JP2013544263A5 - - Google Patents
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- JP2013544263A5 JP2013544263A5 JP2013541210A JP2013541210A JP2013544263A5 JP 2013544263 A5 JP2013544263 A5 JP 2013544263A5 JP 2013541210 A JP2013541210 A JP 2013541210A JP 2013541210 A JP2013541210 A JP 2013541210A JP 2013544263 A5 JP2013544263 A5 JP 2013544263A5
- Authority
- JP
- Japan
- Prior art keywords
- disease
- group
- medicament according
- mucopolysaccharidosis
- gangliosidosis
- 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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- 239000003814 drug Substances 0.000 claims 16
- 101100507655 Canis lupus familiaris HSPA1 gene Proteins 0.000 claims 11
- 208000002678 Mucopolysaccharidoses Diseases 0.000 claims 9
- 201000010099 disease Diseases 0.000 claims 9
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 9
- 206010028093 mucopolysaccharidosis Diseases 0.000 claims 9
- 102100022548 Beta-hexosaminidase subunit alpha Human genes 0.000 claims 6
- 208000008955 Mucolipidoses Diseases 0.000 claims 6
- 208000002537 Neuronal Ceroid-Lipofuscinoses Diseases 0.000 claims 5
- 150000002443 hydroxylamines Chemical class 0.000 claims 5
- 201000008892 GM1 Gangliosidosis Diseases 0.000 claims 4
- 206010056886 Mucopolysaccharidosis I Diseases 0.000 claims 4
- 208000021811 Sandhoff disease Diseases 0.000 claims 4
- 208000022292 Tay-Sachs disease Diseases 0.000 claims 4
- 208000001905 GM2 Gangliosidoses Diseases 0.000 claims 3
- 201000008905 GM2 gangliosidosis Diseases 0.000 claims 3
- 208000009796 Gangliosidoses Diseases 0.000 claims 3
- 101001045440 Homo sapiens Beta-hexosaminidase subunit alpha Proteins 0.000 claims 3
- 201000008333 alpha-mannosidosis Diseases 0.000 claims 3
- 239000012634 fragment Substances 0.000 claims 3
- 201000006440 gangliosidosis Diseases 0.000 claims 3
- 208000007345 glycogen storage disease Diseases 0.000 claims 3
- 208000030159 metabolic disease Diseases 0.000 claims 3
- 108090000790 Enzymes Proteins 0.000 claims 2
- 102000004190 Enzymes Human genes 0.000 claims 2
- 208000006562 Glycogen Storage Disease Type VII Diseases 0.000 claims 2
- 208000032007 Glycogen storage disease due to acid maltase deficiency Diseases 0.000 claims 2
- 208000032008 Glycogen storage disease due to glycogen debranching enzyme deficiency Diseases 0.000 claims 2
- 208000032000 Glycogen storage disease due to muscle glycogen phosphorylase deficiency Diseases 0.000 claims 2
- 208000031926 Glycogen storage disease due to muscle phosphofructokinase deficiency Diseases 0.000 claims 2
- 206010053185 Glycogen storage disease type II Diseases 0.000 claims 2
- 206010053250 Glycogen storage disease type III Diseases 0.000 claims 2
- 206010018462 Glycogen storage disease type V Diseases 0.000 claims 2
- 102100033448 Lysosomal alpha-glucosidase Human genes 0.000 claims 2
- 206010072928 Mucolipidosis type II Diseases 0.000 claims 2
- 239000013543 active substance Substances 0.000 claims 2
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 claims 2
- 201000004502 glycogen storage disease II Diseases 0.000 claims 2
- 201000004543 glycogen storage disease III Diseases 0.000 claims 2
- 201000004534 glycogen storage disease V Diseases 0.000 claims 2
- 201000009339 glycogen storage disease VII Diseases 0.000 claims 2
- 208000017476 juvenile neuronal ceroid lipofuscinosis Diseases 0.000 claims 2
