JP2019502378A5 - - Google Patents
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- JP2019502378A5 JP2019502378A5 JP2018531202A JP2018531202A JP2019502378A5 JP 2019502378 A5 JP2019502378 A5 JP 2019502378A5 JP 2018531202 A JP2018531202 A JP 2018531202A JP 2018531202 A JP2018531202 A JP 2018531202A JP 2019502378 A5 JP2019502378 A5 JP 2019502378A5
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- Prior art keywords
- promoter
- seq
- viral vector
- sequence
- aav
- 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.)
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- 239000013603 viral vector Substances 0.000 claims 20
- 230000014509 gene expression Effects 0.000 claims 18
- 239000000203 mixture Substances 0.000 claims 16
- 239000013598 vector Substances 0.000 claims 14
- 102100022881 Rab proteins geranylgeranyltransferase component A 1 Human genes 0.000 claims 11
- 101710088575 Rab escort protein 1 Proteins 0.000 claims 10
- 101710108890 Rab proteins geranylgeranyltransferase component A 1 Proteins 0.000 claims 10
- 102100029142 Cyclic nucleotide-gated cation channel alpha-3 Human genes 0.000 claims 8
- 101000771071 Homo sapiens Cyclic nucleotide-gated cation channel alpha-3 Proteins 0.000 claims 8
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 8
- 150000007523 nucleic acids Chemical group 0.000 claims 8
- 241000700605 Viruses Species 0.000 claims 7
- 210000000234 capsid Anatomy 0.000 claims 6
- 108020004705 Codon Proteins 0.000 claims 5
- 241000702421 Dependoparvovirus Species 0.000 claims 4
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 4
- 239000002299 complementary DNA Substances 0.000 claims 4
- 239000013612 plasmid Substances 0.000 claims 4
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 3
- 238000004519 manufacturing process Methods 0.000 claims 3
- 238000000034 method Methods 0.000 claims 3
- 108090000623 proteins and genes Proteins 0.000 claims 3
- 102000004169 proteins and genes Human genes 0.000 claims 3
- NCYCYZXNIZJOKI-IOUUIBBYSA-N 11-cis-retinal Chemical group O=C/C=C(\C)/C=C\C=C(/C)\C=C\C1=C(C)CCCC1(C)C NCYCYZXNIZJOKI-IOUUIBBYSA-N 0.000 claims 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 2
- 108091006146 Channels Proteins 0.000 claims 2
- 208000006992 Color Vision Defects Diseases 0.000 claims 2
- 241000282412 Homo Species 0.000 claims 2
- 101000620777 Homo sapiens Rab proteins geranylgeranyltransferase component A 1 Proteins 0.000 claims 2
- 102100038300 Metabotropic glutamate receptor 6 Human genes 0.000 claims 2
- 102000004330 Rhodopsin Human genes 0.000 claims 2
- 108090000820 Rhodopsin Proteins 0.000 claims 2
- 208000003571 choroideremia Diseases 0.000 claims 2
- 201000007254 color blindness Diseases 0.000 claims 2
- 125000004122 cyclic group Chemical group 0.000 claims 2
- 239000003814 drug Substances 0.000 claims 2
- 230000001939 inductive effect Effects 0.000 claims 2
- 108010038450 metabotropic glutamate receptor 6 Proteins 0.000 claims 2
- 238000004806 packaging method and process Methods 0.000 claims 2
- 230000003612 virological effect Effects 0.000 claims 2
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 1
- 239000013607 AAV vector Substances 0.000 claims 1
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 1
- 102000000806 Basic-Leucine Zipper Transcription Factors Human genes 0.000 claims 1
- 108010001572 Basic-Leucine Zipper Transcription Factors Proteins 0.000 claims 1
- 108090000565 Capsid Proteins Proteins 0.000 claims 1
- 102100023321 Ceruloplasmin Human genes 0.000 claims 1
- 208000033810 Choroidal dystrophy Diseases 0.000 claims 1
- 108010003730 Cone Opsins Proteins 0.000 claims 1
