US Manufactures First Personalized mRNA CRISPR Therapy for Infant

Researchers in the United States have successfully manufactured the world’s first personalized mRNA-based CRISPR gene editing therapy to treat an infant suffering from urea cycle disorder (UCD), a condition with no current cure. This milestone, achieved by Aldevron and Integrated DNA Technologies in collaboration with Children's Hospital of Philadelphia and the University of Pennsylvania, involved a novel in vivo base-editing therapy tailored to the patient's unique mutation, developed and approved under an expedited timeline. The case exemplifies a patient-first approach, emphasizing personalized treatment for rare genetic diseases, as highlighted by a study published in the New England Journal of Medicine and presented at the American Association for Gene and Cell Therapy conference. Complementing this, new CRISPR-associated transposase (CAST) technology promises to precisely insert entire genes without cutting DNA, potentially enabling scalable, one-time gene correction therapies for a variety of diseases. Together, these advancements demonstrate significant progress in genome editing, moving towards safer, more efficient, and highly personalized therapeutic options for genetic disorders.