JP2020510433A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2020510433A5 JP2020510433A5 JP2019547457A JP2019547457A JP2020510433A5 JP 2020510433 A5 JP2020510433 A5 JP 2020510433A5 JP 2019547457 A JP2019547457 A JP 2019547457A JP 2019547457 A JP2019547457 A JP 2019547457A JP 2020510433 A5 JP2020510433 A5 JP 2020510433A5
- Authority
- JP
- Japan
- Prior art keywords
- raav
- capsid
- variant
- sequence
- promoter
- 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
Links
- 210000000234 capsid Anatomy 0.000 claims 14
- 241000702421 Dependoparvovirus Species 0.000 claims 7
- 239000007900 aqueous suspension Substances 0.000 claims 6
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 3
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 3
- LOYTUFQOTJYLPX-UHFFFAOYSA-N C1=CC=[Si]C=C1 Chemical compound C1=CC=[Si]C=C1 LOYTUFQOTJYLPX-UHFFFAOYSA-N 0.000 claims 3
- 108091026890 Coding region Proteins 0.000 claims 3
- 241000701022 Cytomegalovirus Species 0.000 claims 3
- 208000036357 GUCY2D-related recessive retinopathy Diseases 0.000 claims 3
- 201000003533 Leber congenital amaurosis Diseases 0.000 claims 3
- 239000008194 pharmaceutical composition Substances 0.000 claims 3
- 239000000725 suspension Substances 0.000 claims 3
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 2
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims 2
- 239000000203 mixture Substances 0.000 claims 2
- 239000002245 particle Substances 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
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 1
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 1
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 1
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 1
- 101001008411 Homo sapiens Lebercilin Proteins 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 102000052659 human LCA5 Human genes 0.000 claims 1
- 238000002347 injection Methods 0.000 claims 1
- 239000007924 injection Substances 0.000 claims 1
- 108020004707 nucleic acids Proteins 0.000 claims 1
- 150000007523 nucleic acids Chemical class 0.000 claims 1
- 102000039446 nucleic acids Human genes 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 229940124597 therapeutic agent Drugs 0.000 claims 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023002799A JP7671790B2 (ja) | 2017-03-01 | 2023-01-12 | 眼疾患の遺伝子治療 |
| JP2025068834A JP2025114604A (ja) | 2017-03-01 | 2025-04-18 | 眼疾患の遺伝子治療 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762465649P | 2017-03-01 | 2017-03-01 | |
| US62/465,649 | 2017-03-01 | ||
| US201762469642P | 2017-03-10 | 2017-03-10 | |
| US62/469,642 | 2017-03-10 | ||
| PCT/US2018/020470 WO2018160849A1 (en) | 2017-03-01 | 2018-03-01 | Gene therapy for ocular disorders |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023002799A Division JP7671790B2 (ja) | 2017-03-01 | 2023-01-12 | 眼疾患の遺伝子治療 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020510433A JP2020510433A (ja) | 2020-04-09 |
| JP2020510433A5 true JP2020510433A5 (enExample) | 2021-03-18 |
| JP7211960B2 JP7211960B2 (ja) | 2023-01-24 |
Family
ID=63370488
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019547457A Active JP7211960B2 (ja) | 2017-03-01 | 2018-03-01 | 眼疾患の遺伝子治療 |
| JP2023002799A Active JP7671790B2 (ja) | 2017-03-01 | 2023-01-12 | 眼疾患の遺伝子治療 |
| JP2025068834A Pending JP2025114604A (ja) | 2017-03-01 | 2025-04-18 | 眼疾患の遺伝子治療 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023002799A Active JP7671790B2 (ja) | 2017-03-01 | 2023-01-12 | 眼疾患の遺伝子治療 |
| JP2025068834A Pending JP2025114604A (ja) | 2017-03-01 | 2025-04-18 | 眼疾患の遺伝子治療 |
Country Status (11)
| Country | Link |
|---|---|
| US (2) | US11564996B2 (enExample) |
| EP (2) | EP3589738A4 (enExample) |
| JP (3) | JP7211960B2 (enExample) |
| KR (3) | KR102545070B1 (enExample) |
| CN (2) | CN119752819A (enExample) |
| AU (2) | AU2018228881B2 (enExample) |
| BR (1) | BR112019017327A2 (enExample) |
| CA (1) | CA3054136A1 (enExample) |
| IL (2) | IL314284A (enExample) |
