JP2014512171A5 - - Google Patents
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- Publication number
- JP2014512171A5 JP2014512171A5 JP2013554938A JP2013554938A JP2014512171A5 JP 2014512171 A5 JP2014512171 A5 JP 2014512171A5 JP 2013554938 A JP2013554938 A JP 2013554938A JP 2013554938 A JP2013554938 A JP 2013554938A JP 2014512171 A5 JP2014512171 A5 JP 2014512171A5
- Authority
- JP
- Japan
- Prior art keywords
- vector
- vector according
- seq
- serotype
- derivative
- 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.)
- Pending
Links
- 102000040430 polynucleotide Human genes 0.000 claims 4
- 108091033319 polynucleotide Proteins 0.000 claims 4
- 239000002157 polynucleotide Substances 0.000 claims 4
- 239000003814 drug Substances 0.000 claims 3
- 238000002347 injection Methods 0.000 claims 3
- 239000007924 injection Substances 0.000 claims 3
- 101710112083 Para-Rep C1 Proteins 0.000 claims 2
- 102100022881 Rab proteins geranylgeranyltransferase component A 1 Human genes 0.000 claims 2
- 101710119887 Trans-acting factor B Proteins 0.000 claims 2
- 239000002245 particle Substances 0.000 claims 2
- 210000001525 retina Anatomy 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
- 208000033810 Choroidal dystrophy Diseases 0.000 claims 1
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 210000000234 capsid Anatomy 0.000 claims 1
- 208000003571 choroideremia Diseases 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 229920001184 polypeptide Polymers 0.000 claims 1
- 102000004196 processed proteins & peptides Human genes 0.000 claims 1
- 108090000765 processed proteins & peptides Proteins 0.000 claims 1
- 230000001105 regulatory effect Effects 0.000 claims 1
- 230000002207 retinal effect Effects 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GB1103062.4 | 2011-02-22 | ||
| GBGB1103062.4A GB201103062D0 (en) | 2011-02-22 | 2011-02-22 | Method |
| PCT/GB2012/050376 WO2012114090A1 (en) | 2011-02-22 | 2012-02-21 | Aav -vectors for use in gene therapy of choroideremia |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017117286A Division JP2017221192A (ja) | 2011-02-22 | 2017-06-14 | コロイデレミアの遺伝子治療に使用するためのａａｖベクター |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2014512171A JP2014512171A (ja) | 2014-05-22 |
| JP2014512171A5 true JP2014512171A5 (enExample) | 2015-04-16 |
Family
ID=43881504
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2013554938A Pending JP2014512171A (ja) | 2011-02-22 | 2012-02-21 | コロイデレミアの遺伝子治療に使用するためのａａｖベクター |
| JP2017117286A Pending JP2017221192A (ja) | 2011-02-22 | 2017-06-14 | コロイデレミアの遺伝子治療に使用するためのａａｖベクター |
| JP2019201092A Pending JP2020054345A (ja) | 2011-02-22 | 2019-11-05 | コロイデレミアの遺伝子治療に使用するためのａａｖベクター |
| JP2022193831A Pending JP2023036618A (ja) | 2011-02-22 | 2022-12-02 | コロイデレミアの遺伝子治療に使用するためのａａｖベクター |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017117286A Pending JP2017221192A (ja) | 2011-02-22 | 2017-06-14 | コロイデレミアの遺伝子治療に使用するためのａａｖベクター |
| JP2019201092A Pending JP2020054345A (ja) | 2011-02-22 | 2019-11-05 | コロイデレミアの遺伝子治療に使用するためのａａｖベクター |
| JP2022193831A Pending JP2023036618A (ja) | 2011-02-22 | 2022-12-02 | コロイデレミアの遺伝子治療に使用するためのａａｖベクター |
Country Status (18)
| Country | Link |
|---|---|
| US (4) | US9834788B2 (enExample) |
| EP (2) | EP2678435B2 (enExample) |
| JP (4) | JP2014512171A (enExample) |
| KR (3) | KR102299430B1 (enExample) |
| CN (2) | CN107881198A (enExample) |
| AU (2) | AU2012220404B2 (enExample) |
| BR (1) | BR112013021318B1 (enExample) |
| CA (1) | CA2827975C (enExample) |
| DK (1) | DK2678435T3 (enExample) |
| ES (1) | ES2676550T5 (enExample) |
| GB (1) | GB201103062D0 (enExample) |
| HK (2) | HK1258917A1 (enExample) |
| IL (1) | IL228031B (enExample) |
| MX (2) | MX356525B (enExample) |
| PL (1) | PL2678435T3 (enExample) |
| PT (1) | PT2678435T (enExample) |
| SG (1) | SG192911A1 (enExample) |
| WO (1) | WO2012114090A1 (enExample) |
Families Citing this family (47)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201103062D0 (en) † | 2011-02-22 | 2011-04-06 | Isis Innovation | Method |
