JP2019529385A5 - - Google Patents
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- Publication number
- JP2019529385A5 JP2019529385A5 JP2019512292A JP2019512292A JP2019529385A5 JP 2019529385 A5 JP2019529385 A5 JP 2019529385A5 JP 2019512292 A JP2019512292 A JP 2019512292A JP 2019512292 A JP2019512292 A JP 2019512292A JP 2019529385 A5 JP2019529385 A5 JP 2019529385A5
- Authority
- JP
- Japan
- Prior art keywords
- use according
- mammal
- cells
- cns
- therapeutic protein
- 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
- 210000003169 central nervous system Anatomy 0.000 claims 16
- 230000001225 therapeutic effect Effects 0.000 claims 16
- 210000004027 cell Anatomy 0.000 claims 15
- 102000004169 proteins and genes Human genes 0.000 claims 15
- 108090000623 proteins and genes Proteins 0.000 claims 15
- 210000001519 tissue Anatomy 0.000 claims 15
- 241000124008 Mammalia Species 0.000 claims 14
- 210000000056 organ Anatomy 0.000 claims 12
- 108010025628 Apolipoproteins E Proteins 0.000 claims 7
- 102000013918 Apolipoproteins E Human genes 0.000 claims 7
- 241000702421 Dependoparvovirus Species 0.000 claims 7
- 102000001708 Protein Isoforms Human genes 0.000 claims 7
- 108010029485 Protein Isoforms Proteins 0.000 claims 7
- 239000013598 vector Substances 0.000 claims 7
- 239000003814 drug Substances 0.000 claims 6
- 239000002245 particle Substances 0.000 claims 6
- 229940124597 therapeutic agent Drugs 0.000 claims 6
- 210000005252 bulbus oculi Anatomy 0.000 claims 5
- 108020004707 nucleic acids Proteins 0.000 claims 5
- 102000039446 nucleic acids Human genes 0.000 claims 5
- 150000007523 nucleic acids Chemical class 0.000 claims 5
- 230000001681 protective effect Effects 0.000 claims 5
- 108090000565 Capsid Proteins Proteins 0.000 claims 4
- 102100023321 Ceruloplasmin Human genes 0.000 claims 4
- 101000901683 Homo sapiens Battenin Proteins 0.000 claims 3
- 101000710215 Homo sapiens Ceroid-lipofuscinosis neuronal protein 6 Proteins 0.000 claims 3
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 3
- 108091081062 Repeated sequence (DNA) Proteins 0.000 claims 3
- CTMZLDSMFCVUNX-VMIOUTBZSA-N cytidylyl-(3'->5')-guanosine Chemical compound O=C1N=C(N)C=CN1[C@H]1[C@H](O)[C@H](OP(O)(=O)OC[C@@H]2[C@H]([C@@H](O)[C@@H](O2)N2C3=C(C(N=C(N)N3)=O)N=C2)O)[C@@H](CO)O1 CTMZLDSMFCVUNX-VMIOUTBZSA-N 0.000 claims 3
- 201000010099 disease Diseases 0.000 claims 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 3
- 210000004185 liver Anatomy 0.000 claims 3
- 201000007607 neuronal ceroid lipofuscinosis 3 Diseases 0.000 claims 3
- 210000003463 organelle Anatomy 0.000 claims 3
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 2
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 2
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 2
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 2
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 2
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 2
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 2
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 2
- 241000649046 Adeno-associated virus 11 Species 0.000 claims 2
- 241000649047 Adeno-associated virus 12 Species 0.000 claims 2
- 102000053187 Glucuronidase Human genes 0.000 claims 2
- 108010060309 Glucuronidase Proteins 0.000 claims 2
- 101000710213 Homo sapiens Protein CLN8 Proteins 0.000 claims 2
- 108020002591 Palmitoyl protein thioesterase Proteins 0.000 claims 2
- 102000005327 Palmitoyl protein thioesterase Human genes 0.000 claims 2
