Regenxbio CEO Ken Mills finds gene therapy's resurgence full of promise

Ken Mills sees a new era for gene therapy. (Image: Regenxbio)

Regenxbio CEO Ken Mills is riding and contributing to the growing momentum that gene therapy is beginning to experience.

The resurgence sped up last December when the U.S. Food and Drug Administration approved the first gene therapy for genetic disease, Spark Therapeutics’ Luxturna, to treat a rare form of blindness.

“Building on that momentum, gene therapy is at the forefront of the scientific and biotech industry’s purview for this year and beyond,” Mills told FierceCEO.

“That’s why you see so much excitement around this space and why I’m so committed to advancing this technology. I’ve been part of this industry for more than 20 years and Regenxbio is one of two gene therapy companies I’ve helped found.”

When Regenxbio was formed in 2009, it was a tricky time to be starting something new in biotech and pharma, Mills said.

That’s because there had been several setbacks in the early development of gene therapies for treating people.

But the promise of gene therapythe idea of curing disease with a single treatment“was captivating,” Mills said. “From curing blindness to hemophilia, this technology has so much potential, and the number of people whose lives could be transformed for the better by gene therapy is astounding.”

Right now, Regenxbio owns a gene therapy technology that allows the transfer of genetic materials to cells and tissues in the body to treat diseases. Regenxbio uses the technology to develop their own products and also licenses it to other companies. “We are developing drugs as are our partners,” Mills said.

Regenxbio also has two treatments in human trials, one for the most common cause of blindness—wet age-related macular degeneration—and another for people who have genetic high cholesterol.

Mills is “also excited by science and discovery. For instance, AAVs [adeno-associated viruses that can be engineered for gene therapy] are viruses that occur in nature, but weren’t known because they don’t cause disease in people. They were only discovered by accident. I love that background, and how something that no one knew existed can have such huge potential. At the foundation, that’s what science is all about.”

Regenxbio spent its first several years as a private company and then went public in 2015. The move was a good one in that “developing treatments is capital intensive, so having access to capital markets is important,” Mills said.

On the other hand, “The way we have to report progress—quarterly—is not consistent with the time it takes to develop drugs, which can be years,” he said.

Mills has always had a keen interest in genetic therapy and participating in operating companies. Before founding Regenxbio, he was CFO and VP of business development at Meso Scale Diagnostics, a privately held life sciences company he helped found.

There, he served as a member of the management team, and worked to establish the company's operations and ongoing business strategy. In this position, Mills supervised company activities, including direct management of corporate and business development, strategic planning, finance and accounting activities.

He left Meso to start Regenxbio.

Prior to Meso, he was director of business development for Igen International, a medical diagnostics company.

Fast Five with Ken Mills

What's the key to finding new opportunities for revenue?

Being innovative and open minded.

When have you had to adapt and what did you learn?

Going from a private to a public company, the learning curve was having to communicate the missions and values to a public audience.

On a scale of 1 to 10 (1 lowest and 10 highest) how much of a priority do you place on the following things at your company: people, process and technology?

  • People—10
  • Process—9
  • Technology—8

What's one tip for motivating employees?

Being transparent and having open communication.

What do you wish you had known 5 years ago that you know now?

Having some predictability around the stability of the economy.