JP2018506585A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2018506585A5 JP2018506585A5 JP2017560479A JP2017560479A JP2018506585A5 JP 2018506585 A5 JP2018506585 A5 JP 2018506585A5 JP 2017560479 A JP2017560479 A JP 2017560479A JP 2017560479 A JP2017560479 A JP 2017560479A JP 2018506585 A5 JP2018506585 A5 JP 2018506585A5
- Authority
- JP
- Japan
- Prior art keywords
- raav
- nucleic acid
- disease
- raav particle
- optionally
- 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
Priority Applications (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021129519A JP2021178861A (ja) | 2015-02-10 | 2021-08-06 | 線条体および皮質へのウイルス粒子の強化された送達 |
| JP2023018846A JP2023058630A (ja) | 2015-02-10 | 2023-02-10 | 線条体および皮質へのウイルス粒子の強化された送達 |
| JP2024228114A JP2025060823A (ja) | 2015-02-10 | 2024-12-25 | 線条体および皮質へのウイルス粒子の強化された送達 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562114544P | 2015-02-10 | 2015-02-10 | |
| US62/114,544 | 2015-02-10 | ||
| US201562220997P | 2015-09-19 | 2015-09-19 | |
| US62/220,997 | 2015-09-19 | ||
| PCT/US2016/017210 WO2016130591A2 (en) | 2015-02-10 | 2016-02-09 | Enhanced delivery of viral particles to the striatum and cortex |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021129519A Division JP2021178861A (ja) | 2015-02-10 | 2021-08-06 | 線条体および皮質へのウイルス粒子の強化された送達 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018506585A JP2018506585A (ja) | 2018-03-08 |
| JP2018506585A5 true JP2018506585A5 (enExample) | 2019-03-22 |
| JP6928558B2 JP6928558B2 (ja) | 2021-09-01 |
Family
ID=56614902
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017560479A Active JP6928558B2 (ja) | 2015-02-10 | 2016-02-09 | 線条体および皮質へのウイルス粒子の強化された送達 |
| JP2021129519A Pending JP2021178861A (ja) | 2015-02-10 | 2021-08-06 | 線条体および皮質へのウイルス粒子の強化された送達 |
| JP2023018846A Pending JP2023058630A (ja) | 2015-02-10 | 2023-02-10 | 線条体および皮質へのウイルス粒子の強化された送達 |
| JP2024228114A Pending JP2025060823A (ja) | 2015-02-10 | 2024-12-25 | 線条体および皮質へのウイルス粒子の強化された送達 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021129519A Pending JP2021178861A (ja) | 2015-02-10 | 2021-08-06 | 線条体および皮質へのウイルス粒子の強化された送達 |
| JP2023018846A Pending JP2023058630A (ja) | 2015-02-10 | 2023-02-10 | 線条体および皮質へのウイルス粒子の強化された送達 |
| JP2024228114A Pending JP2025060823A (ja) | 2015-02-10 | 2024-12-25 | 線条体および皮質へのウイルス粒子の強化された送達 |
Country Status (27)
| Country | Link |
|---|---|
| US (3) | US20190111157A1 (enExample) |
| EP (3) | EP4219728A3 (enExample) |
| JP (4) | JP6928558B2 (enExample) |
| KR (2) | KR102863734B1 (enExample) |
| CN (2) | CN107530447A (enExample) |
| AU (3) | AU2016219398A1 (enExample) |
| BR (1) | BR112017017037A2 (enExample) |
| CA (1) | CA2976082A1 (enExample) |
| CL (1) | CL2017002028A1 (enExample) |
| CR (1) | CR20170407A (enExample) |
| DK (1) | DK3256594T3 (enExample) |
| EA (1) | EA201791803A1 (enExample) |
| ES (1) | ES2904504T3 (enExample) |
| HU (1) | HUE057272T2 (enExample) |
| IL (4) | IL296466B2 (enExample) |
| LT (1) | LT3256594T (enExample) |
| MX (2) | MX388264B (enExample) |
| NZ (1) | NZ772772A (enExample) |
| PH (1) | PH12017501436A1 (enExample) |
| PL (1) | PL3256594T3 (enExample) |
| PT (1) | PT3256594T (enExample) |
| RS (1) | RS62811B1 (enExample) |
| SG (3) | SG10201907399RA (enExample) |
| SI (1) | SI3256594T1 (enExample) |
| TN (1) | TN2017000353A1 (enExample) |
| TW (1) | TWI730952B (enExample) |
| WO (1) | WO2016130591A2 (enExample) |
