Duchenne muscular dystrophy gene therapy new trial with improved formulation and rare disease research
Clinical TrialsBusiness Wire

Pfizer Initiates New Phase 3 Gene Therapy Trial for Duchenne Muscular Dystrophy

Pfizer announces initiation of a new Phase 3 trial evaluating an improved gene therapy formulation for Duchenne muscular dystrophy in ambulatory boys.

Pfizer today announced the initiation of a new Phase 3 trial evaluating an improved formulation of its gene therapy for Duchenne muscular dystrophy (DMD) in ambulatory boys aged 4-7 years. The new trial incorporates lessons learned from the CIFFREO trial and uses an optimized dosing regimen. The trial will evaluate the improved formulation's ability to increase micro-dystrophin expression and improve motor function. Pfizer remains committed to developing a gene therapy for DMD and believes the improved formulation has the potential to demonstrate clinical benefit. The trial is expected to complete enrollment within 18 months.

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