The Gene Genie: Andelyn Biosciences Turns Research Breakthroughs into Patient Treatments

Each time the U.S. Food and Drug Administration approves a new gene therapy treatment, everyone at Columbus-based Andelyn Biosciences celebrates. But only momentarily. With an estimated 7,000 rare and ultra-rare human diseases worldwide without treatments, the biotech company has plenty of work left to do in the growing but still infantile gene therapy industry. 

“Every dose that’s successful, every disease we stop, we’re on cloud nine,” says Andelyn CEO Wade Macedone. “That’s what keeps me going.” 

Andelyn manufactures gene therapy products for the biotechnology and pharmaceutical industries, taking researchers’ gene-therapy discoveries and turning them into viable treatments for patients. The company can produce a single dose or hundreds of doses for a condition, depending upon the client and the rarity of the disease. And because the only treatment for some diseases is palliative care, the development of a gene therapy can literally save lives. 

Andelyn started 17 years ago as the manufacturing arm of Nationwide Children’s Hospital gene therapy research, but was spun out into a for-profit company in 2020.  “All the growth led us to decide that, if we really wanted to help the field advance, the best thing to do was to let our manufacturing core develop into a free-standing company,” says Dr. Dennis Durbin, president of the Abigail Wexner Research Institute at Nationwide Children’s. “That didn’t start as the plan, but the expertise that had developed over the years and the growth of the field led to the decision.” 

Unlike traditional health care, which has been largely built on addressing disease symptoms, the hope and promise of gene therapy lies with cures for diseases, or at least ways to effectively manage them, Durbin says. With gene therapy, normal cells replace missing or defective ones in an effort to correct genetic disorders.   

The work is complicated, specific to each disorder, expensive and time consuming. Manufacturing has been a bottleneck in the process. 

One of Andelyn’s goals has been to speed up production. Since 2020, it has built three facilities in Central Ohio and now its production process, which took two or more years in the past, has shrunk to less than a year, Macedone says. 

Its newest facility officially opened in June at Ohio State University’s Carmenton innovation district on its west campus. At 185,000 square feet, it allows the company to help clients from the development stage through commercial production of a gene therapy. Overall, the company has 240 employees, and its majority owner is Nationwide Children’s. 

Like a host of other gene therapy companies, Andelyn has multiple researchers and scientists who transitioned from their work at Nationwide Children’s to the private sector, bringing with them their knowledge and expertise. Others include Forge Biologics in Grove City and Sarepta Therapeutics Inc. of Cambridge, Massachusetts, which has a research center in Columbus. Researchers at all three companies worked on a much-heralded gene replacement therapy for Duchenne muscular dystrophy that received limited-use FDA approval in June—a first for that form of the disease. The research originated at Nationwide Children’s, as did the research that resulted in FDA approval for gene therapy for spinal muscular atrophy in 2019, the first systemic gene therapy for a neuromuscular disease. 

Andelyn took its name from two patients—Andrew and Evelyn—who participated in experimental gene therapy treatments for each of the diseases at Nationwide Children’s.  

Eddie Pauline, president and chief executive officer of the biotech trade organization Ohio Life Sciences, says the growing concentration of gene therapy companies in Central Ohio is attracting more and more talent and making the area a hub for gene therapy research and production. 

“Two or three years ago, it was a risky proposition for a life science scientist to come here, but now if you work in biotech, you’ve got 10 to 12 opportunities to jump to if you want,” Pauline says. Andelyn, he says, is an anchor for that growth because it’s homegrown, headquartered here and backed by the institutional might of Nationwide Children’s.  

Researchers are working on gene therapy for a host of diseases and conditions, including obesity, alcoholism, Alzheimer’s, Parkinson’s and hemophilia, Macedone says, so he sees plenty of growth in the future for Andelyn. Its facility at 5185 Blazer Parkway in Dublin is designed to double in size, as is the new facility at 1180 Arthur E. Adams Dr. 

“The growth is coming; it’s just a matter of time,” he says. FDA approvals of cell and gene therapy products are accelerating, and that will spur more investment in the industry, Macedone says. 

“For years we’ve worked through every holiday and haven’t taken a day off,” Macedone says. “This isn’t a job for most people; it’s a lifestyle.” 

This story is from the October 2023 issue of Columbus Monthly and the Fall 2023 issue of Columbus CEO.