(COLUMBUS, Ohio) – Nationwide Children’s Hospital has been designated an authorized treatment center for CASGEVY™ (exagamglogene autotemcel (exa-cel)), a gene therapy for the treatment of sickle cell disease.
In December 2023, the U.S. Food and Drug Administration approved CASGEVY, which was developed by pharmaceutical company Vertex Pharmaceuticals and CRISPR Therapeutics, for the treatment of sickle cell disease in adult and pediatric patients 12 years and older who require regular red blood cell tranfusions.
With Vertex Pharmaceuticals’ designation, Nationwide Children’s becomes one of a handful of facilities throughout the U.S. that is certified to have the advanced facilities and specially-trained staff necessary for the administration of CASGEVY.
“We are excited to be able to provide this kind of personalized therapy to patients who have historically had long and difficult clinical journeys managing their sickle cell disease,” said Hemalatha Rangarajan, MD, pediatric stem cell transplant physician at Nationwide Children’s. “Offering families new hope is what we strive to do every day.”
Sickle cell disease (SCD) is an inherited blood disorder that affects red blood cells, caused by hemoglobin S. Instead of red blood cells that are round, smooth and move easily through the body, carrying oxygen to different parts of the body, patients with SCD have red blood cells that are hard, sticky and sickle-shaped, which can clog blood vessels causing infection, pain and anemia.
CASGEVY is a CRISPR-based gene therapy that edits a patient’s own blood stem cells to produce high levels of the healthy, oxygen-carrying hemoglobin that is typically produced in utero.
“We are excited to be able to offer this therapy to our patients with sickle cell disease,” said Anthony Villella, MD, director of the Sickle Cell and Thalassemia Program at Nationwide Children’s. “This is an especially significant milestone because, historically, patients with sickle cell disease have been neglected when it comes to disease awareness and research investment.”
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