A top United States regulator plans to unveil a faster approach to approving custom gene-editing treatments, a move designed to unleash a wave of industry investment that will yield cures for patients with rare diseases.
Vinay Prasad, who oversees gene therapies at the Food and Drug Administration, said scientific advances, like Crispr, have forced the agency to relax some of its strict rules.
“Regulation has to evolve as fast as science evolves,” Prasad said …. The agency is “going to be extremely flexible and work very fast with the scientists who want to bring these therapies to kids who need it.”
Historically, every treatment for every disease has needed its own separate study. The advent of Crispr … has made it theoretically possible for scientists to develop cures for many of the 7,000 rare diseases, a small fraction of which have treatments. But the FDA’s demands made it seem too expensive and time-consuming.
Related on the SLP
Now, the FDA is allowing a combined trial of patients with related genetic disorders …. It’s a fundamental shift in how the agency approaches genetic therapies, potentially freeing them from traditional rules that slowed innovation.
