JP2014512171A5 - - Google Patents
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- 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 |
|---|---|---|---|
| GBGB1103062.4A GB201103062D0 (en) | 2011-02-22 | 2011-02-22 | Method |
| GB1103062.4 | 2011-02-22 | ||
| 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 (OSRAM) | 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 (OSRAM) |
| EP (2) | EP2678435B2 (OSRAM) |
| JP (4) | JP2014512171A (OSRAM) |
| KR (3) | KR102139203B1 (OSRAM) |
| CN (2) | CN107881198A (OSRAM) |
| AU (2) | AU2012220404B2 (OSRAM) |
| BR (1) | BR112013021318B1 (OSRAM) |
| CA (1) | CA2827975C (OSRAM) |
| DK (1) | DK2678435T3 (OSRAM) |
| ES (1) | ES2676550T5 (OSRAM) |
| GB (1) | GB201103062D0 (OSRAM) |
| HK (2) | HK1258917A1 (OSRAM) |
| IL (1) | IL228031B (OSRAM) |
| MX (2) | MX356525B (OSRAM) |
| PL (1) | PL2678435T3 (OSRAM) |
| PT (1) | PT2678435T (OSRAM) |
| SG (1) | SG192911A1 (OSRAM) |
| WO (1) | WO2012114090A1 (OSRAM) |
Families Citing this family (47)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201103062D0 (en) † | 2011-02-22 | 2011-04-06 | Isis Innovation | Method |
| CA2919995A1 (en) | 2013-08-05 | 2015-02-12 | Academisch Ziekenhuis Leiden H.O.D.N. Leids Universitair Medisch Centrum | Recombinant aav-crumbs homologue composition and methods for treating lca-8 and progressive rp |
| MX379027B (es) * | 2013-12-06 | 2025-03-10 | Inst Nat Sante Rech Med | Metodos y composiciones farmaceuticas para la expresion de un polinucleotido de interes en el epitelio pigmentario retiniano de un sujeto. |
| WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| RU2716991C2 (ru) | 2014-11-05 | 2020-03-17 | Вояджер Терапьютикс, Инк. | Полинуклеотиды aadc для лечения болезни паркинсона |
| RU2716422C2 (ru) | 2014-11-14 | 2020-03-11 | Вояджер Терапьютикс, Инк. | Композиции и способы лечения бокового амиотрофического склероза (als) |
| CN112410339A (zh) | 2014-11-14 | 2021-02-26 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| HK1245326A1 (zh) | 2014-12-12 | 2018-08-24 | Voyager Therapeutics, Inc. | 用於生产scaav的组合物和方法 |
| HUE051491T2 (hu) * | 2015-03-06 | 2021-03-01 | Massachusetts Eye & Ear Infirmary | Gén-augmentációs terápiák a PRPF31 gén mutációi által okozott öröklött retina degenerációra |
| EP3795180B1 (en) | 2015-12-14 | 2025-11-05 | The Trustees of The University of Pennsylvania | Gene therapy for ocular disorders |
| GB201604146D0 (en) * | 2016-03-10 | 2016-04-27 | Nightstarx Ltd | Prenylation assay |
| WO2017189964A2 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| CN109831916B (zh) | 2016-05-18 | 2023-07-21 | 沃雅戈治疗公司 | 治疗亨廷顿氏舞蹈病的组合物和方法 |
| CA3252099A1 (en) | 2016-05-18 | 2025-06-05 | Voyager Therapeutics, Inc. | MODULATING POLYNUCLEOTIDES |
| EP3506817A4 (en) | 2016-08-30 | 2020-07-22 | The Regents of The University of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| CN110177579A (zh) * | 2016-11-07 | 2019-08-27 | 星火治疗股份有限公司 | Rab保护蛋白效价测定 |
| WO2018129586A1 (en) * | 2017-01-10 | 2018-07-19 | Children's Medical Research Institute | 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 |
| JP2020518259A (ja) | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | ハンチントン病治療組成物および方法 |
| JP2020518258A (ja) | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | 筋萎縮性側索硬化症（ａｌｓ）治療組成物および方法 |
| WO2018232149A1 (en) | 2017-06-14 | 2018-12-20 | 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 |
| WO2019018342A1 (en) | 2017-07-17 | 2019-01-24 | Voyager Therapeutics, Inc. | NETWORK EQUIPMENT TRACK GUIDE SYSTEM |
| KR102850887B1 (ko) | 2017-08-03 | 2025-08-28 | 보이저 테라퓨틱스, 인크. | Aav의 전달을 위한 조성물 및 방법 |
| 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. |
| CN111465691A (zh) * | 2017-10-03 | 2020-07-28 | 普利维尔治疗公司 | 用于溶酶体障碍的基因疗法 |
| CN119242711A (zh) | 2017-10-16 | 2025-01-03 | 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) |
| SG11202003462SA (en) | 2017-10-17 | 2020-05-28 | Nightstarx Ltd | Prenylation assay |
| KR20200119782A (ko) * | 2017-12-06 | 2020-10-20 | 오비드 테라퓨틱스 인크. | 발작 장애 치료 시 mir101 또는 mir128의 용도 |
