Pfizer’s Expansion in North Carolina May Help Advance Manufacturing of Hemophilia Gene Therapies

Pfizer will invest an additional $500 million into the construction of its new manufacturing facility in Sanford, North Carolina, the company announced.

According to the company, the facility could help advance the manufacturing of one-time gene therapies that use modified adeno-associated viral vectors, such as SB-525, which is being developed with Sangamo Therapeutics for patients with hemophilia A.

“Our purpose is breakthroughs that change patients’ lives,” Angela Hwang, group president of Pfizer Biopharmaceuticals Group, said in a press release. “We’re excited to build this new state-of-the-art facility in Sanford because it will have the potential to help us develop novel methods to deliver transformative treatments to patients.”

SB-525 is intended to provide durable production of coagulation factor VIII (FVIII) — the clotting protein missing in people with hemophilia A. Recent results of the ongoing Phase 1/2 Alta trial (NCT03061201, which is still enrolling) in severe hemophilia A showed that the treatment was generally well-tolerated, led to progressive increases in FVIII levels, and was associated with no spontaneous bleeding events up to 24 weeks of infusion in one patient.

Pfizer is also working with Spark Therapeutics on a Phase 3 program to test a gene therapy known as fidanacogene elaparvovec (SPK-9001) for patients with hemophilia B. A Phase 1/2 study (NCT02484092) in moderate to severe disease showed no serious adverse events, reduced bleeding, and fewer infusions of FIX, the factor missing in these patients.

In North Carolina, Pfizer’s gene therapy development and manufacturing is also done at facilities in Kit Creek and Chapel Hill. At the Kit Creek facility, the investigators work on a small scale – from 2L flasks up to 250L bioreactors – to develop a process that is then optimized in Chapel Hill, where Good Manufacturing Practice standards are being implemented.

Through the use of these processes and the investment in Sanford, Pfizer plans to improve its ability to supply clinical trials and the market with gene therapies for rare diseases.

“This investment will further strengthen Pfizer’s leadership in gene therapy manufacturing technology,” said Mike McDermott, president of Pfizer Global Supply.

The “hundreds of highly skilled jobs” expected at Sanford, McDermott says, are expected to boost the facility’s high-tech manufacturing capability and are part of an overall plan to invest nearly $5 billion over the next several years.

The team at Sanford also manufactures components for vaccines such as Prevnar 13 — to protect from invasive pneumococcal pneumonia caused by Streptococcus pneumoniae. The expansion would add approximately 300 new jobs to a current workforce of 650. More than 3,600 Pfizer employees work in North Carolina.

“North Carolina is a leader in life sciences in part because of our long-standing partnership with Pfizer,” said North Carolina Governor Roy Cooper. “Today we mark another boost to our state and we are proud of the people conducting life-saving research in Sanford.”