Why We Walk

The future of gene therapy is happening at CHOP. Trials here were instrumental in achieving FDA approval for two gene therapies that fix the genetic problem that causes sickle cell disease. But CHOP’s visionary researchers continue to work relentlessly. They are finding ways to make gene therapy treatment even better and without the need for chemotherapy. This approach could expand access to gene therapies — a game-changer for patients.

Your participation can change the lives of children with sickle cell disease and help CHOP provide its world-class, comprehensive care. CHOP offers the fullest range of services, including blood transfusions, an acute care unit that sees children immediately, care for lung/airway conditions, a stroke team for children at risk, state-of-the-art imaging technology, and counseling to help children and families cope with chronic illness.

• Two gene therapies received FDA approval in December 2023. CHOP physician-scientists chaired the international study committee for one of the gene therapy clinical trials, consulted on the design for the other one, and testified before the FDA.
• CHOP was the first to identify the neuroradiologic findings in children with sickle cell disease and stroke, and to describe safe and effective use of transfusion therapy for the prevention of recurrent stroke.
• CHOP discovered novel red cell enzymes and factors regulating globin gene regulation and hemoglobin stability key to advances in gene therapy.

The Cure Sickle Cell Walk & Family Fun Day, presented by West Pharmaceutical Services, is a celebration of all of our sickle cell warriors who have been cared for by CHOP’s Comprehensive Sickle Cell Center. We honor each and every one of them. Our patient ambassadors represent the millions of children worldwide with sickle cell disease. As they share their stories, they help people understand what it’s like living with this disease is and why it’s worth supporting CHOP.