Galgt2 gene therapy - Nationwide Children's Hospital/Sarepta Therapeutics

Drug Profile

Galgt2 gene therapy - Nationwide Children's Hospital/Sarepta Therapeutics

Alternative Names: GalNAc tranferase gene therapies; rAAVrh74.MCK.GALGT2

Latest Information Update: 15 Nov 2017

Price : *
* 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. Adis is an information provider. We do not sell or distribute the pharmaceutical compounds written about in this database.

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

  • 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
  • 01 Nov 2017 Phase-I/II clinical trials in Duchenne muscular dystrophy (In children, In adolescents, In adults) in USA (Intra-arterial) (NCT03333590)
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 so we can help.

Back to top