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Aldevron And Integrated DNA Technologies Manufacture World's First mRNA-Based Personalized CRISPR Gene Editing Drug Product To Treat Infant With Urea Cycle Disorder

Benzinga·05/15/2025 17:03:13
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  • N of 1 therapy uniquely developed, on demand, for infant with life-threatening rare metabolic disorder in six months
  • Results published in The New England Journal of Medicine and showcased at American Society of Gene & Cell Therapy Annual Meeting

Aldevron, a global leader in the production of DNA, RNA and protein, together with Integrated DNA Technologies (IDT), a global leader in genomics solutions, announced the successful manufacture of the world's first personalized CRISPR gene editing drug product to treat an infant with urea cycle disorder (UCD). With no current cure for UCDs, the Children's Hospital of Philadelphia (CHOP) and the University of Pennsylvania (Penn) engaged Aldevron and IDT, both part of Danaher Corporation (NYSE:DHR) to manufacture a novel mRNA-based personalized CRISPR therapy in six months—three times faster than the standard timeline for gene editing drug products.