Investors Revive Shelved Drug

BostonRoche shelved it; RA Capital just paid $175 million for it.

Orphan-drug status buys exclusivity and smaller trials, making a rare-disease therapy viable only at six-figure yearly prices per patient.

The SPAC-backed startup competes against Ultragenyx’s and Ionis’s Phase 3 trials, both reporting results this year.

Sources: STAT