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 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

  • 30 Aug 2017 LYSOGENE plans to initiate a phase I/II trial in Gangliosidoses in January 2019
  • 24 May 2017 Lysogene enters into a manufacturing agreement with Brammer Bio for large scale production of LYS GM101
  • 24 May 2017 Lysogene intends to file an IND application for Gangliosidoses
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