Automation, portable manufacturing and finding the right partner: Industry vets discuss the coming gene therapy wave

Gene therapy
With capacity challenges coming along with the impending gene therapy wave, automation will be key, industry veterans said. (LuckyStep48/Getty Images)

During this week's Fierce Biotech Cell & Gene Therapy event, there were times when the opening panelists sounded like TV marriage counselors Dr. Phil and Dr. Laura. 

“If you’re going to be in this relationship for years, there has to be an understanding that there’s some give and take across the board when things don’t go well. That’s how you preserve a relationship,” said Curran Simpson, the senior VP of product development and chief technology officer at Maryland-based Regenxbio.

No, this wasn’t a bizarre detour into couples counseling. It was part of Simpson’s answer to a question about the top factors cell and gene therapy companies face when selecting a contract manufacturing partner.

Jerry Keybl, the senior director of cell and gene therapy at Boston-based MilliporeSigma, stayed with the theme. 

“There’s many balls in the air at all times and having the transparency to build trust and partnership is the hallmark and the base of any successful relationship,” Keybl said. 

Kim Raineri, the chief manufacturing and technology officer at Boston-based Avrobio, might have had the most Dr. Phil-esque answer.

“It’s not just a transactional relationship,” he said. “[It’s] how they deal with issues when they occur and issues always occur.”

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Joking aside, the three industry veterans provided insights on a variety of cell and gene therapy issues, including the CDMO question. 

Raineri stressed the “right-sizing” of CDMO partners. A small gene therapy company, matched with a large CDMO, can easily become disenchanted by a lack of attention. And sometimes the level of attention is dictated not by the size of the company but by the volume of work the CDMO is performing.   

“These organizations are an extension of your organization and you need to be very comfortable with that group,” Raineri said.

As for Regenxbio, Simpson said his company considers whether a manufacturing contractor will allow Regenxbio's personnel on the floor when batches are produced     

“A person in the plant that’s experienced and can really carry the process knowledge to the CMO is an absolute must in our opinion,” Simpson said.

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Much of the discussion was forward-looking, with panelists addressing the concerns of the looming gene therapy wave and the capacity challenges that will come with it.

So what is the biggest challenge today in gene therapy? 

“Many of these processes are still very bespoke in how they are approached,” Keybl said. “That limits the transfer as we solve problems from one product to another, from one innovator to another. A great reference is the progress that’s happened in monoclonal antibodies. I think as an industry they are more homogenous from a process perspective than we are in the viral vector industry. That’s what we should keep in mind when we think about maximizing efficiency and scale.” 

Regenxbio’s response to the wave is to create its own manufacturing facility, one that will be highly automated and is set to come online early next year.

“People will be able to watch batches from their living room, if they choose to,” Simpson said. “If you don’t automate your plant, then you are signing up for a very, very large workforce to make production happen.”

While also stressing automation, Avrobio took a different approach to the problem, developing a portable manufacturing platform. It’s an off-the-shelf manufacturing unit—the size of a washing machine—customized for a process that is “nimble" and "scalable across our portfolio,” Raineri said. 

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The approach has allowed Avrobio to manufacture and conduct clinical trials over multiple continents and with multiple indications.  

“We have an end-to-end, closed, automated drug product manufacturing process that we can take globally,” Raineri added. “If you look at conventional space in cell and gene therapy, there’s a lot of manual processes, open aseptic manipulation and those are just not scalable."