Beyond being a boon for the city of Sanford, this week’s announcement that Pfizer would pump $500 million into its Lee County facility also highlighted how much of a hub the greater Triangle region has become for gene therapy research.
Though still experimental, gene therapy is one of the most promising forms of treatment in recent years for diseases like cancer and some inherited disorders.
The treatment uses genes to treat diseases multiple ways, like replacing a mutated gene that causes a disease with a healthy one, knocking out mutated genes or adding a new gene to counteract a disease.
Leaders of the FDA said earlier this year, though, that they anticipate the agency will approve 10 to 20 cell and gene therapy products yearly by 2025.
But even in the early stages of its development, the Triangle has attracted a significant cluster of large and small companies working in the gene therapy space. Manufacturing of these therapies has especially become a skill set that the region has benefited from.
Pfizer, which has made a huge investment in gene therapy in recent years, is one of the largest players. With its latest investment, the global company will add 300 jobs to its Sanford plant, where the company will make gene therapy products for both clinical and commercial efforts. Pfizer already had 650 workers based there.
It’s a welcome turnaround for the plant, about 40 miles south of Raleigh-Durham International Airport, which had once employed 1,500 people but had been in decline until recently.
That turnaround coincided with Pfizer’s increased interest in gene therapy. In 2016, Pfizer bought Bamboo Therapeutics, a Chapel Hill startup that was developing gene therapy treatments, in a deal that was worth up to $645 million.
Bamboo’s chief scientific officer was Jude Samulski, the director of UNC’s 20-year-old Gene Therapy Center, which has given the region a hub of expertise on the topic. Most roads related to gene therapy in the Triangle lead back to Samulski, who was originally recruited to UNC from the University of Pittsburgh with the help of a $430,000 Faculty Recruitment Grant from the N.C. Biotechnology Center in 1993, according to the center’s web site.
Bamboo itself was a spinoff of another Chapel Hill company called Asklepios Biopharmaceutical (AskBio), a gene-delivery technology company founded in 2003. Bamboo’s portfolio of gene therapies targeted diseases like Duchenne muscular dystrophy, Friedreich’s ataxia, giant anoxal neuropathy and Canavan disease.
Pfizer has focused its gene therapy treatments so far on hemophilia and Duchenne muscular dystrophy — but it’s also researching its use for other diseases like ALS, also known as Lou Gehrig’s disease, Angela Hwang, Pfizer’s biopharmaceuticals group president, said in Sanford on Wednesday.
Bill Bullock, who works in economic development at the N.C. Biotechnology Center, said Pfizer’s investment underlined how much gene therapy talent has made its home in the Triangle.
“It’s a big deal because Pfizer is one of the largest pharmaceutical companies on the planet,” Bullock said. “And I think it is a strong validation to make a decision to build out clinical and manufacturing in North Carolina.”
“Especially for a new technology like gene therapy,” he added, “you can’t really just make that up -- you have to have an ecosystem (in place).”
Bullock noted that with clinical trials for gene therapy treatments increasing rapidly in recent years, more companies began to put operations down in the Triangle. Often, the Triangle is competing for these companies with Massachusetts and California as well as Ireland and Singapore, he said.
“Those companies are going to look to places that have an established workforce because the skill set for this manufacturing is very high,” Bullock said.
One of those companies was AveXis, a subsidiary of the Swiss pharma company Novartis, which licensed technology from the Chapel Hill-based AskBio.
AveXis, which received approval earlier this year for a gene therapy treatment for pediatric patients less than 2 years old with spinal muscular atrophy, has hired 400 people in Durham after receiving millions of dollars in incentives from the state.
The company brought a variety of manufacturing and engineering positions to Durham and its incentives were contingent on getting FDA approval. While the company did get approved for its SMA treatment, in recent days the company has been mired in a bit of controversy that has led to the ouster of several employees.
The FDA has accused the company of data manipulation during the approval process, The Wall Street Journal reported, though if the manipulation had been known it wouldn’t have affected the final decision to approve the drug, which is called Zolgensma and is considered the world’s most expensive drug.
In total, there are 13 companies now based in the Triangle that are working on gene therapies, according to a list compiled by the Biotech Center. Last year, the region was ranked the eighth best biopharmaceutical cluster in the trade magazine Geneteic Engineering & Biotechnology News’ annual list of Top 10 U.S. Biopharma Clusters.
AskBio hit a significant milestone in April when it got a $225 million influx of capital from investors.
Bluebird Bio opened a new manufacturing facility in Durham, where it plans to hire 70 employees by the end of the year.
Cellectis, a French gene therapy company working on cancer treatments, said in March it would hire 200 people at a manufacturing facility in Wake, in exchange for state incentives.
And Precision BioSciences, a Durham-based gene editing company, raised around $100 million in an initial public offering earlier this year, which it used to complete the clinical trials of one cancer treatment, support development of two other disease treatments, and build out a manufacturing facility in Research Triangle Park.
The combination of small, mainly local businesses and those with origins in some of the biggest pharma companies in the world will be beneficial to the region going forward, Bullock said.
“If you are a guy like me that looks at the ecosystem from a big picture,” he said, “it is great to have the Pfizers and Biogens of the world, but you want to have that innovation piece as well.”
“Having that capacity to grow that (gene therapy) science here is a multiplier,” he added. “As the smaller companies grow … and those companies get into human trials and manufacturing, they know there is a readily available workforce here and they will be more likely to stay here.”
Additionally, several companies in Research Triangle Park are also using gene editing technology applications in agriculture.
This story was produced with financial support from a coalition of partners led by Innovate Raleigh as part of an independent journalism fellowship program. The N&O maintains full editorial control of the work. Learn more; go to bit.ly/newsinnovate