JPWO2020197967A5 - - Google Patents
Download PDFInfo
- Publication number
- 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
- Authority
- JP
- Japan
- Prior art keywords
- composition
- disease
- administered
- effective amount
- subject
- 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
Links
- 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 (3)
| Publication Number | Publication Date |
|---|---|
| JP2022525888A JP2022525888A (ja) | 2022-05-20 |
| JPWO2020197967A5 true JPWO2020197967A5 (https=) | 2023-03-28 |
| JP2022525888A5 JP2022525888A5 (https=) | 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 (https=) |
| EP (1) | EP3941584A4 (https=) |
| JP (1) | JP2022525888A (https=) |
| KR (1) | KR20210143848A (https=) |
| CN (1) | CN113811359A (https=) |
| AU (1) | AU2020245415B2 (https=) |
| CA (1) | CA3132379A1 (https=) |
| EA (1) | EA202192328A1 (https=) |
| WO (1) | WO2020197967A1 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3121785A1 (en) * | 2018-12-05 | 2020-06-11 | Washington University | Methods of detecting, preventing, reversing, and treating neurological diseases |
| CN116723870A (zh) * | 2021-01-20 | 2023-09-08 | 拉什大学医学中心 | 球形细胞脑白质营养不良症或克拉伯病的改良治疗 |
| JP2026504932A (ja) * | 2023-01-18 | 2026-02-10 | ラッシュ・ユニバーシティ・メディカル・センター | Ｃｌｎ３関連若年型神経セロイドリポフスチン症の治療のための経口ゲムフィブロジル |
Family Cites Families (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN100402026C (zh) * | 2002-06-03 | 2008-07-16 | 诺瓦提斯公司 | 取代的氰基吡咯烷和含有它们的组合制剂用于治疗高脂血症和相关疾病的用途 |
| WO2008049058A2 (en) * | 2006-10-18 | 2008-04-24 | 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 |
| WO2016081365A1 (en) * | 2014-11-19 | 2016-05-26 | Rush University Medical Center | Compositions and methods for treating lysosomal disorders |
| CN107249646B (zh) * | 2014-12-16 | 2021-06-29 | 内布拉斯加大学董事会 | 用于青少年巴滕病的基因疗法 |
| HK1256341A1 (zh) * | 2015-05-15 | 2019-09-20 | 明尼苏达大学董事会 | 用於治疗性递送到中枢神经系统的腺相关物 |
| US20180236105A1 (en) * | 2015-10-23 | 2018-08-23 | 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 |
| US20220323612A1 (en) * | 2016-06-13 | 2022-10-13 | Bluebird Bio, Inc. | Gene therapy of neuronal ceroid lipofuscinoses |
| RU2764919C2 (ru) * | 2016-06-13 | 2022-01-24 | Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл | Оптимизированные гены и экспрессионные кассеты cln1, и их применение |
| BR112019010210A2 (pt) * | 2016-12-29 | 2019-09-03 | Rush University Medical Center | melhora na atividade locomotora e aumento da longevidade de indivíduos com lipofuscinose ceroide neuronal infantil tardia por gemfibrozil |
| US12599636B2 (en) * | 2017-01-20 | 2026-04-14 | University of Pittsburgh—of the Commonwealth System of Higher Education | Treatment of Krabbe disease with umbilical cord blood transplantion (UCBT) and increased galactocerebrosidase (GALC) expression |
| US12454701B2 (en) * | 2018-12-05 | 2025-10-28 | Abeona Therapeutics Inc. | Recombinant adeno-associated viral vector for gene delivery |
| MX2021009250A (es) * | 2019-02-01 | 2021-11-04 | Spark Therapeutics Inc | Metodos de tratamiento por vectores aav para la lipofuscinosis ceroidea neuronal infantil tardia tipo 2. |
-
2020
- 2020-03-20 KR KR1020217034175A patent/KR20210143848A/ko active Pending
- 2020-03-20 WO PCT/US2020/023768 patent/WO2020197967A1/en not_active Ceased
- 2020-03-20 EP EP20777092.6A patent/EP3941584A4/en not_active Withdrawn
- 2020-03-20 CA CA3132379A patent/CA3132379A1/en active Pending
- 2020-03-20 JP JP2021555592A patent/JP2022525888A/ja active Pending
- 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 EA EA202192328A patent/EA202192328A1/ru unknown
- 2020-03-20 AU AU2020245415A patent/AU2020245415B2/en active Active
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| EP1492519B1 (en) | Methods for treating lower urinary tract disorders using antimuscarinics and alpha-2-delta subunit calcium channel modulators | |
| TWI843711B (zh) | 用於治療憂鬱症的艾司氯胺酮（ｅｓｋｅｔａｍｉｎｅ） | |
| JP2020158521A5 (https=) | ||
| JP2023120246A5 (https=) | ||
| WO1988004929A1 (en) | Nasal delivery of caffeine | |
| US20250319069A1 (en) | Methods for treating depressive states | |
| US20030060422A1 (en) | Tannate compositions and methods of treatment | |
| US20060264509A1 (en) | Methods for treating pain using smooth muscle modulators and a2 subunit calcium channel modulators | |
| Shapiro et al. | Plasminogen, human‐tvmh for the treatment of children and adults with plasminogen deficiency type 1 | |
| LaForce et al. | A randomized, double-blind, parallel-group, multicenter, placebo-controlled study of the safety and efficacy of extended-release guaifenesin/pseudoephedrine hydrochloride for symptom relief as an adjunctive therapy to antibiotic treatment of acute respiratory infections | |
| JPWO2020197967A5 (https=) | ||
| TW200306804A (en) | Ambroxol for the treatment of painful conditions in the mouth and pharyngeal cavity | |
| US20240091178A1 (en) | Method of treating a person afflicted with a respiratory condition and pharmaceutical formulation including dapsone | |
| WO2025195268A1 (zh) | 药物组合物在治疗糖尿病周围神经病变上的应用 | |
| CN1223338C (zh) | 盐酸西替利嗪凝胶剂 | |
| CN1055620C (zh) | 氢碘酸水溶液在制备催欲药中的用途 | |
| US20060276542A1 (en) | Methods for treating functional bowel disorders using alpha2 subunit calcium channel modulators with smooth muscle modulators | |
| Allenby et al. | PERINAL—a new no-touch spray to relieve the symptoms of pruritus ani | |
| WO2021209620A1 (en) | Cysteine protease inhibitors for use in the prevention and/or treatment of coronavirus | |
| Krenn et al. | Efficacy of solnatide to treat pulmonary permeability edema in SARS-CoV-2 positive patients with moderate to severe ARDS: A randomized controlled pilot-trial | |
| US20250186454A1 (en) | Methods for treating aberrant behavior and motor activity | |
| US20250352554A1 (en) | Intranasal olanzapine formulations and methods of their use | |
| CN109718230A (zh) | 丹酚酸a在制备抗高尿酸血症和抗痛风药物中的应用 | |
| CN1839834A (zh) | 一种防治眼疲劳及过敏的眼药 | |
| CN114831990A (zh) | 青藤碱在制备治疗帕金森病药物组合物中的应用 |