The scientists behind treating Baby KJ say they’ve hit a stumbling block in their efforts to create more custom gene editing treatments for children with rare diseases.
Food and Drug Administration reviewers, they say, are imposing high manufacturing and quality control standards that could make it too expensive and complicated for them — or any academics — to bring such bespoke therapies to approval.
Instead, they warned, such efforts could require the resources of industry.
This article is exclusive to STAT+ subscribers
Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+.
Already have an account? Log in
