Phase |/||

Therapeutic Approach

Restoring or Replacing Dystrophin

GALGT2 is a gene that produces an enzyme that encourages over-production of other proteins that are important to stabilize the muscle cell membrane and ultimately improve muscle function. The GALGT2 gene is introduced through gene therapy using a viral vector, with the aim of compensating for the missing dystrophin.


A phase 1/2 trial is active, not recruiting.


This program is sponsored by Nationwide Children’s Hospital and partially funded by Parent Project Muscular Dystrophy.

Related Studies

Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy

Media Library

JUNE 2018

Kevin Flanigan, MD (Nationwide Children’s) Presents at the PPMD 2018 Annual Conference


Webinar: Understanding Gene Therapy, Part 3 - Galgt2 and Dup2

As part of our ongoing series on Understanding Gene Therapy, PPMD hosted a webinar with Dr. Kevin Flanigan, Director of the Center for Gene Therapy at Nationwide Children’s Hospital. Dr. Flanigan helped explain two gene therapy technologies that he and his team at Nationwide are exploring: GALGT2 and Dup2. This webinar is the third in our series on Gene Therapy where we are bring leaders in gene therapy technology to you to discuss their latest projects and provide an update on this incredibly exciting and expanding field.

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