Our Name
The name “Andelyn Biosciences” represents a hybrid of the name of two gene therapy pioneering patients.
Tracing Our Roots
Our values are founded in our origin story.
The name “Andelyn” is a hybrid of the name of two gene therapy pioneering patients who participated in pivotal Phase I clinical trials at Nationwide Children’s Hospital. Andrew received the first U.S. investigational gene therapy for Duchenne muscular dystrophy in 2006. Evelyn received an experimental gene therapy for spinal muscular atrophy in 2015.
“Andelyn” combines their names, representing all the families who have courageously participated in the research that make today’s gene therapies possible, and inspires our vision of Turning Hope Into Reality™.
Andrew: A Gene Therapy Pioneer
At the age of four, Andrew was diagnosed with Duchenne muscular dystrophy (DMD). Of more than 40 types of muscular dystrophies, DMD is one of the most highly aggressive forms.
More prevalent in males than females, children with DMD lose mobility and strength in their muscles. The disease then progressively affects their heart.
Immediately after hearing his diagnosis, Andrew’s parents, Julie and Mike, began to research doctors who could offer the most help for Andrew. They found Jerry R. Mendell, MD, a world-renowned neurologist right in their backyard in Columbus, Ohio, at Nationwide Children’s Hospital.
Evelyn: An Infant Innovator
The Villareal’s second child, Evelyn, was born in 2015. She also was born with SMA. At just 8 weeks old, Evelyn was given an investigational gene therapy in a Phase I clinical trial led Jerry Mendell, MD, principal investigator in the Abigail Wexner Research Institute at Nationwide Children’s Hospital. This trial was the first to study gene therapy for spinal muscular atrophy type 1, the most severe form of SMA to replace her missing SMN1 gene. The gene therapy worked. Four years later in 2019, the therapy was approved by the FDA as the first systemic gene therapy for a neuromuscular disease. “I forget that she has SMA,” says Evelyn’s mom, Elena. “She’s a healthy girl and does everything a normal child would do.
In 1989, after more than two decades of study and caring for patients with neuromuscular disorders, Dr. Mendell was the first scientist to publish a study showing that corticosteroids were the only effective treatment known to decrease the progression of DMD.
He also is the first doctor to demonstrate that gene therapy could be a safe, effective option for DMD. Through his and his colleagues’ pioneering research, in March 2006, Dr. Mendell was the first to inject microdystrophin, a shorter but functional version of the protein that is missing in DMD, into an arm muscle of a young boy with DMD. That boy was Andrew Kilbarger.
The injection, using an adeno-associated virus (AAV)-mediated vector, delivered microdystrophin to the muscle producing a functional protein that reduced muscle deterioration. “Andrew is so important because his injections proved the safety and efficacy of this therapy,” Dr. Mendell explains.
Thanks to Andrew and his family’s courageous approach to his condition, Dr. Mendell has been able to advance the microdystrophin gene therapy to a later stage clinical trial and expand the study of gene therapy to other neuromuscular disorders. The most prominent success to date has been the discovery of gene therapy to treat spinal muscular atrophy (SMA). The therapy was approved by the FDA in 2019 as the first systemic gene therapy for a neuromuscular disease.