Nemours Children’s Hospital in Wilmington has been designated as a qualified treatment center (QTC) to offer a transformative gene therapy, LYFGENIA, to treat sickle cell disease.
This designation marks a significant milestone in pediatric care for families across the Delaware Valley and beyond. As a QTC, Nemours Children’s is now authorized to collect stem cells and administer the FDA-approved, one-time gene therapy offering new hope to children and adolescents living with the life-threatening blood disorder.
“Being designated as a qualified treatment center represents an extraordinary step forward for children and adolescents living with sickle cell disease and reflects our unwavering commitment to delivering the most innovative therapies to those who need them most,” said Dr. David Brousseau, Pediatrician-in-Chief, Chair of Pediatrics at Nemours Children’s Health, Delaware Valley. “We are proud to bring this groundbreaking treatment to the Delaware Valley and to provide comprehensive care — from inpatient administration to long‑term follow‑up — through the expertise of our team at the Lisa Dean Moseley Foundation Institute for Cancer and Blood Disorders.”
LYFGENIA is designed to address the underlying genetic cause of sickle cell disease, offering the potential for long-term relief and improved quality of life. The therapy is administered through a highly specialized process involving stem cell collection, genetic modification, and reinfusion — requiring the expertise and infrastructure available at Nemours Children’s.
Sickle cell disease is an inherited blood disorder that affects about 100,000 people in the United States; more than 90 percent are non-Hispanic Black or African-American, and an estimated three percent to nine percent are Hispanic or Latino. The estimated life expectancy of people with sickle cell disease in the United States 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 Dr. Emi Caywood, 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.”
LYFGENIA is manufactured by Genetix Biotherapeutics. For more information about eligibility and referrals, visit LYFGENIA.
Learn more about Nemours Children’s Health and how it seeks to transform the health of children by adopting a holistic health model that utilizes innovative, safe, and high-quality care, while also addressing children’s needs well beyond medicine.
