Drug Profile


Alternative Names: AVR-RD-02

Latest Information Update: 28 Mar 2017

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At a glance

  • Originator Lund University
  • Developer AVROBIO
  • Class Gene therapies
  • Mechanism of Action Cell replacements; Glucosylceramidase replacements
  • Orphan Drug Status

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

  • New Molecular Entity No

Highest Development Phases

  • Preclinical Gaucher's disease

Most Recent Events

  • 09 Aug 2018 AVROBIO plans to file an clinical trial application (CTA) for AVR RD 02 in Gaucher disease in second half of 2018
  • 01 Feb 2018 AVROBIO plans a phase I/II trial for Gaucher's disease in USA in June 2019
  • 22 Mar 2017 Preclinical trials in Gaucher's disease in USA (IV)
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