Drug Profile
Arylsulfatase A gene therapy - REGENXBIO
Latest Information Update: 16 Dec 2015
Price :
$50
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At a glance
- Originator ReGenX Biosciences
- Developer Assistance Publique Hopitaux de Paris
- Class Gene therapies
- Mechanism of Action Gene transference
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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)