JP2016511259A5 - - Google Patents
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- JP2016511259A5 JP2016511259A5 JP2015560377A JP2015560377A JP2016511259A5 JP 2016511259 A5 JP2016511259 A5 JP 2016511259A5 JP 2015560377 A JP2015560377 A JP 2015560377A JP 2015560377 A JP2015560377 A JP 2015560377A JP 2016511259 A5 JP2016511259 A5 JP 2016511259A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- subject
- taz1
- expression
- peptide
- 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
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- 108090000765 processed proteins & peptides Proteins 0.000 claims description 16
- 101000835595 Homo sapiens Tafazzin Proteins 0.000 claims description 14
- 102100026508 Tafazzin Human genes 0.000 claims description 14
- 208000011580 syndromic disease Diseases 0.000 claims description 14
- 150000003839 salts Chemical class 0.000 claims description 11
- 239000002327 cardiovascular agent Substances 0.000 claims description 6
- 229940125692 cardiovascular agent Drugs 0.000 claims description 6
- ZGSPNIOCEDOHGS-UHFFFAOYSA-L disodium [3-[2,3-di(octadeca-9,12-dienoyloxy)propoxy-oxidophosphoryl]oxy-2-hydroxypropyl] 2,3-di(octadeca-9,12-dienoyloxy)propyl phosphate Chemical compound [Na+].[Na+].CCCCCC=CCC=CCCCCCCCC(=O)OCC(OC(=O)CCCCCCCC=CCC=CCCCCC)COP([O-])(=O)OCC(O)COP([O-])(=O)OCC(OC(=O)CCCCCCCC=CCC=CCCCCC)COC(=O)CCCCCCCC=CCC=CCCCCC ZGSPNIOCEDOHGS-UHFFFAOYSA-L 0.000 claims description 6
- 239000003087 receptor blocking agent Substances 0.000 claims description 6
- 229940123413 Angiotensin II antagonist Drugs 0.000 claims description 3
- 208000035143 Bacterial infection Diseases 0.000 claims description 3
- 201000005943 Barth syndrome Diseases 0.000 claims description 3
- 229940127291 Calcium channel antagonist Drugs 0.000 claims description 3
- 208000031229 Cardiomyopathies Diseases 0.000 claims description 3
- 229940122202 Thromboxane receptor antagonist Drugs 0.000 claims description 3
- 230000005856 abnormality Effects 0.000 claims description 3
- 239000002333 angiotensin II receptor antagonist Substances 0.000 claims description 3
- 229940044094 angiotensin-converting-enzyme inhibitor Drugs 0.000 claims description 3
- 239000004004 anti-anginal agent Substances 0.000 claims description 3
- 229940124345 antianginal agent Drugs 0.000 claims description 3
- 239000003416 antiarrhythmic agent Substances 0.000 claims description 3
- 239000003524 antilipemic agent Substances 0.000 claims description 3
- 229940127218 antiplatelet drug Drugs 0.000 claims description 3
- 208000022362 bacterial infectious disease Diseases 0.000 claims description 3
- 102000012740 beta Adrenergic Receptors Human genes 0.000 claims description 3
- 108010079452 beta Adrenergic Receptors Proteins 0.000 claims description 3
- 239000008280 blood Substances 0.000 claims description 3
- 210000004369 blood Anatomy 0.000 claims description 3
- 239000000480 calcium channel blocker Substances 0.000 claims description 3
- 239000003246 corticosteroid Substances 0.000 claims description 3
- 230000003111 delayed effect Effects 0.000 claims description 3
- 229940068811 digitalis preparation Drugs 0.000 claims description 3
- 239000002934 diuretic Substances 0.000 claims description 3
- 230000001882 diuretic effect Effects 0.000 claims description 3
- 239000003527 fibrinolytic agent Substances 0.000 claims description 3
