First-Ever Custom CRISPR Therapy Saves Infant With Deadly Genetic Disorder

A pivotal medical milestone has been reached. For the first time ever, researchers have used a personalized CRISPR-based gene therapy to treat an infant’s rare and life-threatening illness. Doctors at the Children’s Hospital of Philadelphia (CHOP) and Penn Medicine detailed their achievement in a study published Thursday in the New England Journal of Medicine. The treated child, named KJ, was born with a metabolic disorder known to kill up to 50% of children in their infancy. Now, three months after his first dose, KJ appears to have responded well to the treatment and is doing better than ever. “Years and years of progress in gene editing and collaboration between researchers and clinicians made this moment possible, and while KJ is just one patient, we hope he is the first of many to benefit from a methodology that can be scaled to fit an individual patient’s needs,” said Rebecca Ahrens-Nicklas, director of the Gene Therapy for Inherited Metabolic Disorders Frontier Program at CHOP ... Read full article.