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6d ago

Gene Therapy Restores Immune Function in Children with Rare Disorder

A groundbreaking trial using lentiviral gene therapy has enabled nine children with severe LAD-1 to survive and thrive, with the treatment now under FDA review.

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Eisa Hussain had pioneering treatment for a rare immunodeficiency as a baby

Overview

  • Nine children with severe leukocyte adhesion deficiency-I (LAD-1) achieved immune restoration following lentiviral gene therapy, avoiding the life-threatening prognosis of the disease.
  • The therapy reprograms patients' stem cells to produce the missing CD18 protein, enabling their immune systems to fight infections effectively.
  • Two-year follow-up data published in the New England Journal of Medicine confirmed all patients survived, with durable CD18 expression levels reaching up to 82% in some cases.
  • The trial, sponsored by Rocket Pharmaceuticals, showed no therapy-related deaths or severe adverse events, with participants experiencing fewer infections and improved wound healing.
  • The U.S. FDA is currently reviewing the therapy's Biologics License Application, raising hopes for its wider availability and potential application to other genetic and degenerative conditions.