SCD gene-editing therapy Casgevy now being offered at CHLA
Hospital also offering Lyfgenia, following its 2023 approval
Children’s Hospital Los Angeles (CHLA) is now offering Casgevy (exagamglogene autotemcel), an approved gene-editing therapy for sickle cell disease (SCD).
“This is a transformative time for Children’s Hospital Los Angeles and the pediatric community we serve,” Alan S. Wayne, MD, pediatrician-in-chief at CHLA, said in a press release. “With the addition of this gene therapy, we are further advancing our mission to create hope and build healthier futures for patients and families by providing access to paradigm-changing therapies that offer the prospect of cure and improved quality of life.”
SCD is caused by mutations in a gene needed to make hemoglobin, the protein that red blood cells use to carry oxygen through the body. The mutated hemoglobin forms clumps that deform red blood cells into the sickle-like shape that gives the disease its name. The deformed cells are prone to getting trapped inside blood vessels, blocking blood flow, which can lead to painful vaso-occlusive crises (VOCs).
Now offering two gene therapies for SCD
Casgevy, which was developed by CRISPR Therapeutics and Vertex Pharmaceuticals, is a gene-editing therapy that uses CRISPR/Cas9 technology to modify a patient’s blood stem cells to boost the production of fetal hemoglobin, an alternative form of the protein that’s normally made during fetal development. The modified stem cells are then transplanted back into the patient, giving rise to red blood cells capable of producing fetal hemoglobin that can compensate for the mutated adult version.
With the addition of Casgevy, CHLA is now offering two gene therapies for sickle cell. The hospital has been offering Lyfgenia (lovotibeglogene autotemcel), a gene therapy that works by providing patients with a gene that encodes a sickle-resistant version of adult hemoglobin.
Both therapies were approved by the U.S. Food and Drug Administration (FDA) in late 2023 to treat people with SCD, ages 12 and older, who have histories of VOCs or other complications related to blood vessel obstruction. Casgevy is also approved for transfusion-dependent beta thalassemia, a disorder where little or no hemoglobin is produced.
Along with these SCD treatments, CHLA is offering eight other gene and cell therapies for other diseases, giving the institution the widest range of pediatric cell and gene therapies on the West Coast, according to the hospital.
“These new therapies represent the next generation of medical innovation,” said Malika Maddison, vice president of service line strategy and operations at CHLA. “Our ability to offer these treatments ensures that our patients have access to world-class care.”
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