JP2018510620A5 - - Google Patents
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- Publication number
- JP2018510620A5 JP2018510620A5 JP2017541749A JP2017541749A JP2018510620A5 JP 2018510620 A5 JP2018510620 A5 JP 2018510620A5 JP 2017541749 A JP2017541749 A JP 2017541749A JP 2017541749 A JP2017541749 A JP 2017541749A JP 2018510620 A5 JP2018510620 A5 JP 2018510620A5
- Authority
- JP
- Japan
- Prior art keywords
- sequence
- rpe
- vector
- seq
- specific promoter
- 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
- 239000013598 vector Substances 0.000 claims description 38
- 210000003583 retinal pigment epithelium Anatomy 0.000 claims description 28
- 239000002773 nucleotide Substances 0.000 claims description 16
- 125000003729 nucleotide group Chemical group 0.000 claims description 16
- 230000000694 effects Effects 0.000 claims description 12
- 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 9
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims description 9
- 208000032578 Inherited retinal disease Diseases 0.000 claims description 8
- 208000032430 Retinal dystrophy Diseases 0.000 claims description 8
- 201000006321 fundus dystrophy Diseases 0.000 claims description 8
- 208000017532 inherited retinal dystrophy Diseases 0.000 claims description 8
- 210000000234 capsid Anatomy 0.000 claims description 6
- 239000008194 pharmaceutical composition Substances 0.000 claims description 5
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims description 4
- 108091026890 Coding region Proteins 0.000 claims description 4
- 101000729271 Homo sapiens Retinoid isomerohydrolase Proteins 0.000 claims description 4
- 102100031176 Retinoid isomerohydrolase Human genes 0.000 claims description 4
- 206010064930 age-related macular degeneration Diseases 0.000 claims description 4
- 102000002717 c-Mer Tyrosine Kinase Human genes 0.000 claims description 4
- 108010018804 c-Mer Tyrosine Kinase Proteins 0.000 claims description 4
- 208000002780 macular degeneration Diseases 0.000 claims description 4
- 108091033319 polynucleotide Proteins 0.000 claims description 4
- 239000002157 polynucleotide Substances 0.000 claims description 4
- 102000040430 polynucleotide Human genes 0.000 claims description 4
- 229920001184 polypeptide Polymers 0.000 claims description 4
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 4
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 4
- 210000001525 retina Anatomy 0.000 claims description 4
- 239000013603 viral vector Substances 0.000 claims description 4
- 239000003814 drug Substances 0.000 claims description 3
- 230000002265 prevention Effects 0.000 claims description 3
- 241000702421 Dependoparvovirus Species 0.000 claims description 2
- 102100033356 Lecithin retinol acyltransferase Human genes 0.000 claims description 2
- 102000026889 Myosin VIIa Human genes 0.000 claims description 2
- 108010009047 Myosin VIIa Proteins 0.000 claims description 2
- 108010025020 Nerve Growth Factor Proteins 0.000 claims description 2
- 102000007072 Nerve Growth Factors Human genes 0.000 claims description 2
- 230000001772 anti-angiogenic effect Effects 0.000 claims description 2
- 230000002424 anti-apoptotic effect Effects 0.000 claims description 2
- 230000007812 deficiency Effects 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 238000002347 injection Methods 0.000 claims description 2
- 239000007924 injection Substances 0.000 claims description 2
- 108010084957 lecithin-retinol acyltransferase Proteins 0.000 claims description 2
- 238000004519 manufacturing process Methods 0.000 claims description 2
- 239000003900 neurotrophic factor Substances 0.000 claims description 2
- 201000003533 Leber congenital amaurosis Diseases 0.000 claims 2
