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-111Latest Information Update: 13 Feb 2024
Price :
$50
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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
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- 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