FDA Approves First Gene Therapy for Severe Leukocyte Adhesion Deficiency Type I
The FDA has approved Kresladi (marnetegragene autotemcel), marking the first gene therapy authorized for the treatment of severe Leukocyte Adhesion Deficiency Type I (LAD-I).
Manufacturers of cell and gene therapies, clinical researchers specialized in primary immunodeficiencies, and regulatory professionals managing autologous biologics.
This approval represents a regulatory milestone as the first genetic intervention for LAD-I. For industry professionals, it confirms the viability of autologous gene therapy platforms for treating rare immunologic disorders, likely necessitating robust post-market monitoring and specialized manufacturing controls.
Regulatory and clinical teams should monitor for long-term follow-up requirements typical of autologous gene therapies and ensure manufacturing protocols for autotemcel products align with current cellular therapy standards.
The U.S. Food and Drug Administration today approved Kresladi (marnetegragene autotemcel), the first gene therapy for the treatment of severe Leukocyte Adhesion Deficiency Type I (LAD-I).
Open in openFDA / FDA.gov