Escaping the Valley of Death

Abstract

A joint project of the American Society of Gene and Cell Therapy (ASGCT) and the Trans–National Institutes of Health (NIH) gene therapy group, the NIH Gene Therapy Symposium took place at the NIH Natcher Center in Bethesda, Maryland, on 26–27 September 2011. More than 400 registrants, primarily from the NIH and the US Food and Drug Administration (FDA), met to review the challenges faced by investigators moving experimental gene and cell therapies into the clinic and to present examples of how technical and regulatory hurdles have been addressed both within the United States and in Europe. The genesis of the symposium was a series of meetings in February 2010 among various NIH institute directors and the then ASGCT President Ken Cornetta and Vice President Barrie Carter, with the aim of discussing how the Society and the NIH could work together to capitalize on the growing successes in gene and cell therapy. A key theme addressed at the symposium was the issue of how to cross the so-called “valley of death”—the critical period in the development pathway of complex biologics that spans the stages between preclinical validation and clinical studies of new therapeutics (Figure 1)—and how the ASGCT could work with the NIH to help facilitate clinical translation of new gene and cell therapies.