by William Lee, PhD
Volume 5, Issue 2 (Summer 2006)
Since the first gene therapy trials were conducted 25 years ago, there have been high expectations from the public, and much attention from investors, that previously incurable diseases would be cured by gene therapy. Still, despite numerous gene therapy clinical trials for many different indications, there are no approved gene therapy drugs in the United States. In 1999, one gene therapy patient died during clinical trials, the first ever. This highly publicized event led to heightened regulatory scrutiny over all such trials. Then in 2003 and 2005, three subjects developed leukemia as a direct consequence of gene therapy; one of them eventually passed away. The regulatory response stemming from these incidents led to greater regulatory oversight in gene therapy, as compared to other investigational drugs and biologics…
Citation:
Lee W. Regulatory Overview of Conducting Gene Therapy Clinical Trials in the United States. BioProcess J, 2006; 5(2): 45-50. https://doi.org/10.12665/J52.Lee