| RU (1) | RU2019130004A (enExample) |
| WO (1) | WO2018160849A1 (enExample) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2021529001A (ja) * | 2018-06-29 | 2021-10-28 | ウーハン ニューロフス バイオテクノロジー リミテッド カンパニーＷｕｈａｎ Ｎｅｕｒｏｐｈｔｈ Ｂｉｏｔｅｃｈｎｏｌｏｇｙ Ｌｉｍｉｔｅｄ Ｃｏｍｐａｎｙ | レーベル遺伝性視神経症を治療するための組成物及び方法 |
| WO2021119544A1 (en) * | 2019-12-12 | 2021-06-17 | Novartis Ag | Injection device and injection solution transferring system |
| EP4284335A1 (en) | 2021-02-01 | 2023-12-06 | RegenxBio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| US20220387627A1 (en) * | 2021-06-03 | 2022-12-08 | Hope for Hasti | Vectors and gene therapy for treating cornelia de lange syndrome |
Family Cites Families (43)
| 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 |
| 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 |
| US6548286B1 (en) | 1997-04-14 | 2003-04-15 | Cell Genesys, Inc. | Methods for increasing the efficiency of recombinant AAV product |
| EP1080218A1 (en) | 1998-05-27 | 2001-03-07 | University of Florida | Method of preparing recombinant adeno-associated virus compositions by using an iodixanol gradient |
| ATE454445T1 (de) | 1998-11-10 | 2010-01-15 | Univ North Carolina | Virusvektoren und verfahren für ihre herstellung und verabreichung. |
| ATE403715T1 (de) | 1999-08-09 | 2008-08-15 | Targeted Genetics Corp | Erhöhung der expression einer einzelsträngigen, heterologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen |
| AU2001268149B2 (en) | 2000-06-01 | 2005-08-18 | University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
| CA2442670A1 (en) * | 2001-04-13 | 2002-10-24 | The Trustees Of The University Of Pennsylvania | Method of treating or retarding the development of blindness |
| CN103555677B (zh) | 2001-11-13 | 2018-01-30 | 宾夕法尼亚大学托管会 | 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法 |
| WO2003052051A2 (en) | 2001-12-17 | 2003-06-26 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 8 sequences |
| AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | 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 |
| EP1660970A4 (en) | 2003-08-01 | 2007-02-14 | Dna Twopointo Inc | SYSTEMS AND METHODS FOR BIOPOLYMER TECHNOLOGY |
| US8005620B2 (en) | 2003-08-01 | 2011-08-23 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
| DK3211085T3 (da) | 2003-09-30 | 2021-06-21 | Univ Pennsylvania | Klader af adeno-associeret virus (aav), sekvenser, vektorer indeholdende disse og anvendelser deraf |
| US8999678B2 (en) | 2005-04-07 | 2015-04-07 | 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 |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| WO2008065430A2 (en) * | 2006-12-01 | 2008-06-05 | Oxford Biomedica (Uk) Limited | Promoter construct |
| WO2010053572A2 (en) | 2008-11-07 | 2010-05-14 | Massachusetts Institute Of Technology | Aminoalcohol lipidoids and uses thereof |
| US20120172419A1 (en) * | 2009-09-15 | 2012-07-05 | Medical College Of Wisconsin Research Foundation Inc. | Reagents and methods for modulating cone photoreceptor activity |
| SG10201908848RA (en) | 2010-03-29 | 2019-10-30 | 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 |
| US9249425B2 (en) * | 2011-05-16 | 2016-02-02 | The Trustees Of The University Of Pennslyvania | Proviral plasmids and production of recombinant adeno-associated virus |
| PT2717893T (pt) | 2011-06-08 | 2019-08-20 | Translate Bio Inc | Composições de nanopartículas lipídicas e métodos para transferência de arnm |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| WO2013123503A1 (en) * | 2012-02-17 | 2013-08-22 | The Children's Hospital Of Philadelphia | Aav vector compositions and methods for gene transfer to cells, organs and tissues |
| US20150126589A1 (en) | 2012-06-08 | 2015-05-07 | Ethris Gmbh | Pulmonary Delivery of Messenger RNA |
| JP6199965B2 (ja) | 2012-07-11 | 2017-09-20 | 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 | Ｒｐｇｒｘ連鎖性網膜変性のａａｖ媒介型遺伝子治療 |
