JP2018506530A5 - - Google Patents

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Publication number
JP2018506530A5
JP2018506530A5 JP2017540569A JP2017540569A JP2018506530A5 JP 2018506530 A5 JP2018506530 A5 JP 2018506530A5 JP 2017540569 A JP2017540569 A JP 2017540569A JP 2017540569 A JP2017540569 A JP 2017540569A JP 2018506530 A5 JP2018506530 A5 JP 2018506530A5
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JP
Japan
Prior art keywords
spinal
aav9
gfp
expression
buffy coat
Prior art date
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Pending
Application number
JP2017540569A
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English (en)
Japanese (ja)
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JP2018506530A (ja
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Publication date
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Priority claimed from PCT/US2015/065704 external-priority patent/WO2016122791A1/en
Publication of JP2018506530A publication Critical patent/JP2018506530A/ja
Publication of JP2018506530A5 publication Critical patent/JP2018506530A5/ja
Pending legal-status Critical Current

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JP2017540569A 2015-01-30 2015-12-15 脊柱軟膜下遺伝子送達システム Pending JP2018506530A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562110340P 2015-01-30 2015-01-30
US62/110,340 2015-01-30
PCT/US2015/065704 WO2016122791A1 (en) 2015-01-30 2015-12-15 Spinal subpial gene delivery system

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2019182624A Division JP2020019796A (ja) 2015-01-30 2019-10-03 脊髄軟膜下遺伝子送達システム

Publications (2)

Publication Number Publication Date
JP2018506530A JP2018506530A (ja) 2018-03-08
JP2018506530A5 true JP2018506530A5 (enExample) 2019-02-14

Family

ID=56544138

Family Applications (5)

Application Number Title Priority Date Filing Date
JP2017540569A Pending JP2018506530A (ja) 2015-01-30 2015-12-15 脊柱軟膜下遺伝子送達システム
JP2019182624A Withdrawn JP2020019796A (ja) 2015-01-30 2019-10-03 脊髄軟膜下遺伝子送達システム
JP2021124373A Pending JP2021181456A (ja) 2015-01-30 2021-07-29 脊髄軟膜下遺伝子送達システム
JP2023036473A Active JP7876202B2 (ja) 2015-01-30 2023-03-09 脊髄軟膜下遺伝子送達システム
JP2024199400A Pending JP2025015673A (ja) 2015-01-30 2024-11-15 脊髄軟膜下遺伝子送達システム

Family Applications After (4)

Application Number Title Priority Date Filing Date
JP2019182624A Withdrawn JP2020019796A (ja) 2015-01-30 2019-10-03 脊髄軟膜下遺伝子送達システム
JP2021124373A Pending JP2021181456A (ja) 2015-01-30 2021-07-29 脊髄軟膜下遺伝子送達システム
JP2023036473A Active JP7876202B2 (ja) 2015-01-30 2023-03-09 脊髄軟膜下遺伝子送達システム
JP2024199400A Pending JP2025015673A (ja) 2015-01-30 2024-11-15 脊髄軟膜下遺伝子送達システム

Country Status (6)

Country Link
US (1) US20180008727A1 (enExample)
EP (1) EP3250279B1 (enExample)
JP (5) JP2018506530A (enExample)
CN (1) CN107427666B (enExample)
CA (1) CA2975447C (enExample)
WO (1) WO2016122791A1 (enExample)

Families Citing this family (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
EP3215191B1 (en) 2014-11-05 2024-08-14 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
CN114717264A (zh) 2014-11-14 2022-07-08 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
US10688285B2 (en) 2015-01-30 2020-06-23 The Regents Of The University Of California Spinal subpial gene delivery system
AU2017241534A1 (en) 2016-03-28 2018-10-04 The Regents Of The University Of California Method and composition for treating neuronal hyper-excitability
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
US10584337B2 (en) 2016-05-18 2020-03-10 Voyager Therapeutics, Inc. Modulatory polynucleotides
IL297576B2 (en) 2016-05-18 2024-02-01 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
EP3831281B1 (en) 2016-08-30 2026-02-25 The Regents of The University of California Devices and systems for biomedical targeting and delivery
MX2019013172A (es) 2017-05-05 2020-09-07 Voyager Therapeutics Inc Composiciones y metodos para tratar la enfermedad de huntington.
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
US11512327B2 (en) 2017-08-03 2022-11-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV
AU2018327253B2 (en) * 2017-09-08 2024-05-16 The Regents Of The University Of California Method and composition for treating neuropathic pain
MX2020003042A (es) 2017-09-29 2020-11-18 Voyager Therapeutics Inc Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa.
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
CN112424359A (zh) 2018-05-15 2021-02-26 沃雅戈治疗公司 用于治疗帕金森氏病的组合物和方法
US12281321B2 (en) 2018-09-28 2025-04-22 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
JP7373226B2 (ja) * 2019-01-10 2023-11-02 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 軟膜下送達システムおよび使用方法
USD963164S1 (en) 2020-01-09 2022-09-06 The Regents Of The University Of California Surgical needle
KR20250133811A (ko) 2022-12-05 2025-09-08 이지 427 경직 치료를 위한 gad를 코딩하는 바이러스 벡터