- 239000003112 inhibitor Substances 0.000 claims description 3
- 230000000297 inotrophic effect Effects 0.000 claims description 3
- 210000003205 muscle Anatomy 0.000 claims description 3
- 208000004235 neutropenia Diseases 0.000 claims description 3
- 150000007524 organic acids Chemical class 0.000 claims description 3
- 235000005985 organic acids Nutrition 0.000 claims description 3
- 239000002516 radical scavenger Substances 0.000 claims description 3
- 239000000932 sedative agent Substances 0.000 claims description 3
- 230000001624 sedative effect Effects 0.000 claims description 3
- 210000002027 skeletal muscle Anatomy 0.000 claims description 3
- 239000000126 substance Substances 0.000 claims description 3
- 230000002889 sympathetic effect Effects 0.000 claims description 3
- 229960000103 thrombolytic agent Drugs 0.000 claims description 3
- 239000002396 thromboxane receptor blocking agent Substances 0.000 claims description 3
- 210000002700 urine Anatomy 0.000 claims description 3
- 229940124549 vasodilator Drugs 0.000 claims description 3
- 239000003071 vasodilator agent Substances 0.000 claims description 3
- QTBSBXVTEAMEQO-UHFFFAOYSA-M Acetate Chemical compound CC([O-])=O QTBSBXVTEAMEQO-UHFFFAOYSA-M 0.000 claims description 2
- DTQVDTLACAAQTR-UHFFFAOYSA-M Trifluoroacetate Chemical compound [O-]C(=O)C(F)(F)F DTQVDTLACAAQTR-UHFFFAOYSA-M 0.000 claims description 2
- -1 cardioglycide Substances 0.000 claims description 2
- 230000002265 prevention Effects 0.000 claims description 2
- 238000007634 remodeling Methods 0.000 claims description 2
- 241000894007 species Species 0.000 claims description 2
- 230000000087 stabilizing effect Effects 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 17
- 238000007918 intramuscular administration Methods 0.000 claims 1
- 238000007912 intraperitoneal administration Methods 0.000 claims 1
- 238000007920 subcutaneous administration Methods 0.000 claims 1
- 238000007910 systemic administration Methods 0.000 claims 1
- IMCGHZIGRANKHV-AJNGGQMLSA-N tert-butyl (3s,5s)-2-oxo-5-[(2s,4s)-5-oxo-4-propan-2-yloxolan-2-yl]-3-propan-2-ylpyrrolidine-1-carboxylate Chemical compound O1C(=O)[C@H](C(C)C)C[C@H]1[C@H]1N(C(=O)OC(C)(C)C)C(=O)[C@H](C(C)C)C1 IMCGHZIGRANKHV-AJNGGQMLSA-N 0.000 claims 1
- 238000011200 topical administration Methods 0.000 claims 1
- 238000000034 method Methods 0.000 description 32
- 230000003203 everyday effect Effects 0.000 description 4
- 159000000021 acetate salts Chemical class 0.000 description 1
- 239000002368 cardiac glycoside Substances 0.000 description 1
- 230000003247 decreasing effect Effects 0.000 description 1
- VFRSADQPWYCXDG-LEUCUCNGSA-N ethyl (2s,5s)-5-methylpyrrolidine-2-carboxylate;2,2,2-trifluoroacetic acid Chemical compound OC(=O)C(F)(F)F.CCOC(=O)[C@@H]1CC[C@H](C)N1 VFRSADQPWYCXDG-LEUCUCNGSA-N 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2019080101A JP6918046B2 (ja) | 2013-03-01 | 2019-04-19 | バース症候群の予防または治療のための方法及び組成物 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361771642P | 2013-03-01 | 2013-03-01 | |
| US201361771534P | 2013-03-01 | 2013-03-01 | |
| US61/771,534 | 2013-03-01 | ||
| US61/771,642 | 2013-03-01 | ||
| US201361839753P | 2013-06-26 | 2013-06-26 | |
| US61/839,753 | 2013-06-26 | ||