- 206010003591 Ataxia Diseases 0.000 claims 1
- 201000007908 Ocular Albinism Diseases 0.000 claims 1
- 241000219422 Urtica Species 0.000 claims 1
- 235000009108 Urtica dioica Nutrition 0.000 claims 1
- 239000000825 pharmaceutical preparation Substances 0.000 claims 1
- 229940127557 pharmaceutical product Drugs 0.000 claims 1
- 238000000034 method Methods 0.000 description 8
- 241001164825 Adeno-associated virus - 8 Species 0.000 description 5
- 238000011084 recovery Methods 0.000 description 3
- 239000012634 fragment Substances 0.000 description 2
- 238000001727 in vivo Methods 0.000 description 2
- 108010054126 retinoid isomerohydrolase Proteins 0.000 description 2
- 201000004384 Alopecia Diseases 0.000 description 1
- 206010007747 Cataract congenital Diseases 0.000 description 1
- 206010048768 Dermatosis Diseases 0.000 description 1
- 241001465754 Metazoa Species 0.000 description 1
- 241000699670 Mus sp. Species 0.000 description 1
- 231100000360 alopecia Toxicity 0.000 description 1
- 230000002950 deficient Effects 0.000 description 1
- 238000011156 evaluation Methods 0.000 description 1
- 230000006870 function Effects 0.000 description 1
- 230000004243 retinal function Effects 0.000 description 1
- 208000017520 skin disease Diseases 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GBGB1502137.1A GB201502137D0 (en) | 2015-02-09 | 2015-02-09 | Treatment |
| GB1502137.1 | 2015-02-09 | ||
| PCT/GB2016/050289 WO2016128722A1 (en) | 2015-02-09 | 2016-02-08 | Optimized rpe65 promoter and coding sequences |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018510620A JP2018510620A (ja) | 2018-04-19 |
| JP2018510620A5 true JP2018510620A5 (enExample) | 2019-03-22 |
| JP6963503B2 JP6963503B2 (ja) | 2021-11-10 |
Family
ID=52746363
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017541749A Active JP6963503B2 (ja) | 2015-02-09 | 2016-02-08 | 最適化されたrpe65プロモーター及びコード配列 |
Country Status (24)
| Country | Link |
|---|---|
| US (3) | US10568973B2 (enExample) |
| EP (2) | EP3256169B1 (enExample) |
| JP (1) | JP6963503B2 (enExample) |
| CN (1) | CN107429252B (enExample) |
| AU (1) | AU2016217654B2 (enExample) |
| BR (1) | BR112017017060A2 (enExample) |
| CA (1) | CA2975850C (enExample) |
| CY (1) | CY1123947T1 (enExample) |
| DK (1) | DK3256169T3 (enExample) |
| ES (1) | ES2830030T3 (enExample) |
| GB (1) | GB201502137D0 (enExample) |
| HR (1) | HRP20201831T1 (enExample) |
| HU (1) | HUE052407T2 (enExample) |
| IL (2) | IL287142B2 (enExample) |
| LT (1) | LT3256169T (enExample) |
| MX (2) | MX383316B (enExample) |
| PH (1) | PH12017501430A1 (enExample) |
| PL (1) | PL3256169T3 (enExample) |
| PT (1) | PT3256169T (enExample) |
| RS (1) | RS61079B1 (enExample) |
| SG (1) | SG11201706520UA (enExample) |
| SI (1) | SI3256169T1 (enExample) |
| SM (1) | SMT202000655T1 (enExample) |
| WO (1) | WO2016128722A1 (enExample) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201502137D0 (en) * | 2015-02-09 | 2015-03-25 | Ucl Business Plc | Treatment |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| MX2018013205A (es) * | 2016-04-28 | 2019-06-24 | Spark Therapeutics Inc | Ensayo de potencia relativa para un vector viral que codifica isomero hidrolasas. |
| WO2019106035A1 (en) * | 2017-11-30 | 2019-06-06 | Friedrich Miescher Institute For Biomedical Research | Synpiii, a promoter for the specific expression of genes in retinal pigment epithelium |
| CN111118017B (zh) * | 2018-11-01 | 2023-10-03 | 上海朗昇生物科技有限公司 | 治疗Leber先天性黑蒙症的载体及其应用 |
| PT3850089T (pt) | 2019-02-04 | 2024-01-24 | Freeline Therapeutics Ltd | Polinucleótidos |
| GB202002202D0 (en) | 2020-02-18 | 2020-04-01 | Ucl Business Plc | Gene therapy |
| CN113952471A (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 |
