Idorsia’s Fabry flop puts focus on gene therapies
But questions about gene therapy safety could benefit the enzyme-replacement therapy incumbents.
Editas falls well short of Brilliance
Data on five patients treated with the Crispr therapy EDIT-101 raise doubts about efficacy and safety.
Pfizer adds to gene therapy woes
But another safety scare for the big pharma’s Duchenne muscular dystrophy project could be good news for Sarepta.
Another gene therapy safety scare hits
The clinical hold for Biomarin’s phenylketonuria project comes on the heels of an FDA adcom to discuss gene therapy toxicity.
Astellas’s gene therapy aspirations crumble
AT132 has prompted more safety concerns, this time at a lower dose than before.
Go or no go? A first for Nefecon
Calliditas is hoping for its first approval, while Abbvie is gearing up to enter the crowded migraine space with an oral anti-CGRP project.
Bluebird exits Europe as another hold hits
The business split is still on, but problems mount for the genetic disease wing of Bluebird.
