JPWO2020028466A5 - - Google Patents
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- Publication number
- JPWO2020028466A5 JPWO2020028466A5 JP2021505674A JP2021505674A JPWO2020028466A5 JP WO2020028466 A5 JPWO2020028466 A5 JP WO2020028466A5 JP 2021505674 A JP2021505674 A JP 2021505674A JP 2021505674 A JP2021505674 A JP 2021505674A JP WO2020028466 A5 JPWO2020028466 A5 JP WO2020028466A5
- Authority
- JP
- Japan
- Prior art keywords
- raav
- medicament according
- mammal
- viral vector
- gene product
- 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
- 108090000623 proteins and genes Proteins 0.000 claims description 58
- 239000003814 drug Substances 0.000 claims description 44
- 238000000034 method Methods 0.000 claims description 41
- 239000013603 viral vector Substances 0.000 claims description 40
- 241000124008 Mammalia Species 0.000 claims description 37
- 241000702421 Dependoparvovirus Species 0.000 claims description 25
- 102000004310 Ion Channels Human genes 0.000 claims description 24
- 108090000862 Ion Channels Proteins 0.000 claims description 24
- 239000003795 chemical substances by application Substances 0.000 claims description 21
- 210000000234 capsid Anatomy 0.000 claims description 17
- 102000004169 proteins and genes Human genes 0.000 claims description 16
- 210000005036 nerve Anatomy 0.000 claims description 15
- 239000013598 vector Substances 0.000 claims description 14
- 210000001186 vagus nerve Anatomy 0.000 claims description 12
- 210000001835 viscera Anatomy 0.000 claims description 12
- 230000000694 effects Effects 0.000 claims description 11
- 231100000331 toxic Toxicity 0.000 claims description 11
- 230000002588 toxic effect Effects 0.000 claims description 11
- 108091027967 Small hairpin RNA Proteins 0.000 claims description 8
- 102000003802 alpha-Synuclein Human genes 0.000 claims description 8
- 108090000185 alpha-Synuclein Proteins 0.000 claims description 8
- 239000000835 fiber Substances 0.000 claims description 8
- 238000002604 ultrasonography Methods 0.000 claims description 8
- 230000009278 visceral effect Effects 0.000 claims description 8
- 206010011224 Cough Diseases 0.000 claims description 7
- 239000000747 designer drug Substances 0.000 claims description 7
- 230000007441 retrograde transport Effects 0.000 claims description 7
- 239000004055 small Interfering RNA Substances 0.000 claims description 7
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 6
- 108091033409 CRISPR Proteins 0.000 claims description 6
- 108700011259 MicroRNAs Proteins 0.000 claims description 6
- 238000009792 diffusion process Methods 0.000 claims description 6
- 210000001035 gastrointestinal tract Anatomy 0.000 claims description 6
- 210000004072 lung Anatomy 0.000 claims description 6
- 239000002679 microRNA Substances 0.000 claims description 6
- 210000005037 parasympathetic nerve Anatomy 0.000 claims description 6
- 102000005962 receptors Human genes 0.000 claims description 6
- 108020003175 receptors Proteins 0.000 claims description 6
- 108091006146 Channels Proteins 0.000 claims description 5
- 210000004556 brain Anatomy 0.000 claims description 5
- 210000004126 nerve fiber Anatomy 0.000 claims description 5
- 210000002569 neuron Anatomy 0.000 claims description 5
- 230000001105 regulatory effect Effects 0.000 claims description 5
- 210000002784 stomach Anatomy 0.000 claims description 5
- 241000701157 Canine mastadenovirus A Species 0.000 claims description 4
- 241000713666 Lentivirus Species 0.000 claims description 4
- 210000000056 organ Anatomy 0.000 claims description 4
- 210000005070 sphincter Anatomy 0.000 claims description 4
- 101710092462 Alpha-hemolysin Proteins 0.000 claims description 3