- 208000020460 mucolipidosis II alpha/beta Diseases 0.000 claims 2
- 208000020468 mucolipidosis III alpha/beta Diseases 0.000 claims 2
- 201000008051 neuronal ceroid lipofuscinosis Diseases 0.000 claims 2
- 108090000623 proteins and genes Proteins 0.000 claims 2
- 102000004169 proteins and genes Human genes 0.000 claims 2
- 238000011277 treatment modality Methods 0.000 claims 2
- SGEIEGAXKLMUIZ-ZPTIMJQQSA-N (3e)-n-[(2r)-2-hydroxy-3-piperidin-1-ylpropoxy]-1-oxidopyridin-1-ium-3-carboximidoyl chloride Chemical compound C([C@H](O)CN1CCCCC1)O\N=C(\Cl)C1=CC=C[N+]([O-])=C1 SGEIEGAXKLMUIZ-ZPTIMJQQSA-N 0.000 claims 1
- QWVRTSZDKPRPDF-UHFFFAOYSA-N 5-(piperidin-1-ylmethyl)-3-pyridin-3-yl-5,6-dihydro-2h-1,2,4-oxadiazine Chemical compound C1CCCCN1CC(N=1)CONC=1C1=CC=CN=C1 QWVRTSZDKPRPDF-UHFFFAOYSA-N 0.000 claims 1
- 208000031277 Amaurotic familial idiocy Diseases 0.000 claims 1
- 206010053249 Glycogen Storage Disease Type IV Diseases 0.000 claims 1
- 208000032003 Glycogen storage disease due to glucose-6-phosphatase deficiency Diseases 0.000 claims 1
- 208000011123 Glycogen storage disease due to glycogen branching enzyme deficiency Diseases 0.000 claims 1
- 206010018464 Glycogen storage disease type I Diseases 0.000 claims 1
- 102000003886 Glycoproteins Human genes 0.000 claims 1
- 108090000288 Glycoproteins Proteins 0.000 claims 1
- 229920002683 Glycosaminoglycan Polymers 0.000 claims 1
- 208000015178 Hurler syndrome Diseases 0.000 claims 1
- 208000015204 Hurler-Scheie syndrome Diseases 0.000 claims 1
- 208000000501 Lipidoses Diseases 0.000 claims 1
- 206010024585 Lipidosis Diseases 0.000 claims 1
- 208000027933 Mannosidase Deficiency disease Diseases 0.000 claims 1
- 206010072929 Mucolipidosis type III Diseases 0.000 claims 1
- 206010072930 Mucolipidosis type IV Diseases 0.000 claims 1
- 102100026502 Mucolipin-1 Human genes 0.000 claims 1
- 206010028095 Mucopolysaccharidosis IV Diseases 0.000 claims 1
- 208000025915 Mucopolysaccharidosis type 6 Diseases 0.000 claims 1
- 229910019142 PO4 Inorganic materials 0.000 claims 1
- 201000002883 Scheie syndrome Diseases 0.000 claims 1
- 208000017460 Sialidosis type 2 Diseases 0.000 claims 1
- 208000026589 Wolman disease Diseases 0.000 claims 1
- 208000017478 adult neuronal ceroid lipofuscinosis Diseases 0.000 claims 1
- 229940035676 analgesics Drugs 0.000 claims 1
- 239000000730 antalgic agent Substances 0.000 claims 1
- 229950011582 arimoclomol Drugs 0.000 claims 1
- -1 aspartyl Chemical group 0.000 claims 1
- 201000006486 beta-mannosidosis Diseases 0.000 claims 1
- 239000012867 bioactive agent Substances 0.000 claims 1
- 210000004556 brain Anatomy 0.000 claims 1
- 230000000747 cardiac effect Effects 0.000 claims 1
- 235000012000 cholesterol Nutrition 0.000 claims 1
- 208000024042 cholesterol ester storage disease Diseases 0.000 claims 1
- 208000013760 cholesteryl ester storage disease Diseases 0.000 claims 1
- 239000003246 corticosteroid Substances 0.000 claims 1
- 229960001334 corticosteroids Drugs 0.000 claims 1
- 230000007547 defect Effects 0.000 claims 1
- 229940079593 drug Drugs 0.000 claims 1
- 230000000694 effects Effects 0.000 claims 1
- 238000002641 enzyme replacement therapy Methods 0.000 claims 1
- 201000008049 fucosidosis Diseases 0.000 claims 1
- 230000014509 gene expression Effects 0.000 claims 1
