Senior Director Passage Bio Philadelphia, Pennsylvania
Cell and gene therapies are now emerging as important treatments for various diseases. Particularly, for rare diseases, cell and gene therapies may hold the only potential option for long term treatment or even cure. Efficient methods for manufacturing, addressing conduct of clinical trials evaluating safety and efficacy in small populations, and ensuring availability are issues that all need to addressed. Adjusting the application of existing regulatory paradigms can facilitate these aspects of the development of these therapies.
Review recent regulatory developments in celll and gene therapy
Understand the importance of therapies for disorders affecting very small numbers of individuals (bespoke therapies)
Describe potential development pathways for evaluating the safety and efficacy of cell and gene therapies for bespoke therapies