JP2018148927A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2018148927A5 JP2018148927A5 JP2018109162A JP2018109162A JP2018148927A5 JP 2018148927 A5 JP2018148927 A5 JP 2018148927A5 JP 2018109162 A JP2018109162 A JP 2018109162A JP 2018109162 A JP2018109162 A JP 2018109162A JP 2018148927 A5 JP2018148927 A5 JP 2018148927A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- administration
- formulated
- composition according
- injection
- 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
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US17498209P | 2009-05-02 | 2009-05-02 | |
| US61/174,982 | 2009-05-02 | ||
| US26805909P | 2009-06-08 | 2009-06-08 | |
| US61/268,059 | 2009-06-08 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015195602A Division JP6397391B2 (ja) | 2009-05-02 | 2015-10-01 | 神経変性障害のための遺伝子治療 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2018148927A JP2018148927A (ja) | 2018-09-27 |
| JP2018148927A5 true JP2018148927A5 (enExample) | 2018-12-27 |
Family
ID=43050324
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2012508474A Active JP5879256B2 (ja) | 2009-05-02 | 2010-04-27 | 神経変性障害のための遺伝子治療 |
| JP2015195602A Active JP6397391B2 (ja) | 2009-05-02 | 2015-10-01 | 神経変性障害のための遺伝子治療 |
| JP2018109162A Pending JP2018148927A (ja) | 2009-05-02 | 2018-06-07 | 神経変性障害のための遺伝子治療 |
Family Applications Before (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2012508474A Active JP5879256B2 (ja) | 2009-05-02 | 2010-04-27 | 神経変性障害のための遺伝子治療 |
| JP2015195602A Active JP6397391B2 (ja) | 2009-05-02 | 2015-10-01 | 神経変性障害のための遺伝子治療 |
Country Status (22)
| Country | Link |
|---|---|
| US (9) | US20130287736A1 (enExample) |
| EP (5) | EP4342992B1 (enExample) |
| JP (3) | JP5879256B2 (enExample) |
| KR (2) | KR101835490B1 (enExample) |
| CN (2) | CN102459611B (enExample) |
| CA (1) | CA2759801C (enExample) |
| CY (1) | CY1120982T1 (enExample) |
| DK (4) | DK3421603T3 (enExample) |
| ES (4) | ES2903127T3 (enExample) |
| FI (2) | FI4342992T3 (enExample) |
| HR (4) | HRP20250897T1 (enExample) |
| HU (4) | HUE072183T2 (enExample) |
| IL (2) | IL216094A0 (enExample) |
| LT (4) | LT3988660T (enExample) |
| MX (4) | MX341073B (enExample) |
| PL (4) | PL4342992T3 (enExample) |
| PT (4) | PT2424991T (enExample) |
| RS (3) | RS67077B1 (enExample) |
| RU (2) | RU2603740C2 (enExample) |
| SG (3) | SG10202109219SA (enExample) |
| SI (4) | SI3988660T1 (enExample) |
| WO (1) | WO2010129021A1 (enExample) |
Families Citing this family (99)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
| US11219696B2 (en) | 2008-12-19 | 2022-01-11 | Nationwide Children's Hospital | Delivery of polynucleotides using recombinant AAV9 |
| SG10202109219SA (en) | 2009-05-02 | 2021-10-28 | Genzyme Corp | Gene therapy for neurodegenerative disorders |
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| DK2826860T3 (en) * | 2010-04-23 | 2018-12-03 | Univ Massachusetts | CNS targeting AAV vectors and methods for their use |
| EP2561075B1 (en) | 2010-04-23 | 2018-06-27 | University of Massachusetts | Aav-based treatment of cholesterol-related disorders |
| EP2640407A4 (en) * | 2010-11-16 | 2014-07-09 | Denis G Kay | PROCESS FOR INCREASING THE EXPRESSION AND ACTIVITY OF NEPRILYSIN |
| GB201103062D0 (en) * | 2011-02-22 | 2011-04-06 | Isis Innovation | Method |
| EP3777843B1 (en) | 2011-04-18 | 2023-01-04 | National Center of Neurology and Psychiatry | Drug delivery particle and method for producing the same |
| EP3318634A1 (en) | 2011-04-21 | 2018-05-09 | University of Massachusetts | Raav-based compositions and methods for treating diseases involving dominant-negative or gain of function mutations |
| US20130039888A1 (en) | 2011-06-08 | 2013-02-14 | Nationwide Children's Hospital Inc. | Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders |
| WO2013190059A1 (en) * | 2012-06-21 | 2013-12-27 | Association Institut De Myologie | Widespread gene delivery of gene therapy vectors |
| EP2879719B1 (en) | 2012-08-01 | 2018-07-04 | Nationwide Children's Hospital | Intrathecal delivery of recombinant adeno-associated virus 9 |
| CN103638604B (zh) * | 2012-12-31 | 2016-05-04 | 深圳先进技术研究院 | 一种助行系统 |
