STAT+: Gene therapies and CRISPR drugs slowed by 'irrational pessimism,' say scientists forging new paths

NEW YORK — To gene therapy pioneer Jim Wilson, the field is currently clouded with “irrational pessimism.” Companies are abandoning ship; approved therapies aren’t being prescribed. Yet both he and University of Pennsylvania researcher Kiran Musunuru are forging ahead, using contrasting approaches, as they explained onstage at STAT’s Breakthrough Summit East. 

Wilson has left academia entirely to become president and CEO of GemmaBio, betting that by partnering with middle-income countries and by making treatments undergirded by the same reusable platform, his company can succeed where others have failed. Musunuru, meanwhile, is using his position within academia to tackle those bespoke, n-of-1 CRISPR-based therapies that industry is now wary of taking on. 

This darker period for gene therapies, in Wilson’s estimation, is an overcorrection for the irrational exuberance of an earlier time. “While we have enormous experience in the safety profile, there still are concerns about long-term safety,” he said, which he thinks can be overcome by focusing on areas of unmet medical need. But the whole project of gene therapy has been even more difficult than he expected. 

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