Indication selection: Lessons learned from the CRISPR-Cas9 story

It’s been over a decade since a small group of biotechs launched to create curative therapies using CRISPR-Cas9. Their track record of strategic decisions, successes, failures and lessons learned provides new insights into the old debate over whether platform companies should validate their technology on established targets or pursue new ones. 

BioCentury discussed these issues with the founding CEOs of the original four CRISPR-Cas9 companies. Their views on pipeline strategy remain mixed, but so far, the more de-risked targets and indications are the ones that have led to clinical success. Yet, those clinical achievements have not translated into commercial success...