Aldevron and IDT Manufacture First Personalised CRISPR Therapy for Infant with UCD

• Aldevron and IDT have successfully manufactured the world’s first personalised CRISPR therapy to treat an infant with urea cycle disorder (UCD).

• The mRNA-based drug product was developed in just six months, three times faster than standard gene editing timelines.

Aldevron, a global supplier of DNA, RNA, and protein, and Integrated DNA Technologies (IDT), a genomics solutions provider, have announced the successful production of the first personalised CRISPR gene editing drug for a paediatric patient with urea cycle disorder. Developed in partnership with the Children’s Hospital of Philadelphia (CHOP) and the University of Pennsylvania, the therapy was delivered in six months—significantly ahead of conventional timelines.

The N-of-1 mRNA-based therapy was designed specifically for a child suffering from neonatal-onset CPS1 deficiency. It included a new guide RNA, a novel mRNA-encoded base editor, custom safety services, and a lipid nanoparticle (LNP) formulation. Manufacturing was completed by Aldevron and IDT, both part of Danaher Corporation, with LNP support from Acuitas Therapeutics.

“This CRISPR therapy was made under exceptional circumstances—not something our industry is built to do consistently,” said Mark Wetzel, VP/GM of mRNA CDMO Services at Aldevron. “The future of rare disease treatment is now brighter as a result.”

The work complements the mission of the Danaher-IGI Beacon for CRISPR Cures, a collaborative initiative launched in January 2024 to accelerate gene-editing medicines for rare diseases. The outcome is detailed in a study published in The New England Journal of Medicine, offering proof of concept for rapid, safe, personalised CRISPR therapies.

The infant was treated at CHOP, with the team securing EIND approval for clinical use. The collaboration is being cited as a model for future integration between academic medicine and industry to accelerate personalised treatment for genetic disorders.