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US 2016/0106728 A1Published application
Methods and compositions for treating beta-thalassemia and sickle cell disease
AssigneeAcademia Sinica
InventorsChe-Kun James Shen, Yu-Chi Chou, Tsann-Long Su
Life of this patent
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Abstract
Compounds, pharmaceutical compositions, and methods for treating anemia β-thalassemia anemia or sickle cell anemia.
Claims27
A pharmaceutical composition comprising a compound of formula (I-a):
- or a pharmaceutically acceptable salt, solvate, or hydrate thereof, and a pharmaceutically acceptable excipient
- wherein:
- R1, R2, R3, R4, R6a, and R6b are each independently selected from the group consisting of hydrogen, halogen, optionally substituted alkyl, optionally substituted alkenyl, optionally substituted alkynyl, optionally substituted heterocyclyl, optionally substituted heteroaryl, optionally substituted aryl, —ORA, —OC(O)RA, —SRA, —N(RB)2, —N(RA)C(O)RA, —C(O)N(RB)2, —CN, —NO2, —C(O)RA, —C(O)ORA, —S(O)RA, —SO2RA, —SO2N(RB)2, and —NHSO2R13;
- R5 is hydrogen, optionally substituted alkyl, optionally substituted alkenyl, optionally substituted alkynyl, optionally substituted heterocyclyl or optionally substituted aryl;
- each instance of RA is independently selected from the group consistinf of hydrogen, optionally substituted alkyl, optionally substituted alkenyl, optionally substituted alkynyl, optionally substituted heterocyclyl, optionally substituted heteroaryl, and optionally substituted aryl;
- each instance of RB is independently selected from the group consisting of hydrogen, optionally substituted alkyl, optionally substituted alkenyl, optionally substituted alkynyl, optionally substituted heterocyclyl, optionally substituted heteroaryl, and optionally substituted aryl, or two RB are taken together with the intervening atoms to form a heterocycle.
3 more elements
A method of inducing γ globin production, comprising:
- contacting a cell with an effective amount compound of formula (I-a):
- or a pharmaceutically acceptable salt thereof,
- wherein:
- R1, R2, R3, R4, R6a, and R6b are each independently selected from the group consisting of hydrogen, halogen, optionally substituted alkyl, optionally substituted alkenyl, optionally substituted alkynyl, optionally substituted heterocyclyl, optionally substituted heteroaryl, optionally substituted aryl, —ORA, —OC(O)RA, —SRA, —N(RB)2, —N(RA)C(O)RA, —C(O)N(RB)2, —CN, —NO2, —C(O)RA, —C(O)ORA, —S(O)RA, —SO2RA, —SO2N(RB)2, and —NHSO2RB;
- R5 is hydrogen, optionally substituted alkyl, optionally substituted alkenyl, optionally substituted alkynyl, optionally substituted heterocyclyl, optionally substituted heteroaryl, or optionally substituted aryl;
- each instance of RA is independently selected from the group consistinf of hydrogen, optionally substituted alkyl, optionally substituted alkenyl, optionally substituted alkynyl, optionally substituted heterocyclyl, optionally substituted heteroaryl, and optionally substituted aryl;
- each instance of RB is independently selected from the group consisting of hydrogen, optionally substituted alkyl, optionally substituted alkenyl, optionally substituted alkynyl, optionally substituted heterocyclyl, optionally substituted heteroaryl, and optionally substituted aryl, or two RB are taken together with the intervening atoms to form a heterocycle.
4 more elements
A method for assessing responsiveness of a subject to a hydroxyurea, the method comprising:
- providing a sample derived from the subject, the sample comprising adult erythroid cells, which are treated by the hydroxyurea,
- measuring the expression level of a hemoglobin gene in the erythroid cells, and
- identifying the subject as being responsive to the hydroxyurea if the expression level of the hemoglobin in the adult erythroid cells is elevated as compared to adult erythroid cells not treated by the hydroxyurea.
Dependencies are inferred from the claim text.