US FDA releases new guidance on targeted cell and gene therapies

US Food and Drug Administration Commissioner Scott Gottlieb has issued a statement outlining the steps his agency is taking to modernize programs and “advance opportunities for developing more targeted therapies.”

These measures are taken, he says, with the aim of making the process of generating pre-clinical and clinical evidence more modern, scientifically rigorous, and efficient.

Dr Gottlieb said the FDA is developing technology- and disease-specific regulatory frameworks for new innovations, such as cell and gene therapies, that “may not have previously had a clear development pathway.”