Families Citing this family (47)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| CN112553229A (zh) | 2014-11-05 | 2021-03-26 | 沃雅戈治疗公司 | 用于治疗帕金森病的aadc多核苷酸 |
| RU2020108189A (ru) | 2014-11-14 | 2020-03-11 | Вояджер Терапьютикс, Инк. | Композиции и способы лечения бокового амиотрофического склероза (als) |
| MX2017006217A (es) | 2014-11-14 | 2018-05-02 | Voyager Therapeutics Inc | Polinucleotidos moduladores. |
| EP3230441A4 (en) | 2014-12-12 | 2018-10-03 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scaav |
| NZ772772A (en) * | 2015-02-10 | 2024-12-20 | Genzyme Corp | Enhanced delivery of viral particles to the striatum and cortex |
| TWI707951B (zh) | 2015-04-08 | 2020-10-21 | 美商健臻公司 | 過大腺相關載體之製造 |
| EP4491733A3 (en) | 2015-04-23 | 2025-03-26 | University of Massachusetts | Modulation of aav vector transgene expression |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| BR112018006074A2 (pt) | 2015-09-24 | 2018-10-09 | Biomarin Pharm Inc | vetores de fator viii de vírus adeno-associado, partículas virais associadas e formulações terapêuticas compreendendo as mesmas |
| EP3448874A4 (en) | 2016-04-29 | 2020-04-22 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| US11299751B2 (en) | 2016-04-29 | 2022-04-12 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| AU2017267665C1 (en) | 2016-05-18 | 2023-10-05 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| BR112018073472A2 (pt) | 2016-05-18 | 2019-08-27 | Voyager Therapeutics, Inc. | composições e métodos de tratamento da doença de huntington |
| WO2018044933A1 (en) | 2016-08-30 | 2018-03-08 | The Regents Of The University Of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| JP7059285B2 (ja) * | 2016-09-30 | 2022-04-25 | エステベ プアルマセウティカルス， エッセ．ア． | ムコ多糖症の治療のためのアデノ随伴ウイルスベクター |
| US20210108197A1 (en) * | 2017-03-31 | 2021-04-15 | Staidson (Beijing) Biopharmaceuticals Co., Ltd. | Shrna expression cassette, polynucleotide sequence carrying same, and application thereof |
| WO2018191450A2 (en) | 2017-04-14 | 2018-10-18 | National Taiwan University Hospital | Gene therapy for aadc deficiency |
| CN111108198A (zh) | 2017-05-05 | 2020-05-05 | 沃雅戈治疗公司 | 治疗亨廷顿病的组合物和方法 |
| CA3061652A1 (en) | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| CN111132626B (zh) | 2017-07-17 | 2024-01-30 | 沃雅戈治疗公司 | 轨迹阵列引导系统 |
| WO2019028306A2 (en) | 2017-08-03 | 2019-02-07 | Voyager Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR ADMINISTRATION OF ADENO-ASSOCIATED VIRUSES |
| AU2018338728B2 (en) | 2017-09-29 | 2025-01-02 | Centre National De La Recherche Scientifique (Cnrs) | Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery |
| CN111479924B (zh) | 2017-10-16 | 2024-06-14 | 沃雅戈治疗公司 | 肌萎缩性侧索硬化症(als)的治疗 |
| US20200237799A1 (en) | 2017-10-16 | 2020-07-30 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| JP2021520811A (ja) | 2018-04-13 | 2021-08-26 | ユニバーシティ オブ マサチューセッツ | ヘキソサミニダーゼアルファおよびベータサブユニットをコードしているバイシストロニックａａｖベクターならびにその使用 |
| AU2019268330A1 (en) | 2018-05-15 | 2020-11-26 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of Parkinson's disease |
| CN113528510A (zh) * | 2018-06-29 | 2021-10-22 | 武汉纽福斯生物科技有限公司 | 治疗遗传性视神经病变的组合物和方法 |
| EP3856762A1 (en) | 2018-09-28 | 2021-08-04 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| KR20210087462A (ko) | 2018-10-05 | 2021-07-12 | 유니버시티 오브 매사추세츠 | Gm1 및 gm2 강글리오시드증의 치료를 위한 raav 벡터 |
| SG11202104295UA (en) * | 2018-11-14 | 2021-06-29 | Regenxbio Inc | Gene therapy for neuronal ceroid lipofuscinoses |
