Astellas Gene Therapy Plant Opens in Sanford

Following two years of development, Astellas’ new late-stage clinical and commercial manufacturing plant officially opened its doors in Sanford today, June 8.

Located in Central Carolina Enterprise Park, the 135,000-square-foot facility is equipped for clinical and commercial-scale manufacturing of Astellas’ pipeline of adeno-associated virus (AAV) gene therapies. It also will help address the Japan-headquartered company’s supply chain needs and provide in-house quality control and testing.

The $100 million site will employ more than 200 people in and around Lee County, with an average annual salary of $83,900. That’s more than twice the county’s current average. So far, the company says, about 50 employees work at the site, which still must receive approval from the U.S. Food and Drug Administration to begin commercial production.

“Our new manufacturing facility symbolizes our company’s continued dedication to the advancement of novel life-changing gene therapies for patients with severe diseases and a significant unmet need, as well as our commitment to the Sanford community,” said Mathew Pletcher, Ph.D., senior vice president and division head of Gene Therapy Research and Technical Operations at Astellas Gene Therapies. “This new facility is a key enabler of our mission to develop safe, effective, and transformative gene therapies as swiftly as possible.”

North Carolina Governor Roy Cooper emphasized that North Carolina is a premier location for Astellas. “Our state’s skilled workforce, leading academic institutions and healthy business environment provide all the resources they need to foster innovation and continued success,” he said.

If the company meets its hiring milestones, it will qualify for a state Job Development Investment Grant worth up to $3.7 million. Lee County and the City of Sanford also are offering up to $5.7 million in incentives. The package includes nearly $400,000 in training support from the North Carolina Community College System.

Gene Therapies Center of Excellence focuses on AAV

The new operation is part of the Astellas Gene Therapies Center of Excellence that was created following the 2020 acquisition of San Francisco-based Audentes Therapeutics, Inc. It is positioned to become a global leader in genetic medicines developed around the AAV vector. AAV is engineered to deliver genetic information that codes for the production of healthy genes to replace missing or deficient ones.

The technology offers the advantages of safety, efficient long-term gene expression, and ease of genetic manipulation. It also results in low or, in many cases, no immune response in people who receive initial doses.

Astellas targets serious rare genetic diseases – particularly neuromuscular disorders – that affect young children. They include Pompe disease, Duchenne muscular dystrophy, and mytonic dystrophy Type 1. The company’s goal is to deliver transformative therapies for patients who have few or no effective treatment options.

Astellas’ lead product candidate, AT132, is designed to treat X-linked Myotubular Myopathy (XLMTM). XLMTM is a serious and rare life-threatening neuromuscular disease characterized by extreme muscle weakness, respiratory failure and early death. It is caused by mutations in the MTM1 gene and affects about one in 40,000 to 50,000 newborn males.

AAV Harnessed by UNC pioneer Jude Samulski

Proprietary AAV vectors were first developed by pioneers in the field that included Richard Jude Samulski, Ph.D. Samulski co-founded Research Triangle Park-based Asklepios Biopharmaceutical, commonly known as AskBio, in 2001 to commercialize AAV gene therapies. The North Carolina Biotechnology Center supported his academic research at the University of North Carolina School of Medicine – and the development of his commercial technologies – with more than $1.5 million in grants and loans.

NCBiotech also helped recruit Astellas to North Carolina – in collaboration with the North Carolina Department of Commerce and the Economic Partnership of North Carolina, the N.C. General Assembly, the North Carolina Community College System, Lee County, the City of Sanford, and the Sanford Area Growth Alliance.

“Astellas Gene Therapies’ new Sanford facility is a testament to the company’s commitment to bringing lifesaving treatments to their patients, said Bill Bullock, NCBiotech’s senior vice president for economic development and statewide operations. “And the passion around which they pursue that goal is a perfect fit for NC’s life sciences community.”

About Astellas

Astellas operates in more than 70 countries around the world. Its Gene Therapies Center of Excellence currently is exploring three gene therapy modalities: gene replacement, exon skipping gene therapy, and vectorized RNA knockdown. The unit is based in San Francisco, with manufacturing and laboratory facilities located there as well as at the new Sanford facility.