| WO2014169087A2 (en) * | 2013-04-10 | 2014-10-16 | The Trustees Of Columbia University In The City Of New York | Treatment of proximal spinal muscular atrophy |
| US10821154B2 (en) | 2013-05-01 | 2020-11-03 | Genzyme Corporation | Compositions and methods for treating spinal muscular atrophy |
| CN116004721A (zh) | 2013-07-12 | 2023-04-25 | 费城儿童医院 | Aav载体和用于抗aav（腺相关病毒）中和抗体的检测 |
| US10072251B2 (en) | 2014-02-19 | 2018-09-11 | University Of Massachusetts | Recombinant AAVS having useful transcytosis properties |
| AU2015231294B2 (en) | 2014-03-18 | 2020-10-29 | University Of Massachusetts | rAAV-based compositions and methods for treating amyotrophic lateral sclerosis |
| WO2015140799A1 (en) | 2014-03-18 | 2015-09-24 | Carmel-Haifa University Economic Corp. Ltd | Methods for improving cognitive function via modulation of quinone reductase 2 |
| EP2933335A1 (en) * | 2014-04-18 | 2015-10-21 | Genethon | A method of treating peripheral neuropathies and motor neuron diseases |
| WO2015164786A1 (en) | 2014-04-25 | 2015-10-29 | University Of Massachusetts | Recombinant aav vectors useful for reducing immunity against transgene products |
| WO2015164723A1 (en) | 2014-04-25 | 2015-10-29 | The Trustees Of The University Of Pennsylvania | Methods and compositions for treating metastatic breast cancer and other cancers in the brain |
| WO2015187825A2 (en) | 2014-06-03 | 2015-12-10 | University Of Massachusetts | Compositions and methods for modulating dysferlin expression |
| WO2016054554A1 (en) | 2014-10-03 | 2016-04-07 | University Of Massachusetts | Heterologous targeting peptide grafted aavs |
| EP3795580A1 (en) | 2014-10-03 | 2021-03-24 | University of Massachusetts | High efficiency library-identified aav vectors |
| WO2016065001A1 (en) | 2014-10-21 | 2016-04-28 | University Of Massachusetts | Recombinant aav variants and uses thereof |
| CN112553229A (zh) | 2014-11-05 | 2021-03-26 | 沃雅戈治疗公司 | 用于治疗帕金森病的aadc多核苷酸 |
| CN107249646B (zh) | 2014-12-16 | 2021-06-29 | 内布拉斯加大学董事会 | 用于青少年巴滕病的基因疗法 |
| SMT201900347T1 (it) * | 2014-12-24 | 2019-07-11 | Uniqure Ip Bv | Soppressione del gene huntingtina indotta da rnai |
| US10584321B2 (en) | 2015-02-13 | 2020-03-10 | University Of Massachusetts | Compositions and methods for transient delivery of nucleases |
| CA3021949C (en) | 2015-04-24 | 2023-10-17 | University Of Massachusetts | Modified aav constructs and uses thereof |
| CN108137664B (zh) * | 2015-08-31 | 2021-11-26 | 宾夕法尼亚州大学信托人 | 用于治疗伴侣动物的aav-epo |
| JP7064214B2 (ja) | 2015-09-28 | 2022-05-10 | ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル | 抗体回避性ウイルスベクターのための方法および組成物 |
| WO2017070525A1 (en) | 2015-10-22 | 2017-04-27 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance in neurodegenerative disease |
| US11426469B2 (en) | 2015-10-22 | 2022-08-30 | University Of Massachusetts | Prostate-targeting adeno-associated virus serotype vectors |
| EP3411059A4 (en) | 2016-02-02 | 2019-10-16 | University Of Massachusetts | METHOD FOR INCREASING THE EFFECTIVENESS OF THE SYSTEMIC DELIVERY OF AVV GENES TO THE CENTRAL NERVOUS SYSTEM |
| EP3413928B1 (en) | 2016-02-12 | 2022-04-20 | University of Massachusetts | Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization |
| EP3423109A4 (en) * | 2016-03-02 | 2019-08-14 | The Children's Hospital of Philadelphia | THERAPY FOR FRONTOTEMPORAL DEMENTIA |
| US11207426B2 (en) | 2016-04-05 | 2021-12-28 | University Of Massachusetts | Compositions and methods for selective inhibition of grainyhead-like protein expression |
| WO2017181105A1 (en) | 2016-04-15 | 2017-10-19 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance |
| EP3467497B1 (en) * | 2016-05-24 | 2021-09-29 | Tokyo Women's Medical University | Method for analyzing expression of smn protein nuclear body |
| WO2017218852A1 (en) | 2016-06-15 | 2017-12-21 | University Of Massachusetts | Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells |
| US10457940B2 (en) | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
| US11578340B2 (en) | 2016-10-13 | 2023-02-14 | University Of Massachusetts | AAV capsid designs |
| WO2018081878A1 (en) * | 2016-11-07 | 2018-05-11 | Macquarie University | Modulation of protein accumulation and uses therefor |
