Drug Profile


Alternative Names: LYS-GM101

Latest Information Update: 23 Feb 2017

Price : $50

At a glance

  • Originator LYSOGENE
  • Developer Auburn University; LYSOGENE; University of Massachusetts Medical School
  • 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.

    Yes - Gangliosidoses
  • On Fast track

    Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.

  • New Molecular Entity No

Highest Development Phases

  • Preclinical Gangliosidoses

Most Recent Events

  • 21 Feb 2017 LYS GM101 receives Orphan Drug status for Gangliosidoses in European Union
  • 07 Feb 2017 LYS GM101 receives Orphan Drug status for Gangliosidoses in USA
  • 18 Jan 2017 US FDA grants Rare Paediatric Disease designation to LYS GM101 for Gangliosidoses
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