Gene Therapy Production and Quality Control

Gene therapy is an emerging field and industry is yet to work out how best to manufacture these life-saving products says expert. Interest in gene therapy is growing, says Andrew Powaleny, public affairs director at the Pharmaceutical Research and Manufacturers of America (PhRMA). “As of December 2018, the last time we did our analysis, there were 289 cell and gene therapies in development either in clinical trials or awaiting FDA approval,” he adds. And the interest is understandable. Gene therapies let doctors treat the root causes of disease, potentially curing patients. Also judging by products like Zynteglo and Zolgensma, which cost $1.7m and $2.1m, respectively, gene therapies will generate significant revenue for firms able to commercialize them successfully.