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) | CN110777124A (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 (22)
| 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 |
| US10004780B2 (en) | 2012-10-17 | 2018-06-26 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods and pharmaceutical compositions for the treatment of age-related macular degeneration (AMD) |
| ES2744565T3 (es) | 2014-04-25 | 2020-02-25 | Genethon | Tratamiento de la hiperbilirrubinemia |
| JP6684343B2 (ja) * | 2015-05-21 | 2020-04-22 | アンスティチュ 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 | ニューロン生存因子の相乗組み合わせ及びその使用 |
| GB201516066D0 (en) | 2015-09-10 | 2015-10-28 | Young & Co Llp D | Treatment of retinitis pigmentosa |
| AU2016370590B2 (en) | 2015-12-14 | 2023-11-02 | 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 |
| KR20260085174A (ko) * | 2016-10-11 | 2026-06-08 | 파마 싱크, 엘엘씨 | 짧은 형태의 간상체 유래 원추체 생존능 인자와 친수성 펩타이드 사이의 융합 단백질 |
| AU2018350700B2 (en) * | 2017-10-16 | 2025-04-24 | VeonGen Therapeutics GmbH | AAV vectors |
| WO2019122403A1 (en) * | 2017-12-22 | 2019-06-27 | Sparingvision | Constructs comprising neuronal viability factors and uses thereof |
| WO2019149648A1 (en) * | 2018-01-30 | 2019-08-08 | Universiteit Gent | Compositions for use to treat cataract |
| 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 |
| KR20250058061A (ko) * | 2018-09-07 | 2025-04-29 | 유니버시티 오브 써던 캘리포니아 | 세포 시딩된 기질로부터의 치료적 상층액 및 관련 방법 |
| KR20260086827A (ko) * | 2018-11-14 | 2026-06-10 | 리젠엑스바이오 인크. | 신경원성 세로이드 리포푸신증에 대한 유전자 요법 |
| JP2022514863A (ja) * | 2018-12-19 | 2022-02-16 | ヴェルサメブ アーゲー | タンパク質をコードするｒｎａ |
| CN114651003A (zh) | 2019-09-10 | 2022-06-21 | 黑曜石疗法公司 | 用于可调调节的ca2-il15融合蛋白 |
| JP7495756B2 (ja) * | 2019-12-09 | 2024-06-05 | 北京中因科技有限公司 | 医薬の製造におけるＣＹＰ４Ｖ２およびＲｄＣＶＦの使用 |
| 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 |
| IL300738A (en) * | 2020-08-28 | 2023-04-01 | Biocad Joint Stock Co | A vaccine for SARS-COV-2 based on AAV5 |
| CN121285568A (zh) * | 2023-03-30 | 2026-01-06 | 制药五有限责任公司 | 编码视杆细胞源性视锥细胞活力因子和人IgK信号序列的载体 |
| WO2026072752A1 (en) | 2024-09-27 | 2026-04-02 | Pharma Cinq, Llc | Rod-derived cone viability factor fusion protein |
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-
2012
- 2012-10-26 CN CN201911125836.3A patent/CN110777124A/zh active Pending
- 2012-10-26 EP EP12843778.7A patent/EP2797613B1/en active Active
- 2012-10-26 BR BR112014010091-8A patent/BR112014010091B1/pt active IP Right Grant
- 2012-10-26 ES ES12843778T patent/ES2774779T3/es active Active
- 2012-10-26 CA CA2853379A patent/CA2853379C/en active Active
- 2012-10-26 AU AU2012321102A patent/AU2012321102C1/en active Active
- 2012-10-26 PL PL12843778T patent/PL2797613T3/pl unknown
- 2012-10-26 JP JP2014539038A patent/JP6293664B2/ja not_active Expired - Fee Related
- 2012-10-26 MX MX2014004949A patent/MX354516B/es active IP Right Grant
- 2012-10-26 KR KR1020147011857A patent/KR102000141B1/ko active Active
- 2012-10-26 US US14/354,415 patent/US9265813B2/en active Active
- 2012-10-26 RU RU2014121256A patent/RU2664673C2/ru active
- 2012-10-26 WO PCT/US2012/062106 patent/WO2013063383A2/en not_active Ceased
- 2012-10-26 CN CN201280064474.1A patent/CN104321069A/zh active Pending
- 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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