World Thalassaemia Day 2025 | FDA's first approved gene therapy patient celebrates two-year treatment anniversary

Rahemeen Nabeel was diagnosed with beta-thalassemia when she was just 2 months old. She had a severe form of the disease, which required blood transfusions every two to five weeks in the hospital.

Children’s Hospital of Philadelphia (CHOP), which has been at the spearhead of gene therapy for beta-thalassemia, made it easy for Rahemeen as now she no longer needs transfusions. 

Thalassemia, a blood disorder, causes the body to have less of the protein haemoglobin than the normal count which will affect the oxygen supply across the body. This can eventually lead to a condition called anaemia.

In August 2022, the US Food and Drug Administration (FDA) approved the first gene therapy for transfusion-dependent beta-thalassemia called Zynteglo.

11-year-old Rahemeen Nabeel was the first one to get the approval from FDA for gene therapy. As per the hospital, after receiving the treatment, she now no longer needs transfusions and has fully recovered. 

“We need to confirm their iron levels are in a safe range because people with thalassemia get iron overload from the transfusions they receive. We need to make sure their kidneys are working well and their heart, lungs and liver are healthy”, said Dr Janet Kwiatkowski to CHOP.

What is gene therapy?

Gene therapy includes collecting stem cells from the body and sending them to the lab while they temporarily add a dysfunctional gene to the body. The modified stem cell is later infused back through chemotherapy enabling the functioning of the production of hemoglobin. 

Medical professionals at CHOP believe that the patients will be able to stop transfusions and maintain a haemoglobin level of 9 g/dL through gene therapy. 

CHOP’s Thalassemia Center in partnership with Sickle Cell and Red Cell Disorders Curative Therapy Centre and the Cellular Therapy and Transplant section offers this groundbreaking treatment. 

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