Gene Therapy Safety Drugs May Reduce Treatment Efficacy in Dravet Syndrome

247 Health News Editorial

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14 May 2026

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2 min read

·healthtech

New research from Encoded Therapeutics reveals that drugs used to prevent dangerous gene therapy side effects may reduce treatment effectiveness in children with Dravet syndrome. The finding highlights a critical safety-efficacy tradeoff in neurological gene therapy applications.

Originally reported by STAT News

References

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  2. 2.Dravet, C. (2011). The core Dravet syndrome phenotype. Epilepsia, 52(2), 3-9. https://doi.org/10.1111/j.1528-1167.2011.02994.x
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  5. 5.Mast, J. (2026). Drug meant to make gene therapy safer may also make it less effective. STAT News. https://www.statnews.com/2026/05/13/encoded-therapeutics-dravet-syndrome-study-sirolimus-safety-tradeoff/
  6. 6.Mendell, J. R., Al-Zaidy, S., Shell, R., Arnold, W. D., Rodino-Klapac, L. R., Prior, T. W., ... & Kaspar, B. K. (2017). Single-dose gene-replacement therapy for spinal muscular atrophy. New England Journal of Medicine, 377(18), 1713-1722. https://doi.org/10.1056/NEJMoa1706198
  7. 7.Mingozzi, F., & High, K. A. (2013). Immune responses to AAV vectors: overcoming barriers to successful gene therapy. Blood, 122(1), 23-36. https://doi.org/10.1182/blood-2013-09-521831
  8. 8.Ronzitti, G., Gross, D. A., & Mingozzi, F. (2020). Human immune responses to adeno-associated virus (AAV) vectors. Frontiers in Immunology, 11, 670. https://doi.org/10.3389/fimmu.2020.00670
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