Nemours Children’s Delaware brings life-changing gene therapy to children with sickle cell disease

A gene therapy called LYFGENIA is transforming the way sickle cell disease is treated in children — but it isn’t easy to administer, and it’s not yet widely available. The treatment involves stem cell collection, gene modification and reinfusion, meaning the procedure requires extensive infrastructure and expertise. But now more children and adolescents with the disease can access the life-changing treatment, as LYFGENIA has come to Nemours Children’s Hospital in Wilmington, Del. 

Sickle cell disease affects around 100,000 people in the United States; more than 90% of those with the inherited blood disorder are Black, and 3%-9% are Latino. Sickle cell disease can cause extreme pain, anemia, mobility and vision issues, and the estimated life expectancy of sickle cell patients is more than 20 years shorter than the average.

“We're proud to bring this gene therapy to our patients and we're committed to making it accessible to those who need it most,” said Emi Caywood, M.D, director of Transplant and Cellular Therapy, Department of Pediatrics, Nemours Children’s Health, Delaware Valley. “This designation is a testament to our team's dedication to delivering cutting-edge care that transforms lives."