• Pfizer intends to spend $500 million expanding its gene therapy operations in North Carolina, the latest big pharma investment in the burgeoning technology.
• The money will go toward building a new manufacturing facility in Sanford, a small city about 50 miles outside of Raleigh, North Carolina. The facility will create about 300 jobs, according to Pfizer, and be responsible for making the recombinant adeno-associated viral vectors needed to supply clinical- and commercial-stage gene therapies.
• Pfizer has three gene therapies in clinical testing. One, which it got through the $150 million acquisition of Bamboo Therapeutics, targets a rare muscular disorder. The other two are partnered programs going after hemophilia.

Curing horrific diseases with a single dose of medicine. It’s a lofty goal, but one that became more realistic as gene therapies started to show remarkable efficacy in clinical testing.

The advancements are catching the attention and investments of large pharmaceutical companies. Novartis shelled out nearly $9 billion last year to acquire AveXis, a biotech focused on gene therapies for neurological disorders. Another Swiss pharma giant, Roche, is trying to lock down a $4.8 billion bid for Spark Therapeutics, which received the first ever U.S. approval for a gene therapy targeting an inherited disease.

Pfizer has made a few investments too. The Bamboo deal, for instance, gave it not only a pipeline of gene therapies for neuromuscular and central nervous system diseases, but also a fully staffed, 11,000-square foot manufacturing facility in Chapel Hill, North Carolina.

Executives noted during the 2016 acquisition how they wanted Pfizer to become a leader in the gene therapy field. Since then, the company devoted $100 million to grow the Bamboo manufacturing facility and secured an exclusive option to acquire Vivet Therapeutics, a privately held biotech based in France.

Pfizer’s ambitions are now leading to another manufacturing expansion. According to an Aug. 21 statement, the Sanford site already has 650 Pfizer employees and produces both gene therapies as well as vaccines, including the blockbuster pneumococcal vaccine Prevnar 13.

The additional investments should expand end-to-end production capabilities, Pfizer said. Another of the company’s sites is located in Kit Creek, North Carolina, where scientists test various manufacturing processes at smaller scales to see what might work best on a broader basis.

The selected processes, according to Pfizer, are then honed further at its Chapel Hill site through build-out of quality control measures.

“Pfizer’s focus on these processes, combined with its existing and new additional investment in Sanford to manufacture gene therapies, is designed to result in a high-quality, efficient supply of gene therapies at clinical- and commercial-scale,” the company said in its Aug. 21 statement.

Ensuring adequate supply and quality control are essential tasks in drugmaking, and especially so with gene therapies.

Already, there have been challenges. Bluebird Bio offers one example, as the biotech had to push back the European launch of its gene therapy Zynteglo because it was still finalizing the commercial manufacturing process. And more notably, the pioneering approval Novartis won for Zolgensma is now under scrutiny after revelations company employees manipulated preclinical testing data.