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Only 20% of Gene Therapy Trials Meet Standards

2/21/2025

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Summary

Story summary

A recent study in JAMA Internal Medicine uncovers a troubling reality: just 20% of cell and gene therapy trials submitted to the FDA and EMA provided consistent evidence, underscoring a critical need for regulatory alignment. Additionally, Pfizer has pulled out of the gene therapy arena, halting its hemophilia treatment Beqvez amid dwindling interest.

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Story summary

A recent study in JAMA Internal Medicine uncovers a troubling reality: just 20% of cell and gene therapy trials submitted to the FDA and EMA provided consistent evidence, underscoring a critical need for regulatory alignment. Additionally, Pfizer has pulled out of the gene therapy arena, halting its hemophilia treatment Beqvez amid dwindling interest.