In a Phase 3 clinical trial, Pfizer’s gene therapy for Duchenne muscular dystrophy did not improve patients’ ability to walk or stand up compared to a placebo. The disappointing results are likely to lead to the end of the program. Duchenne is a rare muscle-wasting disease that eventually affects the lungs and heart, leading to fatality. Pfizer’s gene therapy uses a virus to deliver a mini-version of the gene that codes for dystrophin, but safety concerns have plagued its development. In contrast, Sarepta Therapeutics’ gene therapy has shown more promising results and may receive full FDA approval soon.
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Pfizer & Sarepta Gene Therapies Both Failed Phase 3, But Analysts Expect Sarepta Will Win Approval
