Active
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2023
- 2023-09-19 AU AU2023233078A patent/AU2023233078B2/en active Active
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