Empowered Patient Podcast

Ha Tran, Medical Head, Cell and Gene Therapy at Astellas Pharma is developing gene therapy approaches to fight neuromuscular diseases, central nervous system disorders, and ophthalmology conditions.  There is strong evidence that this approach can be a way to treat and possibly cure rare diseases. Ha explains the challenges of running gene therapy trials for rare diseases and the unique patient journey required to run a successful trial. Ha explains, "Currently, at Astellas, our platform is AAV gene therapy, which is a replacement. Additionally, in our early pipeline, we are looking at both gene therapy replacement and vectorized RNA knockdown. We also have a cell therapy division, which I also cover. For that, we’re looking at in vivo cell therapy." "Honestly speaking, gene and cell therapy trials have their nuances since they are often a single infusion or single injection. So, the preclinical data packages and long-term follow-up plans differ from standard drug development. But the biggest nuances actuall