Pfizer has initiated a Phase 1b trial for treating patients with Duchenne muscular dystrophy (DMD) using Bamboo Therapeutics’ investigational mini-dystrophin gene therapy which it acquired in 2016.
This first-in-human open-label, non-randomized study is designed to evaluate the safety and tolerability of a single intravenous infusion of PF-06939926 in ambulatory subjects with DMD.
The first boy received the treatment last month under the supervision of Dr Edward Smith, Principal Investigator of the study at Duke University Medical Center. The study will enrol approximately 12 ambulatory DMD subjects aged 5-12 at up to four clinical centers in the US. In addition to assessing the safety and tolerability, the trial will also evaluate dystrophin expression and distribution, and conduct assessments of muscle strength, quality, and function in the subjects.
PF-06939926 is a recombinant AAV9 vector carrying a shortened version of the human dystrophin gene (mini-dystrophin) under the control of a human muscle specific promotor. It was granted Orphan designation by the EMA and Orphan Drug and Rare Pediatric Disease designation by the FDA in 2017.
Source: Pfizer puts Bamboo’s muscular dystrophy gene therapy to the test; Press Release