Pfizer has announced the first participant for its US-based gene therapy trial, was recently dosed at Duke University Medical Centre. The trial is assessing the gene therapy (PF-06939926) in approximately 12 boys with Duchenne between the ages of 5 and 12 who are still able to walk. PF-06939926 uses an adeno-associated virus to carry a shortened version of the dystrophin gene (mini-dystrophin) into the body.
In addition to safety, the trial will also assess if PF-06939926 is able to increase dystrophin protein levels and improve muscle strength. Preliminary data from this trial is expected in the first half of 2019.
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