Drug Profile


Alternative Names: Cystinosis gene therapy - AVROBIO

Latest Information Update: 09 Oct 2017

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At a glance

  • Originator GenStem Therapeutics
  • Developer AVROBIO
  • Class Gene therapies
  • Mechanism of Action Gene transference
  • 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 Cystinosis

Most Recent Events

  • 04 Oct 2017 AVROBIO announces intention to submit IND application to the US FDA for Phase-I/II trial in Cystinosis in late 2017
  • 04 Oct 2017 AVROBIO in-licenses Cystinosis gene therapy from GenStem Therapeutics
  • 04 Oct 2017 AVROBIO plans a clinical trial for Cystinosis in early 2018
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