| EP4185335A1 (en) * | 2020-07-21 | 2023-05-31 | Frontera Therapeutics, Inc. | Compositions and methods for the treatment of ocular diseases |
| CN111826378B (zh) * | 2020-08-05 | 2022-05-27 | 武汉纽福斯生物科技有限公司 | 一种编码人受体酪氨酸激酶Mer的核苷酸序列及其应用 |
| CN112522292B (zh) * | 2020-10-29 | 2023-05-02 | 南京启真基因工程有限公司 | 一种用于构建先天性黑蒙症克隆猪核供体细胞的CRISPR/Cas9系统及其应用 |
| EP4399314A4 (en) * | 2021-09-06 | 2025-09-10 | Huidagene Therapeutics Singapore Pte Ltd | TREATMENT OF EYE DISEASES AND DISORDERS ASSOCIATED WITH RPE65 |
| CN120882416A (zh) * | 2023-01-05 | 2025-10-31 | 欧普斯遗传学股份有限公司 | 用于眼部疾病的基因疗法 |
| CN117018235A (zh) * | 2023-06-20 | 2023-11-10 | 浙江大学 | 一种可用于活体的可视化神经环路的方法及工具病毒载体 |
| CN116898991A (zh) * | 2023-06-20 | 2023-10-20 | 浙江大学 | 一种可用于活体的可视化的神经活动状态评估方法及工具病毒载体 |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20080064061A1 (en) | 2004-08-06 | 2008-03-13 | Juridical Foundation The Chemo-Sero-Therapeutic Research Institute | Yeast Promoter |
| US7740836B2 (en) | 2006-05-03 | 2010-06-22 | Fondazione Telethon | Methods and compositions for recovering or improving visual function |
| US20100081707A1 (en) * | 2008-02-21 | 2010-04-01 | Ali Robin R | Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea |
| LT3418300T (lt) | 2011-07-18 | 2021-01-11 | Institute For Research In Biomedicine | Neutralizuojantys antikūnai prieš gripo virusą a ir jų panaudojimas |
| US9163259B2 (en) * | 2012-05-04 | 2015-10-20 | Novartis Ag | Viral vectors for the treatment of retinal dystrophy |
| TWI698240B (zh) * | 2012-05-15 | 2020-07-11 | 澳大利亞商艾佛蘭屈澳洲私營有限公司 | 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd) |
| GB201502137D0 (en) * | 2015-02-09 | 2015-03-25 | Ucl Business Plc | Treatment |
| GB202002073D0 (en) * | 2020-02-14 | 2020-04-01 | Ucl Business Ltd | Horizontal cells |
| GB202002202D0 (en) * | 2020-02-18 | 2020-04-01 | Ucl Business Plc | Gene therapy |
-
2015
- 2015-02-09 GB GBGB1502137.1A patent/GB201502137D0/en not_active Ceased
-
2016
- 2016-02-08 DK DK16703850.4T patent/DK3256169T3/da active
- 2016-02-08 RS RS20201400A patent/RS61079B1/sr unknown
- 2016-02-08 SI SI201630984T patent/SI3256169T1/sl unknown
- 2016-02-08 CA CA2975850A patent/CA2975850C/en active Active
- 2016-02-08 CN CN201680020425.6A patent/CN107429252B/zh active Active
- 2016-02-08 IL IL287142A patent/IL287142B2/en unknown
- 2016-02-08 HU HUE16703850A patent/HUE052407T2/hu unknown
- 2016-02-08 LT LTEP16703850.4T patent/LT3256169T/lt unknown
- 2016-02-08 SG SG11201706520UA patent/SG11201706520UA/en unknown
- 2016-02-08 WO PCT/GB2016/050289 patent/WO2016128722A1/en not_active Ceased
- 2016-02-08 PT PT167038504T patent/PT3256169T/pt unknown
- 2016-02-08 BR BR112017017060-4A patent/BR112017017060A2/pt active Search and Examination
- 2016-02-08 JP JP2017541749A patent/JP6963503B2/ja active Active
- 2016-02-08 ES ES16703850T patent/ES2830030T3/es active Active
- 2016-02-08 SM SM20200655T patent/SMT202000655T1/it unknown
- 2016-02-08 EP EP16703850.4A patent/EP3256169B1/en active Active
- 2016-02-08 AU AU2016217654A patent/AU2016217654B2/en active Active
- 2016-02-08 PL PL16703850T patent/PL3256169T3/pl unknown
- 2016-02-08 EP EP20193488.2A patent/EP3769790A1/en active Pending
- 2016-02-08 MX MX2017010292A patent/MX383316B/es unknown
- 2016-02-08 HR HRP20201831TT patent/HRP20201831T1/hr unknown
- 2016-02-08 US US15/549,549 patent/US10568973B2/en active Active
-
2017
- 2017-08-08 IL IL253915A patent/IL253915B/en unknown
- 2017-08-09 PH PH12017501430A patent/PH12017501430A1/en unknown
- 2017-08-09 MX MX2021006762A patent/MX2021006762A/es unknown
-
2020
- 2020-02-20 US US16/796,439 patent/US11969478B2/en active Active
- 2020-11-26 CY CY20201101129T patent/CY1123947T1/el unknown
-
2024
- 2024-04-25 US US18/646,126 patent/US20240293580A1/en active Pending
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