Clinical Trial Results Look Good for Possible Treatment for Transfusion Dependent Beta Thalassemia

According to a story from Market Screener, the biopharmaceutical company Orchard Therapeutics recently announced that the results of a recent clinical trial testing the company’ investigational product OTL-300 were encouraging. OTL-300 was being tested in the trial as a treatment for transfusion dependent beta thalassemia. Orchard Therapeutics is dedicated to the development of gene therapies for serious, life threatening illnesses.

About Beta Thalassemia

Beta thalassemia is a group of genetic disorders which affect the blood. These disorders are characterized by the abnomal production of hemoglobin, the substance in red blood cells which allows them to transport oxygen. The severity of the disorder can vary widely from person to person and depends on the present mutation. These mutations affect the HBB gene which is found on chromosome 11. These mutations can be easily passed down within families. Symptoms of beta thalassemia major, the most severe form, include spleen problems, skeletal abnormalities, poor growth, anemia, liver problems, diabetes, osteoporosis, and heart failure. Treatment for beta thalassemia may include blood transfusions, surgery, and bone marrow transplant, which can be curative for some children. There is need for improved treatments for patients with severe cases. To learn more about beta thalassemia, click here.

Study Findings

The results look at data from seven patients and follows the long term impact of their treatment with OTL-300 after over a year of follow up. Current treatments for patients with severe beta thalassemia often requires indefinite blood transfusions or bone marrow transplants. Both of these methods carry serious risks and unpleasant side effects.

While this trial represents some of the earliest interim data, the results so far are nevertheless encouraging. The data included came from both adult and child patients. The need for transfusions was substantially reduced in the adult patients. Three of the four pediatric patients who were treated were able to stop transfusions entirely after receiving the gene therapy as of the latest follow-up. Generally, younger patients tended to respond better to the therapy than older patients.

More research will be necessary to learn more about the impact of OTL-300, but so far, this gene therapy has the potential to be a new treatment option for patients with transfusion dependent beta thalassemia.

Find the abstract for this study here.


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