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.
For further information, please see Pfizer's community letter and press release.
To find out more about the latest research in Duchenne muscular dystrophy, please contact our Research team at research@musculardystrophyuk.org