Pfizer Planning To Start Phase III DMD Gene Therapy Trial, With Safety Monitoring Protocols
Updated Phase Ib data showed safety issues due to complement activation, but Pfizer is confident the benefits outweigh the risks and monitoring systems will help as it moves into Phase III.
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The day before an FDA advisory committee meeting about the safety of AAV vector-based gene therapies, Astellas reported liver damage in a patient in its trial of AT132.
Advisory committee will discuss five types of toxicity risks with adeno-associated virus vector-based gene therapy products at a two-day meeting in September, which follows some recent high-profile instances of adverse events that have delayed development programs.
Any time advantage Sarepta's Duchenne muscular dystrophy gene therapy may have had over rivals, notably Pfizer, has disappeared after SRP-9001 failed to significantly improve muscle function in a closely watched trial.