DTX 301

Drug Profile

DTX 301

Alternative Names: DTX 301; scAAV8OTC

Latest Information Update: 08 Nov 2017

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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

  • 07 Nov 2017 Dimension Therapeutics has been acquired by Ultragenyx Pharmaceutical
  • 16 Jul 2017 Phase-I/II clinical trials in Inborn urea cycle disorders in Canada (IV) (NCT02991144)
  • 10 May 2017 DTX 301 receives Fast Track designation for Inborn urea cycle disorders [IV] in USA prior to May 2017 (3256041; 9218976)
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