JP2019511570A5 - - Google Patents
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- Publication number
- JP2019511570A5 JP2019511570A5 JP2018565253A JP2018565253A JP2019511570A5 JP 2019511570 A5 JP2019511570 A5 JP 2019511570A5 JP 2018565253 A JP2018565253 A JP 2018565253A JP 2018565253 A JP2018565253 A JP 2018565253A JP 2019511570 A5 JP2019511570 A5 JP 2019511570A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- composition according
- aav
- ventricle
- progranulin
- 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
- 239000000203 mixture Substances 0.000 claims 47
- 102000019204 Progranulins Human genes 0.000 claims 12
- 108010012809 Progranulins Proteins 0.000 claims 12
- 201000011240 Frontotemporal dementia Diseases 0.000 claims 8
- 210000004027 cell Anatomy 0.000 claims 7
- 239000012634 fragment Substances 0.000 claims 7
- 210000001175 cerebrospinal fluid Anatomy 0.000 claims 6
- 230000007812 deficiency Effects 0.000 claims 6
- 201000010099 disease Diseases 0.000 claims 6
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 6
- 230000006870 function Effects 0.000 claims 6
- 241000702421 Dependoparvovirus Species 0.000 claims 5
- 102100037632 Progranulin Human genes 0.000 claims 5
- 208000024891 symptom Diseases 0.000 claims 5
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 4
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 4
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 4
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 4
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 4
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 4
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 4
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 4
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 4
- 241000649046 Adeno-associated virus 11 Species 0.000 claims 4
- 241000649047 Adeno-associated virus 12 Species 0.000 claims 4
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 4
- 241000124008 Mammalia Species 0.000 claims 4
- 239000002245 particle Substances 0.000 claims 4
- 208000031277 Amaurotic familial idiocy Diseases 0.000 claims 3
- 108090000565 Capsid Proteins Proteins 0.000 claims 3
- 102100023321 Ceruloplasmin Human genes 0.000 claims 3
- 230000002411 adverse Effects 0.000 claims 3
- 230000000694 effects Effects 0.000 claims 3
- 230000004770 neurodegeneration Effects 0.000 claims 3
- 230000016273 neuron death Effects 0.000 claims 3
- 102000039446 nucleic acids Human genes 0.000 claims 3
- 108020004707 nucleic acids Proteins 0.000 claims 3
- 150000007523 nucleic acids Chemical class 0.000 claims 3
- 208000002339 Frontotemporal Lobar Degeneration Diseases 0.000 claims 2
- 206010056677 Nerve degeneration Diseases 0.000 claims 2
- 208000002537 Neuronal Ceroid-Lipofuscinoses Diseases 0.000 claims 2
- 241000283984 Rodentia Species 0.000 claims 2
- 210000004556 brain Anatomy 0.000 claims 2
- 210000000234 capsid Anatomy 0.000 claims 2
- 230000001054 cortical effect Effects 0.000 claims 2
- 210000003618 cortical neuron Anatomy 0.000 claims 2
- 239000003623 enhancer Substances 0.000 claims 2
- 210000000981 epithelium Anatomy 0.000 claims 2
- 238000002347 injection Methods 0.000 claims 2
- 239000007924 injection Substances 0.000 claims 2
- 238000007913 intrathecal administration Methods 0.000 claims 2
- 208000017476 juvenile neuronal ceroid lipofuscinosis Diseases 0.000 claims 2
- 210000002161 motor neuron Anatomy 0.000 claims 2
- 201000007607 neuronal ceroid lipofuscinosis 3 Diseases 0.000 claims 2
- 210000002330 subarachnoid space Anatomy 0.000 claims 2
