JP2019537576A5 - - Google Patents
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- JP2019537576A5 JP2019537576A5 JP2019522377A JP2019522377A JP2019537576A5 JP 2019537576 A5 JP2019537576 A5 JP 2019537576A5 JP 2019522377 A JP2019522377 A JP 2019522377A JP 2019522377 A JP2019522377 A JP 2019522377A JP 2019537576 A5 JP2019537576 A5 JP 2019537576A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- particle according
- aav particle
- disease
- particles
- 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
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- 241000702421 Dependoparvovirus Species 0.000 claims 60
- 239000002245 particle Substances 0.000 claims 57
- 241000124008 Mammalia Species 0.000 claims 10
- 210000000234 capsid Anatomy 0.000 claims 8
- 201000010099 disease Diseases 0.000 claims 8
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 8
- 229920001184 polypeptide Polymers 0.000 claims 7
- 108090000765 processed proteins & peptides Proteins 0.000 claims 7
- 102000004196 processed proteins & peptides Human genes 0.000 claims 7
- 208000015439 Lysosomal storage disease Diseases 0.000 claims 6
- 239000003018 immunosuppressive agent Substances 0.000 claims 6
- 102000039446 nucleic acids Human genes 0.000 claims 6
- 108020004707 nucleic acids Proteins 0.000 claims 6
- 150000007523 nucleic acids Chemical class 0.000 claims 6
- 108010039203 Tripeptidyl-Peptidase 1 Proteins 0.000 claims 5
- 102100034197 Tripeptidyl-peptidase 1 Human genes 0.000 claims 5
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 4
- 210000001175 cerebrospinal fluid Anatomy 0.000 claims 4
- 230000000694 effects Effects 0.000 claims 4
- 229960003444 immunosuppressant agent Drugs 0.000 claims 4
- 230000001861 immunosuppressant effect Effects 0.000 claims 4
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 3
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 3
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 3
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 3
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 3
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 3
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 3
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 3
- 241000649046 Adeno-associated virus 11 Species 0.000 claims 3
- 101710132601 Capsid protein Proteins 0.000 claims 3
- 101710197658 Capsid protein VP1 Proteins 0.000 claims 3
- 101710081079 Minor spike protein H Proteins 0.000 claims 3
- 101710118046 RNA-directed RNA polymerase Proteins 0.000 claims 3
- 241000283984 Rodentia Species 0.000 claims 3
- 101710108545 Viral protein 1 Proteins 0.000 claims 3
- 210000001638 cerebellum Anatomy 0.000 claims 3
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims 2
- 101100261165 Homo sapiens TPP1 gene Proteins 0.000 claims 2
- 101100426960 Homo sapiens TTPA gene Proteins 0.000 claims 2
- 208000002537 Neuronal Ceroid-Lipofuscinoses Diseases 0.000 claims 2
- 208000014060 Niemann-Pick disease Diseases 0.000 claims 2
- 210000004027 cell Anatomy 0.000 claims 2
- 210000004720 cerebrum Anatomy 0.000 claims 2
- 239000003623 enhancer Substances 0.000 claims 2
- 229940125721 immunosuppressive agent Drugs 0.000 claims 2
- 210000003140 lateral ventricle Anatomy 0.000 claims 2
