After Setbacks, Confidence Grows In Likely Success For Sarepta’s Duchenne Gene Therapy
Contrasts With Pfizer Safety Concerns
Executive Summary
CEO tells J.P. Morgan conference that he is confident the pivotal EMBARK study can be completed in 2022 with regulatory filing in 2023.
You may also be interested in...
The Top Five (Non-COVID) Pharma Stories Of 2021
The emergence and rapid spread of the Omicron variant has kept COVID-19 firmly in the headlines but the biopharma industry has had plenty on its plate on top of tackling the pandemic. Scrip has taken a look back at five of the biggest non-COVID-19 story themes of 2021 in no particular order.
Spotlight On High Doses Again After Pfizer Duchenne Gene Therapy Death
The death in Pfizer’s Phase I study could reflect wider safety issues for AAV-based gene therapy and could spur intervention from regulators in 2022.
Sarepta Sinks On Mixed DMD Gene Therapy Study
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.