Either you have JavaScript disabled or your browser does not support Javascript . To work properly, this page requires JavaScript to be enabled.
How to enable JavaScript in your browser?


Drug Profile


Alternative Names: Galgt2 gene therapy - Nationwide Children's Hospital/Sarepta Therapeutics; GalNAc tranferase gene therapies; rAAVrh74.MCK.GALGT2

Latest Information Update: 15 Nov 2017

Price : $50 *
  • Adis is an information provider. We do not sell or distribute actual drugs.
  • Final gross price and currency may vary according to local VAT and billing address.
  • Your purchase entitles you to full access to the information contained in our drug profile at the time of purchase.
  • A link to download a PDF version of the drug profile will be included in your email receipt.

At a glance

  • Originator Nationwide Children's Hospital
  • Developer Nationwide Children's Hospital; Sarepta 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.

  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Duchenne muscular dystrophy

Most Recent Events

  • 06 Nov 2017 Phase-I/II clinical trials in Duchenne muscular dystrophy (In children, In adolescents, In adults) in USA (Intra-arterial) (NCT03333590)
  • 03 Nov 2017 Sarepta Therapeutics plans a phase I/IIa trial for rAAVrh74.MCK.GALGT2 in Duchenne muscular dystrophy in USA in 2017
  • 03 Nov 2017 US FDA approves IND application for rAAVrh74.MCK.GALGT2 in Duchenne muscular dystrophy
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