Edited-CD34+ cells for treatment of Beta thalassemia

Blood diseases Cell & Gene Therapy Clinical Phase 1

Project Title: Automated production of CRISPR-Cas9 gene-edited CD34+ hematopoietic stem cells for clinical treatment of Beta thalassemia

Principal Investigator (PI): Dr. Dao Thi Mai Lan

Objective:

To research and apply gene-editing technology in combination with an automated cell manufacturing system to edit hematopoietic stem cells from patients with beta thalassemia, meeting required standards and establishing a foundation for the implementation of clinical trials and the development of gene therapy in Vietnam.

Project Description (list page – 50 words):

The project applies gene-editing technology and automated cell manufacturing to modify hematopoietic stem cells from patients with beta thalassemia, establishing a foundation for clinical trials and the development of cell and gene therapies for the treatment of beta thalassemia in Vietnam.

Project Description (main page – 250 words):

Beta thalassemia is an inherited anemia caused by impaired synthesis of the beta chain of hemoglobin, resulting in reduced oxygen transport capacity of red blood cells and chronic anemia with diverse clinical manifestations. In recent years, gene therapy has been regarded as a highly promising therapeutic approach for beta thalassemia, particularly due to rapid advances in modern biotechnology. Among these advances, gene-editing technologies enable precise modification of specific genomic targets, offering the potential to address the underlying genetic causes of beta thalassemia as well as other inherited disorders.

This project focuses on the research and application of breakthrough technologies in biomedicine, including gene-editing technology and automated cell manufacturing systems. Specifically, hematopoietic stem cells harvested from patients with beta thalassemia will be gene-edited using an automated cell production platform to restore or improve hemoglobin synthesis. Following gene editing, the modified cells will undergo comprehensive evaluation and stringent quality control to ensure compliance with safety and efficacy standards required for clinical application.

This is the first project in Vietnam to perform gene editing directly on patient-derived cell samples, thereby establishing an essential scientific and technical foundation for subsequent steps, including clinical trials in humans. The outcomes of this project are expected to significantly contribute to the advancement of cell and gene therapies and to enhance national capacity for the research and treatment of genetic diseases in Vietnam.