Trends in cell and gene therapy clinical development for rare and ultra-rare diseases

Sven Kili shares his regular analysis of current clinical trends, this time focusing on the key area of rare diseases. As pressure mounts on patient recruitment, and the relatively few specialist clinical centers currently equipped to deal with advanced therapies, the clinical development community is seeking novel trial designs, endpoints and approaches to generate compelling, robust data from a minimal number of patients. Meanwhile, Covid-19 is presenting a further, hugely significant challenge to the entire field.

Please enter your email to access content Read now