Personalized CRISPR Gene Editing Therapy

Aldevron and Integrated DNA Technologies (IDT) have successfully manufactured a personalized CRISPR gene editing therapy to treat an infant with urea cycle disorder (UCD). Leveraging our combined expertise, we were able to manufacture development of an mRNA-based CRISPR therapy in just six months—three times faster than the standard timeline. The therapy was delivered using in vivo base-editing, and the positive outcome was featured in a study published in The New England Journal of Medicine.

“We are unique in our ability to deliver this innovative treatment in such a short timeline," said Mark Wetzel, VP/GM mRNA CDMO Services at Aldevron. “This CRISPR therapy was made under exceptional circumstances—not something our industry is built to do consistently.”

While we are immensely proud of our role in delivering this transformative therapy, it is crucial to emphasize that we are a manufacturing company, not a therapeutics company. As such, we do not work directly with patients or patient communities.

Currently, mRNA-based personalized CRISPR therapies are in the early stages of development. However, this effort will establish a roadmap for future patients and clinical indications, paving the way for the future of personalized in vivo base editing treatments.

Read the press release announcing the manufacture of the first mRNA-based CRISPR gene editing therapy: Aldevron News: Aldevron and Integrated DNA Technologies Manufacture World’s First mRNA-based Personalized CRISPR Therapy

To learn more about this effort: The New England Journal of Medicine article

To learn more about the advancement of mRNA and next-generation RNA therapies: Alliance for mRNA Medicines