- 210000005171 mammalian brain Anatomy 0.000 claims 2
- 208000011045 mucopolysaccharidosis type 3 Diseases 0.000 claims 2
- 208000025919 mucopolysaccharidosis type 7 Diseases 0.000 claims 2
- 210000001577 neostriatum Anatomy 0.000 claims 2
- 230000001537 neural effect Effects 0.000 claims 2
- 210000002442 prefrontal cortex Anatomy 0.000 claims 2
- 230000004044 response Effects 0.000 claims 2
- 208000024891 symptom Diseases 0.000 claims 2
- 210000001103 thalamus Anatomy 0.000 claims 2
- 102000007469 Actins Human genes 0.000 claims 1
- 108010085238 Actins Proteins 0.000 claims 1
- 241000649047 Adeno-associated virus 12 Species 0.000 claims 1
- 208000031277 Amaurotic familial idiocy Diseases 0.000 claims 1
- 108090000565 Capsid Proteins Proteins 0.000 claims 1
- 206010008025 Cerebellar ataxia Diseases 0.000 claims 1
- 102100023321 Ceruloplasmin Human genes 0.000 claims 1
- PMATZTZNYRCHOR-CGLBZJNRSA-N Cyclosporin A Chemical compound CC[C@@H]1NC(=O)[C@H]([C@H](O)[C@H](C)C\C=C\C)N(C)C(=O)[C@H](C(C)C)N(C)C(=O)[C@H](CC(C)C)N(C)C(=O)[C@H](CC(C)C)N(C)C(=O)[C@@H](C)NC(=O)[C@H](C)NC(=O)[C@H](CC(C)C)N(C)C(=O)[C@H](C(C)C)NC(=O)[C@H](CC(C)C)N(C)C(=O)CN(C)C1=O PMATZTZNYRCHOR-CGLBZJNRSA-N 0.000 claims 1
- 108010036949 Cyclosporine Proteins 0.000 claims 1
- 206010011777 Cystinosis Diseases 0.000 claims 1
- 102000010911 Enzyme Precursors Human genes 0.000 claims 1
- 108010062466 Enzyme Precursors Proteins 0.000 claims 1
- 208000024720 Fabry Disease Diseases 0.000 claims 1
- 201000008892 GM1 Gangliosidosis Diseases 0.000 claims 1
- 208000015872 Gaucher disease Diseases 0.000 claims 1
- 206010053185 Glycogen storage disease type II Diseases 0.000 claims 1
- 108090000604 Hydrolases Proteins 0.000 claims 1
- 102000004157 Hydrolases Human genes 0.000 claims 1
- 206010022520 Intention tremor Diseases 0.000 claims 1
- 201000011442 Metachromatic leukodystrophy Diseases 0.000 claims 1
- 208000008955 Mucolipidoses Diseases 0.000 claims 1
- 206010056893 Mucopolysaccharidosis VII Diseases 0.000 claims 1
- 241000288906 Primates Species 0.000 claims 1
- 208000021811 Sandhoff disease Diseases 0.000 claims 1
- 201000001828 Sly syndrome Diseases 0.000 claims 1
- 206010044565 Tremor Diseases 0.000 claims 1
- 210000004289 cerebral ventricle Anatomy 0.000 claims 1
- 229960001265 ciclosporin Drugs 0.000 claims 1
- 210000003703 cisterna magna Anatomy 0.000 claims 1
- 230000003920 cognitive function Effects 0.000 claims 1
- 229930182912 cyclosporin Natural products 0.000 claims 1
- 230000001934 delay Effects 0.000 claims 1
- 201000004502 glycogen storage disease II Diseases 0.000 claims 1
- 238000002347 injection Methods 0.000 claims 1
- 239000007924 injection Substances 0.000 claims 1
- 238000007913 intrathecal administration Methods 0.000 claims 1
- 238000007914 intraventricular administration Methods 0.000 claims 1
- 208000017476 juvenile neuronal ceroid lipofuscinosis Diseases 0.000 claims 1
- 230000004446 light reflex Effects 0.000 claims 1
- 230000002132 lysosomal effect Effects 0.000 claims 1
- HPNSFSBZBAHARI-UHFFFAOYSA-N micophenolic acid Natural products OC1=C(CC=C(C)CCC(O)=O)C(OC)=C(C)C2=C1C(=O)OC2 HPNSFSBZBAHARI-UHFFFAOYSA-N 0.000 claims 1
- 201000007769 mucolipidosis Diseases 0.000 claims 1
- 229940014456 mycophenolate Drugs 0.000 claims 1
