Children’s Hospital Los Angeles (CHLA) has taken a big step forward in the world of pediatric medicine by introducing its 10th FDA-approved cell and gene therapy. This addition gives CHLA the distinction of offering the most advanced cell and gene therapies for children and young adults on the West Coast, addressing a range of serious and life-threatening conditions.
The newest therapy, Casgevy, focuses on treating sickle cell disease and transfusion-dependent beta thalassemia. It joins a growing list of therapies at CHLA that target conditions such as hemophilia, leukemia, vision loss, muscular dystrophy, and spinal muscular atrophy.
Dr. Alan S. Wayne, Pediatrician-in-Chief at CHLA, called this a “transformative time” for pediatric medicine. “This new therapy gives us another tool to provide hope for families and improve the lives of children dealing with these incredibly challenging conditions” he said.
Malika Maddison, Vice President of Service Line Strategy and Operations at CHLA, echoed the sentiment: “These therapies are pushing the boundaries of what’s possible in medicine. They represent hope, progress, and better futures for the children we serve.”
CHLA now offers a total of 10 cell and gene therapies, including:
- Casgevy – Treats sickle cell disease and transfusion-dependent beta thalassemia
- Elevidys – Treats Duchenne muscular dystrophy
- Hemgenix – Treats hemophilia B
- Kymriah – For leukemia and lymphoma
- Luxturna – Restores vision in some forms of inherited blindness
- Lyfgenia – Treats sickle cell disease
- Omisirge – Helps reduce infection risks during umbilical cord transplants
- Roctavian – Treats hemophilia A
- Zolgensma – Treats spinal muscular atrophy
- Zynteglo – Treats beta thalassemia requiring transfusions
CHLA’s work in gene therapy isn’t new. Back in the 1990s, the hospital partnered with the Keck School of Medicine of USC to open one of the country’s first academic facilities for cell and gene therapy. That work led to breakthroughs like the first treatment for newborns with ADA SCID, a severe immune system disorder.
Fast forward to today, and CHLA is still leading the charge. In 2022, the hospital and USC received an $8 million grant to support research and make these therapies more accessible.
To learn more about the therapies CHLA offers, visit CHLA.org/GeneTherapy.