JP2015501156A5 - - Google Patents
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- JP2015501156A5 JP2015501156A5 JP2014539038A JP2014539038A JP2015501156A5 JP 2015501156 A5 JP2015501156 A5 JP 2015501156A5 JP 2014539038 A JP2014539038 A JP 2014539038A JP 2014539038 A JP2014539038 A JP 2014539038A JP 2015501156 A5 JP2015501156 A5 JP 2015501156A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- cells
- composition
- viral vector
- rdcvf
- 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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- 108020004707 nucleic acids Proteins 0.000 claims description 75
- 102000039446 nucleic acids Human genes 0.000 claims description 75
- 150000007523 nucleic acids Chemical class 0.000 claims description 75
- 210000004027 cell Anatomy 0.000 claims description 68
- 101000597433 Mus musculus Nucleoredoxin-like protein 1 Proteins 0.000 claims description 48
- 239000013603 viral vector Substances 0.000 claims description 44
- 239000002773 nucleotide Substances 0.000 claims description 38
- 125000003729 nucleotide group Chemical group 0.000 claims description 38
- 238000000034 method Methods 0.000 claims description 30
- 239000007924 injection Substances 0.000 claims description 16
- 238000002347 injection Methods 0.000 claims description 16
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 15
- 201000010099 disease Diseases 0.000 claims description 13
- 108091026890 Coding region Proteins 0.000 claims description 12
- 241000124008 Mammalia Species 0.000 claims description 12
- 108010076504 Protein Sorting Signals Proteins 0.000 claims description 10
- 102000004169 proteins and genes Human genes 0.000 claims description 7
- 108090000623 proteins and genes Proteins 0.000 claims description 7
- 239000013598 vector Substances 0.000 claims description 7
- 206010003694 Atrophy Diseases 0.000 claims description 6
- 241000701022 Cytomegalovirus Species 0.000 claims description 6
- 150000001413 amino acids Chemical group 0.000 claims description 6
- 230000037444 atrophy Effects 0.000 claims description 6
- 208000011325 dry age related macular degeneration Diseases 0.000 claims description 6
- 208000011580 syndromic disease Diseases 0.000 claims description 6
- 239000000825 pharmaceutical preparation Substances 0.000 claims description 5
- 230000028327 secretion Effects 0.000 claims description 5
- 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 description 4
- 239000013607 AAV vector Substances 0.000 claims description 4
- 102100026031 Beta-glucuronidase Human genes 0.000 claims description 4
- 102000004219 Brain-derived neurotrophic factor Human genes 0.000 claims description 4
- 108090000715 Brain-derived neurotrophic factor Proteins 0.000 claims description 4
- 108020004705 Codon Proteins 0.000 claims description 4
- 101000599951 Homo sapiens Insulin-like growth factor I Proteins 0.000 claims description 4
- 102000002265 Human Growth Hormone Human genes 0.000 claims description 4
- 108010000521 Human Growth Hormone Proteins 0.000 claims description 4
- 239000000854 Human Growth Hormone Substances 0.000 claims description 4
- 102100037852 Insulin-like growth factor I Human genes 0.000 claims description 4
- 108020005067 RNA Splice Sites Proteins 0.000 claims description 4
- 229940077737 brain-derived neurotrophic factor Drugs 0.000 claims description 4
- 210000003583 retinal pigment epithelium Anatomy 0.000 claims description 4
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 2
- 208000024827 Alzheimer disease Diseases 0.000 claims description 2
- 208000023328 Basedow disease Diseases 0.000 claims description 2
- 241000713704 Bovine immunodeficiency virus Species 0.000 claims description 2
- 208000002177 Cataract Diseases 0.000 claims description 2
- 206010007747 Cataract congenital Diseases 0.000 claims description 2
- 108020004414 DNA Proteins 0.000 claims description 2
- 241000702421 Dependoparvovirus Species 0.000 claims description 2
- 206010012689 Diabetic retinopathy Diseases 0.000 claims description 2
- 208000003098 Ganglion Cysts Diseases 0.000 claims description 2
