Stealth BioTherapeutics Inc.

The present technology is directed to compositions and methods for preventing, ameliorating, or reducing the severity of one or more signs, or symptoms associated with a reduction of function, decreased expression level of, and/or deficiency in one or more of COL4A3, COL4A4 and COL4A5 genes. Also disclosed herein are methods of preventing or treating Alport Syndrome in a mammalian subject, reducing risk factors associated with Alport Syndrome, and/or reducing the likelihood or severity of Alport Syndrome. The methods comprise administering to the subject an effective amount of an aromatic-cationic peptide.

The disclosure provides methods for detecting and diagnosing diseases and conditions associated with defects in cardiolipin remodeling. In some embodiments, the present technology relates to methods for detecting the presence or amount of cardiolipin isoforms and/or the presence or amount of enzymes involved in cardiolipin remodeling.

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.

Disclosed are analogs of SBT-20. The compounds are useful for the treatment and prevention of ischemia-reperfusion injury (e.g., cardiac ischemia-reperfusion injury) or myocardial infarction.

The present disclosure relates to compositions, methods and related uses for preventing, inhibiting, ameliorating or delaying (slowing): (i) the thinning of the retinal outer nuclear layer (ONL), and/or (ii) reduction in total retinal thickness in a subject. The present disclosure further relates generally to compositions, methods and related uses for protecting: (i) the retinal outer nuclear layer (ONL) and/or the retina from light- or age-induced damage, and/or (ii) photoreceptors from light- or age-induced damage. In particular, the present technology relates to administering bevemipretide, or a pharmaceutically acceptable salt, stereoisomer, tautomer, hydrate, and/or solvate thereof to subjects in need thereof.

Disclosed are various crystalline salt forms of D-Arg-Dmt-Lys-Phe-NH 2 .

Disclosed are analogs of elamipretide (MTP-131). The compounds are useful for the treatment and prevention of ischemia-reperfusion injury (e.g., cardiac ischemia-reperfusion injury) or myocardial infarction.

The disclosure provides methods of preventing, treating, or ameliorating LV remodeling in a mammalian subject. The methods comprise administering to the subject a therapeutic amount of an aromatic-cationic peptide such as D-Arg-2,6-Dmt-Lys-Phe-NH2.

The present disclosure provides compounds, compositions, and/or methods for treating, preventing, inhibiting, amelio-rating, or delaying the onset of Huntington's disease and/or a HTT proteinopathy in a subject. The methods can comprise administering to the subject an effective amount of a peptidomimetic compound, such as (R)-2-amino-N—((S)-1-(((S)-5-amino-1-(3-benzyl-1,2,4-oxadiazol-5-yl)pentyl)amino)-3-(4-hydroxy-2,6-dimethylphenyl)-1-oxopropan-2-yl)-5- guanidinopentanamide, or a pharmaceutically acceptable salt, stereoisomer, tautomer, hydrate, and/or solvate thereof, alone or in combination with one or more other therapeutic agents.

The present technology provides methods for treating (wet or dry) age-related macular degeneration (AMD). In particular, the present technology relates to the use of elamipretide, or a pharmaceutically acceptable salt thereof, in effective amounts to treat (wet or dry) age-related macular degeneration (AMD) and thereby decrease the rate of growth of photoreceptor loss over time in a subject, such as a mammal (e.g., a human).