Astellas’ gene therapy manufacturing facility is located in Sanford, North Carolina, US. Credit: Business Wire.
The facility was opened in June 2022. Credit: Astellas Pharma.
The new facility manufactures AAV vectors for gene therapy. Credit: Business Wire.

Astellas Pharma, a pharmaceutical company based in Japan, opened a gene therapy manufacturing facility in Sanford, North Carolina, US, in June 2022.

The facility is used for clinical and commercial production of the company’s adeno-associated virus (AAV) vectors – the gene delivery platforms for gene therapies.

The company invested $100m in the development of the advanced manufacturing facility, which is expected to generate more than 200 new jobs in Lee County through 2026. Upon meeting the hiring goals, the company will be eligible for a state Job Development Investment Grant worth up to $3.7m. Additionally, Lee County and the city of Sanford are providing incentives of up to $5.7m to the company.

Key partners in the project were the North Carolina Department of Commerce, North Carolina Biotechnology Center, North Carolina General Assembly, Economic Partnership of North Carolina, Sanford, Lee County, Sanford Area Growth Alliance, and the North Carolina Community College System.

Astellas’ gene therapy manufacturing facility location

The facility is located at 6074 Enterprise Park Drive within Central Carolina Enterprise Park in Sanford, Lee County, North Carolina. The company chose Lee County as the location for the facility to strengthen its position in the genetic medicines industry.

North Carolina houses influential research centres and the largest biomanufacturing workforce comprising highly skilled personnel and a talent pool of gene therapy researchers and scientists. The company preferred the state to others, including California, Massachusetts, and Colorado.

Astellas’ gene therapy manufacturing facility details

The state-of-the-art facility covers an area of 135,000ft² near Pfizer’s $600m gene therapy manufacturing facility in Sanford.

The good manufacturing practices-compliant facility features manufacturing and laboratory spaces to support both clinical and commercial-scale production of Astellas’ AAV gene therapy products in its development pipeline.

Additionally, it helps in meeting the supply chain requirements of the company while offering internal quality control and testing capabilities.

It serves as a flagship production plant and supports Astellas’ strategy of developing and bringing genetic drugs swiftly to patients. The facility is an integral component of the Astellas Gene Therapies Center of Excellence, which came into being after the acquisition of the US-based biotechnology company Audentes Therapeutics in 2020.

The facility also boosts the company’s growth plans for expanding its geographic and therapeutic scope and creating a global supply chain.

Astellas’ AAV gene therapy technology platform details

AAV vector is a non-enveloped virus that can be engineered as a carrier to deliver DNA to target cells. The process involves gene encoding to produce healthy genes to replace missing or deficient genes.

The company’s AAV gene therapy technology platform includes vector construct engineering, research production, and large-scale manufacturing, as well as quality control testing to enable the delivery of product candidates for clinical and commercial use.

The AAV-based genetic medicines company develops programmes covering modalities such as gene replacement, vectorised exon skipping, and vectorised RNA knockdown.

Astellas’ lead product candidate AT132 development

Astellas developed the lead AAV gene therapy candidate, AT132, for the treatment of X-linked Myotubular Myopathy (XLMTM), mainly observed in infants.

XLMTM is a rare genetic neuromuscular disorder that occurs due to mutations in the MTM1 gene, resulting in a lack of or dysfunction of the protein myotubularin required for the development and function of skeletal muscle cells. Those with the disease experience muscle weakness and decreased muscle tone.

AT132 delivers a functional copy of the MTM1 gene using an AAV8 vector. The vector introduces and expresses the myotubularin protein in the skeletal muscle cells.

The company is currently using a 1,000-litre scale manufacturing facility to support the production of AT132 and plans to expand with the addition of 8,000-litre production capacity in future.

Marketing commentary on Astellas

Astellas is a clinical-stage business engaged in the development of innovative AAV-based genetic medicines. The company focuses on developing and commercialising gene therapy medicines for the treatment of severe rare neuromuscular disorders such as Duchenne muscular dystrophy, myotonic dystrophy Type 1, and Pompe disease.

The company acquired Audentes Therapeutics through its indirect wholly-owned subsidiary Asilomar Acquisition in 2020. It has operations in more than 70 countries across the world.