Marnetegragene Autotemcel: First Approval.
FDA approved the first gene therapy for a rare severe immune disorder, offering children a chance at normal immune function without needing a matched donor.
Marnetegragene autotemcel (KRESLADI, Rocket Pharmaceuticals) received first US FDA approval in March 2026 for pediatric severe leukocyte adhesion deficiency type I (LAD-I) lacking an HLA-matched sibling donor. This record was retained from the prior triage attempt for PubMed pipeline handoff.
What the study was
- Study design
- regulatory milestone review
- Category
- rare_diseases
- Maturity
- Validated
- Journal
- Mol Diagn Ther
Why it surfaced
First FDA approval of a gene therapy for LAD-I—a rare, life-threatening pediatric primary immunodeficiency—in March 2026; significant regulatory milestone for HSC gene therapy and the rare disease pipeline.
A plain-language summary of published research — not medical advice. Talk to a clinician about your own care.