Drug Profile


Alternative Names: SAF302

Latest Information Update: 22 Sep 2017

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At a glance

  • Originator LYSOGENE
  • 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 - Mucopolysaccharidosis III
  • New Molecular Entity No

Highest Development Phases

  • Preclinical Mucopolysaccharidosis III

Most Recent Events

  • 19 Sep 2017 Lysogene reported about working on the Paediatric Investigation Plan (PIP) with the EMA
  • 31 Aug 2017 Preclinical trials in Mucopolysaccharidosis III in France (Intracerebral) (LYSOGENE pipeline, August 2017)
  • 30 Aug 2017 LYSOGENE plans to initiate a phase II/III trial in Mucopolysaccharidosis III in January 2018
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