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Early clinical trial data show gene therapy reversing sickle cell anemia


 

After over a decade of preclinical research and development, a new gene therapy treatment for sickle cell anemia (SCA) is reversing disease symptoms in two adults and showing early potential for transportability to resource-challenged parts of the world where SCA is most common.

Preliminary data from a pilot Phase 1-2 clinical trial testing the gene-addition therapy were presented on Dec. 3 at the American Society of Hematology’s (ASH) annual meeting in San Diego by principal investigator Punam Malik, MD. She is a physician-scientist at the Cincinnati Children’s Cancer and Blood Diseases Institute and director of its Comprehensive Sickle Cell Center. Malik called the data promising.

One year after treatment of our first patient, and six months after treatment of our second patient, both have seen a remarkable improvement in the quality of life due to remarkable reduction in disease symptoms.

 

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