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Rebisufligene etisparvovec - Ultragenyx Pharmaceutical

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Drug Profile

Rebisufligene etisparvovec - Ultragenyx Pharmaceutical

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: 11 Nov 2025

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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

  • Preregistration Mucopolysaccharidosis III

Most Recent Events

  • 04 Nov 2025 Ultragenyx Pharmaceuticals anticipates resubmission and approval of Rebisufligene etisparvovec for Mucopolysaccharidosis III in 2026
  • 11 Jul 2025 Ultragenyx Pharmaceuticals announces intention to resubmit BLA to US FDA for Mucopolysaccharidosis III
  • 11 Jul 2025 Ultragenyx Pharmaceuticals receives complete response letter from the US FDA for BLA submitted of Rebisufligene etisparvovec for Mucopolysaccharidosis III

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