- 108010039203 Tripeptidyl-Peptidase 1 Proteins 0.000 claims 2
- 102100034197 Tripeptidyl-peptidase 1 Human genes 0.000 claims 2
- 230000009286 beneficial effect Effects 0.000 claims 2
- 210000001508 eye Anatomy 0.000 claims 2
- 208000017476 juvenile neuronal ceroid lipofuscinosis Diseases 0.000 claims 2
- 210000005229 liver cell Anatomy 0.000 claims 2
- 239000000203 mixture Substances 0.000 claims 2
- 208000024827 Alzheimer disease Diseases 0.000 claims 1
- 208000031277 Amaurotic familial idiocy Diseases 0.000 claims 1
- 108010060219 Apolipoprotein E2 Proteins 0.000 claims 1
- 101000933465 Homo sapiens Beta-glucuronidase Proteins 0.000 claims 1
- 101000574223 Homo sapiens Palmitoyl-protein thioesterase 1 Proteins 0.000 claims 1
- 101100261165 Homo sapiens TPP1 gene Proteins 0.000 claims 1
- 101100426960 Homo sapiens TTPA gene Proteins 0.000 claims 1
- 208000015439 Lysosomal storage disease Diseases 0.000 claims 1
- 241001465754 Metazoa Species 0.000 claims 1
- 208000002678 Mucopolysaccharidoses Diseases 0.000 claims 1
- 208000002537 Neuronal Ceroid-Lipofuscinoses Diseases 0.000 claims 1
- 208000017478 adult neuronal ceroid lipofuscinosis Diseases 0.000 claims 1
- 230000003542 behavioural effect Effects 0.000 claims 1
- 210000004556 brain Anatomy 0.000 claims 1
- 208000015114 central nervous system disease Diseases 0.000 claims 1
- 230000008021 deposition Effects 0.000 claims 1
- 210000003890 endocrine cell Anatomy 0.000 claims 1
- 239000000945 filler Substances 0.000 claims 1
- 210000003494 hepatocyte Anatomy 0.000 claims 1
- 102000056242 human CLN3 Human genes 0.000 claims 1
- 102000045627 human CLN6 Human genes 0.000 claims 1
- 102000051672 human PPT1 Human genes 0.000 claims 1
- 210000005228 liver tissue Anatomy 0.000 claims 1
- 206010028093 mucopolysaccharidosis Diseases 0.000 claims 1
- 208000025919 mucopolysaccharidosis type 7 Diseases 0.000 claims 1
- 230000001537 neural effect Effects 0.000 claims 1
- 210000004923 pancreatic tissue Anatomy 0.000 claims 1
- 230000007310 pathophysiology Effects 0.000 claims 1
- 108091033319 polynucleotide Proteins 0.000 claims 1
- 102000040430 polynucleotide Human genes 0.000 claims 1
- 239000002157 polynucleotide Substances 0.000 claims 1
- 230000008092 positive effect Effects 0.000 claims 1
- 208000024891 symptom Diseases 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022002163A JP2022050574A (ja) | 2016-09-02 | 2022-01-11 | Ｃｎｓ障害を治療するための方法及びベクター |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662383274P | 2016-09-02 | 2016-09-02 | |
| US62/383,274 | 2016-09-02 | ||
| PCT/US2017/049959 WO2018045347A1 (en) | 2016-09-02 | 2017-09-01 | Methods and vectors for treating cns disorders |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022002163A Division JP2022050574A (ja) | 2016-09-02 | 2022-01-11 | Ｃｎｓ障害を治療するための方法及びベクター |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019529385A JP2019529385A (ja) | 2019-10-17 |
| JP2019529385A5 true JP2019529385A5 (enExample) | 2020-09-17 |
| JP7610923B2 JP7610923B2 (ja) | 2025-01-09 |
Family
ID=61309371
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019512292A Active JP7610923B2 (ja) | 2016-09-02 | 2017-09-01 | Ｃｎｓ障害を治療するための方法及びベクター |
| JP2022002163A Pending JP2022050574A (ja) | 2016-09-02 | 2022-01-11 | Ｃｎｓ障害を治療するための方法及びベクター |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022002163A Pending JP2022050574A (ja) | 2016-09-02 | 2022-01-11 | Ｃｎｓ障害を治療するための方法及びベクター |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20190192693A1 (enExample) |
