-An invited Guest Editorial entitled "Give Cas a Chance," by Fyodor Urnov, PhD, Director of Technology & Translation at the Innovative Genomics Institute (IGI), anchors the October 2024 special issue of The CRISPR Journal on "CRISPR Trials."
As guest editor of the special issue, Dr. Urnov has penned an extraordinary editorial that emphatically defines the magnitude of the crisis in the genome editing arena and offers a path forward. The inherently programmable nature of CRISPR gene editing that makes it so amenable to addressing thousands of rare genetic diseases is in jeopardy of being squandered unless action is taken now.
Despite the excitement over the FDA approval of Casgevy in December 2023, there are only a handful of gene editing therapies on course to win approval over the next 1-3 years. The picture is "akin to a skyscraper in an affluent country in the Middle East: the landscape around them is flat," Dr. Urnov writes.
Dr. Urnov proposes a set of interlocking initiatives, starting by pooling patients by syndrome, rather than tackling one mutation at a time. The next step would be to permit multiple gene editors to be incorporated into a single Investigational New Drug application. A further advance would involve focusing regulatory approval on a platform approach, similar to that used for personalized cancer vaccines.
Dr. Urnov argues passionately that the industry needs to adapt a "CRISPR as a platform" mindset without delay. "In the name of all the patients currently living with clinically editable genetic disease, and those who will be born to live with it-we have to give Cas a chance," he states.
The Guest Editorial by Dr. Urnov anchors the October 2024 special issue of The CRISPR Journal on "CRISPR Trials". The issue contains a Review article from Ross Wilson (IGI) and colleagues on Genome editing therapy for the blood; a Perspective from Jon Rueda, Lluis Montoliu and colleagues on the ethical imperative of affordable pricing for CRISPR therapies; a Perspective from Editor-in-Chief Rodolphe Barrangou and colleagues assessing the current status of CRISPR clinical trials; and five original research articles.
