Aldevron and Integrated DNA Technologies Manufacture World’s First mRNA-based Personalized CRISPR Therapy

• N of 1 therapy uniquely developed, on demand, for infant with life-threatening rare metabolic disorder in six months
• Results published in The New England Journal of Medicine and showcased at American Society of Gene & Cell Therapy Annual Meeting

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.

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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.

“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—given the steadfast focus and dedication of the Aldevron and IDT teams to leverage years of expertise and strong partnerships to do what was needed to improve this patient’s outcome. Collaboration between Aldevron, IDT, and Acuitas allowed for this innovation to happen, and the future of rare disease treatment is now brighter as a result.”

This accomplishment is complementary to the goal of the Danaher-IGI Beacon for CRISPR Cures to develop platform approaches that can be easily modified to develop gene-editing medicines for hundreds of devastating diseases. Launched in January 2024, the Beacon unites the finest scientific minds in gene editing at the IGI, with the R&D and manufacturing talent, technology and expertise from across Danaher’s various operating companies, to create transformative solutions.

“What we’ve accomplished together sets a new gold standard for operationalizing the future of medicine,” said Sandy Ottensmann, VP/GM, Gene Writing & Editing at IDT. “The implications of this work are profound and illuminate how collaborations between academic medicine and industry can enable major science wins. The opportunity ahead lies in continuously leveraging our CRISPR toolbox to innovate and operationalize more potential treatments and help patients in desperate search of cures.”

Aldevron provided the mRNA and worked with Acuitas Therapeutics, a private biotechnology company specializing in the development of LNP delivery systems for nucleic acid therapeutics. Together with the gRNA and safety services provided by IDT, the companies delivered a customized in vivo base-editing therapy, in a significantly compressed timeline, as a transformational therapy for the infant patient. Collaboration between all partners’ quality and regulatory teams also led to successful EIND approvals.

“This study is an important milestone,” said corresponding author of the NEJM study Kiran Musunuru, MD, PhD, MPH, ML, MRA, the Barry J. Gertz Professor for Translational Research and Director of the Genetic and Epigenetic Origins of Disease Program in the Perelman School of Medicine at the University of Pennsylvania. “The impact of this work extends beyond this particular patient and category of clinical indications—it suggests a potential roadmap for transforming CRISPR therapies for other inborn errors of metabolism and life-threatening genetic diseases. It’s an exciting future for personalized medicine.”

The patient was treated at CHOP by co-corresponding author and physician-scientist, Rebecca C. Ahrens-Nicklas, MD, PhD. The infant, who was diagnosed with UCD, suffered from neonatal-onset CPS1 deficiency and was unable to remove ammonia from the body.

To learn more about Aldevron and IDT’s capabilities, visit https://dhrls.co/aldevronIDTworldsfirst.

About Aldevron

Aldevron is a premier manufacturing partner, producing high-quality plasmid DNA, mRNA, proteins, and other key components for the development of vaccines, gene and cell therapies, immunotherapies and other treatments. As a part of the Danaher Corporation family of global science and technology companies, Aldevron supports thousands of scientists who are developing revolutionary, lifesaving treatments for millions of people. To learn more about how Aldevron is advancing biological science, visit www.aldevron.com/about-us and follow the company on LinkedIn, Facebook and YouTube.

About IDT

Building from a strong foundation of innovation, expertise, and reliability, Integrated DNA Technologies (IDT) has evolved from an oligo manufacturer to a leading genomics provider. We work shoulder-to-shoulder with scientific and global health partners to enable genomics breakthroughs at scale. Our vision of enabling researchers to rapidly move from the lab to life-changing advances reflects our ongoing commitment to a healthier, brighter future for all.

IDT is proud to be part of Danaher, a global science and technology leader. Together we combine our capabilities to accelerate the real-life impact of tomorrow's science and technology to improve human health.

For more information about IDT, visit www.idtdna.com and follow the company on LinkedIn, X, Facebook, YouTube, and Instagram.

Disclaimer: CGMP refers to products manufactured under ICHQ7; IDT engineering runs and CGMP gRNA are for development and investigational use only. The performance characteristics of this product have not been established. This product is not intended to be used as final drug product. The purchaser is solely responsible for all decisions regarding the intended use of the product and any associated legal or regulatory obligations.

About Danaher

Danaher is a leading global life sciences and diagnostics innovator, committed to accelerating the power of science and technology to improve human health. Our businesses partner closely with customers to solve many of the most important health challenges impacting patients around the world. Danaher's advanced science and technology - and proven ability to innovate - help enable faster, more accurate diagnoses and help reduce the time and cost needed to sustainably discover, develop and deliver life-changing therapies. Focused on scientific excellence, innovation and continuous improvement, our approximately 63,000 associates worldwide help ensure that Danaher is improving quality of life for billions of people today, while setting the foundation for a healthier, more sustainable tomorrow. Explore more at www.danaher.com.

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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.