CRISPR Gene Therapy Milestones: Pediatric Approval and Functional Cures
Key Questions
What FDA approvals have been granted for CRISPR-based therapies in children?
The FDA approved Casgevy for children as young as 2 with sickle cell disease and beta thalassemia. This marks a key regulatory milestone in pediatric gene therapy applications.
What progress has been reported in functional cures using gene therapy?
A functional cure case for sickle cell disease was reported in Louisiana. Additionally, gene therapy targeting CTLA-4 insufficiency is advancing toward clinical trials in 2028.
How has CRISPR performed in treating hereditary angioedema and other conditions?
A single gene-editing infusion reduced severe swelling attacks by 87 percent in trials for hereditary angioedema. Separately, the first baby treated with custom CRISPR base-editing therapy continues to thrive one year later.
FDA approved Casgevy for children as young as 2 with sickle cell disease and beta thalassemia. A functional cure case reported in Louisiana. Gene therapy for CTLA-4 insufficiency heading to trials in 2028. This is a climaxing story with regulatory and clinical momentum.