Two groundbreaking gene therapies, ExaCel by Vertex Pharmaceuticals and LovoCel by bluebird bio, have been approved by the U.S. Food and Drug Administration (FDA) for the treatment of sickle cell disease. These therapies utilize CRISPR gene editing technology and offer a curative path towards transplantation for individuals affected by this condition. The process involves blood transfusions, extraction and manipulation of blood stem cells, and their subsequent return to the patients.
While these gene therapies are considered potentially more targeted and effective than traditional bone marrow or stem cell transplants, they also carry risks such as long-term side effects, lowered immunity, infection, and infertility. Similar advancements in gene therapy are also being explored for β thalassemia, a monogenic disease causing anemia. Clinical trials for gene addition and gene editing-based treatments for transfusion-dependent β thalassemia are showing promising results, raising hopes of significantly improving the quality of life for patients.
Although gene therapy holds the potential to offer a more permanent solution for genetic disorders, it is still in its early stages and requires further research and clinical trials to fully comprehend its risks and benefits. Despite this, the promise of gene therapy provides a beacon of hope for individuals with genetic disorders, as the medical field continues to refine the technology and expand its applications, moving closer to a future where these diseases can be eradicated.
