AstraZeneca's newly acquired Alexion will buy the remaining equity in rare disease specialist Caelum Biosciences in a deal that could be worth up to $500 million.
Biohaven's key pipeline drug verdiperstat has failed to have any therapeutic effect in patients with the rare neurological disorder multiple system atrophy (MSA) – its lead indication – but
Prospects for Valneva's COVID-19 vaccine are up in the air, but another candidate partnered with Pfizer looks increasingly like it could fill the void of an effective shot for Lyme disease.
Sanofi has said that it won't develop its mRNA-based vaccine for COVID-19 any further, even though it seemed to work in an early-stage trial, and will focus instead on another candidate par
Capricor Therapeutics' bid to find a partner for its Duchenne muscular dystrophy (DMD) cell therapy CAP-1002 could get a boost after final phase 2 data showed a strong effect on disease pro
Roche has highlighted new data with its oral treatment for spinal muscular atrophy (SMA) – Evrysdi – showing its benefits when given to pre-symptomatic babies with the rare disease.