- 201000004541 glycogen storage disease I Diseases 0.000 claims 1
- 239000000411 inducer Substances 0.000 claims 1
- 230000003834 intracellular effect Effects 0.000 claims 1
- 229950004636 iroxanadine Drugs 0.000 claims 1
- 230000002132 lysosomal effect Effects 0.000 claims 1
- 201000002273 mucopolysaccharidosis II Diseases 0.000 claims 1
- 208000005340 mucopolysaccharidosis III Diseases 0.000 claims 1
- 208000000690 mucopolysaccharidosis VI Diseases 0.000 claims 1
- 208000022018 mucopolysaccharidosis type 2 Diseases 0.000 claims 1
- 208000011045 mucopolysaccharidosis type 3 Diseases 0.000 claims 1
- 208000010978 mucopolysaccharidosis type 4 Diseases 0.000 claims 1
- 208000020004 mucopolysaccharidosis type 9 Diseases 0.000 claims 1
- ISGGVCWFTPTHIX-UHFFFAOYSA-N n'-(2-hydroxy-3-piperidin-1-ylpropoxy)pyridine-3-carboximidamide;dihydrochloride Chemical compound Cl.Cl.C1CCCCN1CC(O)CONC(=N)C1=CC=CN=C1 ISGGVCWFTPTHIX-UHFFFAOYSA-N 0.000 claims 1
- 201000007607 neuronal ceroid lipofuscinosis 3 Diseases 0.000 claims 1
- NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 claims 1
- 239000010452 phosphate Substances 0.000 claims 1
- 230000035479 physiological effects, processes and functions Effects 0.000 claims 1
- 239000000758 substrate Substances 0.000 claims 1
- 238000009120 supportive therapy Methods 0.000 claims 1
- 230000001629 suppression Effects 0.000 claims 1
- 208000011580 syndromic disease Diseases 0.000 claims 1
- 210000002435 tendon Anatomy 0.000 claims 1
- 238000002560 therapeutic procedure Methods 0.000 claims 1
- 238000002054 transplantation Methods 0.000 claims 1
- 238000011282 treatment Methods 0.000 claims 1
- 238000011269 treatment regimen Methods 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| DKPA201070520 | 2010-11-30 | ||
| DKPA201070520 | 2010-11-30 | ||
| PCT/DK2011/050444 WO2012072082A1 (en) | 2010-11-30 | 2011-11-22 | Methods for increasing intracellular activity of hsp70 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2013544263A JP2013544263A (ja) | 2013-12-12 |
| JP2013544263A5 true JP2013544263A5 (https=) | 2015-01-15 |
Family
ID=46171210
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2013541210A Pending JP2013544263A (ja) | 2010-11-30 | 2011-11-22 | Ｈｓｐ７０の細胞内活性を上昇させる方法 |
Country Status (15)
| Country | Link |
|---|---|
| US (4) | US9662375B2 (https=) |
| EP (2) | EP3626255A1 (https=) |
| JP (1) | JP2013544263A (https=) |
| CN (1) | CN103338784A (https=) |
| AU (1) | AU2011335545A1 (https=) |
| BR (1) | BR112013013143A2 (https=) |
| CA (1) | CA2817773A1 (https=) |
| DK (1) | DK2646044T3 (https=) |
| ES (1) | ES2753169T3 (https=) |
| HU (1) | HUE046235T2 (https=) |
| IL (1) | IL225941A0 (https=) |
| PL (1) | PL2646044T3 (https=) |
| PT (1) | PT2646044T (https=) |
| RU (1) | RU2013125923A (https=) |
| WO (1) | WO2012072082A1 (https=) |
Families Citing this family (43)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8540985B2 (en) | 2008-06-26 | 2013-09-24 | Orphazyme Aps | Use of Hsp70 as a regulator of enzymatic activity |
| RU2013125923A (ru) | 2010-11-30 | 2015-01-10 | Орфазиме Апс | СПОСОБЫ УВЕЛИЧЕНИЯ ВНУТРИКЛЕТОЧНОЙ АКТИВНОСТИ Hsp70 |
| US10967298B2 (en) | 2012-03-15 | 2021-04-06 | Flodesign Sonics, Inc. | Driver and control for variable impedence load |
| US9752113B2 (en) | 2012-03-15 | 2017-09-05 | Flodesign Sonics, Inc. | Acoustic perfusion devices |
