Betibeglogene Autotemcel

A gene therapy used to treat beta-thalassemia, a genetic blood disorder in which the body produces insufficient functional hemoglobin. It involves modifying the patient's own stem cells to include a functional copy of the beta-globin gene, allowing the production of healthy red blood cells. It is a one-time treatment with the potential to reduce or eliminate the need for lifelong blood transfusions.

Delivers functional copies of a gene to target cells using a viral vector, restoring expression of the deficient protein and correcting the underlying genetic defect.

Yes, Betibeglogene Autotemcel is an approved drug. It has reached clinical phase 4. It is classified as a Gene.