| BR112021015817A2 (pt) * | 2019-02-22 | 2021-10-13 | The Trustees Of The University Of Pennsylvania | Vírus adeno-associado recombinante para tratamento de neurodegeneração com início na idade adulta associado a grn |
| EP3983077A4 (en) * | 2019-06-17 | 2023-12-20 | Alnylam Pharmaceuticals, Inc. | DELIVERY OF OLIGONUCLEOTIDES TO THE STRIATUM |
| WO2021007382A1 (en) * | 2019-07-09 | 2021-01-14 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of parkinson's disease |
| WO2021016505A1 (en) * | 2019-07-24 | 2021-01-28 | Voyager Therapeutics, Inc. | Compositions and methods for treating huntington's disease |
| US12442017B2 (en) | 2020-03-02 | 2025-10-14 | National University Corporation Gunma University | Microglial selective gene expression vector |
| EP4093859A4 (en) * | 2020-03-11 | 2023-06-14 | Remotor Therapeutics, Inc. | Methods and materials for disseminating a protein throughout the central nervous system |
| JP2023532944A (ja) * | 2020-06-30 | 2023-08-01 | セキラス ユーケー リミテッド | 水中油型エマルジョンアジュバントの低温濾過 |
| AU2022212922A1 (en) * | 2021-01-27 | 2023-08-17 | Research Institute At Nationwide Children's Hospital | Materials and methods for the treatment of lysosomal acid lipase deficiency (lal-d) |
| WO2023113806A1 (en) | 2021-12-16 | 2023-06-22 | Affinia Therapeutics, Inc. | Recombinant aav for treatment of neural disease |
| WO2022221193A1 (en) | 2021-04-12 | 2022-10-20 | Affinia Therapeutics Inc. | Recombinant aav for treatment of neural disease |
| CN120187407A (zh) * | 2021-12-15 | 2025-06-20 | 健达九州(北京)生物科技有限公司 | 重组aav载体及其用途 |
| US20250333758A1 (en) * | 2021-12-15 | 2025-10-30 | Genans Biotechnology Co., Ltd | Microglia having car and use thereof |
| EP4605410A1 (en) | 2022-10-19 | 2025-08-27 | Affinia Therapeutics Inc. | Recombinant aavs with improved tropism and specificity |
| WO2025151454A1 (en) * | 2024-01-08 | 2025-07-17 | AskBio Inc. | Method or system of infusing and/or predicting an infusate volume |
| WO2025227063A1 (en) | 2024-04-26 | 2025-10-30 | Affinia Therapeutics Inc. | Recombinant aavs with improved tropism and specificity |
Family Cites Families (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4692147A (en) | 1980-04-02 | 1987-09-08 | Medtronic, Inc. | Drug administration device |
| US5735815A (en) | 1993-07-26 | 1998-04-07 | Sentinel Medical, Inc. | Method of using fluid jet surgical cutting tool |
| EP0931158A1 (en) | 1996-09-06 | 1999-07-28 | The Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
| US6042579A (en) | 1997-04-30 | 2000-03-28 | Medtronic, Inc. | Techniques for treating neurodegenerative disorders by infusion of nerve growth factors into the brain |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6989264B2 (en) | 1997-09-05 | 2006-01-24 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| PT1080202E (pt) | 1998-05-27 | 2006-05-31 | Avigen Inc | Distribuicao de vectores aav codificando aadc intensificada por conveccao |
| 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 |
| NZ522840A (en) | 2000-06-01 | 2004-12-24 | Univ North Carolina | A parvovirus vector that carries a duplexed genome resulting in co-packaging of strands of plus and minus polarity tethered together |
| WO2003004660A1 (en) | 2001-07-03 | 2003-01-16 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Methods of administering vectors to synaptically connected neurons |