- 208000010412 Glaucoma Diseases 0.000 claims description 2
- 108010060309 Glucuronidase Proteins 0.000 claims description 2
- 208000015023 Graves' disease Diseases 0.000 claims description 2
- 102100021519 Hemoglobin subunit beta Human genes 0.000 claims description 2
- 108091005904 Hemoglobin subunit beta Proteins 0.000 claims description 2
- 101000933465 Homo sapiens Beta-glucuronidase Proteins 0.000 claims description 2
- 101000597428 Homo sapiens Nucleoredoxin-like protein 1 Proteins 0.000 claims description 2
- 208000023105 Huntington disease Diseases 0.000 claims description 2
- 208000032578 Inherited retinal disease Diseases 0.000 claims description 2
- FFEARJCKVFRZRR-BYPYZUCNSA-N L-methionine Chemical compound CSCC[C@H](N)C(O)=O FFEARJCKVFRZRR-BYPYZUCNSA-N 0.000 claims description 2
- 102100036205 Nucleoredoxin-like protein 2 Human genes 0.000 claims description 2
- 101710106007 Nucleoredoxin-like protein 2 Proteins 0.000 claims description 2
- 206010030043 Ocular hypertension Diseases 0.000 claims description 2
- 208000018737 Parkinson disease Diseases 0.000 claims description 2
- 208000032430 Retinal dystrophy Diseases 0.000 claims description 2
- 208000007014 Retinitis pigmentosa Diseases 0.000 claims description 2
- 208000027073 Stargardt disease Diseases 0.000 claims description 2
- 208000005400 Synovial Cyst Diseases 0.000 claims description 2
- 208000014769 Usher Syndromes Diseases 0.000 claims description 2
- 206010046851 Uveitis Diseases 0.000 claims description 2
- 208000000208 Wet Macular Degeneration Diseases 0.000 claims description 2
- 206010064930 age-related macular degeneration Diseases 0.000 claims description 2
- 210000002159 anterior chamber Anatomy 0.000 claims description 2
- 238000004113 cell culture Methods 0.000 claims description 2
- 210000004978 chinese hamster ovary cell Anatomy 0.000 claims description 2
- 239000012228 culture supernatant Substances 0.000 claims description 2
- 238000012258 culturing Methods 0.000 claims description 2
- 208000035475 disorder Diseases 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 208000030533 eye disease Diseases 0.000 claims description 2
- 201000006321 fundus dystrophy Diseases 0.000 claims description 2
- 210000002287 horizontal cell Anatomy 0.000 claims description 2
- 102000057445 human NXNL1 Human genes 0.000 claims description 2
- 210000005260 human cell Anatomy 0.000 claims description 2
- 238000000338 in vitro Methods 0.000 claims description 2
- 238000001727 in vivo Methods 0.000 claims description 2
- 208000017532 inherited retinal dystrophy Diseases 0.000 claims description 2
- 230000002401 inhibitory effect Effects 0.000 claims description 2
- 208000029515 lens disease Diseases 0.000 claims description 2
- 210000004962 mammalian cell Anatomy 0.000 claims description 2
- 238000004519 manufacturing process Methods 0.000 claims description 2
- 229930182817 methionine Natural products 0.000 claims description 2
- 238000000746 purification Methods 0.000 claims description 2
- 210000000844 retinal pigment epithelial cell Anatomy 0.000 claims description 2
- 210000001685 thyroid gland Anatomy 0.000 claims description 2
- 210000003501 vero cell Anatomy 0.000 claims description 2
- 238000009736 wetting Methods 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 29
- 230000003376 axonal effect Effects 0.000 claims 1
- 230000008786 sensory perception of smell Effects 0.000 claims 1
- 241000700605 Viruses Species 0.000 description 1
- 210000003050 axon Anatomy 0.000 description 1
- 230000000977 initiatory effect Effects 0.000 description 1
- 239000008194 pharmaceutical composition Substances 0.000 description 1
- 230000003248 secreting effect Effects 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201161552155P | 2011-10-27 | 2011-10-27 | |
| US61/552,155 | 2011-10-27 | ||
| PCT/US2012/062106 WO2013063383A2 (en) | 2011-10-27 | 2012-10-26 | Vectors encoding rod-derived cone viability factor |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2015501156A JP2015501156A (ja) | 2015-01-15 |
| JP2015501156A5 true JP2015501156A5 (enExample) | 2015-12-17 |
