JPWO2020197967A5 - - Google Patents
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- JPWO2020197967A5 JPWO2020197967A5 JP2021555592A JP2021555592A JPWO2020197967A5 JP WO2020197967 A5 JPWO2020197967 A5 JP WO2020197967A5 JP 2021555592 A JP2021555592 A JP 2021555592A JP 2021555592 A JP2021555592 A JP 2021555592A JP WO2020197967 A5 JPWO2020197967 A5 JP WO2020197967A5
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- JP
- Japan
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- 238000000034 method Methods 0.000 claims 21
- 108090000623 proteins and genes Proteins 0.000 claims 4
- 102000004190 Enzymes Human genes 0.000 claims 3
- 108090000790 Enzymes Proteins 0.000 claims 3
- SHGAZHPCJJPHSC-YCNIQYBTSA-N all-trans-retinoic acid Chemical compound OC(=O)\C=C(/C)\C=C\C=C(/C)\C=C\C1=C(C)CCCC1(C)C SHGAZHPCJJPHSC-YCNIQYBTSA-N 0.000 claims 3
- 229940079593 drug Drugs 0.000 claims 3
- 239000003814 drug Substances 0.000 claims 3
- 230000002132 lysosomal effect Effects 0.000 claims 3
- 229930002330 retinoic acid Natural products 0.000 claims 3
- 239000013603 viral vector Substances 0.000 claims 3
- 208000015439 Lysosomal storage disease Diseases 0.000 claims 2
- 208000002537 Neuronal Ceroid-Lipofuscinoses Diseases 0.000 claims 2
- 239000003795 chemical substances by application Substances 0.000 claims 2
- 229940125753 fibrate Drugs 0.000 claims 2
- WBYWAXJHAXSJNI-VOTSOKGWSA-M .beta-Phenylacrylic acid Natural products [O-]C(=O)\C=C\C1=CC=CC=C1 WBYWAXJHAXSJNI-VOTSOKGWSA-M 0.000 claims 1
- 208000031277 Amaurotic familial idiocy Diseases 0.000 claims 1
- 102100022440 Battenin Human genes 0.000 claims 1
- 101710199232 Battenin Proteins 0.000 claims 1
- 102100022548 Beta-hexosaminidase subunit alpha Human genes 0.000 claims 1
- WBYWAXJHAXSJNI-SREVYHEPSA-N Cinnamic acid Chemical compound OC(=O)\C=C/C1=CC=CC=C1 WBYWAXJHAXSJNI-SREVYHEPSA-N 0.000 claims 1
- 208000024720 Fabry Disease Diseases 0.000 claims 1
- 208000001948 Farber Lipogranulomatosis Diseases 0.000 claims 1
- 208000033149 Farber disease Diseases 0.000 claims 1
- 208000015872 Gaucher disease Diseases 0.000 claims 1
- HEMJJKBWTPKOJG-UHFFFAOYSA-N Gemfibrozil Chemical group CC1=CC=C(C)C(OCCCC(C)(C)C(O)=O)=C1 HEMJJKBWTPKOJG-UHFFFAOYSA-N 0.000 claims 1
- 208000010055 Globoid Cell Leukodystrophy Diseases 0.000 claims 1
- 102000016871 Hexosaminidase A Human genes 0.000 claims 1
- 108010053317 Hexosaminidase A Proteins 0.000 claims 1
- 208000028226 Krabbe disease Diseases 0.000 claims 1
- 208000014060 Niemann-Pick disease Diseases 0.000 claims 1
- 108020002591 Palmitoyl protein thioesterase Proteins 0.000 claims 1
- 102100025824 Palmitoyl-protein thioesterase 1 Human genes 0.000 claims 1
- 208000022292 Tay-Sachs disease Diseases 0.000 claims 1
- 108010039203 Tripeptidyl-Peptidase 1 Proteins 0.000 claims 1
- 102100034197 Tripeptidyl-peptidase 1 Human genes 0.000 claims 1
- 230000008499 blood brain barrier function Effects 0.000 claims 1
