- Tokyo-based drugmaker Astellas announced Wednesday the opening of a manufacturing plant in North Carolina capable of supplying gene therapies for late-stage clinical testing as well as commercial-scale production, should any of the company’s experimental treatments gain approval.
- Plans for the 135,000-square-foot facility were initially unveiled in early 2020 by Audentes Therapeutics, a gene therapy developer that Astellas had just acquired for $3 billion. Astellas said it ultimately invested $100 million in the facility, where it expects to create more than 200 jobs by the end of 2026.
- The plant’s opening follows several significant setbacks to Astellas’ most advanced gene therapy program, which is testing a potential treatment for children with a rare neuromuscular disease. A key study of the treatment, called AT132, has been paused multiple times over the past couple years due to safety concerns, including the deaths of four participants.
For years, Astellas has been built around the sale of cancer medications. Among the company’s main products are Xtandi, a prostate cancer drug that’s co-owned by Pfizer, and Padcev, a bladder cancer medicine that Astellas developed in partnership with SeaGen.
But like many of its peers, Astellas has also branched into gene therapy research, attracted by the potential to not only treat countless diseases, but possibly cure them as well. On that front, the company’s biggest investment has been Audentes, which it has since rebranded to Astellas Gene Therapies.
That $3 billion acquisition valued Audentes shares at $60 apiece, or more than double what they were worth at the time. Such a premium wasn’t unheard of for a gene therapy buyout. Yet, in the years since, Astellas has hit a series of setbacks that raise questions about whether it overpaid.
That key study of AT132, for instance, has been delayed at least three times because of safety concerns. The Food and Drug Administration placed a formal hold on the trial in mid-2020, after Audentes reported three children who were given its therapy later developed liver problems that ultimately led to fatal complications. Astellas subsequently received criticism, both for how it disclosed these deaths and for not releasing more safety data.
Then, in September 2021, Astellas said it had paused screening and dosing of more participants in the study after another patient showed unusual liver function. Two weeks later, the company reported that the patient had died.
The four deaths added to broader fears about the potential risks posed by gene therapies. Indeed, just days after Astellas paused screening, the FDA convened a panel of experts in a pre-planned meeting to offer advice on how to make this research safer.
For Astellas, the challenges have extended beyond AT132. In April, the company announced the termination of several programs targeting a different rare neuromuscular disease: Duchenne muscular dystrophy.
Between the setbacks with AT132 and the Duchenne therapies, Astellas has recorded more than $700 million in impairment losses.
Still, the company says it remains committed to developing gene therapies, and that the new North Carolina plant will be a critical tool in its efforts.
“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,” Mathew Pletcher, division head of gene therapy research and technical operations at Astellas Gene Therapies, said in a statement.
“This is critical to rapidly advance our programs and drive the next phase of growth for Astellas,” Pletcher addded.