Compositions and methods for the prevention and/or treatment of mitochondrial disease, including Friedreich's ataxia
AssigneeStealth BioTherapeutics Inc.
InventorsDennis Keefe, Guozhu Zheng
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Abstract
The disclosure provides therapeutic compounds, compositions (e.g., therapeutic agents or medicaments) and methods for preventing or treating mitochondrial disease such as Friedreich's ataxia in a mammalian subject, reducing risk factors, signs and/or symptoms associated with mitochondrial disease, such as Friedreich's ataxia, and/or reducing the likelihood or severity of mitochondrial disease such as Friedreich's ataxia. The disclosure further provides novel intermediates for the production of said therapeutic compositions. In some instances, the intermediates may themselves by therapeutic agents or prodrugs of therapeutic agents (e.g. reduced forms of the therapeutic compounds).
Claims6
A compound of formula X, or a pharmaceutically acceptable salt, hydrate, and/or solvate thereof:
- wherein,
- each of R1′ and R2′ is independently C1-C3 alkyl;
- R3′ is H, D, F, —CH3, —CF3, —OCH3 or —OCF3;
- each Z is independently H, D or F;
- a is 4, 5, 6, 7, 8 or 9;
- and
- each of R30, R31 and R32 is independently H, D or F, provided, however, that at least two of R30, R31 or R32 is F.
Dependencies are inferred from the claim text.