FDA Grants Approval for Gene-Editing Treatment Targeting Sickle Cell Disease

The US Food and Drug Administration has approved a breakthrough gene-editing treatment for sickle cell disease. 

Sickle cell is a debilitating, painful disease that predominantly affects people of color, with about 100,000 people in the United States affected by the condition.

The landmark therapy is poised to repair the gene that leads to the disease, marking a significant innovation for sickle cell patients worldwide

The treatment has been named Casgevy and was developed by Vertex Pharmaceuticals and CRISPR Therapeutics. 

The introduction of this innovative therapy has been hailed as a beacon of hope for sufferers of sickle cell disease.

Johnny Lubin, a 15-year-old from Connecticut who has lived with sickle cell disease since infancy, offered insight into the transformative potential of the treatment.

Lubin inherited the gene responsible for the disease from both his parents and has been in and out of the hospital in pain for more than a decade. Doctors told Lubin he would not live past 40.

However, a clinical trial for gene-silencing therapy complements gene editing and promises to manage complications in the long run, over which gene editing offers hope.

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CRISPR Gene-Editing Offers Hope for Sickle Cell Disease

fda-grants-approval-gene-editing-treatment-targeting-sickle-cell-disease
The US Food and Drug Administration has approved a breakthrough gene-editing treatment for sickle cell disease.

The doctors at the New York-Presbyterian/Columbia University Irving Medical Center coordinated Lubin’s therapy. 

They were able to edit Lubin’s cells and reprogram them to produce fetal hemoglobin, which has been proven to be somewhat protective. 

The editing technology, called CRISPR, was used to increase the number of fetal hemoglobin cells, which diminished for most sufferers of sickle cell after birth. 

Only when the protective layer of hemoglobin cells is reduced does the disease take hold? The edited cells were then replaced back into Lubin’s bloodstream.

The new treatment prompted considerable improvement in Lubin’s health and prospects for a longer, healthier life. 

While experts continue to caution that long-term follow-up treatment is necessary and gene editing is expected to cost several million dollars per patient, the outcome of the trial offers a significant breakthrough in the treatment of sickle cell disease. 

This gene-editing treatment has the potential to change lives, and with more research, it could be the cure that sickle cell disease has been waiting for. 

Further testing is currently underway, and as scientists and researchers continue to monitor the progress of gene-silencing and gene-editing therapies, we can expect even more breakthroughs in the treatment of sickle cell disease.

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