Gene Therapy for Pompe Disease: Barry Byrne, MD, PhD


The director of the Powell Gene Therapy Center at the University of Florida discussed gene therapy programs being conducted for Pompe disease.

“The focus of our gene therapy program is trying to address unmet needs in patients. Enzyme replacement therapy doesn't reach those central nervous system compartments and gene therapy vectors do. So, the focus of the has been to establish assays for CNS expression, both in parts of the brain and lower motor neurons within the spinal cord. That’s been the goal of the studies, to show that we can restore the function of neurons and then improve the strength of the animal models that we study with AAV gene therapy.”

Gene therapies are being investigated for the potential treatment of Pompe disease as a single-dose alternative to enzyme replacement therapy. Current studies investigating gene therapies in this space include the phase 1/2 RESOLUTE study (NCT04093349) of SPK-3006 as well multiple studies from the University of Florida’s Powell Center for Gene Therapy.

The Powell Center is conducting a phase 1 study (NCT02240407) investigating re-administration of intramuscular AAV9 in patients with late-onset Pompe disease (LOPD) as well as an observational study (NCT02354664) evaluating dynamic respiratory motor performance as a valuable measure of pulmonary function in adults with LOPD.

GeneTherapyLive spoke with the director of the Powell Center, Barry Byrne, MD, PhD, to learn more about the gene therapy programs the center is conducting. He also discussed the center’s goals in terms of LOPD.

Spark Therapeutics announces first participant dosed in phase 1/2 study of investigational gene therapy for late-onset Pompe disease. News release. Spark Therapeutics. February 1, 2021.
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