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Phase 1 clinical trial of Angelman syndrome haematopoietic stem cell gene therapy

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Phase 1 clinical trial of Angelman syndrome haematopoietic stem cell gene therapy

Status: Planning
Phase of Trial: Phase I

Latest Information Update: 07 Feb 2025

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At a glance

  • Drugs Angelman-syndrome-haematopoietic-stem-cell-gene-therapy-TransformaTx-Biotherapeutics/University-of-California-at-Los-Angeles (Primary)
  • Indications Angelman syndrome
  • Focus Adverse reactions; Pharmacokinetics

    • Most Recent Events

    • 07 Feb 2025 New trial record
    • 30 Jan 2025 According to University of California media release, securing this critical funding from CIRM is a major step forward, accelerating path to a Phase 1 clinical trial.

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