- 230000004083 survival effect Effects 0.000 claims 2
- 230000002861 ventricular Effects 0.000 claims 2
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims 1
- 108700028146 Genetic Enhancer Elements Proteins 0.000 claims 1
- 208000009668 Neurobehavioral Manifestations Diseases 0.000 claims 1
- 230000003542 behavioural effect Effects 0.000 claims 1
- 210000003038 endothelium Anatomy 0.000 claims 1
- 229940125721 immunosuppressive agent Drugs 0.000 claims 1
- 239000003018 immunosuppressive agent Substances 0.000 claims 1
- 238000007914 intraventricular administration Methods 0.000 claims 1
- 210000003140 lateral ventricle Anatomy 0.000 claims 1
- 210000005240 left ventricle Anatomy 0.000 claims 1
- 210000005171 mammalian brain Anatomy 0.000 claims 1
- 210000002418 meninge Anatomy 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 210000004498 neuroglial cell Anatomy 0.000 claims 1
- 210000002569 neuron Anatomy 0.000 claims 1
- 230000003955 neuronal function Effects 0.000 claims 1
- 230000006576 neuronal survival Effects 0.000 claims 1
- 210000004738 parenchymal cell Anatomy 0.000 claims 1
- 210000005241 right ventricle Anatomy 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022030769A JP7507808B2 (ja) | 2016-03-02 | 2022-03-01 | 前頭側頭型認知症の治療 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662302525P | 2016-03-02 | 2016-03-02 | |
| US62/302,525 | 2016-03-02 | ||
| PCT/US2017/020397 WO2017151884A1 (en) | 2016-03-02 | 2017-03-02 | Therapy for frontotemporal dementia |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022030769A Division JP7507808B2 (ja) | 2016-03-02 | 2022-03-01 | 前頭側頭型認知症の治療 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019511570A JP2019511570A (ja) | 2019-04-25 |
| JP2019511570A5 true JP2019511570A5 (cg-RX-API-DMAC7.html) | 2020-03-19 |
| JP7436089B2 JP7436089B2 (ja) | 2024-02-21 |
Family
ID=59743281
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018565253A Active JP7436089B2 (ja) | 2016-03-02 | 2017-03-02 | 前頭側頭型認知症の治療 |
| JP2022030769A Active JP7507808B2 (ja) | 2016-03-02 | 2022-03-01 | 前頭側頭型認知症の治療 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022030769A Active JP7507808B2 (ja) | 2016-03-02 | 2022-03-01 | 前頭側頭型認知症の治療 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20190328906A1 (cg-RX-API-DMAC7.html) |
| EP (1) | EP3423109A4 (cg-RX-API-DMAC7.html) |
| JP (2) | JP7436089B2 (cg-RX-API-DMAC7.html) |
| AU (1) | AU2017227803B2 (cg-RX-API-DMAC7.html) |
| CA (1) | CA3016314A1 (cg-RX-API-DMAC7.html) |
| WO (1) | WO2017151884A1 (cg-RX-API-DMAC7.html) |
Families Citing this family (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| RU2020109343A (ru) | 2014-11-05 | 2020-03-17 | Вояджер Терапьютикс, Инк. | Полинуклеотиды aadc для лечения болезни паркинсона |
| WO2016094783A1 (en) | 2014-12-12 | 2016-06-16 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scaav |
| CA3016314A1 (en) * | 2016-03-02 | 2017-09-08 | Julianne REIDERS | Therapy for frontotemporal dementia |
| US20240002459A1 (en) * | 2016-07-14 | 2024-01-04 | Emory University | Granulin Compositions and Uses Related Thereto |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| AU2018346104B2 (en) | 2017-10-03 | 2023-07-27 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| KR102709597B1 (ko) | 2017-10-03 | 2024-09-26 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| KR20220010062A (ko) | 2017-10-03 | 2022-01-25 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| US20210261981A1 (en) * | 2017-10-23 | 2021-08-26 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative diseases |