| JOP20190200A1 (ar) * | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| EP3622073A4 (en) | 2017-05-09 | 2021-01-06 | University of Massachusetts | METHOD FOR TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| CA3109159A1 (en) * | 2017-08-16 | 2019-02-21 | Lgv1 S.R.L. | Vtft isoform of a bpifb4 protein for use in neuronal diseases and injuries |
| JP7397488B2 (ja) | 2017-09-22 | 2023-12-13 | ユニバーシティ オブ マサチューセッツ | Ｓｏｄ１二重発現ベクターおよびその使用 |
| WO2019070891A1 (en) | 2017-10-03 | 2019-04-11 | Prevail Therapeutics, Inc. | GENE THERAPIES FOR LYSOSOMAL DISORDERS |
| KR102700963B1 (ko) | 2017-10-03 | 2024-09-02 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| IL274430B2 (en) | 2017-11-08 | 2025-01-01 | Novartis Gene Therapies Inc | Means and method for preparing viral vectors and their uses |
| CN108096243B (zh) * | 2017-11-24 | 2020-05-29 | 江苏康缘药业股份有限公司 | 银杏内酯组合物的医药用途 |
| SG11202006169PA (en) | 2017-12-29 | 2020-07-29 | Helixmith Co Ltd | Adeno-associated virus (aav) vector having hybrid hgf gene introduced thereto |
| AU2019211446B2 (en) * | 2018-01-25 | 2025-07-03 | Biogen Ma Inc. | Methods of treating spinal muscular atrophy |
| US20190256867A1 (en) | 2018-02-01 | 2019-08-22 | Homology Medicines, Inc. | Adeno-associated virus compositions for restoring pah gene function and methods of use thereof |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| KR20200108315A (ko) | 2018-02-09 | 2020-09-17 | 제넨테크, 인크. | Tmem106b의 발현을 조절하기 위한 올리고뉴클레오티드 |
| JP7425738B2 (ja) | 2018-04-03 | 2024-01-31 | ギンコ バイオワークス インコーポレイテッド | 眼組織を標的とするウイルスベクター |
| US12091435B2 (en) | 2018-04-03 | 2024-09-17 | Ginkgo Bioworks, Inc. | Antibody-evading virus vectors |
| MX2020010464A (es) | 2018-04-03 | 2021-01-29 | Vectores de virus que evitan anticuerpos. | |
| CN112334157A (zh) * | 2018-04-17 | 2021-02-05 | 应用干细胞有限公司 | 治疗脊髓肌萎缩症的组合物和方法 |
| AU2019268330A1 (en) | 2018-05-15 | 2020-11-26 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of Parkinson's disease |
| WO2019236949A1 (en) * | 2018-06-08 | 2019-12-12 | Avexis Inc. | Cell-based assay for measuring drug product potency |
| US12163129B2 (en) | 2018-06-08 | 2024-12-10 | University Of Massachusetts | Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells |
| CN108795946B (zh) * | 2018-06-28 | 2022-03-04 | 北京锦篮基因科技有限公司 | 携带设计smn1基因表达框的重组腺相关病毒及应用 |
| EP3856762A1 (en) | 2018-09-28 | 2021-08-04 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| CN109266682B (zh) * | 2018-09-29 | 2022-04-01 | 中国科学院武汉物理与数学研究所 | 一种神经细胞快速逆行跨突触标记的方法及应用 |
| MA54383A (fr) | 2018-12-06 | 2021-10-13 | Biogen Ma Inc | Protéine neurofilamenteuse pour guider une intervention thérapeutique dans la sclérose laterale amyotrophique |
| RU2731514C2 (ru) * | 2018-12-21 | 2020-09-03 | Селл энд Джин Терапи Лтд | Генотерапевтический ДНК-вектор на основе генотерапевтического ДНК-вектора VTvaf17, несущий целевой ген, выбранный из группы генов SHH, CTNNB1, NOG, WNT7A для повышения уровня экспрессии этих целевых генов, способ его получения и применения, штамм Escherichia coli SCS110-AF/VTvaf17-SHH, или Escherichia coli SCS110-AF/VTvaf17-CTNNB1, или Escherichia coli SCS110-AF/VTvaf17-NOG, или Escherichia coli SCS110-AF/VTvaf17-WNT7A, несущий генотерапевтический ДНК-вектор, способ его получения, способ производства в промышленных масштабах генотерапевтического ДНК-вектора |
| US20220111005A1 (en) * | 2019-02-01 | 2022-04-14 | Avrobio, Inc. | Compositions and methods for treating neurocognitive disorders |
| BR112021015817A2 (pt) | 2019-02-22 | 2021-10-13 | The Trustees Of The University Of Pennsylvania | Vírus adeno-associado recombinante para tratamento de neurodegeneração com início na idade adulta associado a grn |
| JP7698583B2 (ja) | 2019-03-21 | 2025-06-25 | ギンコ バイオワークス インコーポレイテッド | 組換えアデノ随伴ウイルスベクター |
| SG11202110607WA (en) | 2019-04-01 | 2021-10-28 | Tenaya Therapeutics Inc | Adeno-associated virus with engineered capsid |
| US11999974B2 (en) | 2019-04-10 | 2024-06-04 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| JP2022526425A (ja) * | 2019-04-12 | 2022-05-24 | エンコーデッド セラピューティクス， インコーポレイテッド | 治療剤の投与のための組成物および方法 |
