MINI-DYSTROPHIN - PF-06939926 Mini-dystrophin Gene Therapy for Duchenne


Frequently Asked Questions (FAQs) about this research:


What stage is this research?

  • This Phase 1b clinical trial will begin recruiting in early 2018.  This is a first-in-human, multi-center, open-label, non-randomized, ascending dose, safety and tolerability study of a single intravenous infusion of PF-06939926 in boys with Duchenne muscular dystrophy.


Where is this research being done and who is funding this research?

  • This research is being conducted and sponsored by Pfizer Inc., following Pfizer’s acquisition of Bamboo Therapeutics in August, 2016.  Pfizer’s Rare Disease Research Unit is located in Cambridge, Massachusetts and is expanding their Glossary Link gene therapy manufacturing capabilities in Chapel Hill, North Carolina.


What is the goal or purpose of this research?

  • The clinical candidate, PF-06939926, consists of a recombinant AAV Glossary Link vector expressing a miniaturized version of the DMD gene, which encodes the domains minimally required for functionality of the dystrophin Glossary Link protein.


Who is eligible to participate in this trial?

  • Boys with Duchenne between the ages of 5 years - 12 years who are ambulatory and on a stable dose of steroids may be able to participate.  Please visit (NCT03362502) for the full inclusion and exclusion criteria.


Where is this clinical trial taking place?

  • This trial is taking place at Duke University in Durham, NC, and 3 other sites to be announced soon.  


Where can I learn more about this research?

  • (NCT03362502)
  • Visit, Pfizer’s online database where you learn more about our portfolio of new medicines and find more about our Research and Development efforts around the world.

(updated 12/18/2017)