Treatment of beta-thalassemia using actrii ligand trap
By administering activin receptor type II signal transduction inhibitors to patients with β-thalassemia, the challenge of treating β-thalassemia has been solved, hemoglobin concentration has been increased, transfusion burden has been reduced, erythropoiesis has been improved, and GDF-11 levels have been lowered.
Patent Information
- Application Number
- CN202610488096.3
- Authority / Receiving Office
- CN · China
- Patent Type
- Applications(China)
- Current Assignee / Owner
- Priority Date
- 2015-10-19
- Filing Date
- 2016-05-12
- Publication Date
- 2026-07-17
AI Technical Summary
Currently, there is a lack of safe and effective drug therapies to treat β-thalassemia, especially transfusion-dependent and non-transfusion-dependent β-thalassemia, leaving patients facing the challenge of complications such as anemia and ineffective erythropoiesis.
Humanized fusion proteins of activin receptor type II signal transduction inhibitors, such as ActRIIA or ActRIIB, are administered subcutaneously to subjects at an initial dose of approximately 0.8 mg/kg or approximately 1.0 mg/kg every 21 days for the treatment of β-thalassemia.
It significantly increased the subjects' hemoglobin concentration, reduced the need for transfusions, improved erythropoiesis, reduced GDF-11 levels, and in some cases increased fetal hemoglobin levels.
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Abstract
Citation Information
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