News Alnylam halts trials of haemophilia drug after patient death Trials will resume once safety protocols have been reviewed.
News Orchard’s gene therapy gains UK ‘promising innovative medici... A UK biotech start-up has gained a sought after ‘promising innovative medicine’ (PIM) designation for its ultra-rare disease gene therapy.
Views & Analysis Orphan drugs: overcoming HTA barriers to access How can orphan drug manufacturers demonstrate value and improve access to drugs?
News Ionis to file rare disease drug after GSK declines option Firm turns down options for inotersen and IONIS-FB-LRX.
News Alexion cuts ties with Moderna and re-focuses Alexion cuts collaborations with Moderna Therapeutics, Blueprint Medicines and Arbutus Biopharma
News Promise of CF combinations lifts Vertex, reignites takeover ... Encouraging results from three pipeline combination therapies suggests Vertex could extend treatment to patients with more severe and difficult to treat cystic fibrosis.
News UK's Mission divests kidney drug to Dimerix for $292m Mission Therapeutics has struck a deal to out-license a drug for acute kidney injury to Australia's Dimerix, sharpening its focus on CNS diseases.
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