
Ash 2021 movers – IGM steals the show, for all the wrong reasons
At a meeting lacking practice-changing studies and featuring many disappointments the downturn in IGM’s fortunes stands out.

4D gets a good start in Fabry
But a serious adverse event and waning enzyme levels raise questions about the data.

Idorsia’s Fabry flop puts focus on gene therapies
But questions about gene therapy safety could benefit the enzyme-replacement therapy incumbents.

Crucial clinical events for the smaller players
Data are due in the fourth quarter for Aldeyra, Aeglea and Radius, among other small developers.

Haemophilia B delay hits Uniqure
One-year data look encouraging, but etranadez will now not be filed until 2022.