CRISPR, Gene-Based Therapeutics, And Clinical Trials

At the border between discovery and clinical development lies a space that is simultaneously unknown yet seemingly full of potential. One such space is that created by the discovery of CRISPR-CAS9 gene editing technique. Never before have researchers had so precise a tool with which to make controlled changes to genetic material—in plants, animals, and humans. This discovery has fueled the imaginations of researchers and innovators around the globe and it has expanded significantly the potential for highly targeted advanced therapy medicinal products (ATMPS) in multiple therapeutic areas.

This series on CRISPR technology explores domains relevant to professional staff accountable for the design in the implementation of strategic clinical development programs involving advanced therapy medicinal products (ATMPS), beginning with translational medicine to the interface of discovery with development.