Andrew’s StoryThe Path to Possibility

From Discovery to Breakthrough: Central Ohio’s Biotech Revolution

In the heart of Central Ohio, a quiet revolution is taking place. Fueled by the groundbreaking research and collaboration within Nationwide Children’s Hospital, Sarepta Therapeutics, Andelyn Bio, and other key players, the region is rapidly emerging as a global biotech hub. At the center of this transformation are the pioneering patients and families whose stories epitomize resilience, collaboration, and hope.

Andrew’s Journey: The Human Face of Gene Therapy

In October 2001, Andrew Kilbarger’s life took an unexpected turn. Diagnosed at the age of four with Duchenne Muscular Dystrophy (DMD), a rare and debilitating condition, his family was thrust into a whirlwind of medical tests, consultations, and uncertainty. “I honestly can’t even remember when I really started realizing something wasn’t right,” Andrew recalls. His mother, Julie Wilson Kilbarger, adds, “We just thought he was delayed. It wasn’t until his preschool teacher noticed he couldn’t run, hop, or jump that we began to understand the gravity of the situation.

Andrew Kilbarger and his mother Julie Wilson Kilbarger at the grand opening of Andelyn Biosciences.

What followed was a journey defined by determination and groundbreaking science. Andrew became the first patient in the world to undergo a dystrophin gene transfer. As Julie describes it, “Dr. Mendell approached us about the research. Andrew was the perfect candidate at the perfect time.” This pivotal moment, conducted entirely at Nationwide Children’s Hospital, marked a significant step forward in gene therapy research and set the stage for future breakthroughs.
“I was in second grade when it happened,” Andrew shared. “I remember missing a lot of school during that time, but looking back, it was worth it knowing what came out of it.”

Central Ohio: The New Frontier of Biotech Innovation

The success of Andrew’s treatment is no anomaly. Central Ohio has become a beacon for cutting-edge biotech and gene therapy advancements. Institutions like Nationwide Children’s Hospital and The Ohio State University collaborate with companies such as Sarepta Therapeutics, Andelyn Bio, Forge Biologics, and others in the gene therapy space to push the boundaries of what’s possible in medicine.

We’re so blessed to have all of this just 30 minutes away. It’s not about one facility—it’s about collaboration. Whether it’s Children’s Hospital or OSU’s Heart Hospital, they’re all working together for the greater good.

Julie

Andrew’s mother

Andelyn Bio, named in part after Andrew and Evelyn, another young patient whose life was saved through gene therapy, embodies this collaborative spirit. Its mission to scale gene therapy production reflects the ambition of the entire region—to make these life-saving treatments accessible to patients worldwide.

Dr. Jerry Mendell, MD with Evelyn Villareal.

Navigating the Roadblocks: Collaboration in Action

While the advancements in Central Ohio are remarkable, the journey has not been without obstacles. Families like Andrew’s often face insurance hurdles, logistical challenges, and emotional strain. Julie recounts the difficulty in navigating these challenges: “We faced so many barriers, but we always felt the doctors were on our side, advocating for what Andrew needed to thrive.”

Despite these roadblocks, the community has rallied around families. “The support we’ve received from doctors, researchers, and even complete strangers is overwhelming,” says Julie. “They’re not just supporting Andrew; they’re supporting our whole family.”

Transforming Lives: The Ripple Effect of Innovation

Andrew’s story is one of many that demonstrate the profound impact of Central Ohio’s biotech innovations.

Evelyn is alive today because of this research. Andrew’s purpose in life has been fulfilled in ways we could never have imagined. And it’s not just about Evelyn or Andrew—these therapies hold the potential to transform countless other lives. Families around the globe, facing conditions once thought untreatable, may now see a path forward thanks to this groundbreaking work.

Julie

Andrew’s mother

Sarepta Therapeutics has been a pioneer in advancing treatments for Duchenne Muscular Dystrophy, driving the research that made Andrew’s groundbreaking therapy possible. Their work continues to inspire hope for families affected by rare diseases, setting a high standard for innovation and commitment.

Beyond the personal stories, the ripple effects of these breakthroughs are positioning Ohio as a global leader in biotech. Companies like Sarepta Therapeutics and Andelyn Bio are expanding their footprints, bringing jobs and further innovation to the region.

Grand opening of Andelyn Corporate Center (ACC) in Columbus (2023).

Inspiring Gratitude: A Call to the Medical Community

When asked about his message to the medical community, Andrew’s response is simple but profound: “Thank you for allowing me to be where I’m at today. Without you, I don’t know where I’d be.”

As Central Ohio continues to advance the frontiers of medicine, stories like Andrew’s serve as a powerful reminder of what’s at stake and what’s possible. With every breakthrough, the region cements its role as a global leader in life sciences, driven by a community that refuses to give up on hope.