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Scalable Genetic Therapies for Rare Diseases: A Path Forward | Yale Department of Genetics posted on the topic | LinkedIn
What will it take to turn genetic therapies designed for a single patient into a scalable path for treating rare diseases? In a new Comment in Nature Genetics, Caroline Hendry and Yong-hui Jiang of the Department of Genetics at Yale School of Medicine examine the FDA’s new draft plausible mechanism framework for individualized therapies targeting rare genetic diseases.
Together with Jiangbing Zhou of Yale Neurosurgery and Biomedical Engineering, they assess both the promise of this framework and the groundwork needed to put it into practice.
They argue that the guidance is best understood not as a green light, but as a detailed map of the work ahead—including building robust natural history data, validating biomarkers and assays, characterizing delivery platforms, defining patient-centered outcome measures, and developing scalable manufacturing infrastructure.
Their perspective highlights the importance of early collaboration among researchers, regulators, clinicians, patient communities, foundations, and industry to realize the potential of individualized genetic medicines.
Read the Comment in Nature Genetics:
https://lnkd.in/ezvnXfxZ
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