| IL292297A (en) | 2019-10-17 | 2022-06-01 | Stridebio Inc | Adeno-associated viral vectors for treatment of niemann-pick disease type c |
| IL292382A (en) * | 2019-10-22 | 2022-06-01 | Applied Genetic Tech Corporation | Adeno-associated virus (aav) systems for treatment of progranulin associated neurodegenerative diseases or disorders |
| WO2021119622A1 (en) * | 2019-12-12 | 2021-06-17 | Northwestern University | Modulation of ubiquitin carboxy-terminal hydrolase ligase 1 expression |
| 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 |
| WO2021216503A1 (en) | 2020-04-20 | 2021-10-28 | Christiana Care Health Services, Inc. | Aav delivery system for lung cancer treatment |
| 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 |
| CA3189878A1 (en) | 2020-08-19 | 2022-02-24 | Colin O'BANION | Adeno-associated virus vectors for treatment of rett syndrome |
| CN112121179A (zh) * | 2020-09-15 | 2020-12-25 | 山东兴瑞生物科技有限公司 | 一种组合物及其在治疗脊髓性肌萎缩症中的应用 |
| CN115698304A (zh) | 2020-10-09 | 2023-02-03 | 特纳亚治疗股份有限公司 | 亲斑蛋白-2基因疗法的方法和组合物 |
| TW202246501A (zh) * | 2021-01-29 | 2022-12-01 | 俄羅斯聯邦商亞那拜恩有限公司 | ＳＭＮ１和ｍｉＲ－２３ａ在治療脊髓性肌萎縮中的協同作用 |
| CN116179605B (zh) * | 2021-08-12 | 2025-11-11 | 蓝图生物医药(广州)有限公司 | 一种重组腺相关病毒载体及其应用 |
| US20250144210A1 (en) * | 2022-02-01 | 2025-05-08 | Seattle Children’s Hospital d/b/a Seattle Children’s Research Institute | Simplified method of preparing cells for patient administration |
| EP4504951A2 (en) * | 2022-04-05 | 2025-02-12 | The Johns Hopkins University | Methods and materials for treating syngap1-associated neurodevelopmental disorders |
| EP4508062A1 (en) | 2022-04-11 | 2025-02-19 | Tenaya Therapeutics, Inc. | Adeno-associated virus with engineered capsid |
| WO2023219394A1 (ko) * | 2022-05-10 | 2023-11-16 | 서울대학교산학협력단 | 인간 smn1 단백질 변이체 및 이의 용도 |
| CN114903010B (zh) * | 2022-05-19 | 2024-06-07 | 暨南大学 | 神经退行性疾病模型的构建方法 |
| WO2023240236A1 (en) | 2022-06-10 | 2023-12-14 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of spinal muscular atrophy related disorders |
| CN116042719B (zh) * | 2022-12-28 | 2025-11-11 | 蓝图生物医药(广州)有限公司 | 一种重组腺相关病毒载体及其应用 |
| WO2024215653A1 (en) | 2023-04-10 | 2024-10-17 | Tenaya Therapeutics, Inc. | Guide rnas, vectors, and virions for targeting mutations in the pln gene |
| WO2024215655A1 (en) | 2023-04-10 | 2024-10-17 | Tenaya Therapeutics, Inc. | Cardioprotective bag3 therapies |
| CN118045206B (zh) * | 2024-04-12 | 2024-07-05 | 四川至善唯新生物科技有限公司 | 一种治疗脊髓型肌肉萎缩的药物组合物及其用途 |
Family Cites Families (91)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| NZ210501A (en) | 1983-12-13 | 1991-08-27 | Kirin Amgen Inc | Erythropoietin produced by procaryotic or eucaryotic expression of an exogenous dna sequence |
| US4703008A (en) | 1983-12-13 | 1987-10-27 | Kiren-Amgen, Inc. | DNA sequences encoding erythropoietin |
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| JPS62171696A (ja) | 1986-01-23 | 1987-07-28 | Sumitomo Chem Co Ltd | ヒトエリスロポエチンの製造方法 |
| US4954437A (en) | 1986-09-15 | 1990-09-04 | Integrated Genetics, Inc. | Cell encoding recombinant human erythropoietin |
| US5328470A (en) | 1989-03-31 | 1994-07-12 | The Regents Of The University Of Michigan | Treatment of diseases by site-specific instillation of cells or site-specific transformation of cells and kits therefor |
| US5399346A (en) | 1989-06-14 | 1995-03-21 | The United States Of America As Represented By The Department Of Health And Human Services | Gene therapy |
| US5605690A (en) | 1989-09-05 | 1997-02-25 | Immunex Corporation | Methods of lowering active TNF-α levels in mammals using tumor necrosis factor receptor |
| US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
| WO1992001070A1 (en) | 1990-07-09 | 1992-01-23 | The United States Of America, As Represented By The Secretary, U.S. Department Of Commerce | High efficiency packaging of mutant adeno-associated virus using amber suppressions |
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| US5747469A (en) | 1991-03-06 | 1998-05-05 | Board Of Regents, The University Of Texas System | Methods and compositions comprising DNA damaging agents and p53 |
| AU663725B2 (en) | 1991-08-20 | 1995-10-19 | United States Of America, Represented By The Secretary, Department Of Health And Human Services, The | Adenovirus mediated transfer of genes to the gastrointestinal tract |