| PCT/US2014/019622 WO2014134554A1 (en) | 2013-03-01 | 2014-02-28 | Methods and compositions for the prevention or treatment of barth syndrome |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019080101A Division JP6918046B2 (ja) | 2013-03-01 | 2019-04-19 | バース症候群の予防または治療のための方法及び組成物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2016511259A JP2016511259A (ja) | 2016-04-14 |
| JP2016511259A5 true JP2016511259A5 (enExample) | 2017-04-06 |
| JP6518197B2 JP6518197B2 (ja) | 2019-05-22 |
Family
ID=51428869
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015560377A Active JP6518197B2 (ja) | 2013-03-01 | 2014-02-28 | バース症候群の予防または治療のための方法及び組成物 |
| JP2019080101A Active JP6918046B2 (ja) | 2013-03-01 | 2019-04-19 | バース症候群の予防または治療のための方法及び組成物 |
| JP2021019903A Active JP7072692B2 (ja) | 2013-03-01 | 2021-02-10 | バース症候群の予防または治療のための方法及び組成物 |
| JP2022077425A Active JP7381652B2 (ja) | 2013-03-01 | 2022-05-10 | バース症候群の予防または治療のための方法及び組成物 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019080101A Active JP6918046B2 (ja) | 2013-03-01 | 2019-04-19 | バース症候群の予防または治療のための方法及び組成物 |
| JP2021019903A Active JP7072692B2 (ja) | 2013-03-01 | 2021-02-10 | バース症候群の予防または治療のための方法及び組成物 |
| JP2022077425A Active JP7381652B2 (ja) | 2013-03-01 | 2022-05-10 | バース症候群の予防または治療のための方法及び組成物 |
Country Status (9)
| Country | Link |
|---|---|
| US (5) | US9687519B2 (enExample) |
| EP (2) | EP2961420B1 (enExample) |
| JP (4) | JP6518197B2 (enExample) |
| CN (3) | CN115990242A (enExample) |
| CA (1) | CA2916880C (enExample) |
| DK (1) | DK2961420T3 (enExample) |
| ES (1) | ES2750258T3 (enExample) |
| HU (1) | HUE046924T2 (enExample) |
| WO (1) | WO2014134554A1 (enExample) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2015507611A (ja) * | 2011-12-09 | 2015-03-12 | ステルス ペプチドズ インターナショナル インコーポレイテッド | 芳香族カチオン性ペプチドおよびその使用 |
| CA2916884C (en) | 2013-03-01 | 2021-02-09 | Stealth Biotherapeutics Corp | Methods for the treatment of mitochondrial disease |
| US9687519B2 (en) | 2013-03-01 | 2017-06-27 | Stealth Biotherapeutics Corp | Methods and compositions for the prevention or treatment of barth syndrome |
| CA2916977A1 (en) | 2013-06-26 | 2014-12-31 | Stealth Biotherapeutics Corp | Methods and compositions for detecting and diagnosing diseases and conditions |
| US9550981B2 (en) | 2014-01-22 | 2017-01-24 | University Of Washington | Modified tafazzin proteins and methods of making and using the same |
| EP3230308B1 (en) | 2014-12-12 | 2019-05-01 | University of Washington | Methods for treating and preventing cardiomyopathy |
| WO2018104172A1 (en) | 2016-12-06 | 2018-06-14 | Centre National De La Recherche Scientifique (Cnrs) | Compounds for treating barth syndrome |
| IL269626B1 (en) | 2017-04-05 | 2025-10-01 | Stealth Biotherapeutics Corp | Crystalline salt forms of boc–d–arg–dmt–lys–(boc)–phe–nh2 |
| US10676506B2 (en) | 2018-01-26 | 2020-06-09 | Stealth Biotherapeutics Corp. | Crystalline bis- and tris-hydrochloride salt of elamipretide |
| CN113383005A (zh) * | 2018-12-18 | 2021-09-10 | 隐形生物治疗公司 | 靶向线粒体疾病的类似物 |
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| US4522811A (en) | 1982-07-08 | 1985-06-11 | Syntex (U.S.A.) Inc. | Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides |
| US5858784A (en) | 1991-12-17 | 1999-01-12 | The Regents Of The University Of California | Expression of cloned genes in the lung by aerosol- and liposome-based delivery |
