Gene therapy (Lentiviral vector) · Gene replacement · developed by Rocket Pharmaceuticals
KRESLADI (marnetegragene autotemcel) is an autologous hematopoietic stem cell gene therapy that delivers functional ITGB2 gene copies via lentiviral vector into patient CD34+ cells. Transduced cells engraft in bone marrow and produce functional CD18 protein, enabling leukocyte adhesion and extravasation. It treats pediatric patients with severe LAD-I lacking HLA-matched sibling donors for transplant.
Summary written with AI from the sources listed at the end of this page.
From the indications section of the FDA label (version of Jul 1, 2026). Read the full label for the exact wording, limitations and safety information.
| Trial | Phase | Condition | Status | Sponsor |
|---|---|---|---|---|
| NCT03812263 | Phase 1/2 | Leukocyte Adhesion Defect - Type I | Completed | RKTO |
Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.
Leukocyte Adhesion Defect - Type I
Updated Oct 3, 2026. Something wrong or missing? Report it on the RKTO page.
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