Research facility for gene therapy opens in Easton
Will start with a footprint of 85,000 square feet
Jessica Evans remembers being 12 years old and receiving a life-changing diagnosis.
Evans has limb-girdle muscular dystrophy, a genetic disease that causes muscle groups around the hips and shoulders to atrophy. Symptoms become progressively worse and can also lead to respiratory and heart conditions.
It’s the fourth most common genetic cause of muscle weakness with an estimated prevalence of about 2 in every 100,000 individuals, according to the Muscular Dystrophy Association.
But there’s hope on the horizon with Massachusetts-based Sarepta Therapeutics Inc. expanding its gene therapy research and development footprint in Columbus. Monday marked a grand opening for an 85,000-square-foot facility at Easton dubbed the Genetic Therapies Center for Excellence. There are two nearby buildings that could potentially lead to a 150,000-square-foot expansion. The company also has other sites in Cambridge, Andover and Burlington, Massachusetts.
“Imagine being a young person coming of age and learning you have a condition for which there are no treatments, and none on the horizon would be a permanent fixture in your life,” Evans told a crowd of Sarepta employees and dignitaries at a ribbon cutting ceremony while sitting in a powered wheelchair. “That news was more than I can fully grasp or comprehend at that time.”
With a successful gene therapy treatment, it’s possible the disease stops progressing and patients show improvement. A best case scenario would be a full recovery. Sarepta uses RNA, gene therapy and gene editing programs for its genetic medicine program.
“The goal of our therapy in simple terms is to use
viruses as a shuttle to deliver a new version of that gene to make that fully functioning protein. We’re doing clinical trials. And over time, we’ll, we’ll know in terms of how much we can cause an improvement,” said Louise Rodino-klapac, Sarepta’s Columbus-based executive vice president and chief scientific officer. “From our early trials that we’ve done so far, we’ve seen not only stabilization of the disease, but also improvement in their function. So that is the hope.”
As of Monday, the company has more than 70 local employees with plans to double that by the end of 2022. The facility will usher in process development and optimization work. It’s that kind of work that takes potential treatments from clinical-scale to commercial-scale manufacturing for use.
“The need for treatment is urgent. There are many forms of LGMD, with devastating effects on both cardiac and respiratory,” said Evans, who is an assistant director for The Speak Foundation, a patient-led nonprofit organization for muscular dystrophy. “But imagine now what it would be like to be diagnosed with the condition and to learn that there’s a treatment available. That is what we as a community are eagerly awaiting.”
The company is in the process of moving from leased lab space in Dublin to the new Easton location. Its first location was opened in 2018. The company has had a long-standing partnership with Nationwide Children’s Hospital. The hospital has led to, in part, making Columbus an emerging hub for biotechnology with gene therapy companies opening locations.
“Columbus is really the quiet giant in the forefront of gene therapy. I think of the gene therapy companies, for example,
spun out from the technologies at Nationwide Children’s Hospital. It’s great because of the infrastructure and the know-how. The scientific community here that understands it. I think we’ve built upon that,” Rodino-klapac said.
Ohio Lt. Gov. Jon Husted said the
state is making every effort to help companies like Sarepta succeed.
“The biggest role that government can play is to first be no impediment for your work. And secondly, to do all we can to be a wind in your sails,” he said, citing Ohio’s so-called innovation districts. “We hope to create a great environment for you to succeed . ... Because when we do it right, in the end, people benefit. That’s the beauty of what you’re doing. It creates economic prosperity. It also creates a new and higher quality of life for the recipients of work.”
The new Sarepta facility at Easton “just really expands exponentially what we can do and the amount of programs that we can do. This is our hub for the company for research and development. We have we do research and other sites, too, but this is the primary site. So it’s really pivotal to everything we do,” Rodino-klapac said.
The company already has three approved drugs for Duchenne muscular dystrophy and more than 40 treatments in development.
“The work that we are doing here will resonate, in my opinion, across health care, as well as lead a revolution in the use of genetic medicine to extend and improve the lives of people living with rare diseases,” said Doug Ingram, president and chief executive officer of Sarepta Therapeutics.
“The exciting aspect of genetic medicine offers not incremental improvements, but potentially giant leaps forward in the treatment of diseases that up to now, over the long arc of human history, have invariably and ruthlessly stolen the lives of those with those genetic diseases.,” he said. “There are more than 7,000 rare diseases, 85% of them are single gene mutations, and yet nearly none of them have any meaningful therapies.”
The potential for changing the lives of those with genetic diseases is not lost on those who work at the company.
“We’ve chosen to be here now, to be part of something bigger than ourselves, to accept the challenge. That’s what scientific innovation is really about. Oftentimes, there’s no formula,” Rodino-klapac said. “No one’s ever done any of these things before.” bharvilla@dispatch.com @Beth_harvilla