Developing Gene Therapies

Gene therapies (GTx) are a broad class of medicines for human use that are based on the alteration of genes, tissues, or cells. These novel treatment modalities hold great promise for patients with high unmet medical needs such as rare genetic diseases. The drug development program for a gene therapy... read moremay be very challenging and nuanced, and it may be quite different compared to low molecular weight or antibody drugs. The goal of the present lecture series is to provide an exposition to various scientific and strategic topics arising in research and development of gene therapies – from therapeutic design and planning to clinical trial design and analysis, commercial adoption, and patient access.