JP2015523379A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2015523379A5 JP2015523379A5 JP2015521593A JP2015521593A JP2015523379A5 JP 2015523379 A5 JP2015523379 A5 JP 2015523379A5 JP 2015521593 A JP2015521593 A JP 2015521593A JP 2015521593 A JP2015521593 A JP 2015521593A JP 2015523379 A5 JP2015523379 A5 JP 2015523379A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- rpgr
- nucleic acid
- seq
- sequence
- 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
- 101700001406 RPGR Proteins 0.000 claims 5
- 102100009234 RPGR Human genes 0.000 claims 5
- 229920001850 Nucleic acid sequence Polymers 0.000 claims 4
- 150000007523 nucleic acids Chemical group 0.000 claims 4
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 2
- 102100007493 CNTF Human genes 0.000 claims 2
- 108010005939 Ciliary Neurotrophic Factor Proteins 0.000 claims 2
- 108020004705 Codon Proteins 0.000 claims 2
- 230000001105 regulatory Effects 0.000 claims 2
- 238000002560 therapeutic procedure Methods 0.000 claims 2
- 241000432074 Adeno-associated virus Species 0.000 claims 1
- 101710028887 CKL_0015 Proteins 0.000 claims 1
- 210000000234 Capsid Anatomy 0.000 claims 1
- 101710029785 DA26 Proteins 0.000 claims 1
- 102100020499 GRK1 Human genes 0.000 claims 1
- 101700083645 GRK1 Proteins 0.000 claims 1
- 101710028938 M1627_0030 Proteins 0.000 claims 1
- 101710011281 MDV056 Proteins 0.000 claims 1
- 101700028338 ORF15 Proteins 0.000 claims 1
- 101710030183 ORF161 Proteins 0.000 claims 1
- 210000000608 Photoreceptor Cells Anatomy 0.000 claims 1
- 102100016799 RBP3 Human genes 0.000 claims 1
- 101700025296 RBP3 Proteins 0.000 claims 1
- 208000007014 Retinitis Pigmentosa Diseases 0.000 claims 1
- 101710043875 Vi01_157c Proteins 0.000 claims 1
- 101700019905 YD2M Proteins 0.000 claims 1
- 201000004569 blindness Diseases 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 230000004393 visual impairment Effects 0.000 claims 1
- 101700030621 ycbP Proteins 0.000 claims 1
Claims (16)
- 正常ＲＰＧＲタンパク質またはそのフラグメントをコードする核酸配列を、該正常ＲＰＧＲ遺伝子産物を光受容細胞中で発現する調節配列の制御下に担持する組換えアデノ随伴ウイルス（ＡＡＶ）。
- 核酸配列がＲＰＧＲエキソン１からＯＲＦ１５までをコードする請求項１に記載のＡＡＶ。
- ＲＰＧＲ配列が配列番号5をコードする請求項1または2に記載のＡＡＶ。
- ＲＰＧＲ配列が配列番号６をコードする請求項1または2に記載のＡＡＶ。
- 核酸配列が配列番号１またはそのコドン最適化配列である請求項１〜３のいずれかに記載のＡＡＶ。
- 核酸配列が配列番号４またはそのコドン最適化配列である請求項１、２および４のいずれかに記載のＡＡＶ。
- 調節配列がＩＲＢＰプロモーターまたはＧＲＫ１プロモーターを含んでなる請求項１〜６のいずれかに記載のＡＡＶ。
- ＡＡＶが偽型ＡＡＶであり、そして場合によりＡＡＶ２／５偽型ＡＡＶである請求項１〜４のいずれかに記載のＡＡＶ。
- ＡＡＶキャプシドがＡＡＶ2又はＡＡＶ5からのものである請求項1に記載のＡＡＶ。
- 請求項1〜9のいずれかに記載の組換えＡＡＶおよび製薬学的に許容され得る担体を含んでなる組成物。
- 治療において使用するための請求項１〜９のいずれかに記載のＡＡＶ。
- 網膜色素変性症に伴う視覚喪失を防ぎ、進行を阻止し、または改善する際に使用するための請求項１〜９のいずれかに記載のＡＡＶ。
- 毛様体神経栄養因子（ＣＮＴＦ）と併用するための請求項１〜９のいずれかに記載のＡＡＶ。
- 請求項１〜９のいずれかに記載のＡＡＶを含有する組成物が同じ目および／または反対側の目に少なくとも１回再投与される療法で使用するための該ＡＡＶ。
- 請求項１〜９のいずれかに記載のＡＡＶであって、用量が１ミリリットル（ｍＬ）当たり１０ 9 〜１０ 13 ベクターゲノム（ｖｇ）の濃度を含む、ＡＡＶ。
- 用量が５０μＬ〜１ｍＬの容量で投与される請求項１〜９及び１５のいずれかに記載のＡＡＶ。
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261670355P | 2012-07-11 | 2012-07-11 | |
| US61/670,355 | 2012-07-11 | ||
| PCT/US2013/022628 WO2014011210A1 (en) | 2012-07-11 | 2013-01-23 | Aav-mediated gene therapy for rpgr x-linked retinal degeneration |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2015523379A JP2015523379A (ja) | 2015-08-13 |
| JP2015523379A5 true JP2015523379A5 (ja) | 2016-03-03 |
| JP6199965B2 JP6199965B2 (ja) | 2017-09-20 |
Family
ID=49916455
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015521593A Active JP6199965B2 (ja) | 2012-07-11 | 2013-01-23 | Ｒｐｇｒｘ連鎖性網膜変性のａａｖ媒介型遺伝子治療 |
Country Status (8)
| Country | Link |
|---|---|
| US (2) | US9770491B2 (ja) |
| EP (1) | EP2872183B1 (ja) |
| JP (1) | JP6199965B2 (ja) |
| CN (1) | CN105120901A (ja) |
| AU (1) | AU2013287281B2 (ja) |
| CA (1) | CA2878171C (ja) |
| NZ (1) | NZ704275A (ja) |
| WO (1) | WO2014011210A1 (ja) |
