Aldevron and Integrated DNA Technologies (IDT) have announced the successful development of the first personalized, on-demand CRISPR gene editing drug product to treat an infant with a rare, life-threatening urea cycle disorder (UCD).
The N of 1 therapy addressed neonatal-onset CPS1 deficiency, a severe form of UCD, for which no curative therapies currently exist. The treatment was manufactured in just six months, one-third the typical timeline for such gene editing therapies, and marks a milestone in precision medicine.
Commissioned by the Children’s Hospital of Philadelphia (CHOP) and the University of Pennsylvania, the project brought together Aldevron’s mRNA manufacturing capabilities, IDT’s guide RNA and safety services, and lipid nanoparticle (LNP) delivery systems from Acuitas Therapeutics. Aldevron and IDT are both part of Danaher Corporation.
The investigational therapy incorporated a novel mRNA-encoded base editor, custom guide RNA, and a clinically validated LNP formulation. It was administered under an emergency IND (EIND) and is detailed in a study published in The New England Journal of Medicine and showcased at the American Society of Gene & Cell Therapy (ASGCT) annual meeting. The case serves as proof-of-concept for future applications of rapid, individualized gene editing approaches.
“What we’ve accomplished together sets a new gold standard for operationalizing the future of medicine,” Sandy Ottensmann, vice president and general manager of Gene Writing & Editing at IDT, said in a statement. “The implications of this work are profound and illuminate how collaborations between academic medicine and industry can enable major science wins.”
The achievement aligns with the broader goals of the Danaher-IGI Beacon for CRISPR Cures initiative, which aims to develop scalable, modifiable platforms for treating a wide range of genetic diseases. The program leverages combined expertise from academic and industry partners to streamline R&D and manufacturing timelines for gene editing-based medicines.