Sarepta Charges Ahead With DMD Gene Therapy Despite Setback
Company Announces Q4 2021 Results
The company plans to start its Phase III trial of SRP-9001 in the middle of this year, with a potential readout by the end of 2022.
You may also be interested in...
The next-generation DMD drug showed dramatic improvement on exon dystrophin and exon skipping, but small numbers and adverse events give pause.
The RNA drug’s accelerated approval brings Sarepta’s exon-skipping market share to nearly 30%.
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.