$DTIL

Interesting call on DMD;

• Lowest vector dose (matching SLDB, half of thay by RGNX, 30% lower vs SRPT)

• >90% full capsid ratio

• larger dystrophin product with known functionality (same as in Becker muscular dystrophy) vs microdyathrophin approaches

• Longer term efficacy expected (in vivo gene editing +transduction of satellite cells) (vs slow decline of viral vector genome from alternative approached)

• transduction of skeletal (including diaphragm) + heart muscle

• compregensive prophylactic protocol (eculizumab, steroids, sirolimus) (so I dont expect any surprise AAV-relates serious AEs)(need to revisit but I think SLDB capsid may be safer. but assuming better and durable efficacy it shouldnt be a problem)

Trully best-in-class potential