CLN2 gene therapy - REGENXBIO

Drug Profile

CLN2 gene therapy - REGENXBIO

Latest Information Update: 01 Mar 2016

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At a glance

  • Originator ReGenX Biosciences
  • Developer Weill Cornell Medical College
  • 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

  • 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)
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