- UPEZCKBFRMILAV-JNEQICEOSA-N Ecdysone Natural products O=C1[C@H]2[C@@](C)([C@@H]3C([C@@]4(O)[C@@](C)([C@H]([C@H]([C@@H](O)CCC(O)(C)C)C)CC4)CC3)=C1)C[C@H](O)[C@H](O)C2 UPEZCKBFRMILAV-JNEQICEOSA-N 0.000 claims 1
- 102000004437 G-Protein-Coupled Receptor Kinase 1 Human genes 0.000 claims 1
- 108091004242 G-Protein-Coupled Receptor Kinase 1 Proteins 0.000 claims 1
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims 1
- ROHFNLRQFUQHCH-YFKPBYRVSA-N L-leucine Chemical compound CC(C)C[C@H](N)C(O)=O ROHFNLRQFUQHCH-YFKPBYRVSA-N 0.000 claims 1
- ROHFNLRQFUQHCH-UHFFFAOYSA-N Leucine Natural products CC(C)CC(N)C(O)=O ROHFNLRQFUQHCH-UHFFFAOYSA-N 0.000 claims 1
- 241000124008 Mammalia Species 0.000 claims 1
- 102100029533 Photoreceptor-specific nuclear receptor Human genes 0.000 claims 1
- 101710164507 Photoreceptor-specific nuclear receptor Proteins 0.000 claims 1
- 108090000799 Rhodopsin kinases Proteins 0.000 claims 1
- 239000004098 Tetracycline Substances 0.000 claims 1
- 108091023040 Transcription factor Proteins 0.000 claims 1
- 102000040945 Transcription factor Human genes 0.000 claims 1
- UPEZCKBFRMILAV-UHFFFAOYSA-N alpha-Ecdysone Natural products C1C(O)C(O)CC2(C)C(CCC3(C(C(C(O)CCC(C)(C)O)C)CCC33O)C)C3=CC(=O)C21 UPEZCKBFRMILAV-UHFFFAOYSA-N 0.000 claims 1
- 210000003161 choroid Anatomy 0.000 claims 1
- 238000012258 culturing Methods 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- UPEZCKBFRMILAV-JMZLNJERSA-N ecdysone Chemical compound C1[C@@H](O)[C@@H](O)C[C@]2(C)[C@@H](CC[C@@]3([C@@H]([C@@H]([C@H](O)CCC(C)(C)O)C)CC[C@]33O)C)C3=CC(=O)[C@@H]21 UPEZCKBFRMILAV-JMZLNJERSA-N 0.000 claims 1
- 239000003623 enhancer Substances 0.000 claims 1
- 229940011871 estrogen Drugs 0.000 claims 1
- 239000000262 estrogen Substances 0.000 claims 1
- 239000000833 heterodimer Substances 0.000 claims 1
- 102000049762 human CHM Human genes 0.000 claims 1
- 208000015181 infectious disease Diseases 0.000 claims 1
- 230000002458 infectious effect Effects 0.000 claims 1
- 230000001537 neural effect Effects 0.000 claims 1
- 239000002773 nucleotide Substances 0.000 claims 1
- 125000003729 nucleotide group Chemical group 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 230000001124 posttranscriptional effect Effects 0.000 claims 1
- ZAHRKKWIAAJSAO-UHFFFAOYSA-N rapamycin Natural products COCC(O)C(=C/C(C)C(=O)CC(OC(=O)C1CCCCN1C(=O)C(=O)C2(O)OC(CC(OC)C(=CC=CC=CC(C)CC(C)C(=O)C)C)CCC2C)C(C)CC3CCC(O)C(C3)OC)C ZAHRKKWIAAJSAO-UHFFFAOYSA-N 0.000 claims 1
- 230000001105 regulatory effect Effects 0.000 claims 1
- 230000002207 retinal effect Effects 0.000 claims 1
- QFJCIRLUMZQUOT-HPLJOQBZSA-N sirolimus Chemical compound C1C[C@@H](O)[C@H](OC)C[C@@H]1C[C@@H](C)[C@H]1OC(=O)[C@@H]2CCCCN2C(=O)C(=O)[C@](O)(O2)[C@H](C)CC[C@H]2C[C@H](OC)/C(C)=C/C=C/C=C/[C@@H](C)C[C@@H](C)C(=O)[C@H](OC)[C@H](O)/C(C)=C/[C@@H](C)C(=O)C1 QFJCIRLUMZQUOT-HPLJOQBZSA-N 0.000 claims 1
- 229960002930 sirolimus Drugs 0.000 claims 1
- 229930101283 tetracycline Natural products 0.000 claims 1
- 229960002180 tetracycline Drugs 0.000 claims 1
- 235000019364 tetracycline Nutrition 0.000 claims 1
- 150000003522 tetracyclines Chemical class 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022063749A JP7878917B2 (ja) | 2015-12-14 | 2022-04-07 | 眼疾患のための遺伝子療法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562266789P | 2015-12-14 | 2015-12-14 | |
| US62/266,789 | 2015-12-14 | ||
| PCT/US2016/066402 WO2017106202A2 (en) | 2015-12-14 | 2016-12-13 | Gene therapy for ocular disorders |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022063749A Division JP7878917B2 (ja) | 2015-12-14 | 2022-04-07 | 眼疾患のための遺伝子療法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019502378A JP2019502378A (ja) | 2019-01-31 |
| JP2019502378A5 true JP2019502378A5 (enExample) | 2020-01-30 |
| JP7057281B2 JP7057281B2 (ja) | 2022-04-19 |
Family
ID=58455636