- 210000001218 blood-brain barrier Anatomy 0.000 claims 1
- 229930016911 cinnamic acid Natural products 0.000 claims 1
- 235000013985 cinnamic acid Nutrition 0.000 claims 1
- 229960002297 fenofibrate Drugs 0.000 claims 1
- YMTINGFKWWXKFG-UHFFFAOYSA-N fenofibrate Chemical compound C1=CC(OC(C)(C)C(=O)OC(C)C)=CC=C1C(=O)C1=CC=C(Cl)C=C1 YMTINGFKWWXKFG-UHFFFAOYSA-N 0.000 claims 1
- 229960003627 gemfibrozil Drugs 0.000 claims 1
- 208000017476 juvenile neuronal ceroid lipofuscinosis Diseases 0.000 claims 1
- WBYWAXJHAXSJNI-UHFFFAOYSA-N methyl p-hydroxycinnamate Natural products OC(=O)C=CC1=CC=CC=C1 WBYWAXJHAXSJNI-UHFFFAOYSA-N 0.000 claims 1
- DDOVBCWVTOHGCU-QMXMISKISA-N n-[(e,2s,3r)-3-hydroxy-1-[(2r,3r,4s,5r,6r)-3,4,5-trihydroxy-6-(hydroxymethyl)oxan-2-yl]oxynonadec-4-en-2-yl]octadecanamide Chemical compound CCCCCCCCCCCCCCCCCC(=O)N[C@H]([C@H](O)\C=C\CCCCCCCCCCCCCC)CO[C@@H]1O[C@H](CO)[C@H](O)[C@H](O)[C@H]1O DDOVBCWVTOHGCU-QMXMISKISA-N 0.000 claims 1
- 201000007607 neuronal ceroid lipofuscinosis 3 Diseases 0.000 claims 1
- FATBGEAMYMYZAF-KTKRTIGZSA-N oleamide Chemical compound CCCCCCCC\C=C/CCCCCCCC(N)=O FATBGEAMYMYZAF-KTKRTIGZSA-N 0.000 claims 1
- FATBGEAMYMYZAF-UHFFFAOYSA-N oleicacidamide-heptaglycolether Natural products CCCCCCCCC=CCCCCCCCC(N)=O FATBGEAMYMYZAF-UHFFFAOYSA-N 0.000 claims 1
- 230000001225 therapeutic effect Effects 0.000 claims 1
- 241000701161 unidentified adenovirus Species 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962822310P | 2019-03-22 | 2019-03-22 | |
| US62/822,310 | 2019-03-22 | ||
| PCT/US2020/023768 WO2020197967A1 (en) | 2019-03-22 | 2020-03-20 | Combination of nasal gene delivery and oral cinnamic acid, oleamide or gemfibrozil for lysosomal stoarge disorders |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022525888A JP2022525888A (ja) | 2022-05-20 |
| JPWO2020197967A5 true JPWO2020197967A5 (enExample) | 2023-03-28 |
Family
ID=72609912
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021555592A Pending JP2022525888A (ja) | 2019-03-22 | 2020-03-20 | リソソーム蓄積症のための、経鼻遺伝子送達と経口の桂皮酸、オレアミド、またはゲムフィブロジルとの組合せ |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20220152165A1 (enExample) |
| EP (1) | EP3941584A4 (enExample) |
| JP (1) | JP2022525888A (enExample) |
| KR (1) | KR20210143848A (enExample) |
| CN (1) | CN113811359A (enExample) |
| AU (1) | AU2020245415B2 (enExample) |
| CA (1) | CA3132379A1 (enExample) |
| EA (1) | EA202192328A1 (enExample) |
| WO (1) | WO2020197967A1 (enExample) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2020118056A1 (en) * | 2018-12-05 | 2020-06-11 | Washington University | Methods of detecting, preventing, reversing, and treating neurological diseases |
| US20240082184A1 (en) * | 2021-01-20 | 2024-03-14 | Rush University Medical Center | Improved treatment for globoid cell leukodsytrophy or krabbe disease |
