SHERIDAN, WYOMING – May 17, 2025 – Danaher Corporation has achieved a historic milestone in genomic medicine, enabling the development and delivery of the world’s first personalized mRNA-based CRISPR therapy for an infant with a life-threatening genetic disorder—in under six months. Powered by the Danaher Business System (DBS), this breakthrough exemplifies how disciplined innovation and cross-company collaboration can dramatically accelerate biopharmaceutical timelines and patient impact.
Compressing an 18-Month Timeline into Six
The urgency was real: an infant diagnosed with neonatal-onset CPS1 deficiency—a rare urea cycle disorder causing fatal ammonia accumulation—needed a cure in weeks, not years. DBS, Danaher’s proprietary system of operational tools and methodologies, allowed teams to strip away inefficiencies and take bold, calculated action.
“From the moment we learned about the case, there was a deep commitment and understanding from everyone involved around connecting this infant patient to a potential cure,” said Demaris Mills, Vice President and Group Executive, Genomics Medicine at Danaher. “Our operating companies utilized DBS to remove barriers, act quickly and take smart, calculated risks to compress the schedule.”
A Coordinated Response Across Institutions
The call came from the Innovative Genomics Institute (IGI), with Chief Scientific Officer Sadik Kassim of Danaher’s Genomics Medicines team quickly recognizing the challenge.
“We were first alerted to this case by our collaborators at the Innovative Genomics Institute,” says Kassim. “The situation was critical: a child had been diagnosed with neonatal-onset CPS1 deficiency and needed treatment fast.”
“To help this child, we would have to turn what is typically an 18 to 24-month process to design and manufacture a CRISPR-based therapy into one that was less than six months,” Kassim says.
“It would push the limits of what was possible, but we believed that with our existing technologies, manufacturing capabilities and collaborations, we could do it.”
“In working with Danaher companies since the launch of the Beacon for CRISPR Cures, I’ve come to appreciate how serious their commitment is to creating an accelerated path to making CRISPR a clinical reality,” said Fyodor Urnov, Professor at UC Berkeley and Director, Technology and Translation at the Innovative Genomics Institute. “I thus suggested to the Penn/CHOP team that they would be excellent partners for this first-of-its-kind journey to treating a child before severe disability sets in.”
Precision Tools and Teamwork
The therapy aimed to correct a point mutation in the gene responsible for detoxifying ammonia. This required a custom combination of gene editor, guide RNA, and a lipid nanoparticle (LNP) delivery vehicle. Three Danaher companies—IDT, Aldevron, and partner Acuitas—played critical roles:
- IDT: Produced the guide RNA
- Aldevron: Supplied the mRNA-based gene editor and formulated the final drug product
- Acuitas: Provided the lipid nanoparticles
“Once we had the teams from CHOP, IDT, Acuitas and Aldevron together, we threw a piece of paper on the wall and started mapping it out, which is classic DBS,” said Mark Wetzel, VP/GM of RNA CDMO Services at Aldevron. “We created the ‘swim lanes,’ we identified the dependencies, we worked out the critical path of what needed to happen by when, and we quickly turned that into an Action Plan.”
“We really keyed into the DBS mindset of focusing on what's possible, not what's blocking our way,” Wetzel continues. “Together as a team, we quickly came up with a path to achieve what was required to support the emergency IND, the efficacy and safety studies and ultimately the drug product.”
“This was a technically challenging project, requiring a new gRNA sequence, new mRNA-encoded base editor, custom safety services and a new LNP formulation, all on a highly compressed timeline,” said Sandy Ottensmann, VP/GM of Gene Writing & Editing at IDT. “We relentlessly prioritized this project and leveraged DBS via Daily Management and Action Plans to drive progress with an ‘all hands on deck’ approach.”
The therapy was administered to the infant at just six months of age. “This was a special case. We're so proud to be involved, especially because this couldn’t have been achieved by just any company,” Wetzel continues. “Danaher’s unique portfolio of connected businesses—Aldevron and IDT, in particular—combined with the DBS mindset allowed us to meaningfully accelerate the process of execution.”
Toward Scalable Personalized Therapies
While this was an “N of 1” treatment, its success opens the door to broader applications. The case was presented at the American Society for Cell and Gene Therapy on May 15 and published in the New England Journal of Medicine. The patient is currently doing well, but longer-term outcomes remain under evaluation.
“We’re just at the beginning of what’s possible,” Ottensmann noted. “This isn’t just about one breakthrough or one patient; it’s about building a platform that can deliver personalized cures on demand, for thousands of diseases that still have no treatment.”
“To deliver this kind of personalized care at scale, we need to invest in turning CRISPR into a platform,” says Kassim. “We often talk about this as creating a CRISPR cookbook, where all of the ingredients and manufacturing processes have been defined so we know how to combine them into a safe and effective treatment. Importantly, we need to work with regulators to approve this cookbook so treatments can reach patients as quickly as possible.”
Learn more about Danaher’s genomic medicine efforts at https://www.danaher.com