- 102000013455 Amyloid beta-Peptides Human genes 0.000 claims description 3
- 108010090849 Amyloid beta-Peptides Proteins 0.000 claims description 3
- 108010035848 Channelrhodopsins Proteins 0.000 claims description 3
- 108010026389 Gramicidin Proteins 0.000 claims description 3
- 102000006541 Ionotropic Glutamate Receptors Human genes 0.000 claims description 3
- 108010008812 Ionotropic Glutamate Receptors Proteins 0.000 claims description 3
- 102000019315 Nicotinic acetylcholine receptors Human genes 0.000 claims description 3
- 108050006807 Nicotinic acetylcholine receptors Proteins 0.000 claims description 3
- 102000003734 Voltage-Gated Potassium Channels Human genes 0.000 claims description 3
- 108090000013 Voltage-Gated Potassium Channels Proteins 0.000 claims description 3
- 150000001413 amino acids Chemical class 0.000 claims description 3
- 210000003169 central nervous system Anatomy 0.000 claims description 3
- 210000005095 gastrointestinal system Anatomy 0.000 claims description 3
- 238000002347 injection Methods 0.000 claims description 3
- 239000007924 injection Substances 0.000 claims description 3
- 210000000936 intestine Anatomy 0.000 claims description 3
- 239000000126 substance Substances 0.000 claims description 3
- 102000013498 tau Proteins Human genes 0.000 claims description 3
- 108010026424 tau Proteins Proteins 0.000 claims description 3
- ZWCXYZRRTRDGQE-LUPIJMBPSA-N valyl gramicidin a Chemical compound C1=CC=C2C(C[C@H](NC(=O)[C@@H](CC(C)C)NC(=O)[C@H](CC=3C4=CC=CC=C4NC=3)NC(=O)[C@@H](CC(C)C)NC(=O)[C@H](CC=3C4=CC=CC=C4NC=3)NC(=O)[C@@H](CC(C)C)NC(=O)[C@H](CC=3C4=CC=CC=C4NC=3)NC(=O)[C@@H](C(C)C)NC(=O)[C@H](C(C)C)NC(=O)[C@@H](C(C)C)NC(=O)[C@H](C)NC(=O)[C@H](NC(=O)[C@H](C)NC(=O)CNC(=O)[C@@H](NC=O)C(C)C)CC(C)C)C(=O)NCCO)=CNC2=C1 ZWCXYZRRTRDGQE-LUPIJMBPSA-N 0.000 claims description 3
- 108090000565 Capsid Proteins Proteins 0.000 claims description 2
- 102100023321 Ceruloplasmin Human genes 0.000 claims description 2
- 102000034573 Channels Human genes 0.000 claims description 2
- 208000006545 Chronic Obstructive Pulmonary Disease Diseases 0.000 claims description 2
- 210000004100 adrenal gland Anatomy 0.000 claims description 2
- 230000002612 cardiopulmonary effect Effects 0.000 claims description 2
- 230000001276 controlling effect Effects 0.000 claims description 2
- 201000010099 disease Diseases 0.000 claims description 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 2
- 210000001198 duodenum Anatomy 0.000 claims description 2
- 230000037406 food intake Effects 0.000 claims description 2
- 235000012631 food intake Nutrition 0.000 claims description 2
- 230000002496 gastric effect Effects 0.000 claims description 2
- 210000002149 gonad Anatomy 0.000 claims description 2
- 210000002216 heart Anatomy 0.000 claims description 2
- 210000003734 kidney Anatomy 0.000 claims description 2
- 210000004185 liver Anatomy 0.000 claims description 2
- 210000000496 pancreas Anatomy 0.000 claims description 2
- 230000000069 prophylactic effect Effects 0.000 claims description 2
- 238000010992 reflux Methods 0.000 claims description 2
- 210000000813 small intestine Anatomy 0.000 claims description 2
- 230000001225 therapeutic effect Effects 0.000 claims description 2
- 210000003448 thoracic nerve Anatomy 0.000 claims description 2
- 210000003932 urinary bladder Anatomy 0.000 claims description 2
- 229940079593 drug Drugs 0.000 claims 7
- 241000700584 Simplexvirus Species 0.000 claims 2
- 239000013607 AAV vector Substances 0.000 claims 1
- 230000007170 pathology Effects 0.000 description 3
- 230000001515 vagal effect Effects 0.000 description 3