| US5674534A (en) | 1992-06-11 | 1997-10-07 | Alkermes, Inc. | Composition for sustained release of non-aggregated erythropoietin |
| US5716644A (en) | 1992-06-11 | 1998-02-10 | Alkermes, Inc. | Composition for sustained release of non-aggregated erythropoietin |
| US5989463A (en) | 1997-09-24 | 1999-11-23 | Alkermes Controlled Therapeutics, Inc. | Methods for fabricating polymer-based controlled release devices |
| US6245740B1 (en) | 1998-12-23 | 2001-06-12 | Amgen Inc. | Polyol:oil suspensions for the sustained release of proteins |
| WO2003062784A2 (en) | 2001-08-06 | 2003-07-31 | Vanderbilt University | Apparatus and methods for monitoring the status of a metabolically active cell |
| BRPI0105509B8 (pt) | 2001-11-05 | 2021-05-25 | Univ Minas Gerais | formulações do peptídeo angiotensina-(1-7) usando as ciclodextrinas, lipossomas e o polímero plga |
| HUE027110T2 (en) | 2003-02-04 | 2016-08-29 | Cornell Res Foundation Inc | Use of an aromatic cationic peptide |
| SI1599216T1 (sl) | 2003-02-04 | 2014-02-28 | Cornell Research Foundation, Inc. | Postopek za preprečevanje mitohondrijskega permeabilnostnega prehoda |
| EP1597276A4 (en) * | 2003-02-10 | 2006-11-29 | Autogen Res Pty Ltd | Therapeutic molecules |
| US20070026090A1 (en) | 2003-09-05 | 2007-02-01 | Oren Tirosh | Treatment and prevention of inflammatory disease and mitochondrial dysfunction with high dose selenium |
| CA2554166C (en) | 2004-01-23 | 2014-07-29 | Cornell Research Foundation, Inc. | Methods for reducing oxidative damage |
| US8500720B2 (en) | 2005-05-09 | 2013-08-06 | Medtronic, Inc | Method and apparatus for treatment of cardiac disorders |
| US20070265216A1 (en) * | 2005-10-05 | 2007-11-15 | Gross Richard W | Enhanced medical treatment in diabetic cardiomyopathy |
| EP2012800A2 (en) * | 2006-04-18 | 2009-01-14 | Cortendo Invest, AB | Methods and compositions for treating barth syndrome, cardiomyopathy, mitochondrial diseases and other conditions |
| WO2008137035A1 (en) | 2007-05-02 | 2008-11-13 | The Mclean Hospital Corporation | Methods and compositions for mitochondrial replacement therapy |
| US20080318909A1 (en) | 2007-06-14 | 2008-12-25 | Sparagna Genevieve C | Use Of Linoleic Compounds Against Heart Failure |
| US20090143279A1 (en) | 2007-06-15 | 2009-06-04 | Vamsi Krishna Mootha | Methods and compositions for treating metabolic disorders |
| CN104056248A (zh) * | 2008-02-26 | 2014-09-24 | 康奈尔大学 | 用于防止和治疗急性肾损伤的方法 |
| US20110197294A1 (en) | 2008-06-04 | 2011-08-11 | Gottlieb Roberta A | Compositions and methods for restoring mitochondrial electron transfer function |
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| EP3906933A1 (en) | 2009-03-20 | 2021-11-10 | The General Hospital Corporation d/b/a Massachusetts General Hospital | D-arg-2'6'-dimethyltyrosine-lys-phe-nh2 for use in the prevention of secondary complications of burn injuries |
| US20120122957A1 (en) | 2009-04-17 | 2012-05-17 | The Salk Institute For Biological Studies | Regulation of aging by modulation of mitochondrial function |
| WO2010132347A2 (en) | 2009-05-11 | 2010-11-18 | University Of Maryland, Baltimore | Docosahexaenoic acid for the treatment of heart failure |
| JP5909182B2 (ja) | 2009-08-12 | 2016-04-26 | コーネル ユニヴァーシティー | 代謝性症候群を予防し又は治療するための方法 |
| US8470784B2 (en) * | 2009-08-24 | 2013-06-25 | Stealth Peptides International, Inc. | Methods and compositions for preventing or treating ophthalmic conditions |