| US5587308A (en) | 1992-06-02 | 1996-12-24 | The United States Of America As Represented By The Department Of Health & Human Services | Modified adeno-associated virus vector capable of expression from a novel promoter |
| US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US6686200B1 (en) | 1993-08-31 | 2004-02-03 | Uab Research Foundation | Methods and compositions for the large scale production of recombinant adeno-associated virus |
| DE69433592T2 (de) | 1993-11-09 | 2005-02-10 | Targeted Genetics Corp., Seattle | Die erzielung hoher titer des rekombinanten aav-vektors |
| WO1995013392A1 (en) | 1993-11-09 | 1995-05-18 | Medical College Of Ohio | Stable cell lines capable of expressing the adeno-associated virus replication gene |
| WO1995028493A1 (en) | 1994-04-13 | 1995-10-26 | The Rockefeller University | Aav-mediated delivery of dna to cells of the nervous system |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| EP0708178A1 (en) | 1994-10-19 | 1996-04-24 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Survival motor neuron (SMN) gene: a gene for spinal muscular atrophy |
| EP0796339A1 (en) | 1994-12-06 | 1997-09-24 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant aav vectors |
| US5756283A (en) | 1995-06-05 | 1998-05-26 | The Trustees Of The University Of Pennsylvania | Method for improved production of recombinant adeno-associated viruses for gene therapy |
| US6281010B1 (en) | 1995-06-05 | 2001-08-28 | The Trustees Of The University Of Pennsylvania | Adenovirus gene therapy vehicle and cell line |
| US5882914A (en) | 1995-06-06 | 1999-03-16 | The Johns Hopkins University School Of Medicine | Nucleic acids encoding the hypoxia inducible factor-1 |
| US5677158A (en) | 1995-06-07 | 1997-10-14 | Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
| US5622856A (en) | 1995-08-03 | 1997-04-22 | Avigen | High efficiency helper system for AAV vector production |
| US6001650A (en) | 1995-08-03 | 1999-12-14 | Avigen, Inc. | High-efficiency wild-type-free AAV helper functions |
| US6004797A (en) | 1995-11-09 | 1999-12-21 | Avigen, Inc. | Adenovirus helper-free recombinant AAV Virion production |
| AU2064297A (en) | 1996-02-29 | 1997-09-16 | Immusol, Inc | Hepatitis c virus ribozymes |
| JP2002514899A (ja) | 1996-03-04 | 2002-05-21 | ターゲティッド ジェネティックス コーポレイション | 組換えａａｖベクターを用いて血管中の細胞を形質導入するための方法 |
| US20040076613A1 (en) * | 2000-11-03 | 2004-04-22 | Nicholas Mazarakis | Vector system |
| US6261823B1 (en) | 1996-12-13 | 2001-07-17 | Schering Corporation | Methods for purifying viruses |
| WO1998027204A2 (en) | 1996-12-18 | 1998-06-25 | Targeted Genetics Corporation | Aav split-packaging genes and cell lines comprising such genes for use in the production of recombinant aav vectors |
| AU5603998A (en) | 1996-12-18 | 1998-07-15 | Targeted Genetics Corporation | Recombinase-activatable AAV packaging cassettes for use in the production of AV vectors |
| AU5927598A (en) | 1997-01-23 | 1998-08-18 | Immusol Incorporated | Gene functional analysis and discovery using randomized or target-specific ribozyme gene vector libraries |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6989264B2 (en) | 1997-09-05 | 2006-01-24 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| WO1999011764A2 (en) | 1997-09-05 | 1999-03-11 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of recombinant aav vectors |
| WO1999020779A1 (en) | 1997-10-21 | 1999-04-29 | Targeted Genetics Corporation | Amplifiable adeno-associated virus (aav) packaging cassettes for the production of recombinant aav vectors |
| ATE435293T1 (de) | 1997-10-21 | 2009-07-15 | Targeted Genetics Corp | Invertierte terminale repeats von aav, die für die transkription aktiviert sind, zur verwendung in rekombinanten aav-vektoren |
| IL136454A0 (en) | 1997-12-04 | 2001-06-14 | Genzyme Corp | Compositions and methods for inducing gene expression |
| DK1054955T3 (da) | 1998-02-17 | 2007-01-15 | Schering Corp | Sammensætninger omfattende vira og fremgangsmåde til at opkoncentrere viruspræparater |
