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ASTCT Response to FDA Approval of Gene Therapies for Treatment of Sickle Cell Disease (SCD)

ASTCT Response to FDA Approval of Gene Therapies for Treatment of Sickle Cell Disease (SCD)

ASTCT logo

The American Society for Transplantation and Cellular Therapy is an international professional membership association.

The American Society for Transplantation and Cellular Therapy® (ASTCT®) recognizes the US Food and Drug Administration’s (FDA) recently granted approval for two gene therapies aimed at treating Sickle Cell Disease (SCD). Casgevy, developed by Vertex Pharmaceuticals Inc., and Lyfgenia, developed by Bluebird Bio Inc., have been granted regulatory clearance for these therapies by the FDA after successful clinical trials and extensive review processes.

SCD is a genetically inherited red blood cell disorder. According to the Centers for Disease Control, SCD disproportionately affects Black or African American and Hispanic Americans in the United States. SCD affects an estimated 100,000 people in the United States, affecting one out of every 365 Black or African American births and one out of every 16,300 Hispanic American births.

ASTCT is a professional organization of health care professionals dedicated to the research, science and application of hematopoietic cell transplantation and related cellular therapies. 

“Gene therapy offers a revolutionary approach to treating genetic diseases like sickle cell disease that have, in many cases, been deemed untreatable,” said Miguel-Angel Perales, MD, ASTCT President. “As is the case for many novel therapies, one of the challenges is ensuring that our patients have access to these potentially life-saving treatments. ASTCT strives to provide research and to address disparities in access to care and treatment for blood-related diseases and disorders.”  

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About ASTCT

The American Society for Transplantation and Cellular Therapy is an international professional membership association of more than 3,700 physicians, investigators and other health care professionals from more than 45 countries. Our mission is dedicated to improving the application and success of hematopoietic cell transplantation and related cellular therapies. We strive to be the leading organization promoting research, education and clinical practice in the field. Visit www.astct.org

Contact Information:
Jennifer Kasowicz
ASTCT Marketing Director
[email protected]
(312) 673-4970


Original Source: ASTCT Response to FDA Approval of Gene Therapies for Treatment of Sickle Cell Disease (SCD)
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