“Give Cas a Chance”: Fyodor Urnov Says He Wants a CRISPR Revolution

In an extraordinary 6,000-word guest editorial in the October 2024 issue of The CRISPR Journal (a sister journal of GEN, published by Mary Ann Liebert, Inc.), Fyodor Urnov, PhD, laid out the urgent case for major reform in the regulatory appraisal of clinical therapies involving CRISPR gene therapies.

“CRISPR-Cas deserves a chance to make a major impact in Mendelian disease treatment space—an impact it is currently not making,” Urnov declared. “This is despite the fact that the technology itself is clinic-grade, delivery to cells and organ systems where such disease is prevalent is also clinic-grade, and the complete toolbox of nonclinical derisking exists.”

The editorial anchors a special issue of The CRISPR Journal devoted to “Clinical Trials,” published this week.

When Urnov speaks (or writes), the genome editing community tends to listen. He has been at the forefront of gene editing research for 25 years, helped coin the term “genome editing” in 2005, played a key role in validating the strategy that led to the development of Casgevy, and has spent the past five years working with...