| AUPP249298A0 (en) | 1998-03-20 | 1998-04-23 | Ag-Gene Australia Limited | Synthetic genes and genetic constructs comprising same I |
| DE69922934T2 (de) | 1998-03-20 | 2005-12-08 | The Trustees Of The University Of Pennsylvania | Zusammensetzungen und Verfahren zur helfer-freien Herstellung von rekombinanten Adeno-assoziierten Viren |
| PT1080202E (pt) | 1998-05-27 | 2006-05-31 | Avigen Inc | Distribuicao de vectores aav codificando aadc intensificada por conveccao |
| EP1080218A1 (en) | 1998-05-27 | 2001-03-07 | University of Florida | Method of preparing recombinant adeno-associated virus compositions by using an iodixanol gradient |
| EP1109892B1 (en) | 1998-09-04 | 2008-11-12 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant aav vectors |
| US6646113B1 (en) * | 1998-09-17 | 2003-11-11 | The Trustees Of The University Of Pennsylvania | Nucleic acid molecule encoding human survival of motor neuron-interacting protein 1 (SIP1) deletion mutants |
| US6759050B1 (en) | 1998-12-03 | 2004-07-06 | Avigen, Inc. | Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith |
| US6893865B1 (en) | 1999-04-28 | 2005-05-17 | Targeted Genetics Corporation | Methods, compositions, and cells for encapsidating recombinant vectors in AAV particles |
| JP2003501043A (ja) | 1999-05-28 | 2003-01-14 | ターゲティッド ジェネティクス コーポレイション | 腫瘍壊死因子（ｔｎｆ）関連障害において、ｔｎｆのレベルを低下させるための方法および組成物 |
| ATE403715T1 (de) | 1999-08-09 | 2008-08-15 | Targeted Genetics Corp | Erhöhung der expression einer einzelsträngigen, heterologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen |
| US20020099025A1 (en) * | 1999-12-30 | 2002-07-25 | Heywood James A. | Treatment of neurological disorders |
| CA2799545A1 (en) | 2000-03-07 | 2001-09-13 | Merck Sharp & Dohme Corp. | Adenovirus formulations |
| US7125705B2 (en) | 2000-04-28 | 2006-10-24 | Genzyme Corporation | Polynucleotides for use in recombinant adeno-associated virus virion production |
| NZ522840A (en) | 2000-06-01 | 2004-12-24 | Univ North Carolina | A parvovirus vector that carries a duplexed genome resulting in co-packaging of strands of plus and minus polarity tethered together |
| US6593123B1 (en) | 2000-08-07 | 2003-07-15 | Avigen, Inc. | Large-scale recombinant adeno-associated virus (rAAV) production and purification |
| DE60115070T2 (de) | 2000-09-18 | 2006-07-27 | Genzyme Corp., Cambridge | Expressionsvektoren mit hybriden ubiquitin-promotoren |
| WO2003004660A1 (en) | 2001-07-03 | 2003-01-16 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Methods of administering vectors to synaptically connected neurons |
| US20030050273A1 (en) * | 2001-08-29 | 2003-03-13 | Keiya Ozawa | Compositions and methods for treating neurodegenerative diseases |
| AU2002348151A1 (en) | 2001-11-05 | 2003-05-19 | Genvec, Inc. | Viral vector production methods and compositions |
| AU2002360429A1 (en) | 2001-11-26 | 2003-06-10 | Avigen, Inc. | Methods for producing stocks of recombinant aav virions |
| PT2573170T (pt) | 2001-12-17 | 2018-03-26 | Univ Pennsylvania | Sequências de um vírus adenoassociado (aav) de serotipo 9, vetor contendo as mesmas, e suas utilizações |
| WO2003089612A2 (en) * | 2002-04-17 | 2003-10-30 | University Of Florida Research Foundation, Inc. | IMPROVED rAAV VECTORS |
| PL1620133T3 (pl) | 2003-05-01 | 2016-05-31 | Genzyme Corp | Terapia genowa dla zaburzeń neurometabolicznych |
| US7765583B2 (en) | 2005-02-28 | 2010-07-27 | France Telecom | System and method for managing virtual user domains |
| BRPI0611540A2 (pt) * | 2005-05-02 | 2010-09-21 | Genzyme Corp | terapia de gene para distúrbios de espinha dorsal |
| PT2420256T (pt) | 2005-05-02 | 2016-09-13 | Genzyme Corp | Terapia genética para distúrbios neurometabólicos |
| AR059089A1 (es) | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal |
| WO2008016391A2 (en) | 2006-01-31 | 2008-02-07 | The Board Of Trustees Of The Leland Stanford Junior University | Self-complementary parvoviral vectors, and methods for making and using the same |
| WO2007120533A2 (en) | 2006-03-30 | 2007-10-25 | The Board Of Trustees Of The Leland Stanford Junior University | Minigene expression cassette |
| BRPI0711965A2 (pt) | 2006-06-07 | 2012-01-24 | Genzyme Corporation | terapia de gene para esclerose lateral amiotrófica e outras enfermidades da medula espinal |
