SAF 301

Drug Profile

SAF 301

Alternative Names: LYS-SAF301; SAF-301

Latest Information Update: 26 Jul 2018

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At a glance

  • Originator LYSOGENE
  • Class Gene therapies
  • Mechanism of Action Gene expression modulators; 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

  • Phase I/II Mucopolysaccharidosis III

Most Recent Events

  • 22 Dec 2017 Lysogene plans a pivotal phase II/III trial for Mucopolysaccharidosis III in USA and Europe in 2018
  • 28 Jun 2017 SAF 301 is still in phase I/II development for Mucopolysaccharidosis III in France
  • 01 Jun 2017 Lysogene completes a phase I/II follow-up trial for Mucopolysaccharidosis III in France (NCT02053064)
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