DK2661494T3 - Promotorer, ekspressionskassetter og vektorer til anvendelse ved behandling af achromatopsi og andre sygdomme - Google Patents
Promotorer, ekspressionskassetter og vektorer til anvendelse ved behandling af achromatopsi og andre sygdomme Download PDFInfo
- Publication number
- DK2661494T3 DK2661494T3 DK12731925.9T DK12731925T DK2661494T3 DK 2661494 T3 DK2661494 T3 DK 2661494T3 DK 12731925 T DK12731925 T DK 12731925T DK 2661494 T3 DK2661494 T3 DK 2661494T3
- Authority
- DK
- Denmark
- Prior art keywords
- achromatopsia
- promoters
- vectors
- diseases
- treatment
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- 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
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
-
- 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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- 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
-
- 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
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201161430710P | 2011-01-07 | 2011-01-07 | |
| PCT/US2012/020423 WO2012094560A2 (en) | 2011-01-07 | 2012-01-06 | Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DK2661494T3 true DK2661494T3 (da) | 2019-09-02 |
Family
ID=46457976
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK12731925.9T DK2661494T3 (da) | 2011-01-07 | 2012-01-06 | Promotorer, ekspressionskassetter og vektorer til anvendelse ved behandling af achromatopsi og andre sygdomme |
Country Status (8)
| Country | Link |
|---|---|
| US (2) | US9982275B2 (da) |
| EP (2) | EP3591055A1 (da) |
| JP (2) | JP6345423B2 (da) |
| CN (2) | CN103608455B (da) |
| AU (4) | AU2012204266C1 (da) |
| CA (1) | CA2823890C (da) |
| DK (1) | DK2661494T3 (da) |
| WO (1) | WO2012094560A2 (da) |
Families Citing this family (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3591055A1 (en) * | 2011-01-07 | 2020-01-08 | Applied Genetic Technologies Corporation | Promoters derived from the 5' nontranslated region (5'-ntr) of the human cyclic nucleotide gated beta 3 (cngb3) gene, expression cassettes and vectors comprising said promoters, for use treating achromatopsia and other diseases |
| TWI775096B (zh) | 2012-05-15 | 2022-08-21 | 澳大利亞商艾佛蘭屈澳洲私營有限公司 | 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd) |
| EP2888368A1 (en) | 2012-08-27 | 2015-07-01 | Friedrich Miescher Institute for Biomedical Research | Retinal off circuit-specific promoter |
| CA2912525A1 (en) * | 2013-05-16 | 2014-11-20 | Applied Genetic Technologies Corporation | Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases |
| CA2942776C (en) * | 2014-03-17 | 2023-01-24 | Adverum Biotechnologies, Inc. | Polyneucleotide cassette and expression vector for expression of a gene in cone cells using truncated m-opsin promoter |
| KR20170137730A (ko) | 2015-03-02 | 2017-12-13 | 애드베룸 바이오테크놀로지스, 인코포레이티드 | 망막 추상체에 폴리뉴클레오타이드의 유리체 내 전달을 위한 조성물 및 방법 |
| WO2017106202A2 (en) * | 2015-12-14 | 2017-06-22 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
| GB2545763A (en) | 2015-12-23 | 2017-06-28 | Adverum Biotechnologies Inc | Mutant viral capsid libraries and related systems and methods |
| EP3210632B1 (en) | 2016-02-23 | 2022-09-28 | EyeServ GmbH | Gene therapy for the treatment of a retinal degeneration disease |
| WO2017144080A1 (en) | 2016-02-23 | 2017-08-31 | Eyeserv Gmbh | Gene therapy for the treatment of a disease of retinal cone cells |
| EP3570895A1 (en) * | 2017-01-17 | 2019-11-27 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods of expressing a polynucleotide of interest in the cone photoreceptors |
| EP3638316A4 (en) | 2017-06-14 | 2021-03-24 | The Trustees Of The University Of Pennsylvania | GENE THERAPY FOR EYE DISORDERS |
| RU2020121408A (ru) | 2017-11-30 | 2021-12-30 | Фридрих Мишер Инститьют Фор Байомедикал Рисерч | ПРОМОТОР SynPIII ДЛЯ СПЕЦИФИЧЕСКОЙ ЭКСПРЕССИИ ГЕНОВ В ПИГМЕНТНОМ ЭПИТЕЛИИ СЕТЧАТКИ |
| GB201800546D0 (en) * | 2018-01-12 | 2018-02-28 | Ucl Business Plc | Treatment |
| JP7227589B2 (ja) * | 2018-08-23 | 2023-02-22 | 学校法人藤田学園 | 疾患の発症に関する診断のための情報を提供する方法 |
| CN111281328B (zh) * | 2020-02-27 | 2022-08-12 | 南京云视郎生物科技有限公司 | 一种弱视验光的方法、装置及电子设备 |
Family Cites Families (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4458066A (en) | 1980-02-29 | 1984-07-03 | University Patents, Inc. | Process for preparing polynucleotides |
| US4401796A (en) | 1981-04-30 | 1983-08-30 | City Of Hope Research Institute | Solid-phase synthesis of polynucleotides |
| US4373071A (en) | 1981-04-30 | 1983-02-08 | City Of Hope Research Institute | Solid-phase synthesis of polynucleotides |
