biotech / news / / Medical Xpress
Dr. Donald Kohn has been developing gene therapies for rare pediatric immune disorders for over 30 years.
Kresladi is the first FDA-approved gene therapy for severe leukocyte adhesion deficiency-I in the U.S.
KEY POINTS
- All nine clinical trial patients survived without bone marrow transplant or immune rejection after Kresladi treatment.
- Kresladi uses patients' own stem cells with a healthy ITGB2 gene, reducing toxicity and immune complications.
- This FDA approval is the first ever for a CIRM-supported gene therapy, marking a major milestone.
- Long-term follow-up is required to confirm Kresladi's clinical benefit through a post-marketing registry.
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