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Phase 1/2 Open-Label, Multicenter Study to Assess the Safety, Tolerability and Efficacy of a Single Dose of PBGM01 Delivered Into the Cisterna Magna of Pediatric Type 1 (Early Onset) and Type 2a (Late Onset) Infantile GM1 Gangliosidosis

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Phase 1/2 Open-Label, Multicenter Study to Assess the Safety, Tolerability and Efficacy of a Single Dose of PBGM01 Delivered Into the Cisterna Magna of Pediatric Type 1 (Early Onset) and Type 2a (Late Onset) Infantile GM1 Gangliosidosis

Status: Active, no longer recruiting
Phase of Trial: Phase I/II

Latest Information Update: 16 Aug 2024

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At a glance

  • Drugs PBGM-01 (Primary)
  • Indications Gangliosidoses
  • Focus Adverse reactions; Therapeutic Use
  • Acronyms Imagine-1
  • Sponsors Passage Bio

    • Most Recent Events

    • 08 Aug 2024 According to a Passage Bio media release, following a restructuring of the original agreement of Passage Bio with the University of Pennsylvania about its Gene Therapy Program, a research, collaboration, and license agreement with GEMMA Biotherapeutics was finalized. Consequently, GEMMA Biotherapeutics received an exclusive, worldwide license for all future development of PBGM-01 for the treatment of GM1 gangliosidosis, and 2 other drugs.
    • 16 Jan 2024 Status changed from recruiting to active, no longer recruiting.
    • 07 Aug 2023 According to a Passage Bio media release, the amended study protocol has been approved at several clinical trial sites in multiple countries, including Brazil, Turkey and the United States.

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