| CN105980569A (zh) | 2013-08-05 | 2016-09-28 | 莱顿大学医学中心附属莱顿教学医院 | 重组aav-crumbs同源物组合物及治疗lca-8和进行性rp的方法 |
| KR102281881B1 (ko) * | 2013-12-06 | 2021-07-27 | 엥스띠뛰 나씨오날 드 라 쌍떼 에 드 라 흐쉐르슈 메디깔 (인쎄름) | 대상체의 망막 색소 상피에서 목적 폴리뉴클레오티드를 발현시키기 위한 방법 및 약학적 조성물 |
| 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 |
| US20180043034A1 (en) * | 2015-03-06 | 2018-02-15 | Massachusetts Eye And Ear Infirmary | Gene augmentation therapies for inherited retinal degeneration caused by mutations in the prpf31 gene |
| CN109069668B (zh) | 2015-12-14 | 2023-04-18 | 宾夕法尼亚州大学信托人 | 用于眼病的基因疗法 |
| GB201604146D0 (en) | 2016-03-10 | 2016-04-27 | Nightstarx Ltd | Prenylation assay |
| EP3448987A4 (en) | 2016-04-29 | 2020-05-27 | 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 |
| US10633689B2 (en) | 2016-11-07 | 2020-04-28 | Spark Therapeutics, Inc. | Rab Escort Protein potency assay |
| US11938197B2 (en) * | 2017-01-10 | 2024-03-26 | The Sydney Children's Hospitals Network (Randwick And Westmead (Incorporating The Royal Alexandra Hospital For Children) | Polynucleotides and vectors for the expression of transgenes |
| US10550405B2 (en) | 2017-03-15 | 2020-02-04 | The University Of North Carolina At Chapel Hill | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
| 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) |
| US11827898B2 (en) | 2017-06-14 | 2023-11-28 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
| 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 |
| 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. |
| MX2020004005A (es) * | 2017-10-03 | 2020-10-05 | Prevail Therapeutics Inc | Terapias genicas para los trastornos lisosomales. |
| 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) |
| AU2018352986A1 (en) | 2017-10-17 | 2020-05-14 | Nightstarx Limited | Prenylation assay |
| KR20200119782A (ko) * | 2017-12-06 | 2020-10-20 | 오비드 테라퓨틱스 인크. | 발작 장애 치료 시 mir101 또는 mir128의 용도 |
| WO2019152841A1 (en) | 2018-02-01 | 2019-08-08 | Homology Medicines, Inc. | Adeno-associated virus compositions for pah gene transfer and methods of use thereof |
| AU2019215152A1 (en) | 2018-02-01 | 2020-08-20 | Homology Medicines, Inc. | Adeno-associated virus compositions for restoring PAH gene function and methods of use thereof |
| US11306329B2 (en) | 2018-02-19 | 2022-04-19 | City Of Hope | Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof |
| EP3775234A1 (en) * | 2018-04-05 | 2021-02-17 | NightstaRx Limited | Aav compositions, methods of making and methods of use |
| AU2018422759B2 (en) * | 2018-05-07 | 2026-01-22 | The University Of North Carolina At Chapel Hill | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
| 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 |
| US20220193261A1 (en) * | 2019-04-30 | 2022-06-23 | The Trustees Of The University Of Pennsylvania | Compositions useful for treatment of pompe disease |
| TW202140791A (zh) | 2020-01-13 | 2021-11-01 | 美商霍蒙拉奇醫藥公司 | 治療苯酮尿症之方法 |
| TW202208632A (zh) | 2020-05-27 | 2022-03-01 | 美商同源醫藥公司 | 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法 |
| IL300526A (en) | 2020-08-17 | 2023-04-01 | Massachusetts Inst Technology | Approaches to SHANK3 gene therapy |
| WO2022109247A1 (en) | 2020-11-20 | 2022-05-27 | Biogen Ma Inc. | Viral vector potency assay |
| EP4482970A1 (en) | 2022-02-23 | 2025-01-01 | Massachusetts Institute of Technology | Methods for upregulating shank3 expression |
| JP2025528188A (ja) | 2022-08-11 | 2025-08-26 | インスティテュート オブ モレキュラー アンド クリニカル オフサルモロジー バーゼル （アイオービー） | ヒト錐体光受容体オプトジェネティクス構築物 |
| AU2023323327A1 (en) | 2022-08-11 | 2025-02-13 | Institute Of Molecular And Clinical Ophthalmology Basel (Iob) | Promoters for specific expression of genes in cone photoreceptors |
| CN121794288A (zh) | 2023-05-05 | 2026-04-03 | 麻省理工学院 | Shank3基因治疗方法 |
| WO2025090942A1 (en) * | 2023-10-26 | 2025-05-01 | University Of Notre Dame Du Lac | Gene therapy for treatment of neurometabolic disease |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP0856585A1 (en) | 1997-01-29 | 1998-08-05 | Introgene B.V. | A conditional replication and expression system |
