Aperture

Neuromuscular · Rare

Duchenne muscular dystrophy

Updated August 2026 · DMD (dystrophin, Xp21)

← Pipeline

REGENXBIO · Gene therapy · Phase 3

RGX-202

Thesis

A second systemic micro-dystrophin, arriving after Elevidys has taught the field about both demand and fatal liver risk. Wins if expression + function are cleaner and the safety narrative is tighter. Loses if it is 'Elevidys-like' on a now-scarred class.

Probability of success

48%

Range 28–64%

  • Pivotal functional package vs EMBARK miss▲ High
  • Class safety prior (AAV liver / ALF)▼ High
  • Elevidys installed base + seroconversion▼ Med
  • Accelerated path still open for expression▲ Med

Time to market

BLA mid-2026 → possible US decision 1H 2027 if file holds

  • 2Q 2026Pivotal topline
  • Mid-2026Planned BLA
  • 2027US decision / EU scientific advice

Peak revenue (US+EU+JP dir.)

$900M

$450M – $1.6B

Peak build

  • Incident ambulatory, 7MM, GT-eligible~1,100 / year
  • Share vs Elevidys + SGT-003 in 203025–40% of new GT starts
  • Net price$2.2–2.8M US; deep EU discount
  • DurationOne-time; no redose. Peak is incident, not prevalent.
  • Penetration limiterSerostatus, del 8/9-class exclusions, centre capacity, consent after ALF

What kills the number

  • A second AAV death in any DMD GT programme re-freezes the class.
  • If topline is expression-only without function, payers will treat it as Elevidys-2.
  • Sarepta/Roche can defend with real-world volume and contracting.