| DK2066791T3 (da) | 2006-10-03 | 2012-10-15 | Genzyme Corp | Genterapi for amyotrofisk lateralsklerose og andre rygmarvssygdomme |
| ITMI20070127A1 (it) | 2007-01-29 | 2008-07-30 | Fond I R C C S | Proteine e-o peptidi per la prevenzione e-o cura di malattie neurodegenerative |
| US20080187512A1 (en) * | 2007-02-07 | 2008-08-07 | Academia Sinica | Treatment for spinal muscular atrophy |
| US8299215B2 (en) | 2007-07-14 | 2012-10-30 | University Of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| EP2019143A1 (en) * | 2007-07-23 | 2009-01-28 | Genethon | CNS gene delivery using peripheral administration of AAV vectors |
| EP2058401A1 (en) * | 2007-10-05 | 2009-05-13 | Genethon | Widespread gene delivery to motor neurons using peripheral injection of AAV vectors |
| US8633019B2 (en) | 2008-05-27 | 2014-01-21 | Ptc Therapeutics, Inc. | Methods for treating spinal muscular atrophy |
| WO2010071832A1 (en) | 2008-12-19 | 2010-06-24 | Nationwide Children's Hospital | Delivery of polynucleotides across the blood brain barrier using recombinant aav9 |
| US9415121B2 (en) | 2008-12-19 | 2016-08-16 | Nationwide Children's Hospital | Delivery of MECP2 polynucleotide using recombinant AAV9 |
| US11219696B2 (en) | 2008-12-19 | 2022-01-11 | Nationwide Children's Hospital | Delivery of polynucleotides using recombinant AAV9 |
| SG10202109219SA (en) * | 2009-05-02 | 2021-10-28 | Genzyme Corp | Gene therapy for neurodegenerative disorders |
| EP2879719B1 (en) | 2012-08-01 | 2018-07-04 | Nationwide Children's Hospital | Intrathecal delivery of recombinant adeno-associated virus 9 |
| FR3002237B1 (fr) | 2013-02-15 | 2017-12-15 | Genethon | Methodes pour la production de particules virales aav double brin |
| US10821154B2 (en) | 2013-05-01 | 2020-11-03 | Genzyme Corporation | Compositions and methods for treating spinal muscular atrophy |
| WO2015168666A2 (en) | 2014-05-02 | 2015-11-05 | Genzyme Corporation | Aav vectors for retinal and cns gene therapy |
| LT3411484T (lt) | 2016-02-05 | 2023-11-27 | Emory University | Viengrandžio arba sau pačiam komplementaraus adeno-asocijuoto viruso 9 injekcija į smegenų skystį |
-
2010
- 2010-04-27 SG SG10202109219S patent/SG10202109219SA/en unknown
- 2010-04-27 MX MX2011011594A patent/MX341073B/es active IP Right Grant
- 2010-04-27 PL PL24154262.0T patent/PL4342992T3/pl unknown
- 2010-04-27 CN CN201080027833.7A patent/CN102459611B/zh active Active
- 2010-04-27 PT PT10772363T patent/PT2424991T/pt unknown
- 2010-04-27 MX MX2016010101A patent/MX356669B/es unknown
- 2010-04-27 HU HUE24154262A patent/HUE072183T2/hu unknown
- 2010-04-27 ES ES18175397T patent/ES2903127T3/es active Active
- 2010-04-27 FI FIEP24154262.0T patent/FI4342992T3/fi active
- 2010-04-27 HR HRP20250897TT patent/HRP20250897T1/hr unknown
- 2010-04-27 WO PCT/US2010/001239 patent/WO2010129021A1/en not_active Ceased
- 2010-04-27 EP EP24154262.0A patent/EP4342992B1/en active Active
- 2010-04-27 KR KR1020117028766A patent/KR101835490B1/ko active Active
- 2010-04-27 DK DK18175397.1T patent/DK3421603T3/da active
- 2010-04-27 LT LTEP21200213.3T patent/LT3988660T/lt unknown
- 2010-04-27 DK DK24154262.0T patent/DK4342992T3/da active
- 2010-04-27 KR KR1020187005929A patent/KR20180027619A/ko not_active Ceased
- 2010-04-27 EP EP25166816.6A patent/EP4585263A3/en active Pending
- 2010-04-27 EP EP18175397.1A patent/EP3421603B1/en active Active
- 2010-04-27 HU HUE10772363A patent/HUE039345T2/hu unknown
- 2010-04-27 SI SI201032158T patent/SI3988660T1/sl unknown
- 2010-04-27 CN CN201610906309.6A patent/CN107083400A/zh active Pending
- 2010-04-27 ES ES10772363.7T patent/ES2686504T3/es active Active
- 2010-04-27 EP EP10772363.7A patent/EP2424991B1/en active Active
- 2010-04-27 SI SI201031748T patent/SI2424991T1/sl unknown
- 2010-04-27 HU HUE21200213A patent/HUE072713T2/hu unknown
- 2010-04-27 JP JP2012508474A patent/JP5879256B2/ja active Active
- 2010-04-27 LT LTEP18175397.1T patent/LT3421603T/lt unknown
- 2010-04-27 ES ES24154262T patent/ES3041882T3/es active Active
- 2010-04-27 DK DK10772363.7T patent/DK2424991T3/en active
- 2010-04-27 RS RS20250772A patent/RS67077B1/sr unknown