- HPNSFSBZBAHARI-RUDMXATFSA-N mycophenolic acid Chemical compound OC1=C(C\C=C(/C)CCC(O)=O)C(OC)=C(C)C2=C1C(=O)OC2 HPNSFSBZBAHARI-RUDMXATFSA-N 0.000 claims 1
- 201000007607 neuronal ceroid lipofuscinosis 3 Diseases 0.000 claims 1
- 201000001119 neuropathy Diseases 0.000 claims 1
- 230000007823 neuropathy Effects 0.000 claims 1
- 230000003472 neutralizing effect Effects 0.000 claims 1
- 206010029864 nystagmus Diseases 0.000 claims 1
- 208000033808 peripheral neuropathy Diseases 0.000 claims 1
- 230000000272 proprioceptive effect Effects 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 230000001179 pupillary effect Effects 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022169285A JP2023002721A (ja) | 2016-11-04 | 2022-10-21 | 神経変性疾患を処置するための遺伝子移入用組成物、遺伝子導入法、及び遺伝子導入の使用 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662418033P | 2016-11-04 | 2016-11-04 | |
| US62/418,033 | 2016-11-04 | ||
| PCT/US2017/059986 WO2018085688A1 (en) | 2016-11-04 | 2017-11-03 | Gene transfer compositions, methods and uses for treating neurodegenerative diseases |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022169285A Division JP2023002721A (ja) | 2016-11-04 | 2022-10-21 | 神経変性疾患を処置するための遺伝子移入用組成物、遺伝子導入法、及び遺伝子導入の使用 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2019537576A JP2019537576A (ja) | 2019-12-26 |
| JP2019537576A5 true JP2019537576A5 (cg-RX-API-DMAC7.html) | 2020-12-03 |
Family
ID=62075988
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019522377A Pending JP2019537576A (ja) | 2016-11-04 | 2017-11-03 | 神経変性疾患を処置するための遺伝子移入用組成物、遺伝子導入法、及び遺伝子導入の使用 |
| JP2022169285A Pending JP2023002721A (ja) | 2016-11-04 | 2022-10-21 | 神経変性疾患を処置するための遺伝子移入用組成物、遺伝子導入法、及び遺伝子導入の使用 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022169285A Pending JP2023002721A (ja) | 2016-11-04 | 2022-10-21 | 神経変性疾患を処置するための遺伝子移入用組成物、遺伝子導入法、及び遺伝子導入の使用 |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20190269797A1 (cg-RX-API-DMAC7.html) |
| EP (1) | EP3534892A4 (cg-RX-API-DMAC7.html) |
| JP (2) | JP2019537576A (cg-RX-API-DMAC7.html) |
| CN (1) | CN110198712A (cg-RX-API-DMAC7.html) |
| AU (1) | AU2017355502B2 (cg-RX-API-DMAC7.html) |
| BR (1) | BR112019009074A2 (cg-RX-API-DMAC7.html) |
| CA (1) | CA3041548A1 (cg-RX-API-DMAC7.html) |
| MX (1) | MX2019005266A (cg-RX-API-DMAC7.html) |
| WO (1) | WO2018085688A1 (cg-RX-API-DMAC7.html) |
Families Citing this family (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9818239B2 (en) | 2015-08-20 | 2017-11-14 | Zendrive, Inc. | Method for smartphone-based accident detection |
| MY198804A (en) | 2017-06-07 | 2023-09-28 | Regeneron Pharma | Compositions and methods for internalizing enzymes |
| WO2019079807A1 (en) | 2017-10-20 | 2019-04-25 | Zendrive, Inc. | METHOD AND SYSTEM FOR VEHICULAR COMMUNICATIONS |
| BR112020016005A2 (pt) | 2018-02-07 | 2020-12-15 | Regeneron Pharmaceuticals, Inc. | Método de administração de uma proteína terapêutica ao sistema nervoso central, proteína terapêutica multidomínio, polinucleotídeo, vetor de terapia gênica, e, uso de um nucleotídeo que codifica a proteína terapêutica multidomínio |
| CN112135624B (zh) | 2018-05-17 | 2024-07-19 | 瑞泽恩制药公司 | 抗cd63抗体、缀合物和其用途 |
