Encoded Therapeutics sirolimus blunts gene therapy
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- Encoded Therapeutics tested a gene therapy for Dravet syndrome in 21 children, assigning most to receive steroids as immune suppression.
- Sirolimus was added for a subset of patients, especially those on the highest dose level, to further suppress immune response.
- Sirolimus may blunt the gene therapy’s effectiveness, according to the trial.
Why it matters: Families of Dravet patients risk slower progress as developers may need to drop sirolimus, potentially increasing reliance on steroids alone and altering trial outcomes; investors in gene‑therapy firms could see valuation shifts as safety‑efficacy trade‑offs are reassessed.
