Phase 1b clinical research study of mini-dystrophin for DMD

CureDuchenne is proud that another one of our funding projects is entering clinical trial.

Pfizer Inc. is planning a Phase 1b, Multicenter, Open-label, Single Ascending Dose Study to Evaluate the Safety and Tolerability of PF-06939926 in Ambulatory Boys with Duchenne Muscular Dystrophy (DMD).  This clinical research study is designed to evaluate the safety and tolerability of PF-06939926 in boys diagnosed with DMD who are ambulatory and 5-12 years of age.  PF-06939926 is an investigational, recombinant, adeno-associated virus, serotype 9 (AAV9) carrying a truncated human dystrophin gene (mini-dystrophin) under the control of a human muscle specific promotor. This clinical trial is the culmination of years of scientific exploration and experimentation to advance gene therapy for the DMD community, reflecting the efforts of Pfizer and Bamboo Therapeutics, Inc. to progress this first-in-human/first-in-patient clinical research study. For more information, go to www.clinicaltrials.gov.

We are grateful for Pfizer’s commitment to the Duchenne community.  We look forward to updates on the progress of this important clinical research study.