JP7766393B2 - 眼疾患のための遺伝子療法 - Google Patents
眼疾患のための遺伝子療法Info
- Publication number
- JP7766393B2 JP7766393B2 JP2019569784A JP2019569784A JP7766393B2 JP 7766393 B2 JP7766393 B2 JP 7766393B2 JP 2019569784 A JP2019569784 A JP 2019569784A JP 2019569784 A JP2019569784 A JP 2019569784A JP 7766393 B2 JP7766393 B2 JP 7766393B2
- Authority
- JP
- Japan
- Prior art keywords
- promoter
- aav
- sequence
- seq
- expression
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Active
Links
Classifications
-
- 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
-
- 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
-
- 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
- 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
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4702—Regulators; Modulating activity
- C07K14/4705—Regulators; Modulating activity stimulating, promoting or activating 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
- 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
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0048—Eye, e.g. artificial tears
-
- 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
- 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
- C12N2750/14171—Demonstrated in vivo effect
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- General Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Cell Biology (AREA)
- Immunology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023098376A JP2023116709A (ja) | 2017-06-14 | 2023-06-15 | 眼疾患のための遺伝子療法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762519821P | 2017-06-14 | 2017-06-14 | |
| US62/519,821 | 2017-06-14 | ||
| PCT/US2018/037592 WO2018232149A1 (en) | 2017-06-14 | 2018-06-14 | Gene therapy for ocular disorders |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023098376A Division JP2023116709A (ja) | 2017-06-14 | 2023-06-15 | 眼疾患のための遺伝子療法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020527335A JP2020527335A (ja) | 2020-09-10 |
| JP2020527335A5 JP2020527335A5 (enExample) | 2021-07-26 |
| JP7766393B2 true JP7766393B2 (ja) | 2025-11-10 |
Family
ID=64660849
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019569784A Active JP7766393B2 (ja) | 2017-06-14 | 2018-06-14 | 眼疾患のための遺伝子療法 |
| JP2023098376A Pending JP2023116709A (ja) | 2017-06-14 | 2023-06-15 | 眼疾患のための遺伝子療法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023098376A Pending JP2023116709A (ja) | 2017-06-14 | 2023-06-15 | 眼疾患のための遺伝子療法 |
Country Status (4)
| Country | Link |
|---|---|
| US (1) | US11827898B2 (enExample) |
| EP (1) | EP3638316A4 (enExample) |
| JP (2) | JP7766393B2 (enExample) |
| WO (1) | WO2018232149A1 (enExample) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3638316A4 (en) * | 2017-06-14 | 2021-03-24 | The Trustees Of The University Of Pennsylvania | GENE THERAPY FOR EYE DISEASES |
| US20220143217A1 (en) * | 2019-03-04 | 2022-05-12 | The Trustees Of The University Of Pennsylvania | Neuroprotective gene therapy targeting the akt pathway |
| CN116806158A (zh) | 2020-09-02 | 2023-09-26 | 4D分子治疗有限公司 | 密码子优化的rep1基因及其用途 |
| EP4284335A1 (en) | 2021-02-01 | 2023-12-06 | RegenxBio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| CN113322281B (zh) * | 2021-05-12 | 2024-01-05 | 成都金唯科生物科技有限公司 | 一种高效组织特异性表达rs1蛋白的重组腺相关病毒及应用 |
| CA3257440A1 (en) * | 2022-07-06 | 2024-01-11 | Adverum Biotechnologies Inc | COMPOSITIONS AND METHODS FOR THE TREATMENT OF ACHROMOTOPSY |
| CN116411024A (zh) * | 2023-03-10 | 2023-07-11 | 上海泰昶生物技术有限公司 | 用于生产重组腺相关病毒的质粒系统 |
Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2014504871A (ja) | 2011-01-07 | 2014-02-27 | アプライド ジェネティック テクノロジーズ コーポレイション | 1色覚および他の疾患の治療のためのプロモーター、発現カセット、ベクター、キット、ならびに方法 |
| JP2016520309A (ja) | 2013-05-16 | 2016-07-14 | アプライド ジェネティック テクノロジーズ コーポレイション | 1色覚および他の疾患の治療のためのプロモーター、発現カセット、ベクター、キット、ならびに方法 |
Family Cites Families (42)
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|---|---|---|---|---|
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
| US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US5741683A (en) | 1995-06-07 | 1998-04-21 | The Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| DE69824859T2 (de) | 1997-04-14 | 2005-08-04 | Cell Genesys, Inc., Foster City | Methoden zur erhöhung der effizienz rekombinanter aav produkte |
| US6146874A (en) | 1998-05-27 | 2000-11-14 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions |
| AU780231B2 (en) | 1998-11-10 | 2005-03-10 | University Of North Carolina At Chapel Hill, The | Virus vectors and methods of making and administering the same |
| DE60039766D1 (de) | 1999-08-09 | 2008-09-18 | Targeted Genetics Corp | Terologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen |
| AU6814901A (en) | 2000-06-01 | 2001-12-11 | Univ North Carolina | Methods and compounds for controlled release of recombinant parvovirus vectors |
| EP1409677A2 (en) | 2001-01-19 | 2004-04-21 | Incyte Genomics, Inc. | Receptors and membrane-associated proteins |