| EP (1) | EP3506930A4 (enExample) |
| JP (2) | JP7610923B2 (enExample) |
| CN (1) | CN110121356A (enExample) |
| AU (2) | AU2017318717B2 (enExample) |
| BR (1) | BR112019004353A2 (enExample) |
| CA (1) | CA3035628A1 (enExample) |
| MX (1) | MX2019002518A (enExample) |
| WO (1) | WO2018045347A1 (enExample) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| BR112020000995A2 (pt) * | 2017-07-17 | 2020-09-08 | Spark Therapeutics, Inc. | métodos e usos de aférese |
| BR112020025268A2 (pt) | 2018-06-28 | 2021-03-09 | The University Of North Carolina At Chapel Hill | Cassetes de expressão e genes cln5 otimizados e seu uso |
| JP7624724B2 (ja) | 2018-09-26 | 2025-01-31 | カリフォルニア インスティテュート オブ テクノロジー | 標的遺伝子療法のためのアデノ随伴ウイルス組成物 |
| SG11202104295UA (en) * | 2018-11-14 | 2021-06-29 | Regenxbio Inc | Gene therapy for neuronal ceroid lipofuscinoses |
| BR112021010234A2 (pt) * | 2018-11-28 | 2021-08-24 | Prevail Therapeutics, Inc. | Terapias de gene para doença neurodegenerativa |
| BR112021015817A2 (pt) | 2019-02-22 | 2021-10-13 | The Trustees Of The University Of Pennsylvania | Vírus adeno-associado recombinante para tratamento de neurodegeneração com início na idade adulta associado a grn |
| WO2021061909A1 (en) * | 2019-09-24 | 2021-04-01 | The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone | Method of targeting neuronal apoe to treat a neurocognitive disorder |
| AU2020367437B2 (en) * | 2019-10-16 | 2024-11-07 | Cornell University | Gene therapy for Alzheimer's disease |
| CN116528887A (zh) * | 2020-10-01 | 2023-08-01 | 美商圣加莫治疗股份有限公司 | 检测抗aav抗体的方法 |
| CN112852881B (zh) * | 2021-02-09 | 2023-08-15 | 南京贝思奥生物科技有限公司 | 利用细胞穿膜肽增强腺相关病毒在中枢神经系统转导效率的方法 |
| TW202426479A (zh) * | 2022-10-10 | 2024-07-01 | 美商星火治療公司 | Apoe基因療法 |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7135339B2 (en) * | 2003-11-20 | 2006-11-14 | University Of Iowa Research Foundation | Methods for producing and using in vivo pseudotyped retroviruses using envelope glycoproteins from lymphocytic choriomeningitis virus (LCMV) |
| US10738326B2 (en) * | 2010-10-27 | 2020-08-11 | Jichi Medical University | Adeno-associated virus vector for gene transfer to nervous system cells |
| BR112014028666B1 (pt) * | 2012-05-18 | 2021-12-07 | The General Hospital Corporation | Uso de uma partícula de raav2, composição farmacêutica e kit |
| US10391184B2 (en) * | 2013-07-26 | 2019-08-27 | University Of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| MX2016006584A (es) * | 2013-11-20 | 2016-09-06 | Univ Iowa Res Found | Métodos y composiciones para tratar los depósitos de amiloide. |
| CN108347932B (zh) * | 2015-02-20 | 2022-09-13 | 衣阿华大学研究基金会 | 用于治疗遗传性眼病的方法和组合物 |
-
2017
- 2017-09-01 BR BR112019004353A patent/BR112019004353A2/pt unknown
- 2017-09-01 MX MX2019002518A patent/MX2019002518A/es unknown
- 2017-09-01 AU AU2017318717A patent/AU2017318717B2/en active Active
- 2017-09-01 CA CA3035628A patent/CA3035628A1/en active Pending
- 2017-09-01 CN CN201780067677.9A patent/CN110121356A/zh active Pending
- 2017-09-01 US US16/329,697 patent/US20190192693A1/en not_active Abandoned
- 2017-09-01 WO PCT/US2017/049959 patent/WO2018045347A1/en not_active Ceased
- 2017-09-01 JP JP2019512292A patent/JP7610923B2/ja active Active
- 2017-09-01 EP EP17847658.6A patent/EP3506930A4/en active Pending
-
2022
- 2022-01-11 JP JP2022002163A patent/JP2022050574A/ja active Pending
-
2024
- 2024-10-06 AU AU2024227138A patent/AU2024227138A1/en not_active Abandoned
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