Path to Developing Gene Therapies for Treating Rare Diseases with Ha Tran Astellas Pharma

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 actually for clinical trial design are in rare diseases. The FDA just finalized its guidance for the industry in December. For a long time it was just a draft guidance, but now it's final and I found it to be quite helpful." "There's this major need to understand the patient population, and I see this as threefold. So, understanding country-specific prevalence, the quality of the existing natural history data, and the different phenotypes to target the correct patient population. And there are major pitfalls here if you rush through the process. So you can grossly overestimate patient population if you're using averages instead of really looking into very specific data. So this can lead to slower recruitment and longer timelines once you actually start your study." #AstellasPharma #GeneTherapy #RareDiseases #Allogeneic #ClinicalTrials #CNS #NeuromuscularDiseases astellas.com/us/ Download the transcript here