Either you have JavaScript disabled or your browser does not support Javascript . To work properly, this page requires JavaScript to be enabled.
How to enable JavaScript in your browser?

Rebisufligene etisparvovec - Ultragenyx Pharmaceutical

Next Previous
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: 05 Feb 2026

Price :
$50 *
Note:
  • Adis is an information provider. We do not sell or distribute actual drugs.
  • Final gross price and currency may vary according to local VAT and billing address.
  • Your purchase entitles you to full access to the information contained in our drug profile at the time of purchase.
  • A link to download a PDF version of the drug profile will be included in your email receipt.

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

  • 30 Jan 2026 Ultragenyx anticipates a PDUFA decision from US FDA by February 2026, with a PDUFA date expected to be in the third quarter of 2026
  • 30 Jan 2026 Ultragenyx Pharmaceutical resubmits the Biologics License Application (BLA) to the US FDA for Rebisufligene etisparvovec (UX 111) in Mucopolysaccharidosis III
  • 04 Nov 2025 Ultragenyx Pharmaceuticals anticipates resubmission of Rebisufligene etisparvovec for Mucopolysaccharidosis III in early 2026

You need to be a logged in or subscribed to view this content

Request demo (opens in a new window)
If your organization or you do not have a subscription, try one of the following: If your organization has a subscription, there are several access options, even while working remotely:
  • Working within your organization’s network
  • with username/password or try to via your institution
  • Persisted access using your organization’s identifier stored in your user browser for 90 days
Back to top