# The first-ever U.S. Food and Drug Administration-approved therapy for severe leukocyte adhesion deficiency-I.

*biotech · news · 2026-03-27 · News-Medical.net*

## Key points

- Kresladi is the first FDA-approved gene therapy for severe leukocyte adhesion deficiency-I in the U.S.
- The therapy uses patients' own blood stem cells modified with a healthy ITGB2 gene, avoiding immune rejection.
- All nine clinical trial patients survived without transplants, with no graft failure or immune rejection reported.
- CIRM co-funded the trial, marking its first FDA approval for a project it supported.
- Kohn's gene therapy for ADA-SCID shows a 95% success rate in long-term follow-up of 62 children.

**Companies:** Rocket Pharmaceuticals
**Countries:** United States, United Kingdom, Spain

[Read the full story on News-Medical.net](https://www.news-medical.net/news/20260327/FDA-approves-first-gene-therapy-for-rare-pediatric-immune-disorder.aspx)

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