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
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- Prior art keywords
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- 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.)
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- 229920000023 polynucleotide Polymers 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
- 102100001794 CHM Human genes 0.000 claims 2
- 101710034152 CHM Proteins 0.000 claims 2
- 101700075404 HFR1 Proteins 0.000 claims 2
- 101700084930 REP1 Proteins 0.000 claims 2
- 210000001525 Retina Anatomy 0.000 claims 2
- 239000002245 particle Substances 0.000 claims 2
- 241000432074 Adeno-associated virus Species 0.000 claims 1
- 210000000234 Capsid Anatomy 0.000 claims 1
- 206010008785 Choroidal dystrophy Diseases 0.000 claims 1
- 208000003571 Choroideremia Diseases 0.000 claims 1
- 108091006028 chimera Proteins 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 229920001184 polypeptide Polymers 0.000 claims 1
- 230000001105 regulatory Effects 0.000 claims 1
- 230000002207 retinal Effects 0.000 claims 1
- 235000020945 retinal Nutrition 0.000 claims 1
Claims (17)
- アデノ随伴ウイルス（ＡＡＶ）ゲノム又はその誘導体及びＲＥＰ１又はその変異体をコードするポリヌクレオチド配列を含む、ベクター。
- 誘導体が、キメラ、シャッフル又はカプシド修飾誘導体である、請求項１に記載のベクター。
- ＡＡＶゲノムが、天然由来の血清型、又はＡＡＶの分離株若しくはクレードである、請求項１又は２に記載のベクター。
- 血清型が、ＡＡＶ血清型２（ＡＡＶ２）である、請求項３に記載のベクター。
- 配列番号１又はその誘導体を含む、請求項４に記載のベクター。
- ポリヌクレオチド配列が、配列番号３の全長配列と少なくとも７０％の相同性を有するポリペプチドをコードするものである、請求項１〜５のいずれか１項に記載のベクター。
- ポリヌクレオチド配列が、配列番号２の全長配列と少なくとも７０％の相同性を有する、請求項６に記載のベクター。
- ＲＥＰ１又はその変異体をコードするポリヌクレオチド配列と作動可能に連結したプロモーター配列を含む、請求項１〜７のいずれか１項に記載のベクター。
- プロモーターが、恒常的に活性である、請求項８に記載のベクター。
- プロモーターからの発現が、網膜細胞特異的である、請求項８に記載のベクター。
- １以上の追加的な調節配列を含む、請求項１〜１０のいずれか１項に記載のベクター。
- 配列番号５の全長配列と少なくとも７０％の相同性を有する配列を含む、請求項１１に記載のベクター。
- ベクター粒子の形態である、請求項１〜１２のいずれか１項に記載のベクター。
- ベクター粒子がＡＡＶ２血清型である、請求項１３に記載のベクター。
- 患者にベクターを投与することで、コロイデレミアを治療又は予防する方法における使用のための、請求項１〜１４のいずれか１項に記載のベクターを含む医薬。
- 直接網膜に注射、網膜下に注射、又は硝子体内注射により患者に投与されるためのものである、請求項１５に記載の医薬。
- コロイデレミアを治療又は予防するための医薬の製造における使用のための、請求項１〜１４のいずれか１項に記載のベクター。
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 (ja) | 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 (ja) |
| EP (2) | EP3428283A1 (ja) |
| JP (4) | JP2014512171A (ja) |
| KR (3) | KR102139203B1 (ja) |
| CN (2) | CN103562396A (ja) |
| AU (2) | AU2012220404B2 (ja) |
| BR (1) | BR112013021318B1 (ja) |
| CA (1) | CA2827975C (ja) |
| DK (1) | DK2678435T3 (ja) |
| ES (1) | ES2676550T5 (ja) |
| GB (1) | GB201103062D0 (ja) |
| HK (2) | HK1258917A1 (ja) |
| IL (1) | IL228031B (ja) |
| MX (1) | MX356525B (ja) |
| PL (1) | PL2678435T3 (ja) |
| PT (1) | PT2678435T (ja) |
| SG (1) | SG192911A1 (ja) |
| WO (1) | WO2012114090A1 (ja) |
Families Citing this family (40)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201103062D0 (en) † | 2011-02-22 | 2011-04-06 | Isis Innovation | Method |
| EP3030665B9 (en) | 2013-08-05 | 2023-10-04 | 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 |
