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Pfizer Planning To Start Phase III DMD Gene Therapy Trial, With Safety Monitoring Protocols

 
• By Jessica Merrill

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.

Caution sign
Positive efficacy signals but safety risks too with Pfizer's DMD gene therapy • Source: Shutterstock

Pfizer Inc. will begin enrolling boys with Duchenne muscular dystrophy in a Phase III clinical trial in the second half of 2020, despite serious cases of complement activation in boys treated with the experimental gene therapy in Phase Ib.

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