| US4598049A (en) | 1983-08-31 | 1986-07-01 | Systec Inc. | General purpose gene synthesizer |
| JP3391484B2 (ja) * | 1992-12-02 | 2003-03-31 | 武田薬品工業株式会社 | ヒト・エンドセリン−２遺伝子のプロモーター活性を有する領域を含有するｄｎａ |
| JPH11137251A (ja) | 1997-11-07 | 1999-05-25 | Chugai Bunshi Igaku Kenkyusho:Kk | ヒトｐ２７Ｋｉｐ１遺伝子のプロモーター |
| CN1252260C (zh) | 2000-07-21 | 2006-04-19 | 上海三维生物技术有限公司 | 肿瘤特异性启动子 |
| US20030003582A1 (en) * | 2001-05-08 | 2003-01-02 | Tranzyme, Inc. | Trans-viral vector mediated gene transfer to the retina |
| JP2003146909A (ja) * | 2001-11-07 | 2003-05-21 | Children's Hospital Of Philadelphia | 治療用ポリペプチドに対する免疫寛容の誘導 |
| US20070036756A1 (en) | 2003-02-19 | 2007-02-15 | Dnavec Research, Inc. | Method for treating ischemic diseases |
| BRPI0415563A (pt) * | 2003-10-20 | 2007-01-02 | Nsgene As | método para o tratamento do mal de parkinson, uso de um vetor viral, vetor de expressão viral, composição farmacêutica, célula hospedeira isolada, linhagem de células de empacotamento, mamìfero não humano quimérico, dispositivo de cultura celular implantável, cápsula biocompatìvel, método para tratamento de uma doença do sistema nervoso, célula de mamìfero, e, método para produzir neurturin ou um seu equivalente funcional |
| EP2021499A4 (en) * | 2005-12-16 | 2010-02-17 | Univ Leland Stanford Junior | FUNCTIONAL ARRAYS FOR THE IDENTIFICATION OF ELEMENTS FOR GENE PRESSION REGULATION WITH HIGH THROUGHPUT |
| US20070161031A1 (en) * | 2005-12-16 | 2007-07-12 | The Board Of Trustees Of The Leland Stanford Junior University | Functional arrays for high throughput characterization of gene expression regulatory elements |
| WO2008073303A2 (en) * | 2006-12-07 | 2008-06-19 | Switchgear Genomics | Transcriptional regulatory elements of biological pathways, tools, and methods |
| ES2751999T3 (es) * | 2008-01-29 | 2020-04-02 | Applied Genetic Tech Corporation | Producción recombinante de virus adeno-asociados usando células BHK en suspensión |
| US20100081707A1 (en) * | 2008-02-21 | 2010-04-01 | Ali Robin R | Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea |
| EP3591055A1 (en) * | 2011-01-07 | 2020-01-08 | Applied Genetic Technologies Corporation | Promoters derived from the 5' nontranslated region (5'-ntr) of the human cyclic nucleotide gated beta 3 (cngb3) gene, expression cassettes and vectors comprising said promoters, for use treating achromatopsia and other diseases |
| WO2017144080A1 (en) * | 2016-02-23 | 2017-08-31 | Eyeserv Gmbh | Gene therapy for the treatment of a disease of retinal cone cells |
-
2012
- 2012-01-06 EP EP19178977.5A patent/EP3591055A1/en active Pending
- 2012-01-06 WO PCT/US2012/020423 patent/WO2012094560A2/en active Application Filing
- 2012-01-06 EP EP12731925.9A patent/EP2661494B1/en active Active
- 2012-01-06 DK DK12731925.9T patent/DK2661494T3/da active
- 2012-01-06 CN CN201280012016.3A patent/CN103608455B/zh active Active
- 2012-01-06 JP JP2013548562A patent/JP6345423B2/ja active Active
- 2012-01-06 CA CA2823890A patent/CA2823890C/en active Active
- 2012-01-06 AU AU2012204266A patent/AU2012204266C1/en active Active
- 2012-01-06 CN CN201810178811.9A patent/CN108410872A/zh active Pending
-
2013
- 2013-07-08 US US13/936,728 patent/US9982275B2/en active Active
-
2016
- 2016-11-04 JP JP2016216478A patent/JP6509182B2/ja active Active
-
2017
- 2017-04-07 AU AU2017202320A patent/AU2017202320B2/en active Active
-
2018
- 2018-04-23 US US15/960,048 patent/US20190002913A1/en active Pending
-
2019
- 2019-10-18 AU AU2019250249A patent/AU2019250249A1/en not_active Abandoned
-
2022
- 2022-03-02 AU AU2022201451A patent/AU2022201451A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP2661494B1 (en) | 2019-06-12 |
| AU2012204266A1 (en) | 2013-07-11 |
| EP3591055A1 (en) | 2020-01-08 |
| JP6509182B2 (ja) | 2019-05-08 |
| AU2017202320B2 (en) | 2019-07-18 |
| JP2014504871A (ja) | 2014-02-27 |
| WO2012094560A3 (en) | 2012-10-26 |
| AU2012204266B2 (en) | 2017-05-11 |
| AU2012204266C1 (en) | 2017-12-21 |
| EP2661494A2 (en) | 2013-11-13 |
| US20130317091A1 (en) | 2013-11-28 |
| CA2823890C (en) | 2020-10-06 |
| WO2012094560A2 (en) | 2012-07-12 |
| US9982275B2 (en) | 2018-05-29 |
| AU2019250249A1 (en) | 2019-11-07 |
| EP2661494A4 (en) | 2015-03-25 |
| US20190002913A1 (en) | 2019-01-03 |
| CN103608455B (zh) | 2019-01-15 |
| JP2017060487A (ja) | 2017-03-30 |
| CN108410872A (zh) | 2018-08-17 |
| NZ612375A (en) | 2015-08-28 |
| AU2022201451A1 (en) | 2022-03-24 |
| AU2017202320A1 (en) | 2017-04-27 |
| CA2823890A1 (en) | 2012-07-12 |
| JP6345423B2 (ja) | 2018-06-20 |
| CN103608455A (zh) | 2014-02-26 |
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