Forge Biologics is Manufacturing Gene Therapies for Patients with Genetic Diseases

Scientists and researchers are hard at work to find treatments and cures for an estimated 300 million people worldwide who live with a rare disease. For many of those who suffer from a rare disease, there are no available treatments, and many diseases receive little research and manufacturing attention. Columbus-based manufacturing and therapeutics expert Forge Biologics is helping to solve that problem by expanding access to life-saving gene therapies.

“I’ve been in the gene therapy industry since the late ‘90s,” CEO, President, and Co-Founder Tim Miller, Ph.D. said. “There’s not always a lot of hope out there for patients who have been diagnosed with a rare genetic disease, so many of us go into this industry with a strong therapeutic development background where manufacturing was secondary. Now that science has caught up with therapeutic needs, a lot of our work focuses on how to manufacture at scale to treat dozens to hundreds of patients.”

The basis of Forge’s manufacturing strategy is a genetically modified, non-pathogenic virus. The company takes an adeno-associated virus or AAV and removes any harmful material. The AAV is then used as a delivery vehicle to administer gene therapies to patients who have been diagnosed with a rare genetic disease.

“For example, kids who were born with Krabbe disease have malfunctions in the Krabbe gene,” Dr. Miller said. “We put the correct functioning copy of the gene into the modified virus and that’s what’s administered to patients. To get there, you need plasmid DNA, which is a starting material to make the virus. So, we brought plasmid manufacturing in-house to serve end-to-end capabilities. First you make plasmids, then you use them to make AAV, then that gets put into patients.”

As more and more gene therapies are approved by regulators, Forge is actively working to scale their production capabilities to keep up with increasing demand. The company wants to further build out its facilities and expand its offerings to gene therapies outside AAV manufacturing, while making a global impact.

“We’ve seen the power of what even one injection of a gene therapy can do to improve a patient’s life,” he said. “Forge is scaling this technology to a point that nobody else has done in the world. With that capacity, we have the potential to treat a lot more patients. We’re scaling 5,000-liter bioreactors right now, and we’re excited in 2023 to roll out some of our data to the industry and our colleagues to show that you can scale to that size. It’s really going to be a game changer for Forge and our clients.”

Part of that expansion is the Hearth, a 200,000-square-foot facility Forge has built that has 20 clean room suites and multiple bioreactors. The ambitious project is further proof of the company’s far-reaching goals and desire for global expansion of their technology. And as Forge scales its business to meet those goals, the startup is passionate about how it can continue to make an impact for local Ohio industry.

“We’ve seen a lot of growth in the biotech ecosystem in Ohio,” Dr. Miller said. “There’s been a lot of support from the state legislature for manufacturing initiatives and for biotech overall, especially with Ohio Life Sciences supporting the biotech and pharma industries. We see Ohio as a center for excellence in the Midwest, and we’re excited to think about how Forge in Columbus is helping to build the greater biotechnology community and providing hope in the heartland.”