| EP1228234A2 (en) * | 2000-03-14 | 2002-08-07 | Neurologix, Inc. | Production of chimeric capsid 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 |
| US20040208847A1 (en) * | 2003-03-28 | 2004-10-21 | Fabienne Rolling | Method and vectors for selectively transducing retinal pigment epithelium cells |
| WO2006090689A1 (ja) * | 2005-02-23 | 2006-08-31 | Dnavec Corporation | Siv-pedfベクターを用いた眼組織細胞におけるアポトーシス変性を伴う疾患の治療薬 |
| AU2006242371A1 (en) * | 2005-04-29 | 2006-11-09 | The University Of North Carolina At Chapel Hill | Methods and compositions for regulated expression of nucleic acid at post-transcriptional level |
| US20090022731A1 (en) * | 2006-08-25 | 2009-01-22 | Wyeth | Arthritis-associated B cell gene expression |
| US20100278843A1 (en) * | 2007-08-16 | 2010-11-04 | St. Vincent's Hospital Sydney Limited | Agents and methods for modulating macrophage inhibitory cytokine (mic-1) activity |
| EP2242840B1 (en) * | 2008-01-29 | 2019-07-24 | Applied Genetic Technologies Corporation | Recombinant adeno-associated virus production using bhk cells in suspension |
| CA2713755A1 (en) * | 2008-02-07 | 2009-08-13 | Ceregene, Inc. | Rescue of photoreceptors by intravitreal administration of an expression vector encoding a therapeutic protein |
| SI3988660T1 (sl) * | 2009-05-02 | 2025-09-30 | Genzyme Corporation | Genska terapija za nevrodegenerativne motnje |
| US9169494B2 (en) | 2010-01-12 | 2015-10-27 | The University Of North Carolina At Chapel Hill | Restrictive inverted terminal repeats for viral vectors |
| GB201103062D0 (en) † | 2011-02-22 | 2011-04-06 | Isis Innovation | Method |
-
2011
- 2011-02-22 GB GBGB1103062.4A patent/GB201103062D0/en not_active Ceased
-
2012
- 2012-02-21 CN CN201710784145.9A patent/CN107881198A/zh active Pending
- 2012-02-21 CA CA2827975A patent/CA2827975C/en active Active
- 2012-02-21 US US14/000,836 patent/US9834788B2/en active Active
- 2012-02-21 EP EP12706308.9A patent/EP2678435B2/en active Active
- 2012-02-21 KR KR1020207021515A patent/KR102299430B1/ko not_active Expired - Fee Related
- 2012-02-21 BR BR112013021318-3A patent/BR112013021318B1/pt not_active IP Right Cessation
- 2012-02-21 PL PL12706308T patent/PL2678435T3/pl unknown
- 2012-02-21 MX MX2013009604A patent/MX356525B/es active IP Right Grant
- 2012-02-21 KR KR1020137024782A patent/KR102139203B1/ko not_active Expired - Fee Related
- 2012-02-21 AU AU2012220404A patent/AU2012220404B2/en not_active Ceased
- 2012-02-21 PT PT127063089T patent/PT2678435T/pt unknown
- 2012-02-21 CN CN201280019547.5A patent/CN103562396A/zh active Pending
- 2012-02-21 ES ES12706308T patent/ES2676550T5/es active Active
- 2012-02-21 WO PCT/GB2012/050376 patent/WO2012114090A1/en not_active Ceased
- 2012-02-21 EP EP18172129.1A patent/EP3428283A1/en not_active Withdrawn
- 2012-02-21 DK DK12706308.9T patent/DK2678435T3/en active
- 2012-02-21 KR KR1020217028082A patent/KR20210112407A/ko not_active Ceased
- 2012-02-21 MX MX2018006733A patent/MX388146B/es unknown
- 2012-02-21 SG SG2013063797A patent/SG192911A1/en unknown
- 2012-02-21 JP JP2013554938A patent/JP2014512171A/ja active Pending
-
2013
- 2013-08-19 IL IL228031A patent/IL228031B/en active IP Right Grant
-
2014
- 2014-05-23 HK HK19101394.9A patent/HK1258917A1/en unknown
-
2017
- 2017-06-14 JP JP2017117286A patent/JP2017221192A/ja active Pending
- 2017-07-20 AU AU2017206254A patent/AU2017206254A1/en not_active Abandoned
- 2017-10-31 US US15/799,852 patent/US20180135078A1/en not_active Abandoned
-
2018
- 2018-07-13 HK HK18109118.8A patent/HK1249548A1/zh unknown
-
2019
- 2019-11-05 JP JP2019201092A patent/JP2020054345A/ja active Pending
-
2020
- 2020-01-24 US US16/751,699 patent/US20210032656A1/en not_active Abandoned
-
2022
- 2022-10-26 US US17/974,466 patent/US20230332176A1/en not_active Abandoned
- 2022-12-02 JP JP2022193831A patent/JP2023036618A/ja active Pending
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