FDA approves Gene Therapy for Severe Leukocyte Adhesion Deficiency-I

Severe leukocyte adhesion deficiency type I (LAD‑I) is a rare genetic disease that compromises immune defenses, leaving children susceptible to frequent and life‑threatening infections.

In a UCLA clinical trial, Dr. Donald Kohn developed a gene therapy that corrects the underlying genetic defect by adding a healthy copy of the gene to a patient’s own blood stem cells.

Now approved by the FDA under the accelerated approval pathway, the therapy may provide a roadmap for bringing gene therapies for other rare diseases from the laboratory to the clinic.

Read the full article in the UCLA Newsroom.