| CN105940109A (zh) * | 2013-12-06 | 2016-09-14 | 国立健康与医学研究所 | 用于在受试者的视网膜色素上皮中表达目的多核苷酸的方法和药物组合物 |
| WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| CN107106689A (zh) | 2014-11-05 | 2017-08-29 | 沃雅戈治疗公司 | 用于治疗帕金森病的aadc多核苷酸 |
| MX2017006216A (es) | 2014-11-14 | 2018-08-29 | Voyager Therapeutics Inc | Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela). |
| SG11201703419UA (en) | 2014-11-14 | 2017-05-30 | Voyager Therapeutics Inc | Modulatory polynucleotides |
| EP3230441A4 (en) | 2014-12-12 | 2018-10-03 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scaav |
| EP3748007B1 (en) * | 2015-03-06 | 2024-06-26 | Massachusetts Eye & 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 |
| US20190017096A1 (en) | 2016-03-10 | 2019-01-17 | Nightstarx Limited | Prenylation assay |
| US11299751B2 (en) | 2016-04-29 | 2022-04-12 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| KR102392236B1 (ko) | 2016-05-18 | 2022-05-03 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| SG11201809643UA (en) | 2016-05-18 | 2018-12-28 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
| EP3831281A1 (en) | 2016-08-30 | 2021-06-09 | The Regents of The University of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| JP2019534018A (ja) | 2016-11-07 | 2019-11-28 | スパーク セラピューティクス インコーポレイテッドＳｐａｒｋ Ｔｈｅｒａｐｅｕｔｉｃｓ， Ｉｎｃ． | Ｒａｂエスコートタンパク質の力価アッセイ |
| EP3568483A4 (en) * | 2017-01-10 | 2020-08-19 | Children's Medical Research Institute | POLYNUCLEOTIDES AND VECTORS OF TRANSGEN EXPRESSION |
| 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 |
| EP3619308A4 (en) | 2017-05-05 | 2021-01-27 | Voyager Therapeutics, Inc. | COMPOSITIONS AND METHODS OF TREATMENT FOR HUNTINGTON'S MORBUS |
| CA3061652A1 (en) | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating 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 |
| JP7229989B2 (ja) | 2017-07-17 | 2023-02-28 | ボイジャー セラピューティクス インコーポレイテッド | 軌道アレイガイドシステム |
| KR20200044793A (ko) | 2017-08-03 | 2020-04-29 | 보이저 테라퓨틱스, 인크. | Aav의 전달을 위한 조성물 및 방법 |
| CN112501208A (zh) * | 2017-10-03 | 2021-03-16 | 普利维尔治疗公司 | 用于溶酶体障碍的基因疗法 |
| JP7502991B2 (ja) | 2017-10-16 | 2024-06-19 | ボイジャー セラピューティクス インコーポレイテッド | 筋萎縮性側索硬化症（ａｌｓ）の治療 |
| 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 |
| EP3703820A4 (en) * | 2017-12-06 | 2020-12-09 | Ovid Therapeutics Inc. | USING THE MIR101 OR MIR128 IN TREATMENT OF SEQUENCE DISEASES |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| CN112041437A (zh) | 2018-02-19 | 2020-12-04 | 同源药物公司 | 用于恢复f8基因功能的腺相关病毒组合物和其使用方法 |
| KR20210005045A (ko) * | 2018-04-05 | 2021-01-13 | 나이트스타엑스 리미티드 | Aav 조성물, 제조 방법 및 사용 방법 |
| JP2021522775A (ja) * | 2018-05-07 | 2021-09-02 | ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒルＴｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｎｏｒｔｈ Ｃａｒｏｌｉｎａ Ａｔ Ｃｈａｐｅｌ Ｈｉｌｌ | 合理的なポリプロイド性アデノ随伴ウイルスベクターならびにその製造法および使用法 |
| 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 |