| AU2018375192B2 (en) * | 2017-12-01 | 2023-11-09 | Encoded Therapeutics, Inc. | Engineered DNA binding proteins |
| CA3099306A1 (en) | 2018-05-15 | 2019-11-21 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of parkinson's disease |
| EP3856762A1 (en) | 2018-09-28 | 2021-08-04 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| EP3866861A4 (en) * | 2018-10-16 | 2022-09-07 | Denali Therapeutics Inc. | METHODS OF TREATMENT AND MONITORING OF PROGRANULIN-ASSOCIATED DISEASES |
| US20220111005A1 (en) * | 2019-02-01 | 2022-04-14 | Avrobio, Inc. | Compositions and methods for treating neurocognitive disorders |
| CA3129672A1 (en) * | 2019-02-22 | 2020-08-27 | The Trustees Of The University Of Pennsylvania | Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration |
| FI3953377T3 (fi) | 2019-04-10 | 2025-11-26 | Prevail Therapeutics Inc | Geeniterapioita lysosomaalisiin sairauksiin |
| EP3953378A1 (en) | 2019-04-10 | 2022-02-16 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| GB201913974D0 (en) | 2019-09-27 | 2019-11-13 | King S College London | Vector |
| EP4048799A4 (en) * | 2019-10-22 | 2023-11-15 | Applied Genetic Technologies Corporation | ADENO-ASSOCIATED VIRUS (AAV) SYSTEMS FOR THE TREATMENT OF PROGRANULIN-ASSOCIATED NEURODEGENERATIVE DISEASES OR DISORDERS |
| EP4146797A1 (en) * | 2020-05-06 | 2023-03-15 | Orchard Therapeutics (Europe) Limited | Treatment for neurodegenerative diseases |
| KR20230018378A (ko) * | 2020-05-27 | 2023-02-07 | 유니버시타트 취리히 | 골수 세포 및 미세아교세포에서 치료 단백질을 특이적으로 발현하는 바이러스 벡터 |
| US20230346979A1 (en) * | 2020-08-10 | 2023-11-02 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative disorders |
| PH12023550145A1 (en) * | 2020-08-12 | 2024-03-18 | UCB Biopharma SRL | Gene therapy using nucleic acid constructs comprising methyl cpg binding protein 2 (mecp2) promoter sequences |
| MX2023002364A (es) * | 2020-08-26 | 2023-05-22 | Univ Pennsylvania | Virus adenoasociado recombinante para el tratamiento de la neurodegeneración iniciada en adultos asociada a grn. |
| JP2024505257A (ja) | 2021-02-01 | 2024-02-05 | レジェンクスバイオ インコーポレーテッド | 神経セロイドリポフスチン症の遺伝子治療 |
| WO2024035649A1 (en) * | 2022-08-08 | 2024-02-15 | Shape Therapeutics Inc. | Compositions for progranulin expression and methods of use thereof |
| GB202216168D0 (en) | 2022-10-31 | 2022-12-14 | UCB Biopharma SRL | Route of administration |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2007146046A2 (en) * | 2006-06-07 | 2007-12-21 | Genzyme Corporation | Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders |
| CN102006882B (zh) * | 2008-01-16 | 2017-06-06 | 神经动力公司 | 利用颗粒体蛋白前体(pgrn)治疗神经变性疾病 |
| LT4342992T (lt) * | 2009-05-02 | 2025-08-25 | Genzyme Corporation | Genų terapija, skirta neurodegeneracinių sutrikimų gydymui |
| US20110203007A1 (en) * | 2010-02-16 | 2011-08-18 | Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College | Assays of neurodegenerative disorders, including frontotemporal dementia and amyotrophic lateral sclerosis |
| CA3016314A1 (en) | 2016-03-02 | 2017-09-08 | Julianne REIDERS | Therapy for frontotemporal dementia |
-
2017
- 2017-03-02 CA CA3016314A patent/CA3016314A1/en active Pending
- 2017-03-02 JP JP2018565253A patent/JP7436089B2/ja active Active
- 2017-03-02 WO PCT/US2017/020397 patent/WO2017151884A1/en not_active Ceased
- 2017-03-02 AU AU2017227803A patent/AU2017227803B2/en active Active
- 2017-03-02 US US16/081,346 patent/US20190328906A1/en active Pending
- 2017-03-02 EP EP17760789.2A patent/EP3423109A4/en active Pending
-
2022
- 2022-03-01 JP JP2022030769A patent/JP7507808B2/ja active Active