Drug Profile
CLN2 gene therapy - REGENXBIO
Latest Information Update: 01 Mar 2016
Price :
$50
*
At a glance
- Originator ReGenX Biosciences
- Developer Weill Cornell Medical College
- 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
- Phase I/II Neuronal ceroid lipofuscinosis
Most Recent Events
- 01 Mar 2016 Phase-I/II development is ongoing in USA (NCT01414985)
- 05 Sep 2013 Phase-I/II clinical trials in Neuronal ceroid lipofuscinosis in USA (Intracerebral)
- 20 Apr 2011 Phase-I clinical trials in Neuronal ceroid lipofuscinosis in USA (Intracerebral)