| PE20212076A1 (es) | 2018-02-01 | 2021-10-26 | Homology Medicines Inc | Composiciones de virus adeno-asociado para restaurar la funcion del gen pah y metodos de uso de las mismas |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer 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 |
| BR112020020174A2 (pt) * | 2018-04-05 | 2021-01-19 | Nightstarx Limited | Composições de aav, métodos de fabricação e métodos de uso |
| WO2019216932A1 (en) * | 2018-05-07 | 2019-11-14 | The University Of North Carolina At Chapel Hill | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
| BR112020023082A2 (pt) | 2018-05-15 | 2021-02-09 | Voyager Therapeutics, Inc. | composições e métodos para o tratamento de doença de parkinson |
| CA3114621A1 (en) | 2018-09-28 | 2020-04-02 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| EP3980548A4 (en) * | 2019-04-30 | 2023-09-06 | 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 |
| KR20230051578A (ko) | 2020-08-17 | 2023-04-18 | 메사추세츠 인스티튜트 오브 테크놀로지 | Shank3 유전자 치료 접근법 |
| 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 |
| JP2025528187A (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） | 錐体光受容体における遺伝子の特異的発現のためのプロモーター |
| WO2024033837A1 (en) | 2022-08-11 | 2024-02-15 | Institute Of Molecular And Clinical Ophthalmology Basel (Iob) | Human cone photoreceptor optogenetic constructs |
| WO2024233422A1 (en) | 2023-05-05 | 2024-11-14 | Massachusetts Institute Of Technology | Shank3 gene therapy approaches |
| 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 |
| CA2373110A1 (en) * | 2000-03-14 | 2001-09-20 | Neurologix, Inc. | Production of chimeric capsid vectors |
| WO2002082904A2 (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 |
| JP4959547B2 (ja) * | 2005-02-23 | 2012-06-27 | ディナベック株式会社 | 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 |
| NZ562780A (en) * | 2005-04-29 | 2010-03-26 | Univ North Carolina | Method 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 |
| CA2694863A1 (en) * | 2007-08-16 | 2009-02-19 | Garvan Institute Of Medical Research | 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 |
| JP2011511090A (ja) * | 2008-02-07 | 2011-04-07 | セレジーン インコーポレイテッド | 治療用タンパク質をコードする発現ベクターの硝子体内投与による光受容体のレスキュー方法 |
| DK3421603T3 (da) * | 2009-05-02 | 2022-01-10 | Genzyme Corp | Genterapi for neurodegenerative forstyrrelser |
| EP2524037B1 (en) | 2010-01-12 | 2018-05-16 | 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 KR KR1020137024782A patent/KR102139203B1/ko not_active Expired - Fee Related
- 2012-02-21 ES ES12706308T patent/ES2676550T5/es active Active
- 2012-02-21 MX MX2013009604A patent/MX356525B/es active IP Right Grant
- 2012-02-21 EP EP12706308.9A patent/EP2678435B2/en active Active
- 2012-02-21 CN CN201710784145.9A patent/CN107881198A/zh active Pending
- 2012-02-21 SG SG2013063797A patent/SG192911A1/en unknown
- 2012-02-21 MX MX2018006733A patent/MX388146B/es unknown
- 2012-02-21 CA CA2827975A patent/CA2827975C/en active Active
- 2012-02-21 KR KR1020217028082A patent/KR20210112407A/ko not_active Ceased
- 2012-02-21 AU AU2012220404A patent/AU2012220404B2/en not_active Ceased
- 2012-02-21 BR BR112013021318-3A patent/BR112013021318B1/pt not_active IP Right Cessation
- 2012-02-21 EP EP18172129.1A patent/EP3428283A1/en not_active Withdrawn
- 2012-02-21 PT PT127063089T patent/PT2678435T/pt unknown
- 2012-02-21 PL PL12706308T patent/PL2678435T3/pl unknown
- 2012-02-21 JP JP2013554938A patent/JP2014512171A/ja active Pending
- 2012-02-21 KR KR1020207021515A patent/KR102299430B1/ko not_active Expired - Fee Related
- 2012-02-21 US US14/000,836 patent/US9834788B2/en active Active
- 2012-02-21 DK DK12706308.9T patent/DK2678435T3/en active
- 2012-02-21 CN CN201280019547.5A patent/CN103562396A/zh active Pending
- 2012-02-21 WO PCT/GB2012/050376 patent/WO2012114090A1/en not_active Ceased
-
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 active Pending
- 2022-12-02 JP JP2022193831A patent/JP2023036618A/ja active Pending
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