Swiss company to appeal regulators’ decision.
Trials will resume once safety protocols have been reviewed.
A UK biotech start-up has gained a sought after ‘promising innovative medicine’ (PIM) designation for its ultra-rare disease gene therapy.
How can orphan drug manufacturers demonstrate value and improve access to drugs?
Firm turns down options for inotersen and IONIS-FB-LRX.
Alexion cuts collaborations with Moderna Therapeutics, Blueprint Medicines and Arbutus Biopharma