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UX 111

Drug Profile

UX 111

Alternative Names: AAV SGSH - Abeona; AAV-SGSH; AAV9 SGSH - Abeona; ABO 102; ABO-102 - Abeona; ABX 102 - Abeona; ABX B - Abeona; SC AAV9 SGSH; scAAV9 SGHG; scAAV9.U1a.hSGSH; UX-111

Latest Information Update: 13 Feb 2024

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At a glance

  • Originator Nationwide Children's Hospital
  • Developer Abeona Therapeutics; Ultragenyx Pharmaceutical
  • Class Gene therapies
  • Mechanism of Action Gene transference; N-sulfoglucosamine sulfohydrolase replacements
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Mucopolysaccharidosis III
  • New Molecular Entity No
  • Available For Licensing Yes

Highest Development Phases

  • Phase III Mucopolysaccharidosis III

Most Recent Events

  • 06 Feb 2024 Updated efficacy and adverse events data from the phase II/III Transpher A trial in Mucopolysaccharidosis III released by Ultragenyx
  • 16 Oct 2023 Ultragenyx Pharmaceutical completes enrolment in its I/II pivotal Transpher A trial for mucopolysaccharidosis IIIA
  • 16 Feb 2023 Ultragenyx plans a meeting with US FDA to discuss a plan for accelerated approval in Q4 2023
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