| US10704021B2 (en) | 2012-03-15 | 2020-07-07 | Flodesign Sonics, Inc. | Acoustic perfusion devices |
| US10322949B2 (en) | 2012-03-15 | 2019-06-18 | Flodesign Sonics, Inc. | Transducer and reflector configurations for an acoustophoretic device |
| US10689609B2 (en) | 2012-03-15 | 2020-06-23 | Flodesign Sonics, Inc. | Acoustic bioreactor processes |
| US9458450B2 (en) | 2012-03-15 | 2016-10-04 | Flodesign Sonics, Inc. | Acoustophoretic separation technology using multi-dimensional standing waves |
| US9950282B2 (en) | 2012-03-15 | 2018-04-24 | Flodesign Sonics, Inc. | Electronic configuration and control for acoustic standing wave generation |
| US9745548B2 (en) | 2012-03-15 | 2017-08-29 | Flodesign Sonics, Inc. | Acoustic perfusion devices |
| US10737953B2 (en) | 2012-04-20 | 2020-08-11 | Flodesign Sonics, Inc. | Acoustophoretic method for use in bioreactors |
| JP6023328B2 (ja) * | 2012-07-31 | 2016-11-09 | ザ カトリック ユニバーシティ オブ コリア インダストリー−アカデミック コーオペレイション ファウンデーション | レバミピドを有効成分として含む高脂血症及びこれと関連した疾患の予防または治療用組成物 |
| WO2014145874A1 (en) * | 2013-03-15 | 2014-09-18 | University Of Iowa Research Foundation | Therapeutic methods |
| US9745569B2 (en) | 2013-09-13 | 2017-08-29 | Flodesign Sonics, Inc. | System for generating high concentration factors for low cell density suspensions |
| WO2015105955A1 (en) | 2014-01-08 | 2015-07-16 | Flodesign Sonics, Inc. | Acoustophoresis device with dual acoustophoretic chamber |
| US10357542B2 (en) | 2014-02-04 | 2019-07-23 | New York University | Progranulin (PGRN) and its derivatives for diagnosis and treatment of lysosomal storage diseases |
| US9744483B2 (en) | 2014-07-02 | 2017-08-29 | Flodesign Sonics, Inc. | Large scale acoustic separation device |
| US10709700B2 (en) * | 2014-09-15 | 2020-07-14 | Orphazyme A/S | Arimoclomol formulation |
| US11021699B2 (en) | 2015-04-29 | 2021-06-01 | FioDesign Sonics, Inc. | Separation using angled acoustic waves |
| US11377651B2 (en) | 2016-10-19 | 2022-07-05 | Flodesign Sonics, Inc. | Cell therapy processes utilizing acoustophoresis |
| US11708572B2 (en) | 2015-04-29 | 2023-07-25 | Flodesign Sonics, Inc. | Acoustic cell separation techniques and processes |
| US11459540B2 (en) | 2015-07-28 | 2022-10-04 | Flodesign Sonics, Inc. | Expanded bed affinity selection |
| US11474085B2 (en) | 2015-07-28 | 2022-10-18 | Flodesign Sonics, Inc. | Expanded bed affinity selection |
| WO2017024137A1 (en) * | 2015-08-04 | 2017-02-09 | New York University | Progranulin (pgrn) fragments and derivatives for treatment or alleviation of lysosomal storage diseases |
| EP3442530A1 (en) * | 2016-04-13 | 2019-02-20 | Orphazyme A/S | Heat shock proteins and cholesterol homeostasis |
| KR20210059796A (ko) | 2016-04-29 | 2021-05-25 | 오르파짐 에이/에스 | 글루코세레브로시다제 연관 장애를 치료하기 위한 아리모클로몰 |
| US11085035B2 (en) | 2016-05-03 | 2021-08-10 | Flodesign Sonics, Inc. | Therapeutic cell washing, concentration, and separation utilizing acoustophoresis |
| US11214789B2 (en) | 2016-05-03 | 2022-01-04 | Flodesign Sonics, Inc. | Concentration and washing of particles with acoustics |
| WO2018075830A1 (en) | 2016-10-19 | 2018-04-26 | Flodesign Sonics, Inc. | Affinity cell extraction by acoustics |
| CN107022553A (zh) * | 2017-06-08 | 2017-08-08 | 扬州大学 | 芍药hsp70基因及其植物表达载体和应用 |
| KR102439221B1 (ko) | 2017-12-14 | 2022-09-01 | 프로디자인 소닉스, 인크. | 음향 트랜스듀서 구동기 및 제어기 |
