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.
CN122399028APending Publication Date: 2026-07-17CELGENE CORP +1
Patent Information
- Authority / Receiving Office
- CN · China
- Patent Type
- Applications(China)
- Current Assignee / Owner
- CELGENE CORP
- Filing Date
- 2016-05-12
- Publication Date
- 2026-07-17
AI Technical Summary
Technical Problem
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.
Method used
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.
Benefits of technology
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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Figure CN122399028A_ABST
Abstract
本文涉及使用ACTRII配体陷阱治疗β‑地中海贫血。本文提供通过皮下给予约0.8 mg / kg ActRII信号转导抑制剂来治疗β‑地中海贫血的方法。本文还提供调节给予对象的ActRII信号转导抑制剂的剂量的方法。
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