medcelerator · clinician one-pager

First FDA-Approved Gene Therapy for Severe Leukocyte Adhesion Deficiency

KRESLADI (marnetegragene autotemcel) — one-time gene therapy for children with severe leukocyte adhesion deficiency type I when no matched sibling donor is available

FDA Approved · United States · September 11, 2026

Condition: Leukocyte Adhesion Deficiency Type I

Who this is for

This is for families of children with severe LAD-I from confirmed ITGB2 gene changes who do not have an HLA-matched sibling donor and who may be candidates for labelled US KRESLADI one-time gene therapy at a specialized treatment center. It explains the first FDA-approved LAD-I gene therapy, the Month-12 and Month-24 CD18/CD11a results from the nine-child study, infection and engraftment watchpoints, Rocket support contacts, and how to talk with a pediatric immunology / transplant team — without treating biomarker improvements as already-proven long-term survival benefit, a DIY import as a plan, or “approved” as funded — and with honest note that commercial launch is phased. Authorized is not funded.

Next clinical step

The US Food and Drug Administration (FDA) has approved this medicine. In the United States, ask your doctor or a specialist whether it suits you. They can write a prescription. Insurance or public drug plan coverage is not guaranteed.

Ways to access this treatment

United States · Prescription
US prescription (FDA-labelled)
https://www.fda.gov/vaccines-blood-biologics/kresladi

General resources for further research

ClinicalTrials.gov
US National Library of Medicine registry of clinical studies. Search by condition, drug, NCT number, and location. A listing is not enrolment, eligibility, or funding. Talk with the trial contact and a clinician before assuming a slot exists.
https://clinicaltrials.gov/

AI-produced discussion aid; not prescribing instructions. Approval does not establish coverage. Full article: https://medcelerator.com/articles/kresladi-marnetegragene-autotemcel