The clinical candidate, PF-06939926, consists of a recombinant AAV Glossary Link vector expressing a miniaturized version of the DMD gene, which encodes the domains minimally required for functionality of the dystrophin Glossary Link protein
Trial will enroll approximately 12 boys aged 5 to 12 years old with Duchenne muscular dystrophy
Pfizer’s first recombinant AAV-based gene therapy program to enter the clinic stemming from Pfizer’s 2016 acquisition of Bamboo Therapeutics