| WO2024155759A1 (en) * | 2023-01-18 | 2024-07-25 | Rush University Medical Center | Oral gemfibrozil for the treatment of cln3-related juvenile neuronal ceroid lipofuscinosis |
Family Cites Families (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DE60326232D1 (de) * | 2002-06-03 | 2009-04-02 | Novartis Ag | Verwendung von substituierten cyanopyrrolidinen zur behandlung von hyperlipidämie |
| US20110166074A1 (en) * | 2006-10-18 | 2011-07-07 | Cornell Research Foundation, Inc. | Cln2 treatment of alzheimer's disease |
| WO2012177997A1 (en) * | 2011-06-22 | 2012-12-27 | The General Hospital Corporation | Treatment of proteinopathies |
| WO2014089449A1 (en) * | 2012-12-07 | 2014-06-12 | Rush University Nedical Center | Composition and method for treating neuronal ceroid lipofuscinosis |
| US20170354666A1 (en) * | 2014-11-19 | 2017-12-14 | Rush University Medical Center | Compositions and methods for treating lysosomal disorders |
| CA2967468A1 (en) * | 2014-12-16 | 2016-06-23 | Board Of Regents Of The University Of Nebraska | Gene therapy for juvenile batten disease |
| MX2017014443A (es) * | 2015-05-15 | 2018-08-01 | Univ Minnesota | Adeno-asociado para administracion terapeutica al sistema nervioso central. |
| AU2016341428B2 (en) * | 2015-10-23 | 2021-12-02 | University Of Iowa Research Foundation | Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection |
| US11045557B2 (en) * | 2016-06-09 | 2021-06-29 | Queen's University At Kingston | Methods and gene therapy constructs for treating GM2 gangliosidoses |
| CN109475619A (zh) * | 2016-06-13 | 2019-03-15 | 蓝鸟生物公司 | 神经元蜡样脂褐质沉积症的基因治疗 |
| US11504435B2 (en) * | 2016-06-13 | 2022-11-22 | The University Of North Carolina At Chapel Hill | Optimized CLN1 genes and expression cassettes and their use |
| CN110167545A (zh) * | 2016-12-29 | 2019-08-23 | 拉什大学医学中心 | 吉非罗齐对晚期婴儿型神经元蜡样质脂褐质沉积症患者寿命的增加和自发活动的改善 |
| CN110168085A (zh) * | 2017-01-20 | 2019-08-23 | 联邦高等教育系统匹兹堡大学 | 用脐带血移植(ucbt)和增加的半乳糖脑苷酯酶(galc)表达治疗克拉伯病 |
| WO2020117898A1 (en) * | 2018-12-05 | 2020-06-11 | Abeona Therapeutics Inc. | Recombinant adeno-associated viral vector for gene delivery |
| CA3127889A1 (en) * | 2019-02-01 | 2020-08-06 | Spark Therapeutics, Inc. | Aav vector treatment methods for late infantile neuronal ceroid lipofuscinosis type 2 |
-
2020
- 2020-03-20 EP EP20777092.6A patent/EP3941584A4/en not_active Withdrawn
- 2020-03-20 JP JP2021555592A patent/JP2022525888A/ja active Pending
- 2020-03-20 WO PCT/US2020/023768 patent/WO2020197967A1/en not_active Ceased
- 2020-03-20 CA CA3132379A patent/CA3132379A1/en active Pending
- 2020-03-20 AU AU2020245415A patent/AU2020245415B2/en active Active
- 2020-03-20 CN CN202080022852.4A patent/CN113811359A/zh active Pending
- 2020-03-20 US US17/441,029 patent/US20220152165A1/en active Pending
- 2020-03-20 KR KR1020217034175A patent/KR20210143848A/ko active Pending
- 2020-03-20 EA EA202192328A patent/EA202192328A1/ru unknown
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