Drug Profile


Alternative Names: LYS-GM101

Latest Information Update: 07 Jun 2017

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At a glance

  • Originator LYSOGENE
  • Developer Auburn University; LYSOGENE; University of Massachusetts Medical School
  • Class Gene therapies
  • Mechanism of Action Beta-galactosidase-expression-stimulants; Gene transference
  • Orphan Drug Status

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

    Yes - Gangliosidoses
  • New Molecular Entity No

Highest Development Phases

  • Preclinical Gangliosidoses

Most Recent Events

  • 23 Jul 2018 Lysogene announces that pre-IND meeting with the US FDA is scheduled in 2018
  • 16 Apr 2018 Lysogene announces that scientific advice or protocol assistance from the EMA is scheduled in the first half of 2018
  • 30 Aug 2017 LYSOGENE plans a phase I/II trial in Gangliosidoses in January 2019
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