| EP3784695B1 (en) * | 2018-04-27 | 2023-08-09 | The Medical College of Wisconsin, Inc. | Use of lentivector-transduced t-rapa cells for amelioration of lysosomal storage disorders |
| BR112020021962A2 (pt) * | 2018-04-30 | 2021-01-26 | Amicus Therapeutics, Inc. | construtos para terapia gênica e métodos de uso |
| KR20210015790A (ko) * | 2018-05-28 | 2021-02-10 | 오르파짐 에이/에스 | 질환의 바이오마커로서 pbmc 샘플에서 hsp70 단백질 수준 |
| US20220087962A1 (en) * | 2019-01-30 | 2022-03-24 | Arizona Board Of Regents On Behalf Of The University Of Arizona | Compositions and methods for treating age-related diseases or conditions |
| WO2021055801A1 (en) | 2019-09-18 | 2021-03-25 | The Medical College Of Wisconsin, Inc. | Improved alpha-galactosidase protein for enzyme replacement therapy (ert) and methods of use |
| CN110974833A (zh) * | 2019-12-02 | 2020-04-10 | 吉林省博大伟业制药有限公司 | 一种单独应用聚普瑞锌治疗萎缩性胃炎药物的医药用途 |
| US12600705B2 (en) | 2020-02-14 | 2026-04-14 | University Of Pittsburgh-Of The Commonwealth System Of Higher Education | Heat shock protein modulators and anti-Huntington disease therapeutic agents |
| EP4125946A4 (en) * | 2020-03-27 | 2024-04-24 | Board of Regents, The University of Texas System | MONOCLONAL ANTIBODIES TARGETING HSP70 AND THEIR THERAPEUTIC USES |
| KR20230128462A (ko) | 2020-11-19 | 2023-09-05 | 제브라 덴마크 에이/에스 | 아리모클로몰 시트레이트 및 이의 중간체를 제조하기 위한 공정 |
| WO2022136640A1 (en) * | 2020-12-24 | 2022-06-30 | Orphazyme A/S | Arimoclomol for the treatment of niemann pick disease, type c, in patients with er type missense mutations |
| CN116549431A (zh) * | 2023-03-27 | 2023-08-08 | 上海市第十人民医院 | 花生四烯酸在制备防治电离辐射损伤药物中的应用 |
| CN116688129A (zh) * | 2023-05-24 | 2023-09-05 | 中山大学附属第一医院 | 靶向苯丙酮酸促进糖尿病伤口愈合以及抑制过度nlrp3炎症小体的激活 |
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-
2011
- 2011-11-22 RU RU2013125923/10A patent/RU2013125923A/ru not_active Application Discontinuation
- 2011-11-22 JP JP2013541210A patent/JP2013544263A/ja active Pending
- 2011-11-22 US US13/885,553 patent/US9662375B2/en active Active
- 2011-11-22 AU AU2011335545A patent/AU2011335545A1/en not_active Abandoned
- 2011-11-22 HU HUE11844130A patent/HUE046235T2/hu unknown
- 2011-11-22 WO PCT/DK2011/050444 patent/WO2012072082A1/en not_active Ceased
- 2011-11-22 CN CN2011800575624A patent/CN103338784A/zh active Pending
- 2011-11-22 CA CA2817773A patent/CA2817773A1/en not_active Abandoned
- 2011-11-22 PT PT118441302T patent/PT2646044T/pt unknown
- 2011-11-22 BR BR112013013143A patent/BR112013013143A2/pt not_active IP Right Cessation
- 2011-11-22 PL PL11844130T patent/PL2646044T3/pl unknown
- 2011-11-22 EP EP19193723.4A patent/EP3626255A1/en not_active Withdrawn
- 2011-11-22 EP EP11844130.2A patent/EP2646044B1/en active Active
- 2011-11-22 ES ES11844130T patent/ES2753169T3/es active Active
- 2011-11-22 DK DK11844130T patent/DK2646044T3/da active
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2013
- 2013-04-25 IL IL225941A patent/IL225941A0/en unknown
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2017
- 2017-05-19 US US15/600,407 patent/US10532085B2/en active Active
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2019
- 2019-11-27 US US16/698,473 patent/US20200188492A1/en not_active Abandoned
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2022
- 2022-06-30 US US17/855,002 patent/US20220374361A1/en not_active Abandoned
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