From the moment Baby KJ entered the world, his life hung in the balance. Born with a rare genetic condition called CPS1 deficiency, his outlook at birth was uncertain. This life-threatening disorder demanded swift action—and ultimately, an extraordinary gene therapy effort. Through a remarkable collaboration of doctors, scientists, and institutions, what began as a desperate situation became a breakthrough in gene therapy and a story of survival and hope.
When the Usual Answers Didn’t Fit
Doctors at the Children’s Hospital of Philadelphia diagnosed KJ at birth. But early on, his care team noticed that something about his case didn’t match what they typically saw with CPS1 deficiency. A nurse closely observed KJ’s reactions to treatment and flagged unusual patterns that standard approaches couldn’t explain.
That small but vital observation prompted a deeper look—and sparked the beginning of a collaborative effort across multiple institutions.
Coming Together to Solve the Puzzle
It quickly became clear that KJ’s case was unique and couldn’t be addressed by conventional methods alone. Doctors at CHOP reached out to genetic researchers at Penn Medicine and the Innovative Genomics Institute (IGI) to help find a solution. The goal: to create a customized gene therapy just for KJ.
In August 2024, his case reached Fyodor Urnov at IGI, who immediately recognized its potential. He contacted partners at Danaher, where lead scientist Sadik Kassim joined the effort within minutes. The Jackson Laboratory responded almost instantly when the team needed a specialized mouse model to safely test the treatment, providing exactly what they needed.
Having worked closely with The Jackson Laboratory’s leadership, I wasn’t surprised—but was deeply impressed—by how quickly they responded. Their rapid support vividly showed what’s possible when people are prepared and ready to act together, fast.
The Right People, Connected Fast
KJ’s breakthrough wasn’t the result of one person or one discovery—it was the outcome of many people moving quickly and in sync. Business thinker C.K. Prahalad captured this idea perfectly: “It’s not what you know, but how fast you can access the information that others know.”
That insight was at the heart of this medical milestone. The solution didn’t come from knowing everything—it came from knowing how to connect the right expertise at the right time.
A New Beginning
In February 2025, KJ received his personalized gene therapy. The results were extraordinary. His condition stabilized, his health improved, and today, KJ McDonald is thriving.
His story is a reminder that in medicine—and in life—real breakthroughs happen when people come together. Collaboration isn’t just a buzzword; it’s the key to solving the toughest problems. And often, the team that works together fastest is the one that changes a life.
Dr. Mark DeVolder is a Top Change Management & Transformation Expert, Award Winning Motivational Keynote Speaker Empowering Confidence through Change. Mark can teach you how to change, anticipate business trends and accelerate future-proof transformation. He’s done it before with industry leaders like Qatar Petroleum, PepsiCo, Royal Bank of Canada and Pfizer.
