Mitofusin activators and methods of use thereof
AssigneeMitochondria Emotion, Inc.
InventorGerald W. Dorn, II
Life of this patent
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Abstract
Compositions including small molecule mitofusin activators are described. The mitofusin activators are useful for treating diseases or disorders associated with a mitochondria-associated disease, disorder, or condition such as diseases or disorders associated with mitofusin 1 (MFN1) and/or mitofusin 2 (MFN2), or mitochondrial dysfunction. Methods of treatment, pharmaceutical formulations, and screening methods for identifying compounds that activate mitochondrial fusion are also described.
Claims21
A method comprising:
- administering a therapeutically effective amount of a composition comprising one or more of a mitofusin activator or a pharmaceutically acceptable salt thereof to a subject having or suspected of having a mitochondria-associated disease, disorder or condition, the mitofusin activator having a formula of
- wherein:
- X is
- Z is optionally substituted phenyl;
- Ra and Rb are independently H, F, alkyl, or C3-7 cycloalkyl, or Ra and Rb taken together form a C3-7 cycloalkyl or heterocycloalkyl;
- Rc and Rd are independently H, F, alkyl, CORg, or C3-7 cycloalkyl, or Rc and Rd taken together form a C3-7 cycloalkyl or heterocycloalkyl;
- Y is 0, CH2, cyclopropyl, or cyclobutyl;
- Rg is H, alkyl, or C3-7 cycloalkyl;
- o is 0 or 1;
- p is 1;
- and
- q is 1, 2, 3, or 4, provided that if Y is cyclopropyl or cyclobutyl, the sum of o+p+q is not less than 3 or greater than 5 and otherwise the sum of o+p+q is 5.
A composition comprising one or more of a mitofusin activator or a pharmaceutically acceptable salt thereof, the mitofusin activator having a formula of
- wherein:
- X is
- Z is optionally substituted phenyl;
- Ra and Rb are independently H, F, alkyl, or C3-7 cycloalkyl, or Ra and Rb taken together form a C3-7 cycloalkyl or heterocycloalkyl;
- Rc and Rd are independently H, F, alkyl, CORg, or C3-7 cycloalkyl, or Rc and Rd taken together form a C3-7 cycloalkyl or heterocycloalkyl;
- Y is 0, CH2, cyclopropyl or cyclobutyl;
- Rg is H, alkyl, or C3-7 cycloalkyl;
- o is 0 or 1;
- p is 1;
- and
- q is 1, 2, 3, or 4, provided that if Y is cyclopropyl or cyclobutyl, the sum of o+p+q is not less than 3 or greater than 5 and otherwise the sum of o+p+q is 5.
Dependencies are inferred from the claim text.