Sarepta cuts its operating loss to $9m, but can new RNA drugs offset Elevidys’ decline?
Sarepta narrows its Q3 operating loss to $9 million as Elevidys sales fall 37%. Examine safety, RNA pipeline data and guidance.
Pharma & Biotech
Pharma & Biotech
Sarepta’s RNA therapy reaches the nucleus in myotonic dystrophy models. Can it restore muscle function?
Sarepta has reported up to 80% knockdown of DMPK RNA in both the nucleus and cytoplasm of patient-derived muscle cells, alongside correction of abnormal RNA splicing in preclinical models. The findings address a central delivery challenge in myotonic dystrophy type 1, while an ongoing Phase 1/2 trial will determine whether the mechanism translates into patient
Pharma & Biotech
Could cTAP’s new Duchenne prognostic score change how DMD trials measure mobility risk?
Find out how cTAP’s Duchenne prognostic score could improve DMD trial design and loss of ambulation prediction.
Features & Analysis
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.
Pharma & Biotech
Can Sarepta Therapeutics prove RNA interference works in FSHD1 and DM1 with early Phase 1/2 data
Read why Sarepta Therapeutics’ first siRNA data in FSHD1 and DM1 could matter for rare disease drug development and investor sentiment.
Pharma & Biotech
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.
Pharma & Biotech
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.
Pharma & Biotech
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.