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

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 - Glycogen storage disease type I
  • On Fast track

    Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.

    No
  • New Molecular Entity No

Highest Development Phases

  • Preclinical Glycogen storage disease type I

Most Recent Events

  • 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
  • 04 Oct 2016 DTX 401 receives Orphan Drug status for Glycogen storage disease type I in USA
  • 24 Mar 2016 Dimension Therapeutics has patent protection for DTX 301 and DTX 401 (Dimension Therapeutics 10-K; March 2016)
Restricted Access

Oops, it looks like you don’t have a valid subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • with a username/password associated to an account with a valid subscription
  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Request a trial

If you are a subscriber to this content then contact us at AsktheExpert.AdisInsight@springer.com so we can help.

Back to top