- 208000009889 Herpes Simplex Diseases 0.000 description 2
- 102000019355 Synuclein Human genes 0.000 description 2
- 108050006783 Synuclein Proteins 0.000 description 2
- 210000005056 cell body Anatomy 0.000 description 2
- 230000001953 sensory effect Effects 0.000 description 2
- 208000003098 Ganglion Cysts Diseases 0.000 description 1
- 208000018737 Parkinson disease Diseases 0.000 description 1
- 208000005400 Synovial Cyst Diseases 0.000 description 1
- 239000000556 agonist Substances 0.000 description 1
- 210000003050 axon Anatomy 0.000 description 1
- 230000001575 pathological effect Effects 0.000 description 1
- 210000000413 sensory ganglia Anatomy 0.000 description 1
- 210000001044 sensory neuron Anatomy 0.000 description 1
- 229940124597 therapeutic agent Drugs 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024001612A JP2024041871A (ja) | 2018-07-31 | 2024-01-10 | 臓器機能を制御するための遺伝子治療方法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862712669P | 2018-07-31 | 2018-07-31 | |
| US62/712,669 | 2018-07-31 | ||
| PCT/US2019/044290 WO2020028466A1 (en) | 2018-07-31 | 2019-07-31 | Gene therapy methods to control organ function |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024001612A Division JP2024041871A (ja) | 2018-07-31 | 2024-01-10 | 臓器機能を制御するための遺伝子治療方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021533126A JP2021533126A (ja) | 2021-12-02 |
| JP2021533126A5 JP2021533126A5 (https=) | 2022-08-01 |
| JPWO2020028466A5 true JPWO2020028466A5 (https=) | 2022-08-01 |
Family
ID=69230802
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021505674A Pending JP2021533126A (ja) | 2018-07-31 | 2019-07-31 | 臓器機能を制御するための遺伝子治療方法 |
| JP2024001612A Pending JP2024041871A (ja) | 2018-07-31 | 2024-01-10 | 臓器機能を制御するための遺伝子治療方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024001612A Pending JP2024041871A (ja) | 2018-07-31 | 2024-01-10 | 臓器機能を制御するための遺伝子治療方法 |
Country Status (14)
| Country | Link |
|---|---|
| US (1) | US20210301306A1 (https=) |
| EP (1) | EP3829718A4 (https=) |
| JP (2) | JP2021533126A (https=) |
| KR (1) | KR20210052450A (https=) |
| CN (1) | CN113301956B (https=) |
| AU (1) | AU2019315445A1 (https=) |
| BR (1) | BR112021001878A2 (https=) |
| CA (1) | CA3108324A1 (https=) |
| EA (1) | EA202190358A1 (https=) |
| IL (1) | IL280531A (https=) |
| MX (1) | MX2021001336A (https=) |
| PH (1) | PH12021550243A1 (https=) |
| SG (1) | SG11202101042SA (https=) |
| WO (1) | WO2020028466A1 (https=) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2013134543A1 (en) | 2012-03-08 | 2013-09-12 | Medtronic Ardian Luxembourg Sarl | Immune system neuromodulation and associated systems and methods |
| JP2017536187A (ja) | 2014-12-03 | 2017-12-07 | メタベンション インコーポレイテッド | 神経または他の組織を調節するためのシステムおよび方法 |
| US10524859B2 (en) | 2016-06-07 | 2020-01-07 | Metavention, Inc. | Therapeutic tissue modulation devices and methods |
| CN115707773A (zh) * | 2021-08-20 | 2023-02-21 | 中国科学院深圳先进技术研究院 | 一种利用交感神经控制血管内皮细胞异质性的方法 |
| WO2023019565A1 (zh) * | 2021-08-20 | 2023-02-23 | 中国科学院深圳先进技术研究院 | 一种利用交感神经控制血管内皮细胞异质性的方法 |
| CN116019909B (zh) * | 2022-09-16 | 2025-07-29 | 上海市第十人民医院 | 一种CRISPR/Cas9纳米系统 |
| CN116115758B (zh) * | 2022-11-22 | 2024-12-03 | 上海市内分泌代谢病研究所 | Gabaa受体作为靶点在制备或筛选降血脂、治疗肥胖和/或改善代谢的药物中的应用 |
| CN120699131B (zh) * | 2024-12-04 | 2025-12-09 | 良渚实验室 | 治疗帕金森病的化学遗传学基因治疗新方案 |
Family Cites Families (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4797368A (en) | 1985-03-15 | 1989-01-10 | The United States Of America As Represented By The Department Of Health And Human Services | Adeno-associated virus as eukaryotic expression vector |
| US4863457A (en) | 1986-11-24 | 1989-09-05 | Lee David A | Drug delivery device |