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018531202A Active JP7057281B2 (ja) | 2015-12-14 | 2016-12-13 | 眼疾患のための遺伝子療法 |
| JP2022063749A Active JP7878917B2 (ja) | 2015-12-14 | 2022-04-07 | 眼疾患のための遺伝子療法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022063749A Active JP7878917B2 (ja) | 2015-12-14 | 2022-04-07 | 眼疾患のための遺伝子療法 |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US11090392B2 (enExample) |
| EP (2) | EP3389724B1 (enExample) |
| JP (2) | JP7057281B2 (enExample) |
| KR (1) | KR20180099719A (enExample) |
| CN (1) | CN109069668B (enExample) |
| AU (1) | AU2016370487C1 (enExample) |
| ES (2) | ES3061210T3 (enExample) |
| MX (2) | MX2018007230A (enExample) |
| RU (1) | RU2762747C2 (enExample) |
| WO (1) | WO2017106202A2 (enExample) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN109069668B (zh) * | 2015-12-14 | 2023-04-18 | 宾夕法尼亚州大学信托人 | 用于眼病的基因疗法 |
| ES2796488T3 (es) | 2016-04-08 | 2020-11-27 | Krystal Biotech Inc | Composiciones para el uso en métodos para el tratamiento de heridas, trastornos y enfermedades de la piel |
| US20190160187A1 (en) * | 2016-07-26 | 2019-05-30 | Cornell University | Gene therapy for the treatment of aldehyde dehydrogenase deficiency |
| US11827898B2 (en) | 2017-06-14 | 2023-11-28 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
| MX2020011257A (es) | 2018-04-27 | 2021-01-15 | Krystal Biotech Inc | Ácidos nucleicos recombinantes que codifican una o más proteínas cosméticas para usos estéticos. |
| EP3856914A1 (en) | 2018-09-26 | 2021-08-04 | Krystal Biotech, Inc. | Compositions and methods for the treatment of skin diseases |
| CN113454105A (zh) | 2019-02-08 | 2021-09-28 | 克里斯托生物技术股份有限公司 | 用于递送cftr多肽的组合物和方法 |
| AU2020232692A1 (en) * | 2019-03-04 | 2021-09-30 | The Trustees Of The University Of Pennsylvania | Neuroprotective gene therapy targeting the AKT pathway |
| CN110889859A (zh) * | 2019-11-11 | 2020-03-17 | 珠海上工医信科技有限公司 | 一种用于眼底图像血管分割的u型网络 |
| JP7862321B2 (ja) * | 2020-04-27 | 2026-05-19 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | Ｃｄｋｌ５欠損症（ｃｄｄ）の治療において有用な組成物 |
| CA3191545A1 (en) * | 2020-09-02 | 2022-03-10 | David H. Kirn | Codon optimized rep1 genes and uses thereof |
| MX2023008826A (es) | 2021-02-01 | 2023-09-15 | Regenxbio Inc | Terapia génica para lipofuscinosis neuronal ceroidea. |
| WO2022182986A1 (en) * | 2021-02-26 | 2022-09-01 | Logicbio Therapeutics, Inc. | Manufacturing and use of recombinant aav vectors |
| IL307350A (en) | 2021-04-02 | 2023-11-01 | Krystal Biotech Inc | Viral vectors for cancer treatment |
| WO2023014892A1 (en) * | 2021-08-04 | 2023-02-09 | Alpine Biotherapeutics Corporation | Sustainable ocular cell-mediated intraocular delivery of cellular therapeutics for treatment of ocular diseases or disorders |
Family Cites Families (46)
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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 |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| 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 |
| EP1007637B1 (en) | 1997-04-14 | 2004-06-30 | Cell Genesys, Inc. | Methods for increasing the efficiency of recombinant aav product |
| US6146874A (en) | 1998-05-27 | 2000-11-14 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions |
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| US6596535B1 (en) | 1999-08-09 | 2003-07-22 | Targeted Genetics Corporation | Metabolically activated recombinant viral vectors and methods for the preparation and use |
| AU6814901A (en) | 2000-06-01 | 2001-12-11 | Univ North Carolina | Methods and compounds for controlled release of recombinant parvovirus vectors |
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| CA2442670A1 (en) | 2001-04-13 | 2002-10-24 | The Trustees Of The University Of Pennsylvania | Method of treating or retarding the development of blindness |
