FDA approves breakthrough gene therapy for a rare immune disorder
FDA approves breakthrough gene therapy for a rare immune disorder
In March 2026, the U.S.
Food and Drug Administration (FDA) reached a historic milestone by granting accelerated approval to Kresladi, a groundbreaking gene therapy designed for children with severe leukocyte adhesion deficiency type I (LAD-I).
This ultra-rare genetic disorder, caused by mutations in the ITGB2 gene, prevents white blood cells from fighting infections, often proving fatal in early childhood.
Kresladi represents a major medical breakthrough as the first-ever approved treatment for this condition.
Developed by Rocket Pharmaceuticals, the therapy is autologous, meaning it uses a patient’s own modified stem cells to restore essential immune function.
By reintroducing healthy copies of the ITGB2 gene, the treatment helps the body effectively clear dangerous bacteria and fungi.
Donald Kohn at UCLA, demonstrated that patients experienced fewer severe infections and sustained biomarker improvements over time.
In recognition of this effort to address a critical pediatric need, the FDA also awarded the company a Rare Pediatric Disease Priority Review Voucher.
