The proposed manufacturing facility will be built in Sanford, North Carolina, US. Credit: Business Wire.
Audentes is focused on developing innovative products based on adeno-associated virus (AAV) vector approach. Credit: Business Wire.
The new facility will manufacture gene therapy based medicines. Credit: Business Wire.

Audentes Therapeutics, a genetic medicines company, is planning to build a gene therapy manufacturing facility in Sanford, North Carolina, US.

The company will invest $109m in the development of the advanced manufacturing facility, with a portion of the capital investment made within a period of 18 months. The project will also involve two expansion phases carried out over two years, with support from the remaining investment.

The manufacturing facility is expected to begin operations in 2021, generating 209 jobs in Lee County. Hiring for the facility will start in 2020 and continue through 2026.

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

Audentes’ gene therapy manufacturing facility location

Audentes’ new gene therapy production plant will be in Sanford, Lee County, North Carolina, US.

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 bio-manufacturing workforce comprising highly skilled personnel, home to life sciences clusters, and a talent pool of gene therapy researchers and scientists. The company preferred the state over other states, including California, Massachusetts and Colorado.

The facility will cover an area of 135,000ft² near Pfizer’s new $600m gene therapy manufacturing facility, expected to be opened in 2020.

Audentes’ gene therapy manufacturing facility details

The facility in Sanford will be engaged in biopharmaceutical manufacturing of genetic medicines by using adeno-associated virus (AAV) vector approach.

It will serve as a flagship production plant and support Audentes’ strategy of developing and bringing genetic drugs swiftly to patients.

The proposed manufacturing facility will also boost the firm’s growth plans for expanding the geographic and therapeutic scope and creating a global supply chain.

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 for 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.

AT132 development

Audentes is developing a lead product candidate, AT132, for the treatment of X-linked Myotubular Myopathy (XLMTM), mainly observed in infants.

“The manufacturing facility is expected to begin operations in 2021, generating 209 jobs in Lee County. Hiring for the facility will start in 2020 and continue through 2026.”

XLMTM is a rare genetic neuromuscular disorder that occurs due to mutations in the MTM1 gene resulting in lack 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 uses an AAV8 vector that carries a functional copy of the MTM1 gene. The vector will introduce and express the myotubularin protein in the skeletal muscle cells.

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

The firm intends to submit a biologics licence application (BLA) for the gene replacement therapy candidate to the US Food and Drug Administration (FDA) in 2020.

Marketing commentary on Audentes

Audentes is a wholly-owned subsidiary of Japanese multinational pharmaceutical company Astellas Pharma. The company is a clinical-stage firm 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 (DMD), mytonic dystrophy Type 1 (DM1), and Pompe disease.

Headquartered in San Francisco, US, Audentes operates as the centre of excellence for Astellas Pharma’s genetic regulation primary focus area.

Audentes was acquired by Astellas to create a leading gene therapy company, in January 2020. The company will leverage Astellas’ scientific capabilities and resources to advance the development of its genetic medicines pipeline.