| US5378475A (en) | 1991-02-21 | 1995-01-03 | University Of Kentucky Research Foundation | Sustained release drug delivery devices |
| US5814618A (en) | 1993-06-14 | 1998-09-29 | Basf Aktiengesellschaft | Methods for regulating gene expression |
| US5464758A (en) | 1993-06-14 | 1995-11-07 | Gossen; Manfred | Tight control of gene expression in eucaryotic cells by tetracycline-responsive promoters |
| US5443505A (en) | 1993-11-15 | 1995-08-22 | Oculex Pharmaceuticals, Inc. | Biocompatible ocular implants |
| US6342390B1 (en) | 1994-11-23 | 2002-01-29 | The United States Of America As Represented By The Secretary Of Health And Human Services | Lipid vesicles containing adeno-associated virus rep protein for transgene integration and gene therapy |
| FR2814642B1 (fr) | 2000-10-03 | 2005-07-01 | Ass Pour Le Dev De La Rech En | Souris transgenique pour la recombinaison ciblee mediee par la cre-er modifiee |
| US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| CA2566257A1 (en) | 2004-05-07 | 2005-11-24 | Applera Corporation | Genetic polymorphisms associated with vascular diseases, methods of detection and uses thereof |
| WO2006110689A2 (en) * | 2005-04-07 | 2006-10-19 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
| EP2826860B1 (en) * | 2010-04-23 | 2018-08-22 | University of Massachusetts | CNS targeting AAV vectors and methods of use thereof |
| RS61055B1 (sr) * | 2012-06-19 | 2020-12-31 | Univ Florida | Kompozicije i postupci za lečenje bolesti |
| GB201403684D0 (en) * | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| US10711270B2 (en) * | 2014-10-03 | 2020-07-14 | University Of Massachusetts | High efficiency library-identified AAV vectors |
| AU2016324317A1 (en) * | 2015-09-17 | 2018-03-08 | Coda Biotherapeutics, Inc. | Compositions and methods for treating neurological disorders |
| US20170218842A1 (en) * | 2016-02-02 | 2017-08-03 | General Electric Company | Adjusting Airflow Distortion in Gas Turbine Engine |
| WO2017136536A1 (en) * | 2016-02-02 | 2017-08-10 | University Of Massachusetts | Method to enhance the efficiency of systemic aav gene delivery to the central nervous system |
| JP6968331B2 (ja) * | 2016-03-09 | 2021-11-17 | アサフ，ファディ | ニューロン性疾患の治療におけるニューロン調節のためのｄｒｅａｄｄの使用 |
| MA44546B1 (fr) * | 2016-06-15 | 2021-03-31 | Univ California | Virus adéno-associés variants et procédés d'utilisation |
| WO2018008770A1 (ja) * | 2016-07-06 | 2018-01-11 | 国立研究開発法人国立循環器病研究センター | 末梢神経を操作する方法、脳および脊髄を除く臓器などにおいて神経細胞機能を変化させる機能を発生させる方法、および新規疾病の予防または治療方法、ならびに末梢神経投与用医薬 |
| 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 |
-
2019
- 2019-07-31 JP JP2021505674A patent/JP2021533126A/ja active Pending
- 2019-07-31 US US17/264,752 patent/US20210301306A1/en active Pending
- 2019-07-31 BR BR112021001878-6A patent/BR112021001878A2/pt unknown
- 2019-07-31 WO PCT/US2019/044290 patent/WO2020028466A1/en not_active Ceased
- 2019-07-31 AU AU2019315445A patent/AU2019315445A1/en not_active Abandoned
- 2019-07-31 MX MX2021001336A patent/MX2021001336A/es unknown
- 2019-07-31 SG SG11202101042SA patent/SG11202101042SA/en unknown
- 2019-07-31 CA CA3108324A patent/CA3108324A1/en active Pending
- 2019-07-31 KR KR1020217005554A patent/KR20210052450A/ko not_active Ceased
- 2019-07-31 EA EA202190358A patent/EA202190358A1/ru unknown
- 2019-07-31 CN CN201980065036.9A patent/CN113301956B/zh active Active
- 2019-07-31 EP EP19845335.9A patent/EP3829718A4/en active Pending
-
2021
- 2021-01-31 IL IL280531A patent/IL280531A/en unknown
- 2021-02-01 PH PH12021550243A patent/PH12021550243A1/en unknown
-
2024
- 2024-01-10 JP JP2024001612A patent/JP2024041871A/ja active Pending
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