But questions about gene therapy safety could benefit the enzyme-replacement therapy incumbents.
Data on five patients treated with the Crispr therapy EDIT-101 raise doubts about efficacy and safety.
But another safety scare for the big pharma’s Duchenne muscular dystrophy project could be good news for Sarepta.
The clinical hold for Biomarin’s phenylketonuria project comes on the heels of an FDA adcom to discuss gene therapy toxicity.
AT132 has prompted more safety concerns, this time at a lower dose than before.
Calliditas is hoping for its first approval, while Abbvie is gearing up to enter the crowded migraine space with an oral anti-CGRP project.
The business split is still on, but problems mount for the genetic disease wing of Bluebird.