| JP6293664B2 JP6293664B2 (ja) | 2018-03-14 |
Family
ID=48168788
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2014539038A Expired - Fee Related JP6293664B2 (ja) | 2011-10-27 | 2012-10-26 | 桿体由来錐体生存因子をコードするベクター |
Country Status (14)
| Country | Link |
|---|---|
| US (2) | US9265813B2 (enExample) |
| EP (1) | EP2797613B1 (enExample) |
| JP (1) | JP6293664B2 (enExample) |
| KR (1) | KR102000141B1 (enExample) |
| CN (2) | CN104321069A (enExample) |
| AU (1) | AU2012321102C1 (enExample) |
| BR (1) | BR112014010091B1 (enExample) |
| CA (1) | CA2853379C (enExample) |
| DK (1) | DK2797613T3 (enExample) |
| ES (1) | ES2774779T3 (enExample) |
| MX (1) | MX354516B (enExample) |
| PL (1) | PL2797613T3 (enExample) |
| RU (1) | RU2664673C2 (enExample) |
| WO (1) | WO2013063383A2 (enExample) |
Families Citing this family (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2027889A1 (en) * | 2007-06-05 | 2009-02-25 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | New neuronal viability factor and use thereof |
| WO2014060517A1 (en) * | 2012-10-17 | 2014-04-24 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods and pharmaceutical compositions for the treatment of age-related macular degeneration (amd) |
| DK3546585T3 (da) * | 2014-04-25 | 2025-11-03 | Genethon | Nukleinsyrer med en modificeret intron til brug i behandlingen af hyperbilirubinæmi |
| WO2016185242A1 (en) | 2015-05-21 | 2016-11-24 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Synergistic combination of neuronal viability factors and uses thereof |
| GB201516066D0 (en) * | 2015-09-10 | 2015-10-28 | Young & Co Llp D | Treatment of retinitis pigmentosa |
| US11241506B2 (en) | 2015-12-14 | 2022-02-08 | The Trustees Of The University Of Pennsylvania | Composition for treatment of Crigler-Najjar syndrome |
| WO2017120294A1 (en) * | 2016-01-05 | 2017-07-13 | The Trustees Of The University Of Pennsylvania | Methods and compositions for treatment of ocular disorders and blinding diseases |
| CN106148528B (zh) * | 2016-07-11 | 2019-10-01 | 赵晨 | 一种遗传性Usher综合征的致病突变及其检测试剂 |
| US10946063B2 (en) * | 2016-10-11 | 2021-03-16 | Welltat Ophthalmics Corporation | Fusion protein between short form rod-derived cone viability factor and a hydrophilic peptide |
| CN111601884A (zh) * | 2017-10-16 | 2020-08-28 | 维格内罗有限责任公司 | Aav载体 |
| SG11202006796RA (en) * | 2017-12-22 | 2020-08-28 | Sparingvision | Constructs comprising neuronal viability factors and uses thereof |
| ES2917628T3 (es) * | 2018-01-30 | 2022-07-11 | Univ Gent | Composiciones a utilizar para tratar cataratas |
| CN108504685A (zh) * | 2018-03-27 | 2018-09-07 | 宜明细胞生物科技有限公司 | 一种利用CRISPR/Cas9系统同源重组修复IL-2RG缺陷基因的方法 |
| US11825839B2 (en) * | 2018-09-07 | 2023-11-28 | University Of Southern California | Cryopreservation of cell-seeded substrates and related methods |
| BR112021009370A2 (pt) * | 2018-11-14 | 2021-08-17 | Regenxbio Inc. | método de tratamento da doença de batten cln2, composição farmacêutica e kit |
| KR20210105382A (ko) * | 2018-12-19 | 2021-08-26 | 베르사멥 아게 | 단백질을 코딩하는 rna |
| WO2021050789A1 (en) | 2019-09-10 | 2021-03-18 | Obsidian Therapeutics, Inc. | Ca2-il15 fusion proteins for tunable regulation |
| US20230146121A1 (en) * | 2019-12-09 | 2023-05-11 | Chigenovo Co., Ltd. | Use of cyp4v2 and rdcvf in the manufacture of medicament |
| CN111733174B (zh) * | 2020-08-07 | 2021-02-09 | 北京大学第三医院（北京大学第三临床医学院） | 一种分离的核酸分子及其用途 |
| WO2022045935A1 (en) * | 2020-08-28 | 2022-03-03 | Joint Stock Company "Biocad" | Aav5-based vaccine against sars-cov-2 |
| WO2024206928A1 (en) | 2023-03-30 | 2024-10-03 | Pharma Cinq, Llc | VECTOR ENCODING ROD-DERIVED CONE VIABILITY FACTOR AND HUMAN IgK SIGNAL SEQUENCE |
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| US5840293A (en) | 1988-11-16 | 1998-11-24 | Advanced Polymer Systems, Inc. | Ionic beads for controlled release and adsorption |
| US5225347A (en) | 1989-09-25 | 1993-07-06 | Innovir Laboratories, Inc. | Therapeutic ribozyme compositions and expression vectors |
| US5670488A (en) | 1992-12-03 | 1997-09-23 | Genzyme Corporation | Adenovirus vector for gene therapy |