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| EP3290433A1 (en) * | 2010-05-03 | 2018-03-07 | Stealth Peptides International, Inc. | Aromatic-cationic peptides and uses of same |
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| EA201300215A1 (ru) | 2010-08-06 | 2013-07-30 | Эдисон Фармасьютикалз, Инк. | Лечение митохондриальных болезней нафтохинонами |
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| US9241933B2 (en) | 2011-03-01 | 2016-01-26 | Pharnext | Compositions for treating amyotrophic lateral sclerosis |
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| CN107496899A (zh) | 2011-09-29 | 2017-12-22 | 梅约医学教育与研究基金会 | 芳族阳离子肽和使用它们的方法 |
| CN106039282A (zh) | 2011-10-17 | 2016-10-26 | 康奈尔大学 | 芳香族阳离子肽及其用途 |
| CN106822868A (zh) * | 2011-10-21 | 2017-06-13 | 斯特姆詹尼克斯公司 | 用于胞内传递生物活性分子的官能化纳米粒子 |
| CA2865317A1 (en) | 2012-02-22 | 2013-08-29 | Stealth Peptides International, Inc. | Methods and compositions for preventing or treating ophthalmic conditions |
| WO2013126775A1 (en) * | 2012-02-23 | 2013-08-29 | Cornell University | Aromatic-cationic peptides and uses of same |
| AU2013329312A1 (en) | 2012-10-09 | 2015-05-28 | Massachusetts Institute Of Technology | Treatment of age-related and mitochondrial diseases by inhibition of HIF-1 alpha function |
| BR112015009072A2 (pt) | 2012-10-22 | 2017-07-04 | Henry Ford Health Systems | métodos para redução dos riscos associados à insuficiência cardíaca e fatores associados à mesma |
| US9687519B2 (en) * | 2013-03-01 | 2017-06-27 | Stealth Biotherapeutics Corp | Methods and compositions for the prevention or treatment of barth syndrome |
| CA2916884C (en) | 2013-03-01 | 2021-02-09 | Stealth Biotherapeutics Corp | Methods for the treatment of mitochondrial disease |
| CA2916977A1 (en) * | 2013-06-26 | 2014-12-31 | Stealth Biotherapeutics Corp | Methods and compositions for detecting and diagnosing diseases and conditions |
-
2014
- 2014-02-28 US US14/771,408 patent/US9687519B2/en active Active
- 2014-02-28 EP EP14756991.7A patent/EP2961420B1/en active Active
- 2014-02-28 HU HUE14756991A patent/HUE046924T2/hu unknown
- 2014-02-28 CN CN202211635437.3A patent/CN115990242A/zh active Pending
- 2014-02-28 JP JP2015560377A patent/JP6518197B2/ja active Active
- 2014-02-28 CN CN201480022767.2A patent/CN105407906A/zh active Pending
- 2014-02-28 ES ES14756991T patent/ES2750258T3/es active Active
- 2014-02-28 CA CA2916880A patent/CA2916880C/en active Active
- 2014-02-28 EP EP19190165.1A patent/EP3626252A1/en not_active Withdrawn
- 2014-02-28 DK DK14756991.7T patent/DK2961420T3/da active
- 2014-02-28 CN CN202211634590.4A patent/CN115990241A/zh active Pending
- 2014-02-28 WO PCT/US2014/019622 patent/WO2014134554A1/en not_active Ceased
-
2017
- 2017-06-19 US US15/626,255 patent/US11083771B2/en active Active
-
2019
- 2019-04-19 JP JP2019080101A patent/JP6918046B2/ja active Active
-
2020
- 2020-07-15 US US16/929,370 patent/US11083772B2/en active Active
-
2021
- 2021-02-10 JP JP2021019903A patent/JP7072692B2/ja active Active
- 2021-07-09 US US17/371,475 patent/US11771734B2/en active Active
-
2022
- 2022-05-10 JP JP2022077425A patent/JP7381652B2/ja active Active
-
2023
- 2023-08-23 US US18/454,319 patent/US12268724B2/en active Active
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