| CN116406305A (zh) | 2020-08-17 | 2023-07-07 | 麻省理工学院 | Shank3基因治疗方法 |
| WO2022109247A1 (en) | 2020-11-20 | 2022-05-27 | Biogen Ma Inc. | Viral vector potency assay |
| WO2023164545A1 (en) | 2022-02-23 | 2023-08-31 | Massachusetts Institute Of Technology | Methods for upregulating shank3 expression |
| WO2024033834A1 (en) | 2022-08-11 | 2024-02-15 | Institute Of Molecular And Clinical Opthalmology Basel (Iob) | Promoters for specific expression of genes in cone photoreceptors |
| WO2024033837A1 (en) | 2022-08-11 | 2024-02-15 | Institute Of Molecular And Clinical Ophthalmology Basel (Iob) | Human cone photoreceptor optogenetic constructs |
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 |
| 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 |
| 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 |
| AU2008286706B2 (en) * | 2007-08-16 | 2014-03-06 | Garvan Institute Of Medical Research | Agents and methods for modulating macrophage inhibitory cytokine (MIC-1) activity |
| AU2009209408B2 (en) * | 2008-01-29 | 2015-06-11 | Beacon Therapeutics Limited | Recombinant virus production using mammalian cells in suspension |
| WO2009100253A1 (en) * | 2008-02-07 | 2009-08-13 | Ceregene, Inc. | Rescue of photoreceptors by intravitreal administation of an expression vector encoding a therapeutic protein |
| SI2424991T1 (sl) * | 2009-05-02 | 2018-11-30 | Genzyme Corporation | Genska terapija za nevrodegenerativne motnje |
| WO2011088081A1 (en) * | 2010-01-12 | 2011-07-21 | 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 EP EP18172129.1A patent/EP3428283A1/en active Pending
- 2012-02-21 US US14/000,836 patent/US9834788B2/en active Active
- 2012-02-21 CN CN201280019547.5A patent/CN103562396A/zh active Pending
- 2012-02-21 CN CN201710784145.9A patent/CN107881198A/zh active Pending
- 2012-02-21 KR KR1020137024782A patent/KR102139203B1/ko active IP Right Grant
- 2012-02-21 MX MX2013009604A patent/MX356525B/es active IP Right Grant
- 2012-02-21 KR KR1020207021515A patent/KR102299430B1/ko active IP Right Grant
- 2012-02-21 KR KR1020217028082A patent/KR20210112407A/ko not_active Application Discontinuation
- 2012-02-21 PT PT127063089T patent/PT2678435T/pt unknown
- 2012-02-21 SG SG2013063797A patent/SG192911A1/en unknown
- 2012-02-21 CA CA2827975A patent/CA2827975C/en active Active
- 2012-02-21 BR BR112013021318-3A patent/BR112013021318B1/pt active IP Right Grant
- 2012-02-21 DK DK12706308.9T patent/DK2678435T3/en active
- 2012-02-21 PL PL12706308T patent/PL2678435T3/pl unknown
- 2012-02-21 ES ES12706308T patent/ES2676550T5/es active Active
- 2012-02-21 WO PCT/GB2012/050376 patent/WO2012114090A1/en active Application Filing
- 2012-02-21 EP EP12706308.9A patent/EP2678435B2/en active Active
- 2012-02-21 AU AU2012220404A patent/AU2012220404B2/en not_active Ceased
- 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/zh 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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