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Actio Biosciences has a precision medicine approach to rare diseases - promising preclinical data in a genetically defined epilepsy has them eagerly awaiting forthcoming clinical results

  • 39 minutes ago
  • 1 min read

CEO David Goldstein, a geneticist, describes his frustration with patient care when medicines were lacking rare genetic diseases, and how it motivated him to embark on drug development. Actio's lead program targets KCNT-1 and is in the clinic - preclinical animal work showed that inhibiting the target worked well, and that it may be less of a developmental condition.




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