Ultragenyx's Gene Therapy for Sanfilippo Syndrome Wins EMA Validation

Impact · against what's priced in

The EMA validation for rebisufligene etisparvovec is a modest positive for Ultragenyx as the market has largely ignored the long-term revenue potential of UX111. For Abeona, the milestone is positive as it moves the company closer to realizing royalties from the in-licensed therapy.

RARE+0.20

Challenges what's priced in

The price assumes

The full long-term revenue potential of DTX401 and UX111, which are largely unpriced beyond immediate launch contributions.