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 (enExample) | 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 (enExample) |
| EP (1) | EP3423109A4 (enExample) |
| JP (2) | JP7436089B2 (enExample) |
| AU (1) | AU2017227803B2 (enExample) |
| CA (1) | CA3016314A1 (enExample) |
| WO (1) | WO2017151884A1 (enExample) |
Families Citing this family (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2016073693A2 (en) | 2014-11-05 | 2016-05-12 | Voyager Therapeutics, Inc. | Aadc polynucleotides for the treatment of parkinson's disease |
| US11697825B2 (en) | 2014-12-12 | 2023-07-11 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scAAV |
| JP7436089B2 (ja) * | 2016-03-02 | 2024-02-21 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィア | 前頭側頭型認知症の治療 |
| 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 |
| BR112020006633A2 (pt) | 2017-10-03 | 2020-10-06 | Prevail Therapeutics, Inc. | terapias genéticas para distúrbios lisossômicos |
| AU2018346102B2 (en) | 2017-10-03 | 2023-05-11 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| JP7361037B2 (ja) * | 2017-10-03 | 2023-10-13 | プリベイル セラピューティクス，インコーポレーテッド | ライソゾーム病の遺伝子治療 |
| KR20200075865A (ko) * | 2017-10-23 | 2020-06-26 | 프리베일 테라퓨틱스, 인크. | 신경변성 질환에 대한 유전자 요법 |
| CN118345115A (zh) * | 2017-12-01 | 2024-07-16 | 编码治疗公司 | 工程化的dna结合蛋白 |
| EP3794126A1 (en) | 2018-05-15 | 2021-03-24 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of parkinson's disease |
| SG11202103151RA (en) | 2018-09-28 | 2021-04-29 | Voyager Therapeutics Inc | Frataxin expression constructs having engineered promoters and methods of use thereof |
| CN113164627B (zh) * | 2018-10-16 | 2024-10-29 | 戴纳立制药公司 | 治疗和监测颗粒体蛋白前体相关病症的方法 |
| EP3917539A4 (en) * | 2019-02-01 | 2022-11-23 | Avrobio, Inc. | COMPOSITIONS AND METHODS FOR THE TREATMENT OF NEUROCOGNITIVE DISORDERS |
| JP7637058B2 (ja) * | 2019-02-22 | 2025-02-27 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | Ｇｒｎ関連成人発症性神経変性の治療のための組換えアデノ随伴ウイルス |
| KR20210150486A (ko) | 2019-04-10 | 2021-12-10 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애에 대한 유전자 요법 |
| MX2021012184A (es) | 2019-04-10 | 2022-01-24 | Prevail Therapeutics Inc | Terapias genicas para trastornos lisosomales. |
| GB201913974D0 (en) | 2019-09-27 | 2019-11-13 | King S College London | Vector |
| AU2020371662A1 (en) * | 2019-10-22 | 2022-05-12 | Applied Genetic Technologies Corporation | Adeno-associated virus (AAV) systems for treatment of progranulin associated neurodegenerative diseases or disorders |
| US20230193212A1 (en) * | 2020-05-06 | 2023-06-22 | Orchard Therapeutics (Europe) Limited | Treatment for neurodegenerative diseases |
| AU2021280943A1 (en) * | 2020-05-27 | 2022-07-28 | Universität Zürich | Viral vectors expressing therapeutic proteins specifically in myeloid cells and microglia |
| EP4192517A1 (en) * | 2020-08-10 | 2023-06-14 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative disorders |
| IL300294A (en) * | 2020-08-12 | 2023-04-01 | UCB Biopharma SRL | Gene therapy using nucleic acid constructs containing methyl CPG-binding protein 2 (MECP2) promoter sequences |
| WO2022046988A1 (en) * | 2020-08-26 | 2022-03-03 | The Trustees Of The University Of Pennsylvania | Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration |
| AU2022214429A1 (en) | 2021-02-01 | 2023-09-14 | Tern Therapeutics, Llc | Gene therapy for neuronal ceroid lipofuscinoses |
| 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 |
|---|---|---|---|---|
| LT2489733T (lt) | 2006-06-07 | 2019-05-27 | Genzyme Corporation | Šoninės amiotrofinės sklerozės ir kitų nugaros smegenų sutrikimų genų terapija |
| CA2712276A1 (en) * | 2008-01-16 | 2009-07-23 | Neurodyn, Inc. | Treating neurodegenerative diseases with progranulin (pgrn) |
| DK3421603T3 (da) * | 2009-05-02 | 2022-01-10 | Genzyme Corp | Genterapi for neurodegenerative forstyrrelser |
| 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 |
| JP7436089B2 (ja) * | 2016-03-02 | 2024-02-21 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィア | 前頭側頭型認知症の治療 |
-
2017
- 2017-03-02 JP JP2018565253A patent/JP7436089B2/ja active Active
- 2017-03-02 EP EP17760789.2A patent/EP3423109A4/en active Pending
- 2017-03-02 US US16/081,346 patent/US20190328906A1/en active Pending
- 2017-03-02 WO PCT/US2017/020397 patent/WO2017151884A1/en not_active Ceased
- 2017-03-02 CA CA3016314A patent/CA3016314A1/en active Pending
- 2017-03-02 AU AU2017227803A patent/AU2017227803B2/en active Active
-
2022
- 2022-03-01 JP JP2022030769A patent/JP7507808B2/ja active Active
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