DTX 301

Drug Profile

DTX 301

Alternative Names: DTX 301; scAAV8OTC

Latest Information Update: 29 Mar 2017

Price : $50

At a glance

  • Originator Dimension Therapeutics
  • 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.

    Yes - Inborn urea cycle disorders
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Inborn urea cycle disorders

Most Recent Events

  • 01 Jan 2017 Phase-I/II clinical trials in Inborn urea cycle disorders in USA (IV) (NCT02991144)
  • 13 Dec 2016 US FDA accepts IND application for DTX 301 in ornithine transcarbamylase (OTC) deficiency
  • 23 Jan 2015 ReGenX Biosciences is now called REGENXBIO
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