| US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| 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 |
| GB2389791B (en) | 2002-04-30 | 2006-12-13 | Steven Gill | Implantable drug delivery pump |
| ES2521682T3 (es) | 2003-05-21 | 2014-11-13 | Genzyme Corporation | Procedimientos para producir preparaciones de viriones de AAV recombinantes sustancialmente exentas de cápsidas vacías |
| EP2572661B1 (en) | 2004-10-05 | 2019-11-20 | Genzyme Corporation | Stepped cannula |
| WO2006119432A2 (en) | 2005-04-29 | 2006-11-09 | The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services | Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes |
| PT2420256T (pt) * | 2005-05-02 | 2016-09-13 | Genzyme Corp | Terapia genética para distúrbios neurometabólicos |
| AU2006283189B2 (en) | 2005-08-23 | 2013-01-31 | The Regents Of The University Of California | Reflux resistant cannula and system for chronic delivery of therapeutic agents using convection-enhanced delivery |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| PT2019683T (pt) | 2006-04-25 | 2017-03-17 | Univ California | Administração de fatores de crescimento para o tratamento de distúrbios do snc |
| US20070259031A1 (en) | 2006-04-26 | 2007-11-08 | The Regents Of The University Of California | Compositions and methods for convection enhanced delivery of high molecular weight neurotherapeutics |
| RU2442617C2 (ru) | 2007-05-17 | 2012-02-20 | Медженезиз Терапьютикс Инк. | Конвекционный доставляющий катетер со съемным элементом жесткости и способ его применения |
| CA2751266A1 (en) * | 2009-01-29 | 2010-08-05 | Krystof Bankiewicz | Methods for distributing high levels of therapeutic agent throughout the cortex to treat neurological disorders |
| HUE040636T2 (hu) | 2009-06-16 | 2019-03-28 | Genzyme Corp | Javított eljárások rekombináns AAV-vektorok tisztítására |
| US8663624B2 (en) | 2010-10-06 | 2014-03-04 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| ES2739804T3 (es) * | 2011-02-12 | 2020-02-04 | Univ Iowa Res Found | Compuestos terapéuticos |
| ES2724800T3 (es) | 2011-02-17 | 2019-09-16 | Univ Pennsylvania | Composiciones y métodos para alterar la especificidad de tejido y mejorar la transferencia génica mediada por AAV9 |
| WO2015168666A2 (en) * | 2014-05-02 | 2015-11-05 | Genzyme Corporation | Aav vectors for retinal and cns gene therapy |
| NZ772772A (en) * | 2015-02-10 | 2024-12-20 | Genzyme Corp | Enhanced delivery of viral particles to the striatum and cortex |
| CN111417231A (zh) | 2019-01-04 | 2020-07-14 | 青岛海尔股份有限公司 | 电磁波发生系统及具有该电磁波发生系统的加热装置 |
-
2016
- 2016-02-09 NZ NZ772772A patent/NZ772772A/en unknown
- 2016-02-09 LT LTEPPCT/US2016/017210T patent/LT3256594T/lt unknown
- 2016-02-09 RS RS20220044A patent/RS62811B1/sr unknown
- 2016-02-09 SG SG10201907399RA patent/SG10201907399RA/en unknown
- 2016-02-09 DK DK16714066.4T patent/DK3256594T3/da active
- 2016-02-09 HU HUE16714066A patent/HUE057272T2/hu unknown
- 2016-02-09 CN CN201680020700.4A patent/CN107530447A/zh active Pending
- 2016-02-09 KR KR1020247000976A patent/KR102863734B1/ko active Active
- 2016-02-09 KR KR1020177025285A patent/KR102625784B1/ko active Active
- 2016-02-09 SI SI201631446T patent/SI3256594T1/sl unknown
- 2016-02-09 ES ES16714066T patent/ES2904504T3/es active Active
- 2016-02-09 CN CN202210805297.3A patent/CN116019934A/zh active Pending
- 2016-02-09 TN TNP/2017/000353A patent/TN2017000353A1/en unknown
