Gene therapy is a relatively new field that involves altering the genes inside the body’s cells in an effort to treat or stop genetic diseases. There’s a lot of excitement about the potential of these therapies and a lot of investment across the U.S. and globally to bring these kinds of transformative medicines to the market. One of the biggest constraints so far has been manufacturing.
Timothy J. Miller, Ph.D., CEO, President and Co-Founder of Forge Biologics, is leading an effort to build one of the world’s largest adeno-associated virus (AAV) gene therapy manufacturing facilities in Columbus, Ohio. Forge has raised $160 million in venture capital and recently landed an $80 million credit facility to speed buildout of its facility, where it plans to grow to 400 high-paying jobs in a couple of years, up from 200 today. In the expanded facility, Forge will be able to make batches of gene therapies large enough for the larger late-stage trials and commercial launch.
To learn more about Forge Biologics, we spoke to Dr. Miller and one of his co-founders, Erandi De Silva, Ph.D., a molecular biologist and Senior Vice President of Product Development.
Forge Biologics uses an adeno-associated virus (AAV) platform to manufacture gene therapies. Can you tell us how that works?
De Silva: 300 million people worldwide are affected by over 7,000+ rare diseases, and only a fraction of those patients have any treatment options. The field of gene therapy offers hope by developing medicines that can correct or provide a working copy of a missing gene. We start with a vector, which will deliver the “fix” to the body—in our case we use a virus to do this. We take out the parts of a virus that make it pathogenic, and replace it with a piece of DNA that encodes functional protein to correct the disease.
Forge develops therapeutics but also manufactures them for other companies, correct?
Miller: Yes. Forge operates under a hybrid business model. On one hand of the business, we are a contract development and manufacturing company (CDMO), where we partner with clients and other innovators in the field to produce gene therapies: scientists, physicians, biotech and pharma companies, and patient groups. On the other side, we are a therapeutics development company, developing our own pipeline of therapies for targeted diseases. With this dual approach, we can be innovative and create new therapies, while also helping to alleviate the manufacturing bottleneck that exists in the gene therapy industry. Our goal is to accelerate gene therapy programs from preclinical stage through clinical and potential commercialization.
You are growing your workforce aggressively. What are your recruitment strategies?
De Silva: We network and recruit extensively and broadly, but we’ve also honed our efforts on a few strategies. For example, we created a program called Forge Forward with the goal of training and attracting top talent with specialized Good Manufacturing Practice (GMP) training that we helped to develop with our inaugural partner Case Western Reserve University. We plan to grow this to other universities in Ohio and continue spreading the word to raise awareness about our company and what we’re doing to attract the best candidates.
How did Ohio lend itself so easily to launching a gene therapy business?
De Silva: You can really think of Ohio as one of the birthplaces of AAV-based gene therapy. One of the two FDA-approved AAV-based gene therapies on the market now was invented right here and launched out of a lab at Nationwide Children’s Hospital. We have some of the pioneers of this type of medicine, both on the manufacturing side as well as the development side, right here in the region. What you’re seeing now is other local companies, like Forge, building on that foundation.
We’ve also been able to leverage the presence of some great research universities locally and across the state of Ohio. Our foundational investor, Drive Capital, also helped to bring our co-founders together, and has been instrumental in providing VC funding to many innovative industries across the region.
What areas of gene therapy are your clients working in?
Miller: We manufacture for global clients serving as a nexus point in the gene therapy industry, whether they’re working on Muscular Dystrophy, cardiovascular disease, liver disorders, etc. With so many rare diseases, there are countless opportunities to meet those patients’ needs and new therapies are being investigated by researchers and companies everywhere. [The company is also partnering with clients such as Ray Therapeutics on the manufacturing process of therapies for patients with blinding disease, including retinal disorders.]
You mentioned that Columbus is the up-and-coming city for gene therapy. What were some of the other advantages of starting the company in Columbus?
Miller: Columbus is attractive in many ways. In addition to the VC support available from firms like Drive Capital, support from the state, and the talent pool that local universities provide, Columbus has a great cost of living compared to the coasts. And it’s within a 12-hour drive from here to 50% of the country’s population, making it a reasonable spot for many. The buzz and growth of the biotech industry here is tough to beat, and that’s also key.
De Silva: The traditional expectation for a venture capital-backed company would be that it’s difficult to raise money here. But we’ve been able to raise over $240 million since we launched the company 20 months ago. I think that speaks to the attractiveness of the business model we have and what we’ve been able to launch in the place that we’re in.
There’s a lot of investment in the potential of these gene therapies worldwide. The thing that has slowed down the industry everywhere is the lack of manufacturing capacity and capability. Our company was envisioned to address that time gap and to speed these therapies forward to the patients who need them. Forge is poised to do that from right here in Ohio.