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| AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | University Of Florida | Production of pseudotyped recombinant aav virions |
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| RU2444977C2 (ru) | 2009-08-03 | 2012-03-20 | Федеральное государственное учреждение "МОСКОВСКИЙ НАУЧНО-ИССЛЕДОВАТЕЛЬСКИЙ ИНСТИТУТ ГЛАЗНЫХ БОЛЕЗНЕЙ ИМЕНИ ГЕЛЬМГОЛЬЦА ФЕДЕРАЛЬНОГО АГЕНТСТВА ПО ВЫСОКОТЕХНОЛОГИЧНОЙ МЕДИЦИНСКОЙ ПОМОЩИ" | Способ диагностики ахроматопсии |
| US20120172419A1 (en) * | 2009-09-15 | 2012-07-05 | Medical College Of Wisconsin Research Foundation Inc. | Reagents and methods for modulating cone photoreceptor activity |
| WO2011126808A2 (en) | 2010-03-29 | 2011-10-13 | The Trustees Of The University Of 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 |
| CN103608455B (zh) * | 2011-01-07 | 2019-01-15 | 应用遗传科技公司 | 用于治疗色盲和其它疾病的启动子、表达盒、载体、药盒和方法 |
| GB201103062D0 (en) * | 2011-02-22 | 2011-04-06 | Isis Innovation | Method |
| US9249425B2 (en) | 2011-05-16 | 2016-02-02 | The Trustees Of The University Of Pennslyvania | Proviral plasmids and production of recombinant adeno-associated virus |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| 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 |
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| CN105120901A (zh) | 2012-07-11 | 2015-12-02 | 宾夕法尼亚大学托管会 | Rpgr x染色体连锁视网膜退化的aav介导的基因治疗 |
| JP6129180B2 (ja) | 2012-08-08 | 2017-05-17 | 日本パーカライジング株式会社 | 金属表面処理液、金属基材の表面処理方法及びそれにより得られた金属基材 |
| WO2014124282A1 (en) | 2013-02-08 | 2014-08-14 | The Trustees Of The University Of Pennsylvania | Enhanced aav-mediated gene transfer for retinal therapies |
| DK2986635T3 (en) | 2013-04-18 | 2019-01-28 | Fond Telethon | EFFECTIVE DELIVERY OF BIG GENES THROUGH DUAL-AAV VECTORS |
| EP2996725A4 (en) | 2013-05-16 | 2016-12-28 | Applied Genetic Tech Corp | PROMOTERS, EXPRESSION CASSETTES, VECTORS, KITS AND METHODS FOR TREATING COLOR BLINDNESS AND OTHER PROBLEMS |
| GB201322798D0 (en) * | 2013-12-20 | 2014-02-05 | Oxford Biomedica Ltd | Production system |
| ES2822551T3 (es) | 2014-03-17 | 2021-05-04 | Adverum Biotechnologies Inc | Composiciones y métodos para la expresión génica mejorada en células de los conos |
| EP3132051B1 (en) * | 2014-04-15 | 2019-03-20 | Applied Genetic Technologies Corporation | Codon optimized nucleic acid encoding a retinitis pigmentosa gtpase regulator (rpgr) |
| US20160206704A1 (en) | 2015-01-16 | 2016-07-21 | Isis Innovation Limited | Method |
| CN109069668B (zh) * | 2015-12-14 | 2023-04-18 | 宾夕法尼亚州大学信托人 | 用于眼病的基因疗法 |
-
2016
- 2016-12-13 CN CN201680081801.2A patent/CN109069668B/zh active Active
- 2016-12-13 ES ES20183330T patent/ES3061210T3/es active Active
- 2016-12-13 EP EP16852859.4A patent/EP3389724B1/en active Active
- 2016-12-13 WO PCT/US2016/066402 patent/WO2017106202A2/en not_active Ceased
- 2016-12-13 KR KR1020187019811A patent/KR20180099719A/ko not_active Ceased
- 2016-12-13 ES ES16852859T patent/ES2826384T3/es active Active
- 2016-12-13 JP JP2018531202A patent/JP7057281B2/ja active Active
- 2016-12-13 EP EP20183330.8A patent/EP3795180B1/en active Active
- 2016-12-13 US US16/061,530 patent/US11090392B2/en active Active
- 2016-12-13 MX MX2018007230A patent/MX2018007230A/es unknown
- 2016-12-13 AU AU2016370487A patent/AU2016370487C1/en active Active
- 2016-12-13 RU RU2018125468A patent/RU2762747C2/ru active
-
2018
- 2018-06-13 MX MX2022010959A patent/MX2022010959A/es unknown
-
2021
- 2021-07-07 US US17/369,525 patent/US12403204B2/en active Active
-
2022
- 2022-04-07 JP JP2022063749A patent/JP7878917B2/ja active Active
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