President's Message

ASGCT's Role in Treating Sickle Cell Disease

Guangping Gao, Ph.D. - September 16, 2019

September is Sickle Cell Awareness Month, and a major goal of ASGCT’s new strategic plan is to expand our outreach to countries outside the U.S.

Sub-Saharan Africa is home to 70 percent of children who live with sickle cell disease (SCD) worldwide, a painful inherited disorder that affects red blood cells. More than 90 percent of children with SCD in resource-poor countries do not survive into adulthood, and many die before they turn five. 

September is Sickle Cell Awareness Month, and a major goal of ASGCT’s new strategic plan is to expand our outreach to countries outside the U.S. In the years ahead, we plan to identify more opportunities to promote and help increase access to gene and cell therapies, especially for patients in developing nations. 

Gene and cell therapies can help enhance the lives of patients living with SCD, and ongoing clinical trials are currently testing these modalities throughout the U.S. However, we need to expand our reach so that the most current information and access to potential treatments can get to the patients that need them.

To help these patients, we plan to engage researchers, health care providers, commercial entities, and professional societies outside of the U.S. in order to bring together as much scientific and educational programming as possible. We will establish new international partnerships and strengthen existing ones. Through our sponsorship of the Foundation for the National Institutes of Health’s International Summit in Human Genetics and Genomics, we are supporting the building of infrastructure and genetic technologies in low- and middle-income countries.

We also want to facilitate increased understanding of the global regulatory landscape, in part through our Policy Summit, so we are able to help advance gene and cell therapies outside of the U.S. We believe that collaborating in research, funding, and outreach efforts will move us toward the goal of achieving equitable access to gene therapy treatments for patients in developing nations.

ASGCT also continues to educate the public about sickle cell disease and other inherited blood disorders. The videos and infographics on the Patient Education section of our website provide dynamic, engaging resources for patients and the public to learn about how these disorders affect the human body and, and read about how gene therapy can help treat them. Through these efforts we hope to help bridge the gap between SCD patients and necessary treatments.

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