Phoenix Children’s Joins First Controlled Clinical Trial of Genetic Therapy for Dravet Syndrome - Summary - MDSpire
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Phoenix Children’s Joins First Controlled Clinical Trial of Genetic Therapy for Dravet Syndrome

The first controlled phase 3 trial of a genetic therapy for Dravet syndrome is now underway – with Phoenix Children's as one of 28 sites nationwide.

  • By

  • August 13, 2026

  • 4 min

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Objective:

To evaluate the efficacy and safety of zorevunersen, a genetic therapy for Dravet syndrome, in a controlled clinical trial.

Approach:
  • Clinical Trial Participation: Phoenix Children’s is one of 28 clinical sites participating in the EMPEROR phase 3 study.
  • Therapy Mechanism: The therapy uses antisense oligonucleotides (ASOs) to modulate gene expression rather than traditional gene replacement.
  • Patient Enrollment: Eight participants were enrolled in the trial, which includes four intrathecal administrations of the ASO.
Key Findings:
  • Dravet syndrome is caused by pathogenic variants in the SCN1A gene, leading to severe seizures and developmental delays.
  • The ASO-based therapy aims to increase Nav1.1 protein levels and restore neuronal function.
  • The primary endpoint of the study is the change in major motor seizure frequency.
Limitations:
  • The trial's results are pending as it is ongoing.
  • The study's findings may not be generalizable beyond the specific patient population enrolled.
Sources:

Original Source(s)

Phoenix Children’s Joins First Controlled Clinical Trial of Genetic Therapy for Dravet Syndrome

  • by

  • August 13, 2026

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