Gene therapy has made significant strides in recent years, with several treatments receiving approval for various genetic disorders. Here are some notable gene therapies currently available on the market:
Zolgensma (onasemnogene abeparvovec-xioi): Approved for spinal muscular atrophy (SMA), this gene therapy targets the survival motor neuron 1 (SMN1) gene, enabling the production of the SMN protein essential for motor neuron health.
Luxturna (voretigene neparvovec): This treatment is designed for patients with inherited retinal diseases caused by mutations in the RPE65 gene. Luxturna delivers a normal copy of the RPE65 gene directly to retinal cells, improving vision.
Kymriah (tisagenlecleucel): An innovative CAR T-cell therapy, Kymriah is used to treat certain types of blood cancers. It modifies a patient’s T cells to better recognize and attack cancer cells.
Yescarta (axicabtagene ciloleucel): Similar to Kymriah, Yescarta is another CAR T-cell therapy approved for treating large B-cell lymphoma. It enhances the immune system's ability to target and destroy cancer cells.
Hemgenix (etranacogene dezaparvovec): This gene therapy is indicated for treating hemophilia B by introducing a copy of the factor IX gene, enabling the body to produce the missing clotting factor.
These gene therapies not only represent advancements in treating previously untreatable conditions but also highlight the potential of genetic medicine in transforming patient care.
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