| WO2020068990A1 (en) | 2018-09-26 | 2020-04-02 | California Institute Of Technology | Adeno-associated virus compositions for targeted gene therapy |
| IL282808B2 (en) * | 2018-11-14 | 2025-02-01 | Regenxbio Inc | Gene therapy for neural ceroid lipofuscinosis |
| JP2022518931A (ja) * | 2019-02-01 | 2022-03-17 | スパーク セラピューティクス インコーポレイテッド | 遅発型小児性神経セロイドリポフスチン沈着症２型に対するａａｖベクター治療法 |
| JP2023504269A (ja) | 2019-12-03 | 2023-02-02 | ゼンドライヴ，インコーポレイテッド | ルートのリスク判定方法およびシステム |
| EP4118219A4 (en) * | 2020-03-11 | 2024-04-17 | Shanghai Belief-Delivery Biomed Co., Ltd. | Novel use of aspirin compound in increasing nucleic acid expression |
| WO2025231406A1 (en) * | 2024-05-02 | 2025-11-06 | The Children's Hospital Of Philadelphia | Methods to increase transduction of ependyma cells in brain |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20070003518A1 (en) * | 2002-04-19 | 2007-01-04 | Atkinson Mark A | Raav vector-based compositions and methods for the prevention and treatment of mammalian diseases |
| US20040258666A1 (en) * | 2003-05-01 | 2004-12-23 | Passini Marco A. | Gene therapy for neurometabolic disorders |
| KR20070004637A (ko) * | 2004-01-22 | 2007-01-09 | 가부시키가이샤 디나벡크 겐큐쇼 | 바이러스 벡터의 제조방법 |
| EP3456331B1 (en) * | 2006-02-08 | 2021-05-26 | Genzyme Corporation | Gene therapy for niemann-pick disease type a |
| ES2635726T3 (es) * | 2007-06-06 | 2017-10-04 | Genzyme Corporation | Terapia génica para enfermedades de almacenamiento lisosómico |
| PT2691529T (pt) * | 2011-03-31 | 2019-09-27 | Univ Iowa Res Found | Partícula de aav2 que compreende uma proteína do capsídeo de aav2 e um vetor que compreende um ácido nucleico que codifica uma tripeptidil peptidase 1 (tpp1) para utilização no tratamento da lipofuscinose ceroide infantil tardia (lincl) num mamífero não roedor por injeção intraventricular ou entrega icv |
| KR102380265B1 (ko) * | 2013-07-22 | 2022-03-29 | 더 칠드런스 호스피탈 오브 필라델피아 | 변종 ａａｖ 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도 |
| EP3024497B1 (en) * | 2013-07-26 | 2021-01-13 | University of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| EP3060669B1 (en) * | 2013-10-24 | 2024-12-11 | uniQure IP B.V. | Aav-5 pseudotyped vector for gene therapy for neurological diseases |
| KR20160079891A (ko) * | 2013-11-20 | 2016-07-06 | 유니버시티 오브 아이오와 리써치 파운데이션 | 아밀로이드 침착의 치료를 위한 방법 및 조성물 |
| SG11201803218PA (en) * | 2015-10-23 | 2018-05-30 | Univ Iowa Res Found | Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection |
| IL270422B2 (en) * | 2017-05-11 | 2025-04-01 | Univ Pennsylvania | Gene therapy for neuronal ceroid lipofuscinosis |
-
2017
- 2017-11-03 JP JP2019522377A patent/JP2019537576A/ja active Pending
- 2017-11-03 BR BR112019009074A patent/BR112019009074A2/pt active Search and Examination
- 2017-11-03 CN CN201780067919.4A patent/CN110198712A/zh active Pending
- 2017-11-03 WO PCT/US2017/059986 patent/WO2018085688A1/en not_active Ceased
- 2017-11-03 EP EP17867272.1A patent/EP3534892A4/en not_active Withdrawn
- 2017-11-03 US US16/344,298 patent/US20190269797A1/en active Pending
- 2017-11-03 MX MX2019005266A patent/MX2019005266A/es unknown
- 2017-11-03 CA CA3041548A patent/CA3041548A1/en active Pending
- 2017-11-03 AU AU2017355502A patent/AU2017355502B2/en not_active Ceased
-
2022
- 2022-10-21 JP JP2022169285A patent/JP2023002721A/ja active Pending