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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The commitment and compassion with which we care for all children and families is matched only by the pioneering spirit of discovery and innovation that drives us to think differently, to find answers, and to build a better tomorrow for children everywhere.

Kevin B. Churchwell, President and CEO

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