Arylsulfatase A gene therapy - REGENXBIO

Drug Profile

Arylsulfatase A gene therapy - REGENXBIO

Latest Information Update: 16 Dec 2015

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At a glance

  • Originator ReGenX Biosciences
  • Developer Assistance Publique Hopitaux de Paris
  • 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.

  • New Molecular Entity No

Highest Development Phases

  • No development reported Metachromatic leukodystrophy

Most Recent Events

  • 16 Dec 2015 No development reported - Phase-I/II for Metachromatic leukodystrophy in France (Intracerebral)
  • 01 Mar 2013 Phase-I/II clinical trials in Metachromatic leukodystrophy in France (Intracerebral)
  • 20 Dec 2011 Preclinical trials in Metachromatic leukodystrophy in USA (Intracerebral)
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