Aldevron, IDT manufacture world’s first mRNA-based personalized CRISPR therapy

Aldevron, together with Integrated DNA Technologies (IDT), announced the successful manufacture of the world’s first personalized CRISPR gene editing drug product to treat an infant with urea cycle disorder (UCD), a life-threatening rare metabolic disorder.

The Children’s Hospital of Philadelphia and the University of Pennsylvania engaged Aldevron and IDT, both part of Danaher Corp., 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 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.

Aldevron provided the mRNA and worked with LNP delivery systems specialist Acuitas Therapeutics. Together with the gRNA and safety services provided by IDT, the companies delivered a customized in vivo base-editing therapy. The patient was treated at CHOP by Rebecca C. Ahrens-Nicklas, MD, Ph.D. The infant, who was diagnosed with UCD, suffered from neonatal-onset CPS1 deficiency and was unable to remove ammonia from the body. 

The outcome is featured in a study published in The New England Journal of Medicine.