Can non-viral gene therapies solve the safety problem holding back genetic medicine?
Viral vectors built gene therapy’s first era. Safety, redosing and manufacturing pressure are pushing non-viral platforms into the spotlight.
Sarepta’s sirolimus gambit: Can enhanced immunosuppression rescue ELEVIDYS for non-ambulatory Duchenne patients?
Sarepta’s ELEVIDYS Cohort 8 targets non-ambulatory Duchenne patients with a sirolimus regimen. What the trial design reveals about the path to label restoration.
Can Sarepta’s ELEVIDYS redefine Duchenne therapy? Inside the 3-year EMBARK data
Sarepta’s gene therapy ELEVIDYS shows 3-year durability in Duchenne muscular dystrophy. Find out what it means for patients, payers, and global rollout.
Can ELEVIDYS meet its promise? Sarepta’s pivotal Duchenne gene therapy trial nears major milestone
Sarepta’s EMBARK 3-year readout could clarify ELEVIDYS’ long-term role in Duchenne care. Find out what’s at stake in this pivotal gene therapy trial.