Research programme: NAV gene therapies - Dimension Therapeutics

Drug Profile

Research programme: NAV gene therapies - Dimension Therapeutics

Alternative Names: AAV gene therapy - Dimension; Adeno-associated virus vector gene therapy - Dimension; DTX 401

Latest Information Update: 30 Nov 2016

$50 / €47 *
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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 - Glycogen storage disease type I
  • New Molecular Entity No

Highest Development Phases

  • Preclinical Glycogen storage disease type I

Most Recent Events

  • 27 Jun 2017 Dimension Therapeutics announces intention to submit IND application for DTX 401 for Glycogen storage disease type Ia in early 2018
  • 27 Jun 2017 Dimension Therapeutics plans a clinical trial of DTX 401 for Glycogen storage disease type Ia in USA in 2018
  • 29 Nov 2016 The European Medicines Agency Committee for Orphan Medicinal Products recommends DTX 401 for designation as an orphan medicinal product for Glycogen storage disease type Ia
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