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5292722A (en) * 1992-11-06 1994-03-08 Brigham And Women's Hospital Intravenous solution that diminishes body protein loss
FR2705361B1 (fr) 1993-05-18 1995-08-04 Centre Nat Rech Scient Vecteurs viraux et utilisation en thérapie génique.
EP0705344B8 (en) 1993-06-24 2006-05-10 Advec Inc. Adenovirus vectors for gene therapy
CA2144040A1 (fr) 1993-07-13 1995-01-26 Michel Perricaudet Vecteurs adenoviraux defectifs et utilisation en therapie genique
EP0733103B1 (en) 1993-11-09 2004-03-03 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
DE69433922T2 (de) 1993-11-09 2005-07-28 Medical College Of Ohio, Toledo Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren
WO1995016772A1 (en) 1993-12-14 1995-06-22 Cornell Research Foundation, Inc. Adenovirus gene expression system
FR2716893B1 (fr) 1994-03-03 1996-04-12 Rhone Poulenc Rorer Sa Virus recombinants, leur préparation et leur utilisation thérapeutique.
AUPM452094A0 (en) 1994-03-17 1994-04-14 University Of Queensland, The Waste treatment plant and process
WO1996017947A1 (en) 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
US20030032614A1 (en) 1995-04-13 2003-02-13 Mcmichael John Method of treating respiratory distress using synthetic polynucleic acids
CA2264482A1 (en) 1996-09-06 1998-03-12 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
US20020007145A1 (en) * 1998-10-23 2002-01-17 Timothy Stivland Catheter having improved bonding region
US7125856B1 (en) 1999-04-15 2006-10-24 St. Elizabeth's Medical Center Of Boston, Inc. Angiogenic growth factors for treatment of peripheral neuropathy
JP2004511266A (ja) 2000-04-12 2004-04-15 チルドレンズ ホスピタル オブ フィラデルフィア 間葉間質細胞に対する治療的利用法
US7011647B2 (en) * 2001-07-13 2006-03-14 Scimed Life Systems, Inc. Introducer sheath
US20070213292A1 (en) * 2005-08-10 2007-09-13 The Rockefeller University Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof
EP3146982B1 (en) * 2006-10-03 2019-08-21 Genzyme Corporation Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
NZ599430A (en) 2007-06-11 2014-03-28 Loch Macdonald R A drug delivery system for the prevention of cerebral vasospasm
WO2009091514A2 (en) * 2008-01-14 2009-07-23 I-V Access Technology, Inc. An apparatus for peripheral vascular access
US9415121B2 (en) 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
WO2010071832A1 (en) 2008-12-19 2010-06-24 Nationwide Children's Hospital Delivery of polynucleotides across the blood brain barrier using recombinant aav9
PT2414322T (pt) 2009-03-20 2022-12-07 Egen Inc Derivados de poliamina
CN102666856B (zh) * 2009-11-08 2016-04-06 夸克制药公司 定向至RhoA靶基因的双链RNA化合物在制造治疗神经性疼痛的药物中的用途
US20120310140A1 (en) * 2010-12-01 2012-12-06 Spinal Modulation, Inc. Directed delivery of agents to neural anatomy
JP6091435B2 (ja) 2011-02-22 2017-03-08 カリフォルニア インスティチュート オブ テクノロジー アデノ随伴ウイルス(aav)ベクターを用いたタンパク質の送達
SG10201604810UA (en) * 2012-02-07 2016-08-30 Global Bio Therapeutics Inc Compartmentalized method of nucleic acid delivery and compositions and uses thereof
CA2896442A1 (en) * 2012-09-24 2014-03-27 The Regents Of The University Of California Spinal cord pulsation-cancelation injection system
GB201308917D0 (en) * 2013-05-17 2013-07-03 Renishaw Plc Delivery

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