- 2016-02-09 WO PCT/US2016/017210 patent/WO2016130591A2/en not_active Ceased
- 2016-02-09 JP JP2017560479A patent/JP6928558B2/ja active Active
- 2016-02-09 AU AU2016219398A patent/AU2016219398A1/en not_active Abandoned
- 2016-02-09 PL PL16714066T patent/PL3256594T3/pl unknown
- 2016-02-09 BR BR112017017037A patent/BR112017017037A2/pt not_active Application Discontinuation
- 2016-02-09 SG SG11201706445SA patent/SG11201706445SA/en unknown
- 2016-02-09 CA CA2976082A patent/CA2976082A1/en active Pending
- 2016-02-09 IL IL296466A patent/IL296466B2/en unknown
- 2016-02-09 PT PT167140664T patent/PT3256594T/pt unknown
- 2016-02-09 IL IL313840A patent/IL313840A/en unknown
- 2016-02-09 MX MX2017010370A patent/MX388264B/es unknown
- 2016-02-09 EP EP23150591.8A patent/EP4219728A3/en active Pending
- 2016-02-09 EP EP21198081.8A patent/EP4023758A1/en not_active Withdrawn
- 2016-02-09 CR CR20170407A patent/CR20170407A/es unknown
- 2016-02-09 US US15/549,962 patent/US20190111157A1/en not_active Abandoned
- 2016-02-09 EA EA201791803A patent/EA201791803A1/ru unknown
- 2016-02-09 EP EP16714066.4A patent/EP3256594B1/en active Active
- 2016-02-10 SG SG10201912926VA patent/SG10201912926VA/en unknown
- 2016-02-15 TW TW105104342A patent/TWI730952B/zh active
-
2017
- 2017-08-08 IL IL253894A patent/IL253894B/en unknown
- 2017-08-08 CL CL2017002028A patent/CL2017002028A1/es unknown
- 2017-08-10 MX MX2021004294A patent/MX2021004294A/es unknown
- 2017-08-10 PH PH12017501436A patent/PH12017501436A1/en unknown
-
2021
- 2021-07-22 IL IL285060A patent/IL285060B2/en unknown
- 2021-08-06 JP JP2021129519A patent/JP2021178861A/ja active Pending
-
2022
- 2022-06-28 AU AU2022204583A patent/AU2022204583A1/en not_active Abandoned
- 2022-07-29 US US17/816,139 patent/US12465657B2/en active Active
-
2023
- 2023-02-10 JP JP2023018846A patent/JP2023058630A/ja active Pending
-
2024
- 2024-12-25 JP JP2024228114A patent/JP2025060823A/ja active Pending
-
2025
- 2025-02-26 AU AU2025201381A patent/AU2025201381A1/en active Pending
- 2025-08-01 US US19/288,561 patent/US20260048149A1/en active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2018506585A5 (enExample) | ||
| JP7698583B2 (ja) | 組換えアデノ随伴ウイルスベクター | |
| JP7574508B2 (ja) | 血管系を通過する遺伝子移入の方法および組成物 | |
| US12465657B2 (en) | Enhanced delivery of viral particles to the striatum and cortex | |
| JP7425738B2 (ja) | 眼組織を標的とするウイルスベクター | |
| JP7385282B2 (ja) | 環状ｒｎａ分子のための方法及び組成物 | |
| US9447433B2 (en) | Synthetic adeno-associated virus inverted terminal repeats | |
| JP2024016041A (ja) | 抗体を回避するウイルスベクター | |
| JP7406783B2 (ja) | パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質 | |
| EP3245220B1 (en) | Methods and compositions for targeted gene transfer | |
| JP2014507145A (ja) | 改変された形質導入プロファイルを有するウイルスベクターならびにその製造および使用の方法 | |
| JP2018500904A (ja) | ウィルソン病の処置に使用するための核酸構築物及び遺伝子治療ベクター | |
| JP2016503405A5 (enExample) | ||
| EP4045525A1 (en) | Adeno-associated viral vectors for treatment of niemann-pick disease type c | |
| US20220298528A1 (en) | Viral particles for use in treating synucleinopathies such as parkinson's diseases by gene therapy | |
| US20230277687A1 (en) | Viral particles for use in treating tauopathies such as alzheimer's diseases by gene therapy | |
| HRP20220042T1 (hr) | Poboljšana isporuka virusnih čestica u strijatum i korteks | |
| CN114746556A (zh) | 用于治疗山菲立普病和其他障碍的组合物和方法 | |
| Broekman | Discussion and conclusion |