Granted Patent
B2
US 12,268,724 · App. 18/454,319 · Granted Apr 8, 2025
Methods and compositions for the prevention or treatment of Barth Syndrome
Assignee:
Stealth BioTherapeutics Inc.
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Abstract
The disclosure provides methods of preventing or treating Barth Syndrome in a mammalian subject, reducing risk factors associated with Barth Syndrome, and/or reducing the likelihood or severity of Barth Syndrome. The methods comprise administering to the subject an effective amount of an aromatic-cationic peptide to increase expression of TAZ1 in subjects in need thereof.
Claims (8)
1. A method for reducing the number of mitochondria with abnormal mitochondrial ultrastructure, ameliorating abnormal mitochondrial ultrastructure, or maintaining mitochondrial organization in a subject having Barth Syndrome, comprising administering to the subject a therapeutically effective amount of the peptide D-Arg-2′6′-Dmt-Lys-Phe-NH 2 or a pharmaceutically acceptable salt thereof.
2. The method of claim 1 , wherein the peptide is administered daily for 6 weeks or more.
3. The method of claim 1 , wherein the peptide is administered daily for 12 weeks or more.
4. The method of claim 1 , wherein the subject is human.
5. The method of claim 1 , wherein the peptide is administered orally, topically, systemically, intravenously, subcutaneously, intraperitoneally, or intramuscularly.
6. The method of claim 1 , further comprising separately, sequentially or simultaneously administering a cardiovascular agent to the subject.
7. The method of claim 6 , wherein the cardiovascular agent is selected from the group consisting of: an anti-arrhythmia agent, a vasodilator, an anti-anginal agent, a corticosteroid, a cardioglycoside, a diuretic, a sedative, an angiotensin converting enzyme (ACE) inhibitor, an angiotensin II antagonist, a thrombolytic agent, a calcium channel blocker, a throboxane receptor antagonist, a radical scavenger, an anti-platelet drug, a β-adrenaline receptor blocking drug, an α-receptor blocking drug, a sympathetic nerve inhibitor, a digitalis formulation, an inotrope, and an antihyperlipidemic drug.
8. The method of claim 1 , wherein the pharmaceutically acceptable salt comprises acetate or trifluoroacetate salt.
Assignments (5)
ASSIGNMENT OF ASSIGNOR'S INTEREST
Recorded Feb 6, 2024
From: WILSON, D. TRAVIS
To: STEALTH PEPTIDES INTERNATIONAL, INC.
Reel/Frame 066393/0318 →
CHANGE OF NAME
Recorded Feb 6, 2024
From: STEALTH PEPTIDES INTERNATIONAL, INC.
To: STEALTH BIOTHERAPEUTICS CORP
Reel/Frame 066393/0738 →
ASSIGNMENT OF ASSIGNOR'S INTEREST
Recorded Feb 6, 2024
From: STEALTH BIOTHERAPEUTICS CORP
To: STEALTH BIOTHERAPEUTICS INC.
Reel/Frame 066505/0962 →
CHANGE OF ADDRESS
Recorded Feb 6, 2024
From: STEALTH BIOTHERAPEUTICS INC.
To: STEALTH BIOTHERAPEUTICS INC.
Reel/Frame 066506/0100 →
ASSIGNMENT OF ASSIGNOR'S INTEREST
Recorded Feb 6, 2024
From: WILSON, D. TRAVIS; BAMBERGER, MARK
To: STEALTH PEPTIDES INTERNATIONAL, INC.
Reel/Frame 066507/0781 →
Continuity (8)
Continuation
14771408
Provisional Application
61839753
· Jun 26, 2013
Provisional Application
61771534
· Mar 1, 2013
Provisional Application
61771642
· Mar 1, 2013
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