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Phase I/II/III Gene Transfer Clinical Trial of ScAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA

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Phase I/II/III Gene Transfer Clinical Trial of ScAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA

Status: Recruiting
Phase of Trial: Phase II/III

Latest Information Update: 25 Feb 2025

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At a glance

  • Drugs Rebisufligene etisparvovec (Primary)
  • Indications Mucopolysaccharidosis III
  • Focus Registrational; Therapeutic Use
  • Acronyms Transpher A
  • Sponsors Ultragenyx Pharmaceutical

    • Most Recent Events

    • 18 Feb 2025 According to an Ultragenyx Pharmaceutical media release, the U.S. FDA has accepted for review the Biologics License Application seeking accelerated approval for UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA). The FDA granted Prescription Drug User Fee Act (PDUFA) action date of August 18, 2025. The application is supported by latest data from Transpher A and long-term follow-up studies presented at WORLDSymposium 2025.
    • 13 Feb 2025 According to an Ultragenyx Pharmaceutical media release, New clinical data were recently presented at WORLDSymposium™ 2025.
    • 13 Feb 2025 Results presented in the Ultragenyx Pharmaceutical media release.

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