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In vitro cell culture methods for beta-thalassemia using activin type ii receptor ligand traps

An in vitro cell culture, activin receptor technology, applied in cell culture active agents, receptors/cell surface antigens/cell surface determinants, chemical instruments and methods, etc., can solve problems such as unmet medical needs

Pending Publication Date: 2018-07-31
CELGENE CORP +1
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

[0008] Given the current lack of safe and effective drug therapies for the treatment of β-thalassemias, e.g., transfusion-dependent and non-transfusion-dependent Significant unmet medical needs remain for novel therapies for β-thalassemia and for complications of ineffective erythropoiesis, methods for diagnosing β-thalassemia, and methods for monitoring treatment for β-thalassemia

Method used

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  • In vitro cell culture methods for beta-thalassemia using activin type ii receptor ligand traps
  • In vitro cell culture methods for beta-thalassemia using activin type ii receptor ligand traps
  • In vitro cell culture methods for beta-thalassemia using activin type ii receptor ligand traps

Examples

Experimental program
Comparison scheme
Effect test

Embodiment 1

[0380] 8.1 Example 1. Erythropoietic responses of ligand wells for activin receptors in culture from β-thalassemia patients

8.1.1 Background technology

[0381] The hallmark of beta-thalassemia is ineffective erythropoiesis leading to anemia and tissue hypoxia. Activins have been shown to affect erythropoiesis in late maturation. ActRIIA-hFc (SEQ ID NO:7) is a recombinant type IIA activin receptor (ActRIIA) ligand trap that binds activin A / B and other transforming growth factors with high affinity. In animal models, ActRIIA-hFc (SEQ ID NO:7) reverses bone loss and increases hemoglobin and hematocrit through mechanisms that are not fully understood.

[0382] This example studies the effect of ActRIIA-mFc (see, e.g., U.S. Patent No. 8,173,601 and Carrancio et al., 2014, British Journal of Haematology, 165:870-882) on different stages of differentiation and maturation from β-thalassemia patients. Molecular mechanisms underlying the effects on erythropoiesis.

[0383] 8.1.2 M...

Embodiment 2

[0415]8.2 Example 2. Research on the regulation of erythropoiesis by Sotatercept (ACE-011) in human normal and β-thalassemia erythrocyte liquid culture system

[0416] 8.2.1 Introduction

[0417] This example provides a more detailed description of some of the experiments described in Example 1 (Section 8.1) and others compared to Example 1 (Section 8.1).

[0418] 8.2.2 Materials and methods

[0419] (a) CD34 + Cell Separation

[0420] Peripheral blood obtained from 5 β-thalassemia patients and 5 healthy donors and prepared CD34-enriched by using lymphocyte separation medium (Cappel, Aurora, OH) + Cell. CD34 was positively selected by a small MACS immunomagnetic separation system (Miltenyi Biotec, Auburn, CA) according to the manufacturer's instructions + cell. Briefly, to obtain normal CD34 + cells, will be 10 8 Mononuclear cells of 1 or less were washed twice, and then suspended in 300 L of sorting buffer consisting of 1× phosphate-buffered saline (PBS), 2 mM EDTA (e...

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Abstract

Provided herein are methods of treating beta-thalassemia in a subject comprising administering to the subject an activin type II receptor (ActRII) signaling inhibitor (e.g., an activin ligand trap) and utilizing one or more in vitro cell culture methods provided herein in (i) selection of the subject to be treated according to the methods provided herein; and / or (ii) monitoring of the subject being treated according to the methods provided herein.

Description

[0001] 1. Cross-references to related applications [0002] This application claims priority to U.S. Provisional Patent Application No. 62 / 164,367, filed May 20, 2015, and U.S. Provisional Patent Application No. 62 / 320,032, filed April 8, 2016, each in its entirety It is hereby incorporated by reference and for all purposes. [0003] 2. Sequence Listing [0004] The present application is filed together with a Sequence Listing filed with the filename "12827_965_228_SeqListing.txt" created on May 16, 2016, with a size of 93 kilobytes. This Sequence Listing is hereby incorporated by reference in its entirety and for all purposes. 3. Technical field [0005] Provided herein are methods of treating beta-thalassemia in a subject comprising administering to the subject an activin receptor type II (ActRII) signaling inhibitor (e.g., an activin ligand Trap) and using the present invention if One or more in vitro cell culture methods are provided: (i) selection of a subject to be tr...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): C07K14/705C07K14/52C07K14/495C07K14/71C07K14/535C07K14/545C12N1/00C12N5/077
CPCC12N5/0641C12N2501/16C07K14/71C07K2319/30A61P7/00A61K38/179A61K35/18A61K39/39541A61K39/3955A61K2039/505A61K2039/515C12Q1/025G01N33/4833
Inventor 玛丽亚·卡佩利尼维多利亚·松
Owner CELGENE CORP
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