TheTechno Optimist.

ElevidysSarepta Therapeutics—Three-year Phase 3 EMBARK data shows Elevidys slows disease progression by 70-73% in ambulatory Duchenne patientsElevidysSarepta Therapeutics—FDA adds boxed warning to Elevidys for acute liver injury; non-ambulatory indication removedSarepta Therapeutics—Michael Severino appointed CEO of Sarepta Therapeutics

Sarepta Therapeutics

Gene therapy, Elevidys, Duchenne muscular dystrophy

USpublic~1,370 staffest. 1980sarepta.com (opens sarepta.com)Checked 10 Oct

The brief

Analyst view
  • Elevidys is FDA-approved for ambulatory Duchenne children 4+ years old.
  • Three-year Phase 3 data from January 2026 showed 70% disease progression slowing.
  • Non-ambulatory indication removed November 2025 after liver safety events; boxed warning added.

Technical approach

As reported
Delivery

AAV vector to muscle cells

AAV vectors carry genetic instructions into muscle cells where they make dystrophin to restore muscle function.

Dystrophin

Restoring the missing protein

Duchenne lacks dystrophin, a structural protein for muscle stability. Elevidys delivers a shortened gene so cells build functional dystrophin.

Patient stage

For ambulatory children

Approved for ambulatory children 4+ with Duchenne mutations. Non-ambulatory indication removed after liver safety events.

Primer