Cell and Gene Therapy at Children’s Hospital Los Angeles

Children's Hospital Los Angeles is one of the nation's leaders in pediatric cell and gene therapy research and treatment. We offer more state-of-the-art cell and gene therapy treatments than any other pediatric hospital on the West Coast, providing life-changing solutions for children.

As a clinical trial site, we have a long history of bringing gene therapy and cell therapy innovations to children, adolescents, and young adults:

CHLA played a critical role in the clinical trial that paved the way for Hemgenix®, the FDA approved gene therapy that treats hemophilia B.
Since its FDA approval, we have treated more patients with Elevidys® for Duchenne muscular dystrophy than almost any other center in the country.
We pioneered retinal gene therapy on the West Coast and have performed the most Luxturna® procedures in the country.
CHLA played a pivotal role in two multicenter trials leading to the approval of Kymriah® for the treatment of B-cell acute lymphoblastic leukemia.
In partnership with the Keck School of Medicine of USC, we constructed one of the first academic cell and gene manufacturing facilities in the nation.

The Children’s Hospital Los Angeles Difference

CHLA's specialists have extensive expertise in pediatric cell and gene therapy, helping to ensure the best possible outcomes for children and young adults. Our teams work together across the hospital to provide comprehensive and personalized care to each patient and family—all at one location.

We are also committed to advancing next-generation cell and gene therapies, giving patients the opportunity to participate in leading-edge clinical trials.

Conditions We Treat

Duchenne Muscular Dystrophy (DMD): This genetic condition causes progressive muscle degeneration, but gene therapy can help slow progression by introducing healthy genes to strengthen muscles.
Sickle Cell Disease: By replacing defective genes, gene therapy can reduce or eliminate the painful episodes associated with this blood disorder, offering a chance for a more normal life.
Hemophilia A & B: For patients with these blood-clotting disorders, gene therapy helps the body produce the necessary proteins to reduce or even stop the need for regular infusions.
Spinal Muscular Atrophy (SMA): This life-threatening condition affects muscle strength, but gene therapy can halt disease progression by addressing the underlying genetic cause.
Congenital Blindness: Gene therapy offers the potential to restore vision by delivering healthy genes to retinal cells, making it one of the few options for those with inherited retinal diseases.
Transfusion-Dependent Beta Thalassemia: Patients with this blood disorder rely on frequent transfusions, but gene therapy can help them become transfusion-independent by correcting the faulty genes.
B-cell Acute Lymphoblastic Leukemia (ALL) and Diffuse Large B-Cell Lymphoma (DLBCL): Cell therapy strengthens the immune system to fight cancer cells, offering a new approach for patients with aggressive cancers including B-cell ALL and diffuse large B-cell lymphoma (DLBCL).

Treatments We Offer

CHLA provides comprehensive care through our hospital Infusion Center, Bone Marrow Transplantation Unit, surgery centers, pharmacy, and stem cell labs. We currently offer the following FDA-approved cell and gene therapies:

Casgevy® (treats sickle cell disease and transfusion-dependent beta thalassemia)
Elevidys® (treats Duchenne muscular dystrophy)
Hemgenix® (treats hemophilia B)
Kymriah® (treats B-cell acute lymphoblastic leukemia and diffuse large B-cell lymphoma)
Luxturna® (treats inherited retinal disease)
Lyfgenia™ (treats sickle cell disease)
Omisirge® (treats patients with leukemia who need a bone marrow transplant)
Roctavian™ (treats hemophilia A)
Zolgensma® (treats spinal muscular atrophy)
Zynteglo™ (treats transfusion-dependent beta thalassemia)

Learn more about cell and gene therapy treatment at CHLA.

Clinical Trials and Research

CHLA is a leading pediatric academic medical center where our focus is on innovative research to improve the health of children. We are an active clinical trial site, currently enrolling patients in numerous studies that contribute to the development of transformational cell and gene therapies. We are proud to have played a critical role in clinical trials for cell and gene therapies that helped gain FDA approval for drugs, such as Hemgenix to treat hemophilia B and Kymriah to treat B-cell acute lymphoblastic leukemia and diffuse large B-cell lymphoma. Learn more about The Saban Research Institute and our clinical trials.

Contact Us

Our specialists at CHLA are here to support you. To see if your child might qualify for cell and gene therapy, please contact the appropriate team based on your child’s condition:

For hemophilia A & B, sickle cell disease, transfusion-dependent beta thalassemia, B-cell acute lymphoblastic leukemia, and diffuse large B-cell lymphoma, contact the Cancer and Blood Disease Institute at 323-361-4100.
For Duchenne muscular dystrophy and spinal muscular atrophy, contact the Neurological Institute at 323-361-2471.
For congenital blindness, contact The Vision Center at 323-361-2347.