An emerging economic sector in Central Ohio is transforming the region into a manufacturing epicenter for one of the world's most technologically sophisticated industries.
Regulators, educators and consumers alike have a role in nurturing that growth, representatives of the gene and cell therapy fields said in Wednesday's Columbus Metropolitan Club forum. Central Ohio has built a biotech ecosystem that includes inventors and researchers at Nationwide Children's Hospital and Ohio State University, numerous pharmaceutical development companies and the country's third-largest healthcare distributor, Cardinal Health Inc.
Making the tiniest snippets of engineered DNA – in the massive quantities needed for a clinical trial or commercial use – requires sealed vessels as much as 16 feet tall in huge factories, and there just aren't enough of them.
"One of the biggest constraints has been manufacturing," said Timothy Miller, CEO of startup Forge Biologics Inc.
For example, Duchenne Muscular Dystrophy is the most common and among the most aggressive forms of the inherited condition, fatal by early adulthood. If there were a genetic therapy approved today, Miller said, treating just one-fourth of patients would use up almost half the current manufacturing capacity in the U.S.
Although the U.S. Food and Drug Administration is poised to approve two dozen new therapies this decade, he said, there are about 7,000 rare inherited diseases – and many have no treatments under investigation.
"That’s not just because of the science but because of the manufacturing," he said.
Companies like Forge are working to ease that bottleneck. In addition to developing therapies it has licensed, the Grove City startup is building out a 200,000-square-foot manufacturing facility meeting the FDA's strictest standards for purity and safety of biologic materials. Forge has raised $160 million in venture capital and landed an $80 million credit facility toward the construction; at full capacity it's expected to create 400 high-paying jobs.
"What drives a lot of the innovation is going to be fundraising, whether private or public, academic or otherwise," Miller said.
Also in the region, contract gene therapy manufacturer Andelyn Biosciences Inc. expects to start moving in this summer to a 185,000-square-foot manufacturing facility under construction on OSU's west campus. The Children's spinoff also has a research and temporary production facility in Dublin.
The manufacturing shortage also has meant the difference between life and death in cellular therapy, said Dr. Samantha Jaglowski, Ohio State associate professor of hematology and medical director of its blood and marrow transplant program. She treats patients with transplanted stem cells to treat blood cancers, in contrast to gene therapy in which modified viruses deliver corrected copies of genes to cells in the body.
In one study, Jaglowski said, seven of 12 patients enrolled died waiting for enough stem cells to be reproduced.
"These patients deteriorate in that time," she said. "You’ve got an exacerbation between your haves and your have-nots."
Central Ohio educational institutions can help the sector through developing a skilled workforce, the panelists said. The growing number of biotech companies either starting here or establishing satellites helps, such as Sarepta Therapeutics Inc. opening its genetic R&D center at Easton or biotech giant Amgen building a high-tech pharmaceutical assembly and packaging plant in New Albany.
"Those people are going to be able to jump back and forth at some point," Miller said.
Everyone in the region can contribute to recruiting talent in the small and tight-knit field, and persuading those who left the state for career opportunities to return, said Eli Phillips Jr., vice president for specialty solutions at Dublin-based Cardinal.
"Continue to make this an awesome place to live," Phillips said.
