You know that stock footage on the network news every time there’s a story about the Covid vaccine? There are thousands of glass vials with colorful caps weaving along an automated assembly line.
That’s not the mass-production scenario planned for Ultragenyx Pharmaceuticals, one of Bedford’s newest corporate citizens.
That’s because Ultragenyx specializes in rare diseases, defined as those with fewer than 200,000 patients worldwide. The federal government established that criterion “to encourage companies to work in this space,” explained Thomas Lauzon, the firm’s Vice President of Manufacturing-Gene Therapy.
Indeed, even one dose of a genetically engineered drug can change a lifetime spent fighting an illness.
Ultragenyx, based in California, is building a manufacturing facility on the campus of Bedford Woods, along the northern segment of Middlesex Turnpike.
Town officials, led by former Economic Development Director Alyssa Sandoval and Town Manager Sarah Stanton, along with the developer Gutierrez Cos., worked overtime to attract the company, culminating with a commitment just days before the arrival of Covid-19 in March 2020.
When it opens sometime in the first half of 2023, the plant will be a state-of-the-art manufacturing facility for the production of the firm’s clinical and commercial drugs, said Lauzon, a University of Massachusetts Amherst graduate who has been with Ultragenyx for four years.
Lauzon said the legislation that opened the door to firms like Ultragenyx is known as the Orphan Drug Act. Passed in 1983, the law incentivized the development of drugs to treat rare diseases. “It provided periods of exclusivity to companies to develop these orphan drugs and expedited drug review,” Lauzon related.
Ultragenyx was established in 2010 by Dr. Emil D. Kakkis, now CEO and president. “He is the most prolific rare-disease drugmaker—he personally developed many of the drugs,” said Jeffrey Blake, senior director of corporate communications. “That’s his passion, and the culture comes from the top.”
“We work in MRNA, in small-molecule modalities, that enable us to select the right technology for the disease and hopefully fast-track the therapies needed for rare-disease patients,” Lauzon explained. “We collaborate with a number of scientific agencies. We send our scientists to conferences worldwide for information sharing.”
Scientists advise on the rare diseases and potential treatments, and then Ultragenyx’s research and development group, based in Cambridge, designs the process. “They will develop the cell lines and genetic material,” he said.
That outcome is transferred to a pharmaceutical development lab, and “they will do some initial scale-up work and produce the product. Then it will be transferred (eventually to Bedford) to manufacture and bring to market.”
The building shell is complete, and the interior fit-out is well underway: air and water systems, walls, utilities. “The equipment that’s needed is starting to be delivered as well,” Lauzon said. “It’s pretty extensive. After installation, we have to have a validation process to prove that the equipment works as intended. It’s rally detailed quality assurance.”
The work is overseen by a large engineering group. “Designs were reviewed from a regulatory perspective by a number of consultants,” Lauzon explained.
The environment inside, he said, is specialized. “We need to monitor on a daily basis for microbial growth. We use clean-air systems to purify the area. Operators have to wear specialized garb; they need to be trained. All of this is done under manufacturing procedures which are in the code of federal regulations.”
Specialized equipment grows cell culture components from 20 to 2,000 liters. “We use specialized purification techniques to remove some of the waste,” Lauzon said. “You can think of it as high-tech farming. We need to purify the product. We use over 100 different assays on an individual product. Everything is governed by our quality assurance unit.”
“Really unique is the isolator and filler we use to fill the glass vials,” he stressed. That’s because the product emerges in small lots – only up to about 1,200. The filler is “not a device or piece of equipment that every facility has.”
Blake said there’s “something different about gene therapy that explains why the volumes are lower. Often it’s just one dose—something you take one time and, if it does the job properly, it corrects an underlying genetic issue rather than the symptoms of a disease.”
The plant will employ between 100 and 150 and the workforce will be “highly skilled. “We have a pretty elaborate training system to make sure they understand what they are working with and how the KOB can affect the quality of the drug product,” Lauzon said. Candidates hold bachelor’s and engineering degrees, as well as master’s degrees in biology and chemistry.
He also noted that Ultragenyx has a relationship with Middlesex Community College that spans more than 20 years, supporting the company’s current facility in Woburn. “We are doing a lot of work with Learn and Earn at Middlesex, with includes six months’ training with us. This is a resource to work as manufacturing operators.”
Like any pharmaceutical manufacturer, Lauzon said, Ultragenyx will need to use a lot of water—so much that the town is planning to replace the existing sewer main along Middlesex Turnpike with a larger capacity line. That project is already received a $500,000 state grant and contributions from Ultragenyx and Gutierrez. “We use highly purified water in all of the stages of manufacturing,” he said.
He also emphasized that the firm “is committed to reducing our carbon footprint and our environmental impact—it’s something we take very seriously.” The new building’s certification will be LEED Silver, he said.
“Ultragenyx is especially passionate about diversity,” Lauzon observed. “We are doing a large amount of work in that area. Building a brand-new facility gives you the opportunity to walk the walk in regard to your values. We have done a huge amount of work on our hiring practices to ensure that we target diverse candidates.”
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