Gene Therapy Program

Gene Therapy Program | Conditions We Treat

We currently have multiple open and enrolling clinical trials. In addition, we are a certified treatment center for providing the FDA-approved CAR T-cell therapy called KYMRIAH™ for relapsed B-cell acute lymphoblastic leukemia (ALL) and for the gene replacement therapy called for Zolgensma® for spinal muscular atrophy (SMA).

Dr. David Williams posing with 2 patients and their family

Adrenoleukodystrophy (ALD)

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A tattoo of a protein strain on the inside of the upper arm

Beta Thalassemia

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Two scientists working in a lab together. One observes while the other uses a piping tool..

Hemophilia A

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Stock photo of a scientist looking at a Petrie dish.

Ornithine Transcarbamylase (OTC) Deficiency

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Manny all smiles as he gets his heart rate taken.

Sickle Cell Disease (SCD)

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Artistic illustration of DNA strands inside of a medicine capsule

X-linked Chronic Granulomatous Disease (CGD)

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Gallagher family whose son, Paul, underwent SCID gene therapy

X-Linked Severe Combined Immunodeficiency (SCID-X1)

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We are proud to serve a broad and diverse patient population that often comes to us for answers they can't find anywhere else. This top ranking is a credit and a tribute to our extraordinary and deeply committed clinicians, researchers and so many others who dedicate every day to improving and advancing the health and well-being of all children.

Sandra L. Fenwick, CEO

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