FDA Clears First Gene Therapy for Severe LAD-I in Children
TL;DR Summary
The FDA approved Kresladi (marnetegragene autotemcel), the first gene therapy for severe Leukocyte Adhesion Deficiency Type I in pediatric patients without an HLA-matched donor, using autologous gene-modified stem cells to restore ITGB2 function and CD18/CD11a on white blood cells. The treatment is given as a single IV infusion after conditioning, based on biomarker improvements in an open-label trial, with post-marketing confirmatory studies required and safety monitoring for cytopenias and infections.
- FDA Approves First Gene Therapy for Severe Leukocyte Adhesion Deficiency Type I fda.gov
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- Rocket Pharmaceuticals Announces FDA Approval of KRESLADI™ for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I) Yahoo Finance
- Why Is Rocket Pharma Stock Soaring Friday? Benzinga
- Rocket’s Gene Therapy Wins FDA Greenlight, Clearing Way for Future Products BioSpace
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