SAF 301

Drug Profile

SAF 301

Alternative Names: SAF-301

Latest Information Update: 27 Sep 2016

Price : $50

At a glance

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

  • 21 Jul 2015 SAF 301 is still in phase I/II trials for Mucopolysaccharidosis III in France
  • 21 Jul 2015 LYSOGENE and Alcyone agree to co-develop rAAV based gene therapy using the Alcyone MEMS Cannula (AMC) targeted delivery platform for Mucopolysaccharidosis III
  • 31 Jan 2014 Lysogene completes enrolment in a phase I/II follow-up trial for Mucopolysaccharidosis III in France (NCT02053064)
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