CRISPR genome editing

Behind the breakthrough: How Integrated DNA Technologies and Aldevron enabled the world’s first personalized CRISPR therapy

The world’s first personalized CRISPR therapy was delivered to a newborn in just six months and was made possible with the contribution of coordinated expertise of IDT and Aldevron, setting a new standard for precision medicine. IDT synthesized the therapeutic-grade guide RNA used in the CRISPR therapy and led a comprehensive off-target safety analysis across over 300 genomic sites, ensuring precision and safety at record speed. Aldevron manufactured the custom mRNA encoding the base editor and completed full drug product formulation, packaging, and quality control. Danaher—IGI Beacon for CRISPR Cures initiative, a collaboration between Innovative Genomic Institute (IGI) and Danaher Corporation, aims to create a scalable framework for delivering N-of-1 gene-editing therapies for rare genetic diseases.