Cortellis Drugs to Watch in 2020 report

6. BioMarin Pharmaceutical’s Valrox is an injectable gene therapy that corrects the genetic defect underlying haemophilia A – a genetic disorder caused by a missing or defective clotting protein called factor ... Even with a relatively low worldwide

Presidential hopeful Bernie Sanders criticises BioMarin’s gene therapy price

of the response to what BioMarin is claiming is a one-shot therapy. ... The uncertainty of the durability of BioMarin’s drug has left space for other haemophilia A hopefuls from rival developers.

UK’s CMA clears Roche’s $4.3bn takeover of Spark

Athough, analysts at Jefferies have previously suggested that with a number of other companies developing gene therapies – including BioMarin, Sangamo and UniQure – that should not be considered overly dominant.

Sangamo steps up BioMarin chase with haemophilia gene therapy data

Updated results presented at ASH 2019. Sangamo has been playing catch-up with BioMarin in the race to develop a gene therapy for haemophilia A, but new data reported at the ... Sangamo is adamant that it is still in contention in the haemophilia A gene

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