Gene Therapy for Sickle Cell Now at Nemours Children's

Children with sickle cell disease in the Delaware Valley just gained access to a treatment that seemed like science fiction only years ago.

Nemours Children's Hospital in Wilmington has been designated as a qualified treatment center to offer LYFGENIA, a one-time gene therapy that addresses the underlying genetic cause of sickle cell disease. The hospital can now collect stem cells, modify them genetically, and reinfuse them into young patients seeking relief from this painful, life-threatening blood disorder.

Sickle cell disease affects about 100,000 people in the United States, with more than 90 percent being non-Hispanic Black or African American. The condition shortens life expectancy by more than 20 years compared to the average American.

"Being designated as a qualified treatment center represents an extraordinary step forward for children and adolescents living with sickle cell disease," said Dr. David Brousseau, Pediatrician-in-Chief at Nemours Children's Health, Delaware Valley. The hospital's Lisa Dean Moseley Foundation Institute for Cancer and Blood Disorders will provide comprehensive care from initial treatment through long-term follow-up.

The therapy requires highly specialized expertise and infrastructure that only qualified treatment centers can provide. Patients undergo stem cell collection, followed by genetic modification of those cells, and finally reinfusion of the corrected cells back into their bodies.

Dr. Emi Caywood, Director of Transplant and Cellular Therapy at Nemours, emphasized the team's commitment to accessibility. "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," she said.

The Ripple Effect

This designation transforms care options for families across the entire Delaware Valley and surrounding regions. Parents who once faced limited treatment options now have access to cutting-edge therapy close to home, reducing the burden of traveling to distant medical centers while their children battle a serious illness.

The approval also reflects growing momentum in gene therapy research and implementation nationwide. As more hospitals gain qualified treatment center status, children from communities historically underserved by advanced medical care gain access to treatments that could fundamentally change their futures.

Families interested in learning more about eligibility can visit the LYFGENIA website or contact Nemours Children's Health directly.