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
- Authority
- JP
- Japan
- 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.)
- Granted
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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 JP2022088656A (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 JP2022088656A (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 (https=) | 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 Pending JP2022088656A (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 Pending JP2022088656A (ja) | 2015-12-14 | 2022-04-07 | 眼疾患のための遺伝子療法 |
Country Status (12)
| Country | Link |
|---|---|
| US (2) | US11090392B2 (https=) |
| EP (2) | EP3389724B1 (https=) |
| JP (2) | JP7057281B2 (https=) |
| KR (1) | KR20180099719A (https=) |
| CN (1) | CN109069668B (https=) |
| AU (1) | AU2016370487C1 (https=) |
| BR (1) | BR112018011838A2 (https=) |
| CA (1) | CA3008264A1 (https=) |
| ES (2) | ES2826384T3 (https=) |
| MX (2) | MX2018007230A (https=) |
| RU (1) | RU2762747C2 (https=) |
| WO (1) | WO2017106202A2 (https=) |
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11090392B2 (en) * | 2015-12-14 | 2021-08-17 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
| PT3377637T (pt) | 2016-04-08 | 2020-06-17 | Krystal Biotech Inc | Composições para uso em métodos para o tratamento de ferimentos, transtornos e doenças da pele |
| AU2017301819B2 (en) * | 2016-07-26 | 2024-09-26 | 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 |
| SG11202009895TA (en) | 2018-04-27 | 2020-11-27 | Krystal Biotech Inc | Recombinant nucleic acids encoding cosmetic protein(s) for aesthetic applications |
| WO2020163703A1 (en) | 2019-02-08 | 2020-08-13 | Krystal Biotech, Inc. | Compositions and methods for delivering cftr polypeptides |
| EP3934699A4 (en) * | 2019-03-04 | 2022-12-21 | 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 |
| IL297635A (en) * | 2020-04-27 | 2022-12-01 | Univ Pennsylvania | Compositions useful in treatment of cdkl5 deficiency disorder (cdd) |
| IL300752A (en) * | 2020-09-02 | 2023-04-01 | 4D Molecular Therapeutics Inc | Codon-optimized REP1 genes and their uses |
| WO2022165313A1 (en) | 2021-02-01 | 2022-08-04 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| AU2022227005B2 (en) * | 2021-02-26 | 2025-09-11 | Logicbio Therapeutics, Inc. | Manufacturing and use of recombinant aav vectors |
| BR112023020209A2 (pt) | 2021-04-02 | 2023-12-19 | Krystal Biotech Inc | Genoma do vírus do herpes recombinante, vírus do herpes, composição farmacêutica, uso do vírus do herpes ou da composição farmacêutica, método para expressar, realçar, aumentar, ampliar e/ou suplementar os níveis de um polipeptídeo imunomodulatório, método para prover alívio profilático, paliativo ou terapêutico, e, método para tratar câncer |
| US20240335562A1 (en) * | 2021-08-04 | 2024-10-10 | Alpine Biotherapeutics Corporation | Sustainable ocular cell-mediated intraocular delivery of cellular therapeutics for treatment of ocular diseases or disorders |
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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 |
| US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | 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 |
| WO1998046728A1 (en) | 1997-04-14 | 1998-10-22 | Cell Genesys, Inc. | Methods for increasing the efficiency of recombinant aav product |
| WO1999061643A1 (en) | 1998-05-27 | 1999-12-02 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions by using an iodixananol gradient |
| ES2340230T3 (es) | 1998-11-10 | 2010-05-31 | University Of North Carolina At Chapel Hill | Vectores viricos y sus procedimientos de preparacion y administracion. |
| PT1916258E (pt) | 1999-08-09 | 2014-07-29 | Genzyme Corp | Aumento da expressão de uma sequência nucleotídica heteróloga de cadeia simples a partir de vectores virais recombinantes por concepção da sequência de maneira que esta forme pares de bases intracadeia |
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| DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| 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 |
| WO2011034947A2 (en) * | 2009-09-15 | 2011-03-24 | University Of Washington | Reagents and methods for modulating cone photoreceptor activity |
| US20130023033A1 (en) | 2010-03-29 | 2013-01-24 | 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 |
| 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 |
| 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 |
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| US20140314740A1 (en) * | 2011-12-09 | 2014-10-23 | The Johns Hopkins University | Compositions and methods for the prevention or treatment of diabetic complications |
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| EP2872183B1 (en) | 2012-07-11 | 2018-09-26 | The Trustees Of The University Of Pennsylvania | Aav-mediated gene therapy for rpgr x-linked retinal degeneration |
| 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 |
| US9567376B2 (en) | 2013-02-08 | 2017-02-14 | The Trustees Of The University Of Pennsylvania | Enhanced AAV-mediated gene transfer for retinal therapies |
| CA2909733C (en) * | 2013-04-18 | 2023-10-17 | Fondazione Telethon | Effective delivery of large genes by dual aav vectors |
| AU2014265799B2 (en) * | 2013-05-16 | 2019-10-31 | Applied Genetic Technologies Corporation | Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases |
| GB201322798D0 (en) * | 2013-12-20 | 2014-02-05 | Oxford Biomedica Ltd | Production system |
| KR102537394B1 (ko) | 2014-03-17 | 2023-05-30 | 애드베룸 바이오테크놀로지스, 인코포레이티드 | 원추세포에서 증강된 유전자 발현을 위한 조성물 및 방법 |
| CA2945965C (en) * | 2014-04-15 | 2022-11-01 | 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 |
| US11090392B2 (en) * | 2015-12-14 | 2021-08-17 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
-
2016
- 2016-12-13 US US16/061,530 patent/US11090392B2/en active Active
- 2016-12-13 ES ES16852859T patent/ES2826384T3/es active Active
- 2016-12-13 AU AU2016370487A patent/AU2016370487C1/en active Active
- 2016-12-13 ES ES20183330T patent/ES3061210T3/es active Active
- 2016-12-13 MX MX2018007230A patent/MX2018007230A/es unknown
- 2016-12-13 KR KR1020187019811A patent/KR20180099719A/ko not_active Ceased
- 2016-12-13 EP EP16852859.4A patent/EP3389724B1/en active Active
- 2016-12-13 CN CN201680081801.2A patent/CN109069668B/zh active Active
- 2016-12-13 BR BR112018011838-9A patent/BR112018011838A2/pt active IP Right Grant
- 2016-12-13 RU RU2018125468A patent/RU2762747C2/ru active
- 2016-12-13 EP EP20183330.8A patent/EP3795180B1/en active Active
- 2016-12-13 CA CA3008264A patent/CA3008264A1/en active Pending
- 2016-12-13 WO PCT/US2016/066402 patent/WO2017106202A2/en not_active Ceased
- 2016-12-13 JP JP2018531202A patent/JP7057281B2/ja active 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/JP2022088656A/ja active Pending
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