- 2010-04-27 PL PL18175397T patent/PL3421603T3/pl unknown
- 2010-04-27 LT LTEP10772363.7T patent/LT2424991T/lt unknown
- 2010-04-27 EP EP21200213.3A patent/EP3988660B1/en active Active
- 2010-04-27 SI SI201032101T patent/SI3421603T1/sl unknown
- 2010-04-27 DK DK21200213.3T patent/DK3988660T3/da active
- 2010-04-27 PT PT241542620T patent/PT4342992T/pt unknown
- 2010-04-27 RS RS20220002A patent/RS62779B1/sr unknown
- 2010-04-27 PT PT212002133T patent/PT3988660T/pt unknown
- 2010-04-27 HR HRP20250892TT patent/HRP20250892T1/hr unknown
- 2010-04-27 SI SI201032157T patent/SI4342992T1/sl unknown
- 2010-04-27 RS RS20250773A patent/RS67078B1/sr unknown
- 2010-04-27 PL PL10772363T patent/PL2424991T3/pl unknown
- 2010-04-27 CA CA2759801A patent/CA2759801C/en active Active
- 2010-04-27 ES ES21200213T patent/ES3038109T3/es active Active
- 2010-04-27 RU RU2011149094/10A patent/RU2603740C2/ru not_active IP Right Cessation
- 2010-04-27 RU RU2016140850A patent/RU2743398C2/ru active
- 2010-04-27 HU HUE18175397A patent/HUE057606T2/hu unknown
- 2010-04-27 PL PL21200213.3T patent/PL3988660T3/pl unknown
- 2010-04-27 SG SG10201404528SA patent/SG10201404528SA/en unknown
- 2010-04-27 FI FIEP21200213.3T patent/FI3988660T3/fi active
- 2010-04-27 HR HRP20212024TT patent/HRP20212024T1/hr unknown
- 2010-04-27 PT PT181753971T patent/PT3421603T/pt unknown
- 2010-04-27 LT LTEP24154262.0T patent/LT4342992T/lt unknown
- 2010-04-27 HR HRP20181423TT patent/HRP20181423T1/hr unknown
- 2010-04-27 SG SG2011079837A patent/SG175409A1/en unknown
-
2011
- 2011-11-01 MX MX2018006955A patent/MX2018006955A/es unknown
- 2011-11-01 IL IL216094A patent/IL216094A0/en not_active IP Right Cessation
- 2011-11-01 MX MX2023006169A patent/MX2023006169A/es unknown
- 2011-11-02 US US13/287,583 patent/US20130287736A1/en not_active Abandoned
-
2015
- 2015-10-01 JP JP2015195602A patent/JP6397391B2/ja active Active
- 2015-10-06 US US14/876,683 patent/US9415119B2/en active Active
-
2016
- 2016-05-20 US US15/160,949 patent/US10369193B2/en active Active
-
2018
- 2018-06-07 JP JP2018109162A patent/JP2018148927A/ja active Pending
- 2018-06-07 IL IL259898A patent/IL259898A/en unknown
- 2018-09-06 CY CY181100934T patent/CY1120982T1/el unknown
-
2020
- 2020-02-18 US US16/794,031 patent/US20200384076A1/en not_active Abandoned
-
2022
- 2022-10-13 US US18/046,363 patent/US11975043B2/en active Active
- 2022-11-10 US US18/054,500 patent/US11911440B2/en active Active
-
2023
- 2023-10-17 US US18/488,346 patent/US20240033323A1/en not_active Abandoned
-
2024
- 2024-03-26 US US18/616,511 patent/US12350311B2/en active Active
-
2025
- 2025-06-04 US US19/228,044 patent/US20250288644A1/en active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2018148927A5 (enExample) | ||
| JP2022068181A5 (enExample) | ||
| RU2018116076A (ru) | Модифицированные гены атаксии фридрейха и векторы для генной терапии | |
| HRP20250897T1 (hr) | Genska terapija za neurodegenerativne poremećaje | |
| JP2020522269A5 (enExample) | ||
| JP5704361B2 (ja) | 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン | |
| RU2017125234A (ru) | Конструкции нуклеиновой кислоты и векторы для генотерапии для применения для лечения болезни вильсона и других состояний | |
| JP2009526067A5 (enExample) | ||
| IL273261B2 (en) | Adeno-associated virus variant capsids and methods of using them | |
| JP2020528734A5 (enExample) | ||
| IL320714A (en) | Gene therapy for limb-girdle muscular dystrophy type 2c | |
| JP2018531609A5 (enExample) | ||
| JP2021003120A5 (enExample) | ||
| JP2019511570A5 (enExample) | ||
| JP2017518271A5 (enExample) | ||
| RU2017115477A (ru) | Полинуклеотиды aadc для лечения болезни паркинсона | |
| EP2660325A3 (en) | AAV vectors and corresponding nucleotide sequences and methods | |
| JP2017529395A5 (enExample) | ||
| HRP20241483T1 (hr) | Sastavi i postupci za liječenje poremećaja mrežnice | |
| JPWO2020214609A5 (enExample) | ||
| JP2020510433A5 (enExample) | ||
| JPWO2021053222A5 (enExample) | ||
| JPWO2021050614A5 (enExample) | ||
| FI3177313T3 (fi) | Telomeraasi-käänteistranskriptaasipohjaisia hoitoja sydäninfarktiin liittyvien tilojen hoitamiseksi | |
| JP2023059858A5 (enExample) |