Aldevron and Integrated DNA Technologies Manufacture World’s First mRNA-based Personalized CRISPR Therapy
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.
The technically complex, N of 1 therapy required a new guide RNA (gRNA) sequence, new mRNA-encoded base editor, custom off-target safety services and a clinically validated lipid nanoparticle (LNP) formulation, marking an industry milestone that demonstrates how the U.S. continues to lead the way in mRNA gene editing therapies to improve human health for all. The outcome is featured in a study published today in The New England Journal of Medicine and provides proof of concept for the potential of safe, and effective, personalized CRISPR therapy in the future.
