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Ex Vivo Gene-editing Platform for Hematopoietic Stem Cells

The hematopoietic stem cell platform aims to develop innovative therapies for β-thalassemia and other anemias based on the autologous hematopoietic stem cells through gene editing.

Focusing on key clinical issues closely related to the treatment through hematopoietic stem cell transplantation including R&D of novel drugs for treatment through gene editing of hematopoietic stem cells, ex vivo amplification and development of self-renewal technology of hematopoietic stem cells, characteristic biomarker exploration of hematopoietic stem cells, and research of homing mechanism of hematopoietic stem cells, the platform has developed a series of innovative technologies with proprietary intellectual property rights.

The platform has attracted talents focusing on gene editing, hematology, immunology, cell therapy and other fields, forming an internationally competitive R&D team.

With advanced gene editing technology (academic achievements) and quality clinical resources (hospitals), the platform aims to develop novel drugs for hematopoietic stem cells, in order to bring treatments to patients with related diseases.