| EP1381276A4 (en) * | 2001-04-13 | 2005-02-02 | Univ Pennsylvania | METHOD FOR TREATMENT OR DEVELOPMENT SLUDGE DEGRADATION |
| CA2915124C (en) | 2001-11-13 | 2018-08-14 | The Trustees Of The University Of Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| ES2602352T3 (es) | 2001-12-17 | 2017-02-20 | The Trustees Of The University Of Pennsylvania | Secuencias de serotipo 8 de virus adenoasociado (VAA), vectores que las contienen y usos de las mismas |
| WO2003104413A2 (en) | 2002-06-05 | 2003-12-18 | University Of Florida | Production of pseudotyped recombinant aav virions |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| EP2298926A1 (en) | 2003-09-30 | 2011-03-23 | The Trustees of The University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| EP2357010B1 (en) | 2005-04-07 | 2013-06-12 | The Trustees of The University of Pennsylvania | Method of increasing the function of an AAV vector |
| US7456683B2 (en) | 2005-06-09 | 2008-11-25 | Panasonic Corporation | Amplitude error compensating device and quadrature skew error compensating device |
| EP2007795B1 (en) | 2006-03-30 | 2016-11-16 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid proteins |
| WO2010005533A2 (en) | 2008-06-30 | 2010-01-14 | The Johns Hopkins University | Compositions and methods for the treatment of ocular oxidative stress and retinitis pigmentosa |
| WO2010099383A2 (en) | 2009-02-26 | 2010-09-02 | The Johns Hopkins University | Compositions and methods for ex vivo hepatic nucleic acid delivery |
| US20120172419A1 (en) | 2009-09-15 | 2012-07-05 | Medical College Of Wisconsin Research Foundation Inc. | Reagents and methods for modulating cone photoreceptor activity |
| SG10201502270TA (en) | 2010-03-29 | 2015-05-28 | Univ Pennsylvania | Pharmacologically induced transgene ablation system |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| GB201103062D0 (en) | 2011-02-22 | 2011-04-06 | Isis Innovation | Method |
| WO2012158757A1 (en) | 2011-05-16 | 2012-11-22 | The Trustees Of The University Of Pennsylvania | Proviral plasmids for production of recombinant adeno-associated virus |
| US9375491B2 (en) | 2011-10-28 | 2016-06-28 | University Of Florida Research Foundation, Inc. | Chimeric promoter for cone photoreceptor targeted gene therapy |
| WO2013086515A1 (en) | 2011-12-09 | 2013-06-13 | The Johns Hopkins University | Compositions and methods for the prevention or treatment of diabetic complications |
| DE18200782T1 (de) | 2012-04-02 | 2021-10-21 | Modernatx, Inc. | Modifizierte polynukleotide zur herstellung von proteinen im zusammenhang mit erkrankungen beim menschen |
| CA2878171C (en) | 2012-07-11 | 2021-04-27 | The Trustees Of The University Of Pennsylvania | Aav-mediated gene therapy for rpgr x-linked retinal degeneration |
| US9567376B2 (en) | 2013-02-08 | 2017-02-14 | The Trustees Of The University Of Pennsylvania | Enhanced AAV-mediated gene transfer for retinal therapies |
| US10494645B2 (en) | 2013-04-18 | 2019-12-03 | Fondazione Telethon | Effective delivery of large genes by dual AAV vectors |
| GB201322798D0 (en) | 2013-12-20 | 2014-02-05 | Oxford Biomedica Ltd | Production system |
| KR102288849B1 (ko) * | 2014-03-17 | 2021-08-12 | 애드베룸 바이오테크놀로지스, 인코포레이티드 | 원추세포에서 증강된 유전자 발현을 위한 조성물 및 방법 |
| JP6635942B2 (ja) | 2014-04-15 | 2020-01-29 | アプライド ジェネティック テクノロジーズ コーポレイション | 網膜色素変性症gtpアーゼレギュレーター(rpgr)をコードするコドン最適化核酸 |
| US20160206704A1 (en) | 2015-01-16 | 2016-07-21 | Isis Innovation Limited | Method |
| EP3389724B1 (en) | 2015-12-14 | 2020-07-01 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
| EP3638316A4 (en) * | 2017-06-14 | 2021-03-24 | The Trustees Of The University Of Pennsylvania | GENE THERAPY FOR EYE DISEASES |
-
2018
- 2018-06-14 EP EP18817402.3A patent/EP3638316A4/en active Pending
- 2018-06-14 WO PCT/US2018/037592 patent/WO2018232149A1/en not_active Ceased
- 2018-06-14 JP JP2019569784A patent/JP7766393B2/ja active Active
- 2018-06-14 US US16/621,627 patent/US11827898B2/en active Active
-
2023
- 2023-06-15 JP JP2023098376A patent/JP2023116709A/ja active Pending
Patent Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2014504871A (ja) | 2011-01-07 | 2014-02-27 | アプライド ジェネティック テクノロジーズ コーポレイション | 1色覚および他の疾患の治療のためのプロモーター、発現カセット、ベクター、キット、ならびに方法 |
| JP2016520309A (ja) | 2013-05-16 | 2016-07-14 | アプライド ジェネティック テクノロジーズ コーポレイション | 1色覚および他の疾患の治療のためのプロモーター、発現カセット、ベクター、キット、ならびに方法 |
Non-Patent Citations (2)
| Title |
|---|
| Hum Gene Ther, 2016, Vol.27, No.1, pp.72-82, doi: 10.1089/hum.2015.130 |
| Human Molecular Genetics, 2011, Vol.20, Issue 16, pp.3161-3175, https://doi.org/10.1093/hmg/ddr218 |
Also Published As
| Publication number | Publication date |
|---|---|
| US20200172929A1 (en) | 2020-06-04 |
| JP2020527335A (ja) | 2020-09-10 |
| EP3638316A1 (en) | 2020-04-22 |
| EP3638316A4 (en) | 2021-03-24 |
| JP2023116709A (ja) | 2023-08-22 |
| WO2018232149A1 (en) | 2018-12-20 |
| US11827898B2 (en) | 2023-11-28 |
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