| US20150299864A1 (en) | 2012-08-08 | 2015-10-22 | Nihon Parkerizing Co., Ltd. | Metal surface treatment liquid, surface treatment method for metal bases, and metal base obtained by surface treatment method for metal bases |
| US9567376B2 (en) | 2013-02-08 | 2017-02-14 | The Trustees Of The University Of Pennsylvania | Enhanced AAV-mediated gene transfer for retinal therapies |
| CN105209619B (zh) * | 2013-03-11 | 2018-10-12 | 泰莱托恩基金会 | miR-204和miR-211及其用途 |
| HK1220488A1 (zh) * | 2013-03-15 | 2017-05-05 | The Children's Hospital Of Philadelphia | 含有填充者/填充物多核苷酸序列的载体及其使用方法 |
| US9158889B2 (en) * | 2013-04-26 | 2015-10-13 | Oral4D Systems Ltd. | Electronic dental charting |
| US9719106B2 (en) | 2013-04-29 | 2017-08-01 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof |
| WO2015009575A1 (en) * | 2013-07-16 | 2015-01-22 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of disorders related to cep290 |
| WO2016019364A1 (en) * | 2014-08-01 | 2016-02-04 | The Trustees Of The University Of Pennsylvania | Compositions and methods for self-regulated inducible gene expression |
-
2018
- 2018-03-01 CN CN202411354454.9A patent/CN119752819A/zh active Pending
- 2018-03-01 EP EP18760861.7A patent/EP3589738A4/en not_active Withdrawn
- 2018-03-01 KR KR1020197027823A patent/KR102545070B1/ko active Active
- 2018-03-01 RU RU2019130004A patent/RU2019130004A/ru unknown
- 2018-03-01 CN CN201880029279.2A patent/CN110582572A/zh active Pending
- 2018-03-01 BR BR112019017327A patent/BR112019017327A2/pt not_active Application Discontinuation
- 2018-03-01 IL IL314284A patent/IL314284A/en unknown
- 2018-03-01 CA CA3054136A patent/CA3054136A1/en active Pending
- 2018-03-01 WO PCT/US2018/020470 patent/WO2018160849A1/en not_active Ceased
- 2018-03-01 AU AU2018228881A patent/AU2018228881B2/en active Active
- 2018-03-01 JP JP2019547457A patent/JP7211960B2/ja active Active
- 2018-03-01 KR KR1020247020928A patent/KR20240104196A/ko active Pending
- 2018-03-01 US US16/489,770 patent/US11564996B2/en active Active
- 2018-03-01 EP EP25194444.3A patent/EP4656244A3/en active Pending
- 2018-03-01 KR KR1020237020037A patent/KR20230093072A/ko not_active Ceased
-
2019
- 2019-08-27 IL IL26894619A patent/IL268946A/en unknown
-
2022
- 2022-12-05 US US18/061,633 patent/US20230233709A1/en active Pending
-
2023
- 2023-01-12 JP JP2023002799A patent/JP7671790B2/ja active Active
-
2024
- 2024-04-24 AU AU2024202693A patent/AU2024202693A1/en active Pending
-
2025
- 2025-04-18 JP JP2025068834A patent/JP2025114604A/ja active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US20220331409A1 (en) | Factor ix gene therapy | |
| JP7162021B2 (ja) | 筋ジストロフィーを治療するためのマイクロジストロフィン断片のアデノ随伴ウイルスベクター送達 | |
| JP2021106619A5 (enExample) | ||
| FI3684423T3 (fi) | Adenoassesioidun viruksen kapsidivariantteja ja niiden käyttömenetelmiä | |
| JP2021087431A5 (enExample) | ||
| JP2020528734A5 (enExample) | ||
| JP2020514286A5 (enExample) | ||
| RU2018116076A (ru) | Модифицированные гены атаксии фридрейха и векторы для генной терапии | |
| JP2020510428A5 (enExample) | ||
| JP2019503649A5 (enExample) | ||
| JP2018148927A5 (enExample) | ||
| JP2020513811A5 (enExample) | ||
| JP2020522269A5 (enExample) | ||
| JP2018537984A5 (enExample) | ||
| RU2017125234A (ru) | Конструкции нуклеиновой кислоты и векторы для генотерапии для применения для лечения болезни вильсона и других состояний | |
| JP2018506261A5 (enExample) | ||
| JP2020510433A5 (enExample) | ||
| AU2022407655A1 (en) | Methods and compositions for treating bag-3 related cardiomyopathy with a viral vector | |
| AU2023234604A1 (en) | Methods and compositions for treating tnnt2 related cardiomyopathy with a viral vector | |
| JP2018526994A5 (enExample) | ||
| RU2019130004A (ru) | Генная терапия при глазных заболеваниях | |
| JPWO2021108809A5 (enExample) | ||
| JPWO2022015715A5 (enExample) | ||
| WO2023111106A1 (en) | Fukutin related protein gene transfer increase using modified itr sequences | |
| EP4437117A2 (en) | Compositions comprising kozak sequences selected for enhanced expression |