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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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.
Durable responses have continued to be seen two years after administration of Sarepta’s candidate gene therapy to four boys with Duchenne muscular dystrophy.
Positive functional results from Sarepta’s SRP-9003 gene therapy in limb-girdle muscular dystrophy type 2E bodes well for a pivotal study, and for the company’s studies of other potential gene therapies in Duchenne muscular dystrophy.