Invention Application
- Patent Title: COMPOUNDS FOR USES IN PHARMACOLOGICAL INDUCTION OF HBF FOR TREATMENT OF SICKLE CELL DISEASE AND ß-THALASSEMIA
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Application No.: US17439630Application Date: 2020-03-25
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Publication No.: US20220152036A1Publication Date: 2022-05-19
- Inventor: Weir-Torn JIAANG , Che-Kun James SHEN
- Applicant: Academia Sinica , National Health Research Institutes
- Applicant Address: TW Taipei City; TW Miaoli County
- Assignee: Academia Sinica,National Health Research Institutes
- Current Assignee: Academia Sinica,National Health Research Institutes
- Current Assignee Address: TW Taipei City; TW Miaoli County
- International Application: PCT/US2020/024727 WO 20200325
- Main IPC: A61K31/52
- IPC: A61K31/52 ; A61P7/06 ; C07D473/16 ; C07D473/18 ; A61K31/5377

Abstract:
Provided herein are compounds of Formula (I). The compounds described herein are useful in treating a disease associated with the expression of endogenous embryonic/fetal globin (e.g., γ globin) in erythrocytes (e.g., treating β-thalassemia and/or sickle cell disease through at least the induction of the globin gene expression in erythrocytes), and/or anemia (β-thalassemia and/or sickle cell anemia). The compounds described herein are useful in treating, delaying, and/or preventing the adverse effects of β-thalassemia and/or sickle cell disease, inducing γ globin production, and/or inducing the expression of embryonic/fetal globin genes in a subject, cell, tissue, or biological sample. Also provided in the present disclosure are pharmaceutical compositions, kits, and methods of using the compounds for inducing γ globin production described herein and for treating any of the target diseases described herein.
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