CRISPR: taking the shortcut to a healthy genome editing enterprise.

Abstract

For a field primed to deliver, can we envision the translational, policy & business paths for a long-term success? Methods and tools for ‘genetic engineering’ or ‘genetic modification’ have been used successfully for more than three decades in animal models, as well as treated a number of patients in early gene therapy trials. Until recently, specificity challenges and downstream laborious processes have been upping the stakes in curing genetic disease. However, a number of scientific breakthroughs have led researchers to build tools that edit genomes with unprecedented efficiency and precision, enabling the modification of any part of the genome. The use of precision genome modification has exploded, swelling hopes for the elucidation, modeling and treatment of disease, also creating a surge of companies looking to build and capitalize on the technologies as research tools, novel approaches in agriculture and transformative treatments. In under 3 years, the CRISPR system in specific has seen unprecedented rise in funding, patents and publications, with first ideas already progressing to the clinic. In light of growing global excitement and ongoing scientific success in countries like the USA and UK, there is a timeliness and urgency to support the evolution of a sustainable gene editing field and deliver its promise for a clear economic and social benefit. Building off of this momentum and drawing upon a rapidly growing body of literature and public forums, this report aims to identify critical parameters impacting the acceleration of genome editing technologies, particularly the CRISPR–Cas9 system, and the development of transformative tools and products.