Families Citing this family (24)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| NZ704275A (en) * | 2012-07-11 | 2016-09-30 | Univ Florida | Aav-mediated gene therapy for rpgr x-linked retinal degeneration |
| SI3119437T1 (sl) * | 2014-03-21 | 2020-01-31 | Genzyme Corporation | Genska terapija za retinitis pigmentoza |
| WO2015160893A1 (en) * | 2014-04-15 | 2015-10-22 | Applied Genetic Technologies Corporation | Codon optimized nucleic acid encoding a retinitis pigmentosa gtpase regulator (rpgr) |
| GB201412011D0 (en) * | 2014-07-04 | 2014-08-20 | Ucl Business Plc | Treatments |
| US10314924B2 (en) | 2014-07-24 | 2019-06-11 | Massachusetts Eye & Ear Infirmary | RPGR gene therapy for retinitis pigmentosa |
| EP3748007A1 (en) * | 2015-03-06 | 2020-12-09 | Massachusetts Eye & Ear Infirmary | Gene augmentation therapies for inherited retinal degeneration caused by mutations in the prpf31 gene |
| CA2979229A1 (en) * | 2015-03-11 | 2016-09-15 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Rp2 vectors for treating x-linked retinitis pigmentosa |
| EP3302521A1 (en) * | 2015-06-04 | 2018-04-11 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Improved methods for treating ocular diseases by gene therapy |
| GB201516066D0 (en) | 2015-09-10 | 2015-10-28 | Young & Co Llp D | Treatment of retinitis pigmentosa |
| WO2017087900A1 (en) | 2015-11-19 | 2017-05-26 | The Trustees Of The University Of Pennsylvania | Compositions and methods for correction of heritable ocular disease |
| ES2826384T3 (es) | 2015-12-14 | 2021-05-18 | Univ Pennsylvania | Terapia génica para trastornos oculares |
| JP7140683B2 (ja) | 2016-04-15 | 2022-09-21 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 新規なａａｖ８変異カプシド及びそれを含有する組成物 |
| BR112019000171A2 (pt) | 2016-07-08 | 2019-10-01 | Univ Pennsylvania | métodos e composições para tratamento de distúrbios e doenças envolvendo rdh12 |
| EP3589738A4 (en) | 2017-03-01 | 2021-01-06 | The Trustees of The University of Pennsylvania | GENE THERAPY FOR EYE DISORDERS |
| US11879133B2 (en) | 2017-04-24 | 2024-01-23 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
| CA3098592A1 (en) | 2017-05-31 | 2018-12-06 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating peroxisomal disorders |
| EP3638316A4 (en) | 2017-06-14 | 2021-03-24 | The Trustees Of The University Of Pennsylvania | GENE THERAPY FOR EYE DISORDERS |
| IL308645A (en) * | 2017-11-15 | 2024-01-01 | Univ Michigan Regents | Viral vectors comprising RDH12 coding regions and methods for the treatment of retinal dystrophies |
| WO2019204514A1 (en) | 2018-04-17 | 2019-10-24 | Limelight Bio, Inc. | Trans-splicing molecules |
| CN113952472A (zh) * | 2020-07-21 | 2022-01-21 | 英斯培瑞有限公司 | 用于治疗眼部疾病的组合物和方法 |
| KR20230061372A (ko) * | 2020-09-02 | 2023-05-08 | 4디 몰레큘러 테라퓨틱스 아이엔씨. | 코돈 최적화된 rpgrorf15 유전자 및 이의 용도 |
| JP2024505257A (ja) | 2021-02-01 | 2024-02-05 | レジェンクスバイオ インコーポレーテッド | 神経セロイドリポフスチン症の遺伝子治療 |
| CN117165596A (zh) * | 2022-05-27 | 2023-12-05 | 武汉纽福斯生物科技有限公司 | 编码rpgr的核酸及其应用 |
| WO2024013224A2 (en) * | 2022-07-14 | 2024-01-18 | Fondation Asile Des Aveugles | Gene therapy for fam161a-associated retinopathies and other ciliopathies |
Family Cites Families (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| JP3768257B2 (ja) * | 1993-12-29 | 2006-04-19 | 大日本住友製薬株式会社 | 改変タンパク質 |
| AU1282895A (en) | 1993-12-29 | 1995-07-17 | Sumitomo Pharmaceuticals Company, Limited | Novel human ciliary neurotrophic factor |
| US6372250B1 (en) * | 2000-04-25 | 2002-04-16 | The Regents Of The University Of California | Non-invasive gene targeting to the brain |
| CA2442670A1 (en) | 2001-04-13 | 2002-10-24 | The Trustees Of The University Of Pennsylvania | Method of treating or retarding the development of blindness |
