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FDA Advisers Set To Review New Cure For Sickle Cell Disease Soon 


A potential breakthrough in #sicklecell disease treatment emerges as a groundbreaking gene therapy faces FDA review. The therapy, using the revolutionary CRISPR gene-editing tool, aims to address the genetic root of the inherited blood disorder, which predominantly impacts Black people in the U.S.

Set for evaluation by the #FDA advisers, this pioneering gene therapy, if approved, would mark the first CRISPR-based treatment available in the U.S. The decision is anticipated by early December.

This therapy, named "exa-cel" and developed by Vertex Pharmaceuticals and CRISPR Therapeutics, presents a promising alternative to the current standard, bone marrow transplants, the only existing cure for sickle cell disease. It works by permanently altering the patient's DNA in their blood cells.

Sickle cell disease, characterized by misshapen red blood cells due to a genetic mutation, causes severe pain, organ damage, and various health complications. Traditional treatments involve medications, transfusions, or high-risk bone marrow transplants.

The gene therapy, tested on a limited number of patients, demonstrated encouraging results in reducing pain crises and hospitalizations. One patient, Victoria Gray, described feeling transformed and freed from the agony of sickle cell disease after undergoing the therapy, enabling her to engage in activities previously hindered by her condition.


Despite its potential, concerns regarding unintended genetic alterations, known as "off-target effects," linger. The FDA seeks advice from experts on the adequacy of research conducted by the companies to assess these risks.

While the potential costs of these therapies remain undisclosed, estimated prices suggest they could be notably high. Nevertheless, the impact on patients' quality of life and reduced healthcare expenses might outweigh the initial cost.

Dr. Allison King from Washington University School of Medicine expresses enthusiasm for these advancements, acknowledging the financial implications but emphasizing the immeasurable value of alleviating suffering and reducing hospitalizations for those affected by sickle cell disease.


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Link: NBCNews

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