Pfizer Said Looking for Gene Therapy Site

After acquiring the remaining stake in Bamboo Therapeutics, a privately held gene therapy group based in Chapel Hill, North Carolina, USA, last year in a drive to be a major player in gene therapies, US pharmaceuticals giant is said to be looking for a location to build a new production facility.

According to a report by the Triangle Business Journal, which covers the Raleigh-Durham-Chapel Hill, North Carolina tri-cities area, Pifizer has been searching for a site in the area and has spoken to state and local officials about a potential $100 million expansion project. At the same time, the paper sources said the drugmaker may also consider a site in the state of Massachusetts, where it is currently is building a $200 million biologics and vaccines production facility at Andover. 

Bamboo already has a manufacturing facility in Sanford, North Carolina, acquired last year from the University of North Carolina, where it is said to have produced phase I and II materials. The gene therapy firm currently is working on recombinant adeno-associated virus (rAAV)-based gene therapies for rare diseases.

Pfizer first entered the field in 2014, striking a deal with Spark Therapeutics in hemophilia. Simultaneously, it also established a dedicated gene therapy research center in London known as the Genetic Medicines Institute, which is part of the company’s Rare Disease Research Unit.

Up to now, no gene therapies have been approved yet in the US. A  Dutch company, uniQure, has developed Glybera, the first gene therapy approved in Europe, designed to cure the ultra-rare disease called lipoprotein lipase deficiency. Priced at more than $1.2 million. It has been called the world’s most expensive drug.

According to reports, uniQure is now focused on a hemophilia B program, competing with the gene therapy being developed by Spark Therapeutics and Pfizer. The Dutch company has a $25 million gene therapy manufacturing facility in Lexington, Massachusetts, but is said to have had to eliminate jobs to cut costs.

The UK’s GlaxoSmithKline recently won approval in Europe for Strimvelis, its gene therapy for severe combined immunodeficiency, sometimes called bubble boy disease, a genetic disorder which results in an extreme vulnerability to infectious diseases. The one-time treatment costs about $665,000, but is said to offer a reimbursement if the treatment fails.