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| DK0585368T3 (da) | 1991-04-25 | 1998-03-16 | Univ Brown Res Found | Implanterbart biokompatibelt immunisolatorisk vehikel til afgivelse af udvalgte terapeutiske produkter |
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| CA2146747C (en) | 1992-10-09 | 2006-12-19 | Brian A. Naughton | Liver reserve cells |
| GB9223084D0 (en) | 1992-11-04 | 1992-12-16 | Imp Cancer Res Tech | Compounds to target cells |
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| DE19541450C2 (de) | 1995-11-07 | 1997-10-02 | Gsf Forschungszentrum Umwelt | Genkonstrukt und dessen Verwendung |
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| ES2602352T3 (es) | 2001-12-17 | 2017-02-20 | The Trustees Of The University Of Pennsylvania | Secuencias de serotipo 8 de virus adenoasociado (VAA), vectores que las contienen y usos de las mismas |
| AU2003225544A1 (en) * | 2002-02-04 | 2003-09-02 | Novartis Ag | Recombinant bovine immunodeficiency virus based gene transfer system |
| ATE536405T1 (de) | 2002-07-25 | 2011-12-15 | Scripps Research Inst | Hämatopoetische stammzellen und verfahren zur behandlung von neovaskulären augenerkrankungen damit |
| DE10260805A1 (de) | 2002-12-23 | 2004-07-22 | Geneart Gmbh | Verfahren und Vorrichtung zum Optimieren einer Nucleotidsequenz zur Expression eines Proteins |
| EP1624758A4 (en) * | 2003-05-02 | 2007-03-07 | Scripps Research Inst | HEMOPOETIC STEM CELLS AND METHOD FOR THE TREATMENT OF EYE-RELATED VASCULAR DISEASES USING THESE STEM CELLS |
| FR2870241B1 (fr) | 2004-05-13 | 2015-02-27 | Novartis Ag | Facteur de viabilite des cones derive des batonnets ou rdcvf et applications |
| JP5247468B2 (ja) | 2005-12-30 | 2013-07-24 | ニューロテック ユーエスエー， インコーポレイテッド | 生物活性分子の送達のための微粒子化デバイスおよびその使用方法 |
| KR20090122465A (ko) | 2007-03-01 | 2009-11-30 | 어드벤스드 비젼 테라피스, 인코포레이티드 | 염증성 질환의 치료 |
| EP2027889A1 (en) * | 2007-06-05 | 2009-02-25 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | New neuronal viability factor and use thereof |
| JP5710265B2 (ja) * | 2007-12-06 | 2015-04-30 | アンセルム（アンスチチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル） | 網膜変性疾患の治療のための栄養因子 |
| EP2281047B1 (en) | 2008-04-15 | 2020-04-08 | Genzyme Corporation | Methods to produce rod-derived cone viability factor (rdcvf) |
| WO2009134681A2 (en) | 2008-04-30 | 2009-11-05 | The Trustees Of The University Of Pennsylvania | Aav7 viral vectors for targeted delivery of rpe cells |
| WO2010029130A1 (en) | 2008-09-10 | 2010-03-18 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Neuronal viability factor and use thereof |
| US20120088819A1 (en) * | 2008-10-31 | 2012-04-12 | Makoto Inoue | Method for enhancing expression of recombinant protein |
| DE102009016934A1 (de) | 2009-04-08 | 2010-10-14 | Daimler Ag | Brennstoffzelle, Brennstoffzellenstapel und Verfahren zum Abdichten einer Brennstoffzelle |
| JP2013507373A (ja) | 2009-10-08 | 2013-03-04 | ニューロテック ユーエスエー， インコーポレイテッド | 被包された細胞ベースの送達系におけるｐｅｄｆの使用 |
-
2012
- 2012-10-26 WO PCT/US2012/062106 patent/WO2013063383A2/en not_active Ceased
- 2012-10-26 KR KR1020147011857A patent/KR102000141B1/ko active Active
- 2012-10-26 CN CN201280064474.1A patent/CN104321069A/zh active Pending
- 2012-10-26 ES ES12843778T patent/ES2774779T3/es active Active
- 2012-10-26 JP JP2014539038A patent/JP6293664B2/ja not_active Expired - Fee Related
- 2012-10-26 CN CN201911125836.3A patent/CN110777124A/zh active Pending
- 2012-10-26 CA CA2853379A patent/CA2853379C/en active Active
- 2012-10-26 BR BR112014010091-8A patent/BR112014010091B1/pt active IP Right Grant
- 2012-10-26 PL PL12843778T patent/PL2797613T3/pl unknown
- 2012-10-26 EP EP12843778.7A patent/EP2797613B1/en active Active
- 2012-10-26 MX MX2014004949A patent/MX354516B/es active IP Right Grant
- 2012-10-26 AU AU2012321102A patent/AU2012321102C1/en active Active
- 2012-10-26 RU RU2014121256A patent/RU2664673C2/ru active
- 2012-10-26 US US14/354,415 patent/US9265813B2/en active Active
- 2012-10-26 DK DK12843778.7T patent/DK2797613T3/da active
-
2015
- 2015-12-08 US US14/962,337 patent/US10040835B2/en active Active
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