| US20030003582A1 (en) * | 2001-05-08 | 2003-01-02 | Tranzyme, Inc. | Trans-viral vector mediated gene transfer to the retina |
| FI3517134T3 (fi) | 2001-12-17 | 2024-04-03 | Univ Pennsylvania | Adeno-assosioituneen viruksen (aav) serotyypin 8 sekvenssejä, niitä sisältäviä vektoreita ja niiden käyttöjä |
| DE60333035D1 (de) | 2002-12-23 | 2010-07-29 | Vical Inc | Impfstoffe gegen infektionen mit dem humanen zytomegalivirus auf grundlage von codonoptimierten polynukleotiden |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| EP3211085B1 (en) | 2003-09-30 | 2021-03-31 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
| ES2331342B1 (es) | 2006-05-22 | 2010-10-13 | Consejo Superior Investg.Cientificas | Uso de la proinsulina para la elaboracion de una composicion farmaceutica neuroprotectora, composicion terapeutica que la contiene y sus aplicaciones. |
| 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 |
| WO2009105690A2 (en) * | 2008-02-21 | 2009-08-27 | Targeted Genetics Corporation | Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea |
| US7561972B1 (en) | 2008-06-06 | 2009-07-14 | Dna Twopointo, Inc. | Synthetic nucleic acids for expression of encoded proteins |
| US7561973B1 (en) | 2008-07-31 | 2009-07-14 | Dna Twopointo, Inc. | Methods for determining properties that affect an expression property value of polynucleotides in an expression system |
| ITMI20090284A1 (it) * | 2009-02-26 | 2010-08-27 | Consiglio Nazionale Ricerche | Uso di inibitori della serina palmitoiltrasferasi per la prevenzione e il rallentamento delle degenerazioni retiniche ereditarie e relative composizioni |
| GB0911870D0 (en) * | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
| NZ704275A (en) * | 2012-07-11 | 2016-09-30 | Univ Florida | Aav-mediated gene therapy for rpgr x-linked retinal degeneration |
-
2013
- 2013-01-23 NZ NZ704275A patent/NZ704275A/en unknown
- 2013-01-23 EP EP13816245.8A patent/EP2872183B1/en active Active
- 2013-01-23 US US14/413,884 patent/US9770491B2/en active Active
- 2013-01-23 CA CA2878171A patent/CA2878171C/en active Active
- 2013-01-23 JP JP2015521593A patent/JP6199965B2/ja active Active
- 2013-01-23 AU AU2013287281A patent/AU2013287281B2/en active Active
- 2013-01-23 WO PCT/US2013/022628 patent/WO2014011210A1/en active Application Filing
- 2013-01-23 CN CN201380045636.1A patent/CN105120901A/zh active Pending
-
2017
- 2017-09-08 US US15/699,262 patent/US10383922B2/en active Active
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2015523379A5 (ja) | ||
| RU2020137429A (ru) | Лечение пигментного ретинита | |
| JP6767483B2 (ja) | 遺伝子療法のための、改変されたフリードライヒ運動失調症遺伝子およびベクター | |
| JP2023036618A (ja) | 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 | |
| NZ704275A (en) | Aav-mediated gene therapy for rpgr x-linked retinal degeneration | |
| EA038695B1 (ru) | Способы и композиции для переноса генов по сосудистой сети | |
| JP2017532966A5 (ja) | ||
| US10413598B2 (en) | Factor IX gene therapy | |
| JP2022174192A (ja) | 多重ベクターシステム及びその使用 | |
| JP2017536116A5 (ja) | ||
| JP2019506445A (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 | |
| JP2020073536A5 (ja) | ||
| JP2014512171A5 (ja) | ||
| CA3024449A1 (en) | Compositions and methods of treating huntington's disease | |
| JP2017518271A5 (ja) | ||
| RU2016149206A (ru) | Аденоассоциированные вирусные векторы для лечения лизосомных болезней накопления | |
| JP2021508241A5 (ja) | ||
| RU2018125468A (ru) | Генная терапия офтальмологических нарушений | |
| JP6824169B2 (ja) | 改変ｇ６ｐｃをコードするアデノ随伴ウイルスベクターおよびその使用 | |
| JP2019502378A5 (ja) | ||
| JP2019524090A5 (ja) | ||
| JP2017523239A5 (ja) | ||
| JP2022530824A (ja) | ポンペ病の治療のために有用な組成物 | |
| JP2011511090A5 (ja) | ||
| JP2022116045A (ja) | アルデヒドデヒドロゲナーゼ欠損症の治療のための遺伝子治療 |