Experimental Treatment for Beta Thalassemia Earns Fast Track Designation

According to a story from globenewswire.com, Vertex Pharmaceuticals Inc. and CRISPR Therapeutics have recently announced that the US Food and Drug Administration (FDA) has awarded CTX001, an experimental gene-edited stem cell therapy, Fast Track Designation. This designation is given as a treatment for beta thalassemia, a rare blood disorder.

About Beta Thalassemia

Beta thalassemia is a group of genetic disorders which affect the blood. These disorders are characterized by the abnomal production of hemoglobin, the substance in red blood cells which allows them to transport oxygen. The severity of the disorder can vary widely from person to person and depends on the present mutation. These mutations affect the HBB gene which is found on chromosome 11. These mutations can be easily passed down within families. Symptoms of beta thalassemia major, the most severe form, include spleen problems, skeletal abnormalities, poor growth, anemia, liver problems, diabetes, osteoporosis, and heart failure. Treatment for beta thalassemia may include blood transfusions, surgery, and bone marrow transplant, which can be curative for some children. There is a need for improved treatments for patients with severe cases. To learn more about beta thalassemia, click here.

About Fast Track Designation

Fast Track designation is a program from the FDA that aims to accelerate the development process of investigational treatments that have the potential to treat serious, life-threatening diseases or fulfill unmet medical needs. This designation can provide meaningful benefits to the recipient, such as more frequent meetings and collaboration with the agency and potential eligibility for other incentive programs such as Rolling Review, Priority Review, or Accelerated Approval if other relevant criteria for them are also satisfied.

Treatment of a patient with CTX001 in the clinical trial setting first began in February of this year. This marked the first time that a patient had been treated in a trial with a therapy that was created using CRISPR/Cas9 technology. CRISPR has gained significant media attention because of its potential impact in treating genetically based diseases. This is not the first time that CTX001 has earned Fast Track Designation as it was also given to the therapy in January as a treatment for sickle cell disease, another rare blood disorder. 


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