CA2916880C - Methods and compositions for the prevention or treatment of barth syndrome - Google Patents

Methods and compositions for the prevention or treatment of barth syndrome Download PDF

Info

Publication number
CA2916880C
CA2916880C CA2916880A CA2916880A CA2916880C CA 2916880 C CA2916880 C CA 2916880C CA 2916880 A CA2916880 A CA 2916880A CA 2916880 A CA2916880 A CA 2916880A CA 2916880 C CA2916880 C CA 2916880C
Authority
CA
Canada
Prior art keywords
arg
lys
phe
dmt
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.)
Active
Application number
CA2916880A
Other languages
English (en)
French (fr)
Other versions
CA2916880A1 (en
Inventor
D. Travis Wilson
Mark BAMBERGER
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Stealth Biotherapeutics Inc
Original Assignee
Stealth Biotherapeutics Corp
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Stealth Biotherapeutics Corp filed Critical Stealth Biotherapeutics Corp
Publication of CA2916880A1 publication Critical patent/CA2916880A1/en
Application granted granted Critical
Publication of CA2916880C publication Critical patent/CA2916880C/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/04Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
    • A61K38/06Tripeptides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/04Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
    • A61K38/07Tetrapeptides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/04Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Organic Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Diabetes (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Pulmonology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Hospice & Palliative Care (AREA)
  • Hematology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
CA2916880A 2013-03-01 2014-02-28 Methods and compositions for the prevention or treatment of barth syndrome Active CA2916880C (en)

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
US61/771,642 2013-03-01
US61/771,534 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

Publications (2)

Publication Number Publication Date
CA2916880A1 CA2916880A1 (en) 2014-09-04
CA2916880C true CA2916880C (en) 2021-02-09

Family

ID=51428869

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2916880A Active CA2916880C (en) 2013-03-01 2014-02-28 Methods and compositions for the prevention or treatment of barth syndrome

Country Status (9)

Country Link
US (5) US9687519B2 (cg-RX-API-DMAC7.html)
EP (2) EP3626252A1 (cg-RX-API-DMAC7.html)
JP (4) JP6518197B2 (cg-RX-API-DMAC7.html)
CN (3) CN115990242A (cg-RX-API-DMAC7.html)
CA (1) CA2916880C (cg-RX-API-DMAC7.html)
DK (1) DK2961420T3 (cg-RX-API-DMAC7.html)
ES (1) ES2750258T3 (cg-RX-API-DMAC7.html)
HU (1) HUE046924T2 (cg-RX-API-DMAC7.html)
WO (1) WO2014134554A1 (cg-RX-API-DMAC7.html)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107312066A (zh) * 2011-12-09 2017-11-03 康德生物医疗技术公司 芳香族阳离子肽及其用途
EP3626252A1 (en) 2013-03-01 2020-03-25 Stealth Biotherapeutics Corp Methods and compositions for the prevention or treatment of barth syndrome
DK2961378T3 (da) 2013-03-01 2019-11-25 Stealth Biotherapeutics Corp Fremgangsmåder til behandlingen af mitochondrisk sygdom
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
US10086040B2 (en) 2014-12-12 2018-10-02 University Of Washington Methods for treating and preventing cardiomyopathy with a fusion protein of tafazzin and a cellular permeability peptide
WO2018104172A1 (en) 2016-12-06 2018-06-14 Centre National De La Recherche Scientifique (Cnrs) Compounds for treating barth syndrome
IL322723A (en) 2017-04-05 2025-10-01 Stealth Biotherapeutics Inc 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
WO2020131283A1 (en) * 2018-12-18 2020-06-25 Stealth Biotherapeutics Corp. Analogs that target mitochondrial diseases

Family Cites Families (50)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US5716644A (en) 1992-06-11 1998-02-10 Alkermes, Inc. Composition for sustained release of non-aggregated erythropoietin
US5674534A (en) 1992-06-11 1997-10-07 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
EP1423501A4 (en) 2001-08-06 2008-08-06 Univ Vanderbilt DEVICE AND METHOD FOR MEASURING THE RESPONSE OF AT LEAST ONE CELL TO A MEDIUM
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
CN101440124B (zh) 2003-02-04 2012-07-18 科内尔研究基金会 用于防止线粒体通透性改变的方法
PT1599216E (pt) 2003-02-04 2013-12-17 Cornell Res Foundation Inc Métodos de prevenção da transição de permeabilidade mitocondrial
US20070135335A1 (en) * 2003-02-10 2007-06-14 Autogen Research Pty Ltd. Therapeutic molecules
WO2005023274A1 (en) 2003-09-05 2005-03-17 Yissum Research Development Company Of The Hebrew University Of Jerusalem Treatment and prevention of inflammatory disease and mitochondrial dysfunction with high dose selenium
CA2971931C (en) 2004-01-23 2019-01-08 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
AU2007237899A1 (en) * 2006-04-18 2007-10-25 Cortendo Invest, Ab Methods and compositions for treating barth syndrome, cardiomyopathy, mitochondrial diseases and other conditions
CA2688125A1 (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
WO2008156654A2 (en) 2007-06-15 2008-12-24 Massachusetts Institute Of Technology Cytoskeleton modulators for treating metabolic disorders
CN101951936A (zh) 2008-02-26 2011-01-19 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
CN104231070B (zh) 2008-08-07 2017-09-01 益普生制药股份有限公司 N‑端修饰的葡萄糖依赖性促胰岛素多肽(gip)类似物
CN102573881A (zh) 2009-03-20 2012-07-11 通用医疗公司以马萨诸塞州通用医疗公司名义经营 预防和治疗烧伤损伤和继发性并发症的方法
WO2010121268A2 (en) 2009-04-17 2010-10-21 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
EP3338788A1 (en) * 2009-08-24 2018-06-27 Stealth Peptides International, Inc. Methods and copositions for preventing or treating opthalmic conditions
CN102711785A (zh) * 2009-10-05 2012-10-03 康奈尔大学 预防或治疗心力衰竭的方法
US8719939B2 (en) 2009-12-31 2014-05-06 Mcafee, Inc. Malware detection via reputation system
EP3090754A1 (en) * 2009-12-31 2016-11-09 Stealth Peptides International, Inc. Methods for the prevention or treatment of vessel occlusion injury
CA2797644A1 (en) 2009-12-31 2011-07-07 Edison Pharmaceuticals, Inc. Treatment of leigh syndrome and leigh-like syndrome with tocotrienol quinones
JP4643749B1 (ja) 2010-02-03 2011-03-02 昭和電工株式会社 表面被覆サーメット部材の耐酸化膜形成用の処理液
US20130040901A1 (en) * 2010-02-26 2013-02-14 University Of Florida Research Foundation Inc. Mitochondrial-targeted antioxidants protect against mechanical ventilation-induced diaphragm dysfunction and skeletal muscle atrophy
EP3040080A1 (en) 2010-03-15 2016-07-06 Stealth Peptides International, Inc. Combination therapies using cyclosporine and aromatic cationic peptides
EP2942354A1 (en) 2010-05-03 2015-11-11 Stealth Peptides International, Inc. Aromatic-Cationic Peptides and Uses of Same
US20130288985A1 (en) 2010-07-15 2013-10-31 The Schepens Eye Research Institute Inc. Compositions and methods of treatment of corneal endothelium disorders
CA2807507A1 (en) 2010-08-06 2012-02-09 Edison Pharmaceuticals, Inc. Treatment of mitochondrial diseases with naphthoquinones
JP2013538799A (ja) 2010-08-06 2013-10-17 アンペア ライフ サイエンシーズ,インコーポレイテッド ビタミンkを用いたミトコンドリア病の処置
US9241933B2 (en) 2011-03-01 2016-01-26 Pharnext Compositions for treating amyotrophic lateral sclerosis
WO2012129427A2 (en) * 2011-03-24 2012-09-27 Cornell University Aromatic-cationic peptides and uses of same
JP6157481B2 (ja) 2011-09-29 2017-07-05 メイヨ ファンデーション フォア メディカル エディケイション アンド リサーチ 芳香族陽イオン性ペプチドおよびその使用方法
CN104114181B (zh) 2011-10-17 2017-06-09 康奈尔大学 芳香族阳离子肽及其用途
AU2012325723A1 (en) * 2011-10-21 2014-05-15 Stemgenics, Inc Functionalized nanoparticles for intracellular delivery of biologically active molecules
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
US20150266946A1 (en) 2012-10-09 2015-09-24 President And Fellows Of Harvard College Treatment of age-related and mitochondrial diseases by inhibition of hif-1 alpha function
CN110193077A (zh) * 2012-10-22 2019-09-03 康德生物医疗技术公司 降低与心脏衰竭相关的风险和与其相关的因素的方法
DK2961378T3 (da) 2013-03-01 2019-11-25 Stealth Biotherapeutics Corp Fremgangsmåder til behandlingen af mitochondrisk sygdom
EP3626252A1 (en) * 2013-03-01 2020-03-25 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

Also Published As

Publication number Publication date
EP2961420A1 (en) 2016-01-06
JP2016511259A (ja) 2016-04-14
US20180147251A1 (en) 2018-05-31
JP6518197B2 (ja) 2019-05-22
US11083772B2 (en) 2021-08-10
EP3626252A1 (en) 2020-03-25
CN115990242A (zh) 2023-04-21
EP2961420B1 (en) 2019-09-11
CN115990241A (zh) 2023-04-21
DK2961420T3 (da) 2019-10-07
JP7072692B2 (ja) 2022-05-20
JP2022116008A (ja) 2022-08-09
US20210401923A1 (en) 2021-12-30
EP2961420A4 (en) 2016-08-17
WO2014134554A1 (en) 2014-09-04
US12268724B2 (en) 2025-04-08
US11083771B2 (en) 2021-08-10
JP2019147815A (ja) 2019-09-05
JP2021098704A (ja) 2021-07-01
US20160228487A1 (en) 2016-08-11
ES2750258T3 (es) 2020-03-25
US11771734B2 (en) 2023-10-03
US20240156892A1 (en) 2024-05-16
US9687519B2 (en) 2017-06-27
HUE046924T2 (hu) 2020-03-30
CN105407906A (zh) 2016-03-16
CA2916880A1 (en) 2014-09-04
JP7381652B2 (ja) 2023-11-15
JP6918046B2 (ja) 2021-08-11
US20200345803A1 (en) 2020-11-05

Similar Documents

Publication Publication Date Title
US12268724B2 (en) Methods and compositions for the prevention or treatment of Barth Syndrome
US11266709B2 (en) Methods for the regulation of matrix metalloproteinase expression
US20200113966A1 (en) Methods and compositions for regulating srca2a expression levels in myocardial infarction
HK40019493A (en) Methods and compositions for the prevention or treatment of barth syndrome
US11931397B2 (en) Methods and compositions for the treatment of Sengers syndrome
AU2019392670B2 (en) Methods and compositions for the treatment of Sengers syndrome
HK1219673B (en) Methods and compositions for the prevention or treatment of barth syndrome
HK1219673A1 (en) Methods and compositions for the prevention or treatment of barth syndrome

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20190222