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 Enzyme replacements; Gene modulators; Glucosylceramidase replacements; Immunogenetic stimulants
  • 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

  • 27 Mar 2017 AVROBIO plans a phase I/II trial for Gaucher's disease in USA
  • 22 Mar 2017 Preclinical trials in Gaucher's disease in USA (IV)
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