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The first-ever U.S. Food and Drug Administration-approved therapy for severe leukocyte adhesion deficiency-I.
Kresladi is the first FDA-approved gene therapy for severe leukocyte adhesion deficiency-I in the U.S.
KEY POINTS
- 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.
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