JP2020510433A5 - - Google Patents
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- 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) | EP4656244A3 (enExample) |
| JP (3) | JP7211960B2 (enExample) |
| KR (3) | KR102545070B1 (enExample) |
| CN (2) | CN110582572A (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 |
|---|---|---|---|---|
| EP3814492A4 (en) * | 2018-06-29 | 2022-02-23 | Wuhan Neurophth Biotechnology Limited Company | Compositions and methods for treating leber's hereditary optic neuropathy |
| US12697439B2 (en) * | 2019-12-12 | 2026-08-04 | Novartis Ag | Injection device and injection solution transferring system |
| MX2023008826A (es) | 2021-02-01 | 2023-09-15 | Regenxbio Inc | Terapia génica para lipofuscinosis neuronal ceroidea. |
| US20220387627A1 (en) * | 2021-06-03 | 2022-12-08 | Hope for Hasti | Vectors and gene therapy for treating cornelia de lange syndrome |
Family Cites Families (48)
| 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 |
| 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 |
| 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 |
| EP1135468B1 (en) | 1998-11-10 | 2010-01-06 | University Of North Carolina At Chapel Hill | Virus vectors and methods of making and administering the same |
| 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 |
| CA2442670A1 (en) * | 2001-04-13 | 2002-10-24 | The Trustees Of The University Of Pennsylvania | Method of treating or retarding the development of blindness |
| CN103589692B (zh) | 2001-11-13 | 2018-07-24 | 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 |
| 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 |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| WO2005012877A2 (en) | 2003-08-01 | 2005-02-10 | Dna Twopointo Inc. | Systems and methods for antibody engineering |
| US8005620B2 (en) | 2003-08-01 | 2011-08-23 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
| EP1668143B1 (en) | 2003-09-30 | 2013-03-20 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
| DE23193606T1 (de) | 2005-04-07 | 2024-02-15 | The Trustees Of The University Of Pennsylvania | Verfahren zur erhöhung der funktion eines aav-vektors |
| JP4495210B2 (ja) | 2005-06-09 | 2010-06-30 | パナソニック株式会社 | 振幅誤差補償装置及び直交度誤差補償装置 |
| 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 |
| CA2672203A1 (en) * | 2006-12-21 | 2008-07-03 | Genentech, Inc. | Compositions and methods for the expression of nucleic acids |
| 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 |
| 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 |
| 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 |
| HUE041571T2 (hu) * | 2010-04-23 | 2019-05-28 | Univ Florida | RAAV-guanilát-cikláz-készítmények és ejárások 1-es típusú Leber-féle veleszületett vakság (LCA1) kezelésére |
| US9249425B2 (en) * | 2011-05-16 | 2016-02-02 | The Trustees Of The University Of Pennslyvania | Proviral plasmids and production of recombinant adeno-associated virus |
| KR20220025112A (ko) | 2011-06-08 | 2022-03-03 | 샤이어 휴먼 지네틱 테라피즈 인크. | Mrna 전달을 위한 지질 나노입자 조성물 및 방법 |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| BR112014020325A2 (pt) * | 2012-02-17 | 2017-08-08 | Childrens Hospital Philadelphia | composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos |
| US9163259B2 (en) * | 2012-05-04 | 2015-10-20 | Novartis Ag | Viral vectors for the treatment of retinal dystrophy |
| EA037448B1 (ru) | 2012-06-08 | 2021-03-30 | Этрис Гмбх | Способ доставки в легкие терапевтической мрнк с pei и фармацевтическая композиция |
| 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 |
| CN105209619B (zh) * | 2013-03-11 | 2018-10-12 | 泰莱托恩基金会 | miR-204和miR-211及其用途 |
| JP2016517278A (ja) * | 2013-03-15 | 2016-06-16 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ | スタッファー／フィラーポリヌクレオチド配列を含むベクターおよびその使用方法 |
| 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 |
| US10155794B2 (en) * | 2013-07-16 | 2018-12-18 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of disorders related to CEP290 |
| ES2822551T3 (es) * | 2014-03-17 | 2021-05-04 | Adverum Biotechnologies Inc | Composiciones y métodos para la expresión génica mejorada en células de los conos |
| 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 WO PCT/US2018/020470 patent/WO2018160849A1/en not_active Ceased
- 2018-03-01 CA CA3054136A patent/CA3054136A1/en active Pending
- 2018-03-01 CN CN201880029279.2A patent/CN110582572A/zh active Pending
- 2018-03-01 AU AU2018228881A patent/AU2018228881B2/en active Active
- 2018-03-01 IL IL314284A patent/IL314284A/en unknown
- 2018-03-01 JP JP2019547457A patent/JP7211960B2/ja active Active
- 2018-03-01 RU RU2019130004A patent/RU2019130004A/ru unknown
- 2018-03-01 US US16/489,770 patent/US11564996B2/en active Active
- 2018-03-01 CN CN202411354454.9A patent/CN119752819A/zh active Pending
- 2018-03-01 KR KR1020197027823A patent/KR102545070B1/ko active Active
- 2018-03-01 EP EP25194444.3A patent/EP4656244A3/en active Pending
- 2018-03-01 KR KR1020247020928A patent/KR20240104196A/ko active Pending
- 2018-03-01 KR KR1020237020037A patent/KR20230093072A/ko not_active Ceased
- 2018-03-01 BR BR112019017327A patent/BR112019017327A2/pt not_active Application Discontinuation
- 2018-03-01 EP EP18760861.7A patent/EP3589738A4/en not_active Withdrawn
-
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
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