BR112015000161A2 - composições de vetor de vírus adeno-associado modificado - Google Patents
composições de vetor de vírus adeno-associado modificadoInfo
- Publication number
- BR112015000161A2 BR112015000161A2 BR112015000161A BR112015000161A BR112015000161A2 BR 112015000161 A2 BR112015000161 A2 BR 112015000161A2 BR 112015000161 A BR112015000161 A BR 112015000161A BR 112015000161 A BR112015000161 A BR 112015000161A BR 112015000161 A2 BR112015000161 A2 BR 112015000161A2
- Authority
- BR
- Brazil
- Prior art keywords
- associated virus
- virus vector
- vector compositions
- modified adeno
- adeno
- Prior art date
Links
Classifications
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- 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
- 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
- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
-
- 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
-
- 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
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/38—Vector systems having a special element relevant for transcription being a stuffer
Abstract
resumo patente de invenção: "composições de vetor de vírus adeno-associado modificado". a presente invenção refere-se a um componente de agente de enchimento do vírus adeno-associado, compreendendo um ácido nucleico entre 3300 e 4200 nucleotídeos de comprimento descrito.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201261668839P | 2012-07-06 | 2012-07-06 | |
PCT/US2013/031644 WO2014007858A1 (en) | 2012-07-06 | 2013-03-14 | Modified adeno-associated virus vector compositions |
Publications (1)
Publication Number | Publication Date |
---|---|
BR112015000161A2 true BR112015000161A2 (pt) | 2017-06-27 |
Family
ID=49882407
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112015000161A BR112015000161A2 (pt) | 2012-07-06 | 2013-03-14 | composições de vetor de vírus adeno-associado modificado |
Country Status (12)
Country | Link |
---|---|
US (2) | US9540659B2 (pt) |
EP (1) | EP2870237B1 (pt) |
JP (1) | JP6366581B2 (pt) |
CN (1) | CN104619832B (pt) |
AU (1) | AU2013287261B2 (pt) |
BR (1) | BR112015000161A2 (pt) |
CA (1) | CA2878401C (pt) |
ES (1) | ES2703177T3 (pt) |
HK (1) | HK1210214A1 (pt) |
IN (1) | IN2015KN00074A (pt) |
RU (1) | RU2679843C2 (pt) |
WO (1) | WO2014007858A1 (pt) |
Families Citing this family (30)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
ES2703177T3 (es) * | 2012-07-06 | 2019-03-07 | Univ Iowa Res Found | Composiciones de vector viral adenoasociado modificado |
CA2949437C (en) * | 2014-05-20 | 2023-08-15 | University Of Iowa Research Foundation | Huntington's disease therapeutic compounds |
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 |
US20190000940A1 (en) * | 2015-07-31 | 2019-01-03 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of aadc deficiency |
AU2017234929A1 (en) | 2016-03-18 | 2018-10-04 | The Children's Hospital Of Philadelphia | Therapeutic for treatment of diseases including the central nervous system |
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 |
WO2017201258A1 (en) | 2016-05-18 | 2017-11-23 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
EP3458588A4 (en) | 2016-05-18 | 2020-01-15 | Voyager Therapeutics, Inc. | MODULATING POLYNUCLEOTIDES |
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 |
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 |
WO2018204803A1 (en) | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
US11497576B2 (en) | 2017-07-17 | 2022-11-15 | Voyager Therapeutics, Inc. | Trajectory array guide system |
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) |
US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
EP3755795A4 (en) | 2018-02-19 | 2022-07-20 | Homology Medicines, Inc. | ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR RECOVERING F8 GENE FUNCTION AND METHODS OF USE THEREOF |
JP2021529513A (ja) * | 2018-07-02 | 2021-11-04 | 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および脊髄に関連する障害の治療 |
JP2022531809A (ja) * | 2019-05-14 | 2022-07-11 | デューク ユニバーシティ | ATPase媒介性疾患の処置のための組成物および方法 |
US11905523B2 (en) | 2019-10-17 | 2024-02-20 | Ginkgo Bioworks, Inc. | Adeno-associated viral vectors for treatment of Niemann-Pick Disease type-C |
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 |
EP4150102A2 (en) * | 2020-05-11 | 2023-03-22 | Gordian Biotechnology, Inc. | Viral delivery vehicle selection |
US20240093189A1 (en) * | 2021-02-05 | 2024-03-21 | Maze Therapeutics, Inc. | Vectors comprising stuffer polynucleotide sequences |
WO2022235614A2 (en) * | 2021-05-04 | 2022-11-10 | Regenxbio Inc. | Novel aav vectors and methods and uses thereof |
WO2024033329A2 (en) * | 2022-08-09 | 2024-02-15 | University Of Sheffield | Viral vector |
Family Cites Families (12)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US5139941A (en) * | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
JPH1033175A (ja) * | 1996-07-24 | 1998-02-10 | Hisamitsu Pharmaceut Co Inc | 組換えアデノ随伴ウイルスベクターの製造方法 |
US6171855B1 (en) * | 1998-05-28 | 2001-01-09 | The Regents Of The University Of Michigan | Vectors |
US6943153B1 (en) | 1999-03-15 | 2005-09-13 | The Regents Of The University Of California | Use of recombinant gene delivery vectors for treating or preventing diseases of the eye |
US6995010B1 (en) * | 1999-10-29 | 2006-02-07 | Takara Bio Inc. | Gene transfer method |
US7132277B1 (en) * | 2000-01-31 | 2006-11-07 | Merck & Co., Inc. | Helper dependent vector system for gene therapy |
US7387896B2 (en) | 2003-03-26 | 2008-06-17 | The Regents Of The University Of Michigan | MicroRNA vectors |
DE602004025959D1 (de) * | 2003-10-20 | 2010-04-22 | Nsgene As | Viraler Vektor zur Verwendung in der in-vivo-Gentherapie von Morbus Parkinson |
WO2009134681A2 (en) * | 2008-04-30 | 2009-11-05 | The Trustees Of The University Of Pennsylvania | Aav7 viral vectors for targeted delivery of rpe cells |
CA2833905C (en) * | 2010-04-23 | 2019-09-10 | University Of Massachusetts | Multicistronic expression constructs |
US9181544B2 (en) | 2011-02-12 | 2015-11-10 | University Of Iowa Research Foundation | Therapeutic compounds |
ES2703177T3 (es) * | 2012-07-06 | 2019-03-07 | Univ Iowa Res Found | Composiciones de vector viral adenoasociado modificado |
-
2013
- 2013-03-14 ES ES13812649T patent/ES2703177T3/es active Active
- 2013-03-14 RU RU2015103901A patent/RU2679843C2/ru active
- 2013-03-14 BR BR112015000161A patent/BR112015000161A2/pt not_active Application Discontinuation
- 2013-03-14 WO PCT/US2013/031644 patent/WO2014007858A1/en active Application Filing
- 2013-03-14 EP EP13812649.5A patent/EP2870237B1/en active Active
- 2013-03-14 AU AU2013287261A patent/AU2013287261B2/en active Active
- 2013-03-14 CA CA2878401A patent/CA2878401C/en active Active
- 2013-03-14 US US14/410,983 patent/US9540659B2/en active Active
- 2013-03-14 JP JP2015520166A patent/JP6366581B2/ja active Active
- 2013-03-14 CN CN201380035802.XA patent/CN104619832B/zh active Active
- 2013-03-14 IN IN74KON2015 patent/IN2015KN00074A/en unknown
-
2015
- 2015-11-04 HK HK15110883.2A patent/HK1210214A1/xx unknown
-
2017
- 2017-07-07 US US15/644,491 patent/US20170306355A1/en not_active Abandoned
Also Published As
Publication number | Publication date |
---|---|
ES2703177T3 (es) | 2019-03-07 |
AU2013287261A1 (en) | 2015-01-22 |
CA2878401A1 (en) | 2014-01-09 |
WO2014007858A1 (en) | 2014-01-09 |
JP2015522280A (ja) | 2015-08-06 |
US9540659B2 (en) | 2017-01-10 |
RU2015103901A (ru) | 2016-08-27 |
EP2870237A1 (en) | 2015-05-13 |
JP6366581B2 (ja) | 2018-08-08 |
IN2015KN00074A (pt) | 2015-07-31 |
CN104619832B (zh) | 2021-03-16 |
CA2878401C (en) | 2022-07-19 |
US20150184197A1 (en) | 2015-07-02 |
EP2870237A4 (en) | 2015-12-23 |
RU2679843C2 (ru) | 2019-02-13 |
US20170306355A1 (en) | 2017-10-26 |
AU2013287261B2 (en) | 2019-03-07 |
CN104619832A (zh) | 2015-05-13 |
EP2870237B1 (en) | 2018-09-26 |
HK1210214A1 (en) | 2016-04-15 |
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Legal Events
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B06F | Objections, documents and/or translations needed after an examination request according [chapter 6.6 patent gazette] | ||
B06U | Preliminary requirement: requests with searches performed by other patent offices: procedure suspended [chapter 6.21 patent gazette] | ||
B06F | Objections, documents and/or translations needed after an examination request according [chapter 6.6 patent gazette] | ||
B06A | Patent application procedure suspended [chapter 6.1 patent gazette] | ||
B09B | Patent application refused [chapter 9.2 patent gazette] | ||
B12B | Appeal against refusal [chapter 12.2 patent gazette] |