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Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA

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Trial Profile

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: 26 Apr 2026

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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

  • 02 Apr 2026 According to an Ultragenyx Pharmaceutical media release, the U.S. Food and Drug Administration (FDA or the Agency) has accepted for review the resubmitted Biologics License Application (BLA) seeking accelerated approval for UX111 (rebisufligene etisparvovec) AAV9 gene therapy as a treatment for patients with Sanfilippo syndrome Type A (MPS IIIA). The FDA set a Prescription Drug User Fee Act (PDUFA) action date of September 19, 2026.
  • 13 Feb 2026 According to an Ultragenyx Pharmaceutical media release, earlier today the company received an Incomplete Response Letter (IRL) regarding its resubmitted BLA. The IRL requests additional supportive documentation related to its CRL CMC responses, which the company will provide in a resubmission.
  • 30 Jan 2026 According to an Ultragenyx Pharmaceutical media release, company announced that the detailed updates will be presented next week at the WORLDSymposium™ 2026 in San Diego.

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