STAT+: FDA chiefs offer roadmap to expand custom gene-editing treatments like Baby KJ’s
Top Food and Drug Administration officials on Wednesday detailed a roadmap for approving the world’s first personalized gene-editing treatments.
The perspective, published in the New England Journal of Medicine, comes six months after researchers announced they had crafted a custom gene-editing treatment to fix a unique mutation in KJ, an infant born with an ultra-rare, life-threatening liver disease.
KJ’s therapy was hailed as a milestone in the history of medicine, but one of debatable relevance to other patients facing devastating genetic diseases. It required dozens of scientists across several institutions and companies, working for free or at-cost, to come together around a single patient. Researchers have declined to disclose the exact cost, though it likely ran into the millions, if not tens of millions.
Jason Mast
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