The two highest-ranking Food and Drug Administration officials on Wednesday detailed a roadmap for approving the world’s first personalized gene-editing treatments. This initiative marks a significant step towards expanding access to innovative therapies tailored to individual genetic profiles.
The perspective, published in the New England Journal of Medicine, follows the groundbreaking announcement six months ago regarding a custom gene-editing treatment developed for KJ, an infant suffering from an ultra-rare, life-threatening liver disease. While KJ’s therapy is celebrated as a milestone in medicine, it raises questions about scalability and accessibility for broader patient populations. The collaborative effort required dozens of scientists from various institutions, working pro bono or at-cost, with undisclosed expenses likely reaching millions.
As the FDA lays out this roadmap, industry stakeholders in regulatory, QA/QC, CMC, and sourcing sectors must consider the implications for future gene-editing therapies, including the potential for increased investment and innovation in personalized medicine.
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