Astellas Gene Therapies is an Astellas Center of Excellence developing genetic medicines with the potential to deliver transformative value for patients. Based on an innovative scientific approach and industry leading internal manufacturing capability and expertise, we are currently exploring three gene therapy modalities: gene replacement, exon skipping gene therapy, and vectorized RNA knockdown and will also advance additional Astellas gene therapy programs toward clinical investigation. We are based in San Francisco, with manufacturing and laboratory facilities in South San Francisco and Sanford, North Carolina.

We use a focused set of criteria to select target indications, which we believe increases the chances of success for each of our programs. These criteria include:

  • High potential for meaningful clinical benefit
  • Monogenic diseases with well-understood biology
  • Disease characteristics well-suited for treatment with our AAV gene therapy technology platform
  • Clear measures for evaluation in clinical trials

We believe the quality, reliability and scalability of our gene therapy development and manufacturing approach is critical to our long-term success.

We are leveraging our proprietary AAV gene therapy technology platform, consisting of end-to-end internal expertise from vector construct engineering to state-of the-art large scale cGMP manufacturing, to rapidly advance our programs from discovery through clinical development. Internal process and analytical development, cGMP plasmid production and QC testing capabilities complete the fully integrated approach to production and release of our product candidates for clinical and commercial use. Our current 2×500 -liter scale manufacturing operations in South San Francisco provide sufficient capacity for global commercialization of our lead program AT132, as well as continued clinical development of pipeline programs, and the facility is capable of expanding to include at least 4,000 liters of added production.

Our new facility in Sanford, North Carolina is being built with an initial capacity of 4,000 liters of production and is designed for future expansion to accommodate an additional 16,000 liters of capacity. The Sanford facility will not only provide important manufacturing redundancy to our South San Francisco operations, but will also support the expanding and diverse pipeline of AAV programs resulting from our merger with Astellas.

We believe our strong relationships with key opinion leaders, leading academic institutions, other rare disease companies and patient advocacy groups will support our product development efforts and our potential for future commercial success.

Leveraging our collaborations with these parties allows us to better understand the diseases we target and optimize our research, clinical development and commercial plans.

Our new facility

In February 2020, we announced plans to invest $109 million to build a new 135,000 square foot, state-of-the-art gene therapy manufacturing facility in Sanford, North Carolina. The plant is expected to be GMP ready and fully operational in 2022. The remaining investment will